Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Feature
  • Published:

Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019

Bone Marrow Transplantation volume 54, pages 1525–1552 (2019)Cite this article

Subjects

Abstract

This is the seventh special EBMT report on the indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders. Our aim is to provide general guidance on transplant indications according to prevailing clinical practice in EBMT countries and centres. In order to inform patient decisions, these recommendations must be considered together with the risk of the disease, the risk of the transplant procedure and the results of non-transplant strategies. In over two decades since the first report, the EBMT indications manuscripts have incorporated changes in transplant practice coming from scientific and technical developments in the field. In this same period, the establishment of JACIE accreditation has promoted high quality and led to improved outcomes of patient and donor care and laboratory performance in transplantation and cellular therapy. An updated report with operating definitions, revised indications and an additional set of data with overall survival at 1 year and non-relapse mortality at day 100 after transplant in the commonest standard-of-care indications is presented. Additional efforts are currently underway to enable EBMT member centres to benchmark their risk-adapted outcomes as part of the Registry upgrade Project 2020 against national and/or international outcome data.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

References

  1. Schmitz N, Gratwohl A, Goldman JM. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders. Current practice in Europe in 1996 and proposals for an operational classification. Accreditation Sub-Committee of the European Group for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 1996;17:471–7.

    CAS  PubMed  Google Scholar 

  2. Goldman JM, Schmitz N, Niethammer D, Gratwohl A. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe in 1998. Accreditation Sub-Committee of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 1998;21:1–7.

    Article  CAS  PubMed  Google Scholar 

  3. Urbano-Ispizua A, Schmitz N, de Witte T, Frassoni F, Rosti G, Schrezenmeier H, et al. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: definitions and current practice in Europe. Bone Marrow Transplant. 2002;29:639–46.

    Article  CAS  PubMed  Google Scholar 

  4. Ljungman P, Urbano-Ispizua A, Cavazzana-Calvo M, Demirer T, Dini G, Einsele H, et al. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: definitions and current practice in Europe. Bone Marrow Transplant. 2006;37:439–49.

    Article  CAS  PubMed  Google Scholar 

  5. Ljungman P, Bregni M, Brune M, Cornelissen J, de Witte T, Dini G, et al. Allogeneic and autologous transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe 2009. Bone Marrow Transplant. 2010;45:219–34.

    Article  CAS  PubMed  Google Scholar 

  6. Sureda A, Bader P, Cesaro S, Dreger P, Duarte RF, Dufour C, et al. Indications for allo- and auto-SCT for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2015. Bone Marrow Transplant. 2015;50:1037–56.

    Article  CAS  PubMed  Google Scholar 

  7. Lee SJ, Klein J, Haagenson M, Baxter-Lowe LA, Confer DL, Eapen M, et al. High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood. 2007;110:4576–83.

    Article  CAS  PubMed  Google Scholar 

  8. Ferrara GB, Bacigalupo A, Lamparelli T, Lanino E, Delfino L, Morabito A, et al. Bone marrow transplantation from unrelated donors: the impact of mismatches with substitutions at position 116 of the human leukocyte antigen class I heavy chain. Blood. 2001;98:3150–5.

    Article  CAS  PubMed  Google Scholar 

  9. Bacigalupo A. A closer look at permissive HLA mismatch. Blood. 2013;122:3555–6.

    Article  CAS  PubMed  Google Scholar 

  10. Pidala J, Wang T, Haagenson M, Spellman SR, Askar M, Battiwalla M, et al. Amino acid substitution at peptide-binding pockets of HLA class I molecules increases risk of severe acute GVHD and mortality. Blood. 2013;122:3651–8.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  11. Fleischhauer K, Locatelli F, Zecca M, Orofino MG, Giardini C, De Stefano P, et al. Graft rejection after unrelated donor hematopoietic stem cell transplantation for thalassemia is associated with nonpermissive HLA-DPB1 disparity in host-versus-graft direction. Blood. 2006;107:2984–92.

    Article  CAS  PubMed  Google Scholar 

  12. Weisdorf D, Spellman S, Haagenson M, Horowitz M, Lee S, Anasetti C, et al. Classification of HLA-matching for retrospective analysis of unrelated donor transplantation: revised definitions to predict survival. Biol Blood Marrow Transplant. 2008;14:748–58.

    Article  PubMed  PubMed Central  Google Scholar 

  13. Weisdorf D, Cooley S, Wang T, Trachtenberg E, Haagenson MD, Vierra-Green C, et al. KIR donor selection: feasibility in identifying better donors. Biol Blood Marrow Transplant. 2019;25:e28–e32.

    Article  CAS  PubMed  Google Scholar 

  14. Gale RP, Eapen M. Who is the best alternative allotransplant donor? Bone Marrow Transplant. 2015;50(Suppl 2):S40–2.

    Article  PubMed  PubMed Central  Google Scholar 

  15. Ballen KK. Is there a best graft source of transplantation in acute myeloid leukemia? Best Pract Res Clin Haematol. 2015;28:147–54.

    Article  PubMed  Google Scholar 

  16. Fuchs EJ. Related haploidentical donors are a better choice than matched unrelated donors: point. Blood Adv. 2017;1:397–400.

    Article  PubMed  PubMed Central  Google Scholar 

  17. Shaw BE. Related haploidentical donors are a better choice than matched unrelated donors: counterpoint. Blood Adv. 2017;1:401–6.

    Article  PubMed  PubMed Central  Google Scholar 

  18. Milano F, Gooley T, Wood B, Woolfrey A, Flowers ME, Doney K, et al. Cord-blood transplantation in patients with minimal residual disease. N Engl J Med. 2016;375:944–53.

    Article  PubMed  PubMed Central  Google Scholar 

  19. Passweg JR, Baldomero H, Bader P, Bonini C, Cesaro S, Dreger P, et al. Hematopoietic SCT in Europe 2013: recent trends in the use of alternative donors showing more haploidentical donors but fewer cord blood transplants. Bone Marrow Transplant. 2015;50:476–82.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  20. Passweg JR, Baldomero H, Bader P, Bonini C, Cesaro S, Dreger P, et al. Hematopoietic stem cell transplantation in Europe 2014: more than 40 000 transplants annually. Bone Marrow Transplant. 2016;51:786–92.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  21. Passweg JR, Baldomero H, Bader P, Bonini C, Duarte RF, Dufour C, et al. Use of haploidentical stem cell transplantation continues to increase: the 2015 European Society for Blood and Marrow Transplant activity survey report. Bone Marrow Transplant. 2017;52:811–7.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  22. Passweg JR, Baldomero H, Bader P, Basak GW, Bonini C, Duarte R, et al. Is the use of unrelated donor transplantation leveling off in Europe? The 2016 European Society for Blood and Marrow Transplant activity survey report. Bone Marrow Transplant. 2018;53:1139–48.

    CAS  PubMed  PubMed Central  Google Scholar 

  23. Passweg JR, Baldomero H, Basak GW, Chabannon C, Corbacioglu S, Duarte RF, et al. The EBMT activity survey report 2017: a focus on allogeneic HCT for non-malignant indications and on the use of non-HCT cell therapies. Bone Marrow Transplant. 2018. https://doi.org/10.1038/s41409-019-0465-9.

  24. Cornelissen JJ, Blaise D. Hematopoietic stem cell transplantation for patients with AML in first complete remission. Blood. 2016;127:62–70.

    Article  CAS  PubMed  Google Scholar 

  25. Papaemmanuil E, Döhner H, Campbell PJ. Genomic classification in acute myeloid leukemia. N Engl J Med. 2016;375:900–1.

    Article  PubMed  Google Scholar 

  26. Döhner H, Estey E, Grimwade D, Amadori S, Appelbaum FR, Büchner T, et al. Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel. Blood. 2017;129:424–47.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  27. Shouval R, Labopin M, Bondi O, Mishan-Shamay H, Shimoni A, Ciceri F, et al. Prediction of allogeneic hematopoietic stem-cell transplantation mortality 100 days after transplantation using a machine learning algorithm: a European Group for Blood and Marrow Transplantation Acute Leukemia Working Party Retrospective Data Mining Study. J Clin Oncol. 2015;33:3144–51.

    Article  PubMed  Google Scholar 

  28. Gooley TA, Chien JW, Pergam SA, Hingorani S, Sorror ML, Boeckh M, et al. Reduced mortality after allogeneic hematopoietic-cell transplantation. N Engl J Med. 2010;363:2091–101.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  29. Arber DA, Orazi A, Hasserjian R, Thiele J, Borowitz MJ, Le Beau MM, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127:2391–405.

    Article  CAS  PubMed  Google Scholar 

  30. Balsat M, Renneville A, Thomas X, de Botton S, Caillot D, Marceau A, et al. Postinduction minimal residual disease predicts outcome and benefit from allogeneic stem cell transplantation in acute myeloid leukemia with NPM1 mutation: a study by the Acute Leukemia French Association Group. J Clin Oncol. 2017;35:185–93.

    Article  CAS  PubMed  Google Scholar 

  31. Gorin NC, Labopin M, Frassoni F, Milpied N, Attal M, Blaise D, et al. Identical outcome after autologous or allogeneic genoidentical hematopoietic stem-cell transplantation in first remission of acute myelocytic leukemia carrying inversion 16 or t(8;21): a retrospective study from the European Cooperative Group for Blood and Marrow Transplantation. J Clin Oncol. 2008;26:3183–8.

    Article  PubMed  Google Scholar 

  32. Schlenk RF, Taskesen E, van Norden Y, Krauter J, Ganser A, Bullinger L, et al. The value of allogeneic and autologous hematopoietic stem cell transplantation in prognostically favorable acute myeloid leukemia with double mutant CEBPA. Blood. 2013;122:1576–82.

    Article  CAS  PubMed  Google Scholar 

  33. Savani BN, Labopin M, Kröger N, Finke J, Ehninger G, Niederwieser D, et al. Expanding transplant options to patients over 50 years. Improved outcome after reduced intensity conditioning mismatched-unrelated donor transplantation for patients with acute myeloid leukemia: a report from the Acute Leukemia Working Party of the EBMT. Haematologica. 2016;101:773–80.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  34. Rubio MT, Savani BN, Labopin M, Polge E, Niederwieser D, Ganser A, et al. The impact of HLA-matching on reduced intensity conditioning regimen unrelated donor allogeneic stem cell transplantation for acute myeloid leukemia in patients above 50 years-a report from the EBMT acute leukemia working party. J Hematol Oncol. 2016;9:65.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  35. Schmid C, Labopin M, Socié G, Daguindau E, Volin L, Huynh A, et al. Outcome of patients with distinct molecular genotypes and cytogenetically normal AML after allogeneic transplantation. Blood. 2015;126:2062–9.

    Article  CAS  PubMed  Google Scholar 

  36. Brands-Nijenhuis AV, Labopin M, Schouten HC, Volin L, Socié G, Cornelissen JJ, et al. Monosomal karyotype as an adverse prognostic factor in patients with acute myeloid leukemia treated with allogeneic hematopoietic stem-cell transplantation in first complete remission: a retrospective survey on behalf of the ALWP of the EBMT. Haematologica. 2016;101:248–55.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  37. Cornelissen JJ, Versluis J, Passweg JR, van Putten WL, Manz MG, Maertens J, et al. Comparative therapeutic value of post-remission approaches in patients with acute myeloid leukemia aged 40-60 years. Leukemia. 2015;29:1041–50.

    Article  CAS  PubMed  Google Scholar 

  38. Yanada M, Tsuzuki M, Fujita H, Fujimaki K, Fujisawa S, Sunami K, et al. Phase 2 study of arsenic trioxide followed by autologous hematopoietic cell transplantation for relapsed acute promyelocytic leukemia. Blood. 2013;121:3095–102.

    Article  CAS  PubMed  Google Scholar 

  39. Holter Chakrabarty JL, Rubinger M, Le-Rademacher J, Wang HL, Grigg A, Selby GB, et al. Autologous is superior to allogeneic hematopoietic cell transplantation for acute promyelocytic leukemia in second complete remission. Biol Blood Marrow Transplant. 2014;20:1021–5.

    Article  PubMed  Google Scholar 

  40. Watts JM, Tallman MS. Acute promyelocytic leukemia: what is the new standard of care? Blood Rev. 2014;28:205–12.

    Article  PubMed  Google Scholar 

  41. Ganzel C, Mathews V, Alimoghaddam K, Ghavamzadeh A, Kuk D, Devlin S, et al. Autologous transplant remains the preferred therapy for relapsed APL in CR2. Bone Marrow Transplant. 2016;51:1180–3.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  42. Beldjord K, Chevret S, Asnafi V, Huguet F, Boulland ML, Leguay T, et al. Oncogenetics and minimal residual disease are independent outcome predictors in adult patients with acute lymphoblastic leukemia. Blood. 2014;123:3739–49.

    Article  CAS  PubMed  Google Scholar 

  43. Bassan R, Bourquin JP, DeAngelo DK, Chiaretti S. New approaches to the management of adult acute lymphoblastic lekaemia. J Clin Oncol. 2018. https://doi.org/10.1200/JCO.2017.77.3648

  44. Gupta V, Richards S, Rowe J. Allogeneic, but not autologous, hematopoietic cell transplantation improves survival only among younger adults with acute lymphoblastic leukemia in first remission: an individual patient data meta-analysis. Blood. 2013;121:339–50.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  45. Giebel S, Czyz A, Ottmann O, Baron F, Brissot E, Ciceri F, et al. Use of tyrosine kinase inhibitors to prevent relapse after allogeneic hematopoietic stem cell transplantation for patients with Philadelphia chromosome-positive acute lymphoblastic leukemia: a position statement of the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Cancer. 2016;122:2941–51.

    Article  CAS  PubMed  Google Scholar 

  46. Park JH, Rivière I, Gonen M, Wang X, Sénéchal B, Curran KJ, et al. Long-term follow-up of CD19 CAR therapy in acute lymphoblastic leukemia. N Engl J Med. 2018;378:449–59.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  47. Maude SL, Laetsch TW, Buechner J, Rives S, Boyer M, Bittencourt H, et al. Tisagenlecleucel in children and young adults with B-cell lymphoblastic leukemia. N Engl J Med. 2018;378:439–48.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  48. Wen S, Niu Z, Xing L, Wang Y, Li H, Kuang N, et al. CAR-T bridging to allo-HSCT as a treatment strategy for relapsed adult acute B-lymphoblastic leukemia: a case report. BMC Cancer. 2018;18:1143.

    Article  PubMed  PubMed Central  Google Scholar 

  49. Chabannon C, Kuball J, Mcgrath E, Bader P, Dufour C, Lankester A, et al. CAR-T cells: the narrow path between hope and bankruptcy? Bone Marrow Transplant. 2017;52:1588–9.

    Article  CAS  PubMed  Google Scholar 

  50. Kansagra AJ, Frey NV, Bar M, Laetsch TW, Carpenter PA, Savani BN, et al. Clinical utilization of Chimeric Antigen Receptors T-cells (CAR-T) in B-cell acute lymphoblastic leukemia (ALL) - an expert opinion from the European Society for Blood and Marrow Transplantation (EBMT) and the American Society for Blood and Marrow Transplantation (ASBMT). Biol Blood Marrow Transplant. 2018. https://doi.org/10.1016/j.bbmt.2018.12.068.

  51. Rousselot P, Huguet F, Rea D, Legros L, Cayuela JM, Maarek O, et al. Imatinib mesylate discontinuation in patients with chronic myelogenous leukemia in complete molecular remission for more than 2 years. Blood. 2007;109:58–60.

    Article  CAS  PubMed  Google Scholar 

  52. Mahon FX, Réa D, Guilhot J, Guilhot F, Huguet F, Nicolini F, et al. Discontinuation of imatinib in patients with chronic myeloid leukaemia who have maintained complete molecular remission for at least 2 years: the prospective, multicentre Stop Imatinib (STIM) trial. Lancet Oncol. 2010;11:1029–35.

    Article  CAS  PubMed  Google Scholar 

  53. Alchalby H, Zabelina T, Stübig T, van Biezen A, Bornhäuser M, Di Bartolomeo P, et al. Allogeneic stem cell transplantation for myelofibrosis with leukemic transformation. A study of the MPN-Subcommittee of the CMWP of the EBMT. Biol Blood Marrow Transplant. 2014;20:279–81.

    Article  PubMed  Google Scholar 

  54. Kröger N, Holler E, Kobbe G, Bornhäuser M, Schwerdtfeger R, Baurmann H, et al. Allogeneic stem cell transplantation after reduced-intensity conditioning in patients with myelofibrosis: a prospective, multicenter study of the Chronic Leukemia Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2009;114:5264–70.

    Article  CAS  PubMed  Google Scholar 

  55. Kröger N, Giorgino T, Scott BL, Ditschkowski M, Alchalby H, Cervantes F, et al. Impact of allogeneic stem cell transplantation on survival of patients less than 65 years of age with primary myelofibrosis. Blood. 2015;125:3347–50.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  56. Kröger NM, Deeg JH, Olavarria E, Niederwieser D, Bacigalupo A, Barbui T, et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia. 2015;29:2126–33.

    Article  CAS  PubMed  Google Scholar 

  57. Scott BL, Gooley TA, Sorror ML, Rezvani AR, Linenberger ML, Grim J, et al. The dynamic International Prognostic Scoring System for myelofibrosis predicts outcomes after hematopoietic cell transplantation. Blood. 2012;119:2657–64.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  58. Stübig T, Alchalby H, Ditschkowski M, Wolf D, Wulf G, Zabelina T, et al. JAK inhibition with ruxolitinib as pretreatment for allogeneic stem cell transplantation in primary or post-ET/PV myelofibrosis. Leukemia. 2014;28:1736–8.

    Article  CAS  PubMed  Google Scholar 

  59. Kröger N. Allogeneic stem cell transplantation for elderly patients with myelodysplastic syndrome. Blood. 2012;119:5632–9.

    Article  CAS  PubMed  Google Scholar 

  60. Malcovati L, Porta MG, Pascutto C, Invernizzi R, Boni M, Travaglino E, et al. Prognostic factors and life expectancy in myelodysplastic syndromes classified according to WHO criteria: a basis for clinical decision making. J Clin Oncol. 2005;23:7594–603.

    Article  PubMed  Google Scholar 

  61. Kröger N, Zabelina T, van Biezen A, Brand R, Niederwieser D, Martino R, et al. Allogeneic stem cell transplantation for myelodysplastic syndromes with bone marrow fibrosis. Haematologica. 2011;96:291–7.

    Article  PubMed  Google Scholar 

  62. Bejar R, Stevenson K, Abdel-Wahab O, Galili N, Nilsson B, Garcia-Manero G, et al. Clinical effect of point mutations in myelodysplastic syndromes. N Engl J Med. 2011;364:2496–506.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  63. Bejar R, Stevenson KE, Caughey B, Lindsley RC, Mar BG, Stojanov P, et al. Somatic mutations predict poor outcome in patients with myelodysplastic syndrome after hematopoietic stem-cell transplantation. J Clin Oncol. 2014;32:2691–8.

    Article  PubMed  PubMed Central  Google Scholar 

  64. de Witte T, Bowen D, Robin M, Malcovati L, Niederwieser D, Yakoub-Agha I, et al. Allogeneic hematopoietic stem cell transplantation for MDS and CMML: recommendations from an International Expert Panel. Blood. 2017;129:1753–62.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  65. Dreger P, Ghia P, Schetelig J, van Gelder M, Kimby E, Michallet M, et al. High-risk chronic lymphocytic leukemia in the era of pathway inhibitors: integrating molecular and cellular therapies. Blood. 2018;132:892–902.

    Article  CAS  PubMed  Google Scholar 

  66. Cwynarski K, van Biezen A, de Wreede L, Stilgenbauer S, Bunjes D, Metzner B, et al. Autologous and allogeneic stem-cell transplantation for transformed chronic lymphocytic leukemia (Richter's Syndrome): a retrospective analysis from the Chronic Lymphocytic Leukemia Subcommittee of the Chronic Leukemia Working Party and Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2012;30:2211–7.

    Article  PubMed  Google Scholar 

  67. Gisselbrecht C, Schmitz N, Mounier N, Singh GD, Linch DC, Trneny M, et al. Rituximab maintenance therapy after autologous stem-cell transplantation in patients with relapsed CD20(+) diffuse large B-cell lymphoma: final analysis of the collaborative trial in relapsed aggressive lymphoma. J Clin Oncol. 2012;30:4462–9.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  68. Mounier N, Canals C, Gisselbrecht C, Cornelissen J, Foa R, Conde E, et al. High-dose therapy and autologous stem cell transplantation in first relapse for diffuse large B cell lymphoma in the rituximab era: an analysis based on data from the European Blood and Marrow Transplantation Registry. Biol Blood Marrow Transplant. 2012;18:788–93.

    Article  PubMed  Google Scholar 

  69. Ghielmini M, Vitolo U, Kimby E, Montoto S, Walewski J, Pfreundschuh M, et al. ESMO Guidelines consensus conference on malignant lymphoma 2011 part 1: diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL) and chronic lymphocytic leukemia (CLL). Ann Oncol. 2013;24:561–76.

    Article  CAS  PubMed  Google Scholar 

  70. van Kampen RJ, Canals C, Schouten HC, Nagler A, Thomson KJ, Vernant JP, et al. Allogeneic stem-cell transplantation as salvage therapy for patients with diffuse large B-cell non-Hodgkin's lymphoma relapsing after an autologous stem-cell transplantation: an analysis of the European Group for Blood and Marrow Transplantation Registry. J Clin Oncol. 2011;29:1342–8.

    Article  PubMed  Google Scholar 

  71. Rigacci L, Puccini B, Dodero A, Iacopino P, Castagna L, Bramanti S, et al. Allogeneic hematopoietic stem cell transplantation in patients with diffuse large B cell lymphoma relapsed after autologous stem cell transplantation: a GITMO study. Ann Hematol. 2012;91:931–40.

    Article  PubMed  Google Scholar 

  72. Glass B, Hasenkamp J, Wulf G, Dreger P, Pfreundschuh M, Gramatzki M, et al. Rituximab after lymphoma-directed conditioning and allogeneic stem-cell transplantation for relapsed and refractory aggressive non-Hodgkin lymphoma (DSHNHL R3): an open-label, randomised, phase 2 trial. Lancet Oncol. 2014;15:757–66.

    Article  CAS  PubMed  Google Scholar 

  73. Schorb E, Kasenda B, Atta J, Kaun S, Morgner A, Hess G, et al. Prognosis of patients with primary central nervous system lymphoma after high-dose chemotherapy followed by autologous stem cell transplantation. Haematologica. 2013;98:765–70.

    Article  PubMed  PubMed Central  Google Scholar 

  74. Ferreri AJM, Cwynarski K, Pulczynski E, Fox CP, Schorb E, La RP, et al. Whole-brain radiotherapy or autologous stem-cell transplantation as consolidation strategies after high-dose methotrexate-based chemoimmunotherapy in patients with primary CNS lymphoma: results of the second randomisation of the International Extranodal Lymphoma Study Group-32 phase 2 trial. Lancet Haematol. 2017;4:e510–e523.

    Article  PubMed  Google Scholar 

  75. Neelapu SS, Locke FL, Bartlett NL, Lekakis LJ, Miklos DB, Jacobson CA, et al. Axicabtagene ciloleucel CAR T-cell therapy in refractory large B-cell lymphoma. N Engl J Med. 2017;377:2531–44.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  76. Schuster SJ, Svoboda J, Chong EA, Nasta SD, Mato AR, Anak O, et al. Chimeric antigen receptor T cells in refractory B-cell lymphomas. N Engl J Med. 2017;377:2545–54.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  77. Montoto S, Corradini P, Dreyling M, Ghielmini M, Kimby E, Lopez-Guillermo A, et al. Indications for hematopoietic stem cell transplantation in patients with follicular lymphoma: a consensus project of the EBMT-Lymphoma Working Party. Haematologica. 2013;98:1014–21.

    Article  PubMed  PubMed Central  Google Scholar 

  78. Buske C, Leblond V. How to manage Waldenstrom's macroglobulinemia. Leukemia. 2013;27:762–72.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  79. Kyriakou C, Canals C, Sibon D, Cahn JY, Kazmi M, Arcese W, et al. High-dose therapy and autologous stem-cell transplantation in Waldenstrom macroglobulinemia: the lymphoma working party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010;28:2227–32.

    Article  CAS  PubMed  Google Scholar 

  80. Dimopoulos M, Kastrdis E, Owen RG, Kyle RA, Landgren O, Morra E, et al. Treatment recommendations for patients with Waldenstrom macroglobyulinemia (WM) and related disorders: IWWM-7 consensus. Blood. 2014;124:1404–11.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  81. Kyriakou C, Canals C, Cornelissen JJ, Socie G, Willemze R, Ifrah N, et al. Allogeneic stem-cell transplantation in patients with waldenstrom macroglobulinemia: report from the lymphoma working party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010;28:4926–34.

    Article  PubMed  Google Scholar 

  82. Dreyling M, Jurczak W, Jerkeman M, Silva RS, Rusconi C, trneny M, et al. Ibrutinib versus temsirolimus in patients with relapsed or refractory mantle-cell lymphoma: an international, randomised, open-label, phase 3 study. Lancet. 2016;387:770–8.

    Article  CAS  PubMed  Google Scholar 

  83. Martin P, Maddocks K, Leonard JP, Ruan J, Goy A, Wagner-Johnston N, et al. Post-ibrutinib outcomes in patients with mantle cell lymphoma. Blood. 2016;127:1559–63.

    Article  CAS  PubMed  Google Scholar 

  84. Dreger P, Michallet M, Bosman P, Dietrich S, Sobh M, Boumendil A, et al. Ibrutinib for bridging to allogeneic hematopoietic cell transplantation in patients with chronic lymphocytic leukemia or mantle cell lymphoma: a study by the EBMT Chronic Malignancies and Lymphoma Working Parties. Bone Marrow Transplant. 2018. https://doi.org/10.1038/s41409-018-0207-4.

  85. Fenske TS, Zhang MJ, Carreras J, Ayala E, Burns LJ, Cashen A, et al. Autologous or reduced-intensity conditioning allogeneic hematopoietic cell transplantation for chemotherapy-sensitive mantle-cell lymphoma: analysis of transplantation timing and modality. J Clin Oncol. 2013;32:273–81.

    Article  PubMed  PubMed Central  Google Scholar 

  86. Schmitz N, Nickelsen M, Altmann B, Ziepert M, Bouabdallah K, Gisselbrecht C, et al. Allogeneic or autologous transplantation as first-line therapy for younger patients with peripheral T-cell lymphoma: results of the interim analysis of the AATT trial. J Clin Oncol. 2015;33(15_suppl):8507–8507.

    Google Scholar 

  87. Whittaker SJ, Marsden JR, Spittle M, Russell-Jones R. Joint British Association of Dermatologists and U.K. Cutaneous Lymphoma Groups guidelines for the management of primary cutaneous T-cell lymphomas. Br J Dermatol. 2003;149:1095–107.

    Article  CAS  PubMed  Google Scholar 

  88. Jawed SI, Myskowski PL, Horwitz S, Moskowitz A, Querfeld C. Primary cutaneous T-cell lymphoma (mycosis fungoides and Sézary syndrome): part II. Prognosis, management, and future directions. J Am Acad Dermatol. 2014;70:223.e1–17.

    Article  Google Scholar 

  89. Trautinger F, Eder J, Assaf C, Bagot M, Cozzio A, Dummer R, et al. European Organisation for Research and Treatment of Cancer consensus recommendations for the treatment of mycosis fungoides/Sézary syndrome - update 2017. Eur J Cancer. 2017;77:57–74.

    Article  PubMed  Google Scholar 

  90. Duarte RF, Schmitz N, Servitje O, Sureda A. Haematopoietic stem cell transplantation for patients with primary cutaneous T-cell lymphoma. Bone Marrow Transplant. 2008;41:597–604.

    Article  CAS  PubMed  Google Scholar 

  91. Duarte RF, Canals C, Onida F, Gabriel IH, Arranz R, Arcese W, et al. Allogeneic hematopoietic cell transplantation for patients with mycosis fungoides and Sézary syndrome: a retrospective analysis of the Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol. 2010;28:4492–9.

    Article  PubMed  Google Scholar 

  92. Duarte RF, Boumendil A, Onida F, Gabriel I, Arranz R, Arcese W, et al. Long-term outcome of allogeneic hematopoietic cell transplantation for patients with mycosis fungoides and sézary syndrome: a European society for blood and marrow transplantation lymphoma working party extended analysis. J Clin Oncol. 2014;32:3347–8.

    Article  PubMed  Google Scholar 

  93. Linch DC, Winfield D, Goldstone AH, Moir D, Hancock B, McMillan A, et al. Dose intensification with autologous bone-marrow transplantation in relapsed and resistant Hodgkin's disease: results of a BNLI randomised trial. Lancet. 1993;341:1051–4.

    Article  CAS  PubMed  Google Scholar 

  94. Schmitz N, Pfistner B, Sextro M, Sieber M, Carella AM, Haenel M, et al. Aggressive conventional chemotherapy compared with high-dose chemotherapy with autologous haemopoietic stem-cell transplantation for relapsed chemosensitive Hodgkin's disease: a randomised trial. Lancet. 2002;359:2065–71.

    Article  CAS  PubMed  Google Scholar 

  95. Sureda A, Canals C, Arranz R, Caballero D, Ribera JM, Brune M, et al. Allogeneic stem cell transplantation after reduced intensityconditioning in patients with relapsed or refractory Hodgkin'slymphoma. Results of the HDR-ALLO study - a prospectiveclinical trial by the Grupo Espanol de Linfomas/Trasplante deMedula Osea (GEL/TAMO) and the Lymphoma Working Party ofthe European Group for Blood and Marrow Transplantation. Haematologica. 2012;97:310–7.

    Article  PubMed  PubMed Central  Google Scholar 

  96. Sarina B, Castagna L, Farina L, Patriarca F, Benedetti F, Carella AM, et al. Allogeneic transplantation improves the overall and progression-free survival of Hodgkin lymphoma patients relapsing after autologous transplantation: a retrospective study based on the time of HLA typing and donor availability. Blood. 2010;115:3671–7.

    Article  CAS  PubMed  Google Scholar 

  97. Messer M, Steinzen A, Vervolgyi E, Lerch C, Richter B, Dreger P, et al. Unrelated and alternative donor allogeneic stem cell transplantation in patients with relapsed or refractory Hodgkin's lymphoma: a systematic review. Leuk Lymphoma. 2014;55:296–306.

    Article  CAS  PubMed  Google Scholar 

  98. Dhakal B, Szabo A, Chhabra S, Hamadani M, D'Souza A, Usmani SZ, et al. Autologous transplantation for newly diagnosed multiple myeloma in the era of novel agent induction: a systematic review and meta-analysis. JAMA Oncol. 2018;4:343–50.

    Article  PubMed  PubMed Central  Google Scholar 

  99. Mina R, Petrucci MT, Corradini P, Spada S, Patriarca F, Cerrato C, et al. Treatment intensification with autologous stem cell transplantation and lenalidomide maintenance improves survival outcomes of patients with newly diagnosed multiple myeloma in complete response. Clin Lymphoma Myeloma Leuk. 2018;18:533–40.

    Article  PubMed  Google Scholar 

  100. Rodriguez TE, Hari P, Stiff PJ, Smith SE, Sterrenberg D, Vesole DH. Busulfan, melphalan, and bortezomib versus high-dose melphalan as a conditioning regimen for autologous hematopoietic stem cell transplantation in multiple myeloma. Biol Blood Marrow Transplant. 2016;22:1391–6.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  101. Cavo M, Petrucci MT, Di Raimondo F, Zamagni E, Gamberi B, Crippa C, et al. Upfront single versus double autologous stem cell transplantation for newly diagnosed multiple myeloma: an intergroup, multicenter, phase III study of the European Myeloma Network (EMN02/HO95 MM Trial). Blood. 2016;128:991.

    Google Scholar 

  102. Cavo M, Beksac M, Dimopoulos MA, Pantani L, Gay F, Hájek R, et al. Intensification therapy with bortezomib-melphalan-prednisone versus autologous stem cell transplantation for newly diagnosed multiple myeloma: an intergroup, multicenter, phase III study of the European Myeloma Network (EMN02/HO95 MM Trial). Blood. 2016;128:673.

    Google Scholar 

  103. Attal M, Lauwers-Cances V, Marit G, Caillot D, Moreau P, Facon T, et al. Lenalidomide maintenance after stem-cell transplantation for multiple myeloma. N Engl J Med. 2012;366:1782–91.

    Article  CAS  PubMed  Google Scholar 

  104. McCarthy PL, Owzar K, Hofmeister CC, Hurd DD, Hassoun H, Richardson PG, et al. Lenalidomide after stem-cell transplantation for multiple myeloma. N Engl J Med. 2012;366:1770–81.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  105. Jimenez-Zepeda VH, Mikhael J, Winter A, Franke N, Masih-Khan E, Trudel S, et al. Second autologous stem cell transplantation as salvage therapy for multiple myeloma: impact on progression-free and overall survival. Biol Blood Marrow Transplant. 2012;18:773–9.

    Article  PubMed  Google Scholar 

  106. Sobh M, Michallet M, Gahrton G, Iacobelli S, van Biezen A, Schönland S, et al. Allogeneic hematopoietic cell transplantation for multiple myeloma in Europe: trends and outcomes over 25 years. A study by the EBMT Chronic Malignancies Working Party. Leukemia. 2016;30:2047–54.

    Article  CAS  PubMed  Google Scholar 

  107. Bruno B, Rotta M, Patriarca F, Mordini N, Allione B, Carnevale-Schianca F, et al. A comparison of allografting with autografting for newly diagnosed myeloma. N Engl J Med. 2007;356:1110–20.

    Article  CAS  PubMed  Google Scholar 

  108. Gahrton G, Iacobelli S, Bjorkstrand B, Hegenbart U, Gruber A, Greinix H, et al. Autologous/reduced-intensity allogeneic stem cell transplantation vs autologous transplantation in multiple myeloma: long-term results of the EBMT-NMAM2000 study. Blood. 2013;121:5055–63.

    Article  CAS  PubMed  Google Scholar 

  109. Rosinol L, Perez-Simon JA, Sureda A, de la Rubia J, de Arriba F, Lahuerta JJ, et al. A prospective PETHEMA study of tandem autologous transplantation versus autograft followed by reduced-intensity conditioning allogeneic transplantation in newly diagnosed multiple myeloma. Blood. 2008;112:3591–3.

    Article  CAS  PubMed  Google Scholar 

  110. Patriarca F, Einsele H, Spina F, Bruno B, Isola M, Nozzoli C, et al. Allogeneic stem cell transplantation in multiple myeloma relapsed after autograft: a multicenter retrospective study based on donor availability. Biol Blood Marrow Transplant. 2012;18:617–26.

    Article  CAS  PubMed  Google Scholar 

  111. Ghosh N, Ye X, Tsai HL, Bolaños-Meade J, Fuchs EJ, Luznik L, et al. Allogeneic blood or marrow transplantation with post-transplantation cyclophosphamide as graft-versus-host disease prophylaxis in multiple myeloma. Biol Blood Marrow Transplant. 2017;23:1903–9.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  112. Dispenzieri A, Kyle RA, Lacy MQ, Therneau TM, Larson DR, Plevak MF, et al. Superior survival in primary systemic amyloidosis patients undergoing peripheral blood stem cell transplantation: a case-control study. Blood. 2004;103:3960–3.

    Article  CAS  PubMed  Google Scholar 

  113. Jaccard A, Moreau P, Leblond V, Leleu X, Benboubker L, Hermine O, et al. High-dose melphalan versus melphalan plus dexamethasone for AL amyloidosis. N Engl J Med. 2007;357:1083–93.

    Article  CAS  PubMed  Google Scholar 

  114. D'Souza A, Dispenzieri A, Wirk B, Zhang MJ, Huang J, Gertz MA, et al. Improved outcomes after autologous hematopoietic cell transplantation for light chain amyloidosis: a Center for International Blood and Marrow Transplant Research Study. J Clin Oncol. 2015;33:3741–9.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  115. Sidiqi MH, Aljama MA, Buadi FK, Warsame RM, Lacy MQ, Dispenzieri A, et al. Stem cell transplantation for light chain amyloidosis: decreased early mortality over time. J Clin Oncol. 2018;36:1323–9.

    Article  PubMed  Google Scholar 

  116. Bochtler T, Hegenbart U, Kunz C, Benner A, Kimmich C, Seckinger A, et al. Prognostic impact of cytogenetic aberrations in AL amyloidosis patients after high-dose melphalan: a long-term follow-up study. Blood. 2016;128:594–602.

    Article  CAS  PubMed  Google Scholar 

  117. Schönland SO, Dreger P, de Witte T, Hegenbart U. Current status of hematopoietic cell transplantation in the treatment of systemic amyloid light-chain amyloidosis. Bone Marrow Transplant. 2012;47:895–905.

    Article  CAS  PubMed  Google Scholar 

  118. Marsh JC, Ball SE, Cavenagh J, et al. Guidelines for the diagnosis and management of aplastic anaemia. Br J Haematol. 2009;147:43–70.

    Article  CAS  PubMed  Google Scholar 

  119. Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120:1185–96.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  120. Giammarco S, Peffault de Latour R, Sica S, Dufour C, Socie G, Passweg J, et al. Transplant outcome for patients with acquired aplastic anemia over the age of 40: has the outcome improved? Blood. 2018;131:1989–92.

    Article  CAS  PubMed  Google Scholar 

  121. Bacigalupo A, Socié G, Schrezenmeier H, Tichelli A, Locasciulli A, Fuehrer M, et al. Bone marrow versus peripheral blood matched sibling transplants, in acquired aplastic anemia: survival advantage for marrow in all age groups. Haematologica. 2012;97:1142–8.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  122. Marsh JC, Gupta V, Lim Z, Ho AY, Ireland RM, Hayden J, et al. Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anaemia. Blood. 2011;118:2351–7.

    Article  CAS  PubMed  Google Scholar 

  123. Marsh JC, Pearce RM, Koh MB, Lim Z, Pagliuca A, Mufti GJ, et al. Retrospective study of Alemtuzumab versus ATG based conditioning without irradiation for unrelated and matched sibling donor transplants in acquired severe aplastic anaemia: a study from the British Society for Blood and Marrow Transplantation (BSBMT). Bone Marrow Transplant. 2014;49:42–48.

    Article  CAS  PubMed  Google Scholar 

  124. Bacigalupo A, Marsh JC. Unrelated donor search and unrelated donor transplantation in the adult aplastic anaemia patient aged 18-40 years without an HLA-identical sibling and failing immunosuppression. Bone Marrow Transplant. 2013;48:198–200.

    Article  CAS  PubMed  Google Scholar 

  125. de Latour RP. Transplantation for bone marrow failure: current issues. Hematology Am Soc Hematol Educ Program. 2016;2016:90–8.

  126. Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012;367:11–19.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  127. Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, et al. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014;123:1818–25.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  128. Lengline E, Drenou B, Peterlin P, Tournilhac O, Abraham J, Berceanu A, et al. Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia. Haematologica. 2018;103:212–20.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  129. DeZern AE, Zahurak M, Symons H, Cooke K, Jones RJ, Brodsky RA. Alternative donor transplantation with high-dose post-transplantation cyclophosphamide for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2017;23:498–504.

    Article  CAS  PubMed  Google Scholar 

  130. Peffault de Latour R, Purtill D, Ruggeri A, Sanz G, Michel G, Gandemer V, et al. Influence of nucleated cell dose on overall survival of unrelated cord blood transplantation for patients with severe acquired aplastic anaemia. A study by Eurocord and the Aplastic Anaemia Working Party of the European Group for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2010;1:78–85.

    Google Scholar 

  131. De Latour RP, Rocha V, Socie G. Cord blood transplantation in aplastic anaemia. Bone Marrow Transplant. 2013;48:201–2.

    Article  Google Scholar 

  132. Ciceri F, Lupo-Stanghellini MT, Korthof ET. Haploidentical transplantation in patients with acquired aplastic anaemia. Bone Marrow Transplant. 2013;48:183–5.

    Article  CAS  PubMed  Google Scholar 

  133. Clay J, Kulasekarara AG, Potter V, Grimaldi F, McLornan D, Raj K, et al. Non-myeloablative peripheral blood haploidentical stem cell transplantation for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2014;20:1711–6.

    Article  PubMed  Google Scholar 

  134. Pagliuca S, Peffault de Latour R, Volt F, Locatelli F, Zecca M, Dalle JH, et al. Long-term outcomes of cord blood transplantation from an HLA-identical sibling for patients with bone marrow failure syndromes: a Report from Eurocord, Cord Blood Committee and Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2017;23:1939–48.

    Article  PubMed  Google Scholar 

  135. Bacigalupo A. Alternative donor transplants for severe aplastic anemia. Hematol Am Soc Hematol Educ Program. 2018;2018:467–73.

    Article  Google Scholar 

  136. Peffault de Latour R, Schrezenmeier H, Bacigalupo A, Blaise D, de Souza CA, Vigouroux S, et al. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica. 2012;97:1666–73.

    Article  PubMed  Google Scholar 

  137. Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124:2804–11.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  138. Medeiros C, Zaris-Neto J, Pasquini R. Bone marrow transplantation for patients with Fanconi anaemia: reduced doses of cyclophosphamide without irradiation as conditioning. Bone Marrow Transplant. 1999;24:849–52.

    Article  CAS  PubMed  Google Scholar 

  139. De Latour RP, Porcher R, Dalle J-H, Aljurf M, Korthof ET, Svahn J, et al. Allogeneic haemopoietic stem cell transplantation in Fanconi anaemia: the European Group for Blood and Bone Marrow Transplantation experience. Blood. 2013;122:4279–86.

    Article  CAS  Google Scholar 

  140. Mehta PA, Davies SM, Leemhuis T, Myers K, Kernan NA, Prockop SE, et al. Radiation-free, alternative-donor HCT for Fanconi anemia patients: results from a prospective multi-institutional study. Blood. 2017;129:2308–15.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  141. Fioredda F, Iacobelli S, Korthof ET, Knol C, van Biezen A, Bresters D, et al. Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita. Br J Haematol. 2018;183:110–8.

    Article  CAS  PubMed  Google Scholar 

  142. Gadalla SM, Sales-Bonfim C, Carreras J, Alter BP, Antin JH, Ayas M, et al. Outcomes of allogeneic hematopoietic cell transplantation in patients with dyskeratosis congenital. Biol Blood Marrow Transplant. 2013;19:1238–43.

    Article  PubMed  PubMed Central  Google Scholar 

  143. Ayas M, Nassar A, Hamidieh AA, Kharfan-Dabaja M, Othman TB, Elhaddad A, et al. Reduced intensity conditioning is effective for hematopoietic SCT in dyskeratosis congenita-related BM failure. Bone Marrow Transplant. 2013;48:1168–72.

    Article  CAS  PubMed  Google Scholar 

  144. Barkholt L, Bregni M, Remberger M, Blaise D, Peccatori J, Massenkeil G, et al. Allogeneic haematopoietic stem cell transplantation for metastatic renal carcinoma in Europe. Ann Oncol. 2006;17:1134–40.

    Article  CAS  PubMed  Google Scholar 

  145. Carnevale-Schianca F, Cignetti A, Capaldi A, Vitaggio K, Vallario A, Ricchiardi A, et al. Allogeneic nonmyeloablative hematopoietic cell transplantation in metastatic colon cancer: tumor-specific T cells directed to a tumor-associated antigen are generated in vivo during GVHD. Blood. 2006;107:3795–803.

    Article  CAS  PubMed  Google Scholar 

  146. Dudley ME, Yang JC, Sherry R, Hughes MS, Royal R, Kammula U, et al. Adoptive cell therapy for patients with metastatic melanoma: evaluation of intensive mieloablative chemoradiation preparative regimens. J Clin Oncol. 2008;26:5233–9.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  147. Robbins PF, Morgan RA, Feldman SA, Yang JC, Sherry RM. Dudley ME, et al. Tumor regression in patients with metastatic synovial cell sarcoma and melanoma using genetically engineered lymphocytes reactive with NY-ESO-1. J Clin Oncol. 2011;29:917–24.

    Article  PubMed  PubMed Central  Google Scholar 

  148. Comoli P, Pedrazzoli P, Maccario R, Basso S, Carminati O, Labirio M, et al. Cell therapy of stage IV nasopharyngeal carcinoma with autologous EBV-targeted cytotoxic T-lymphocytes. J Clin Oncol. 2005;23:8942–9.

    Article  CAS  PubMed  Google Scholar 

  149. Demirer T, Barkholt L, Blaise D, Pedrazzoli P, Aglietta M, Carella AM, et al. Transplantation of allogeneic hematopoietic stem cells: an emerging treatment modality for solid tumors. Nat Clin Pract Oncol. 2008;5:256–67.

    Article  CAS  PubMed  Google Scholar 

  150. Bregni M, Badoglio M, Pedrazzoli P, Lanza F. Is allogeneic transplant for solid tumors still alive? Bone Marrow Transplant. 2016;51:751–2.

    Article  CAS  PubMed  Google Scholar 

  151. Martino M, Bottini A, Rosti G, Generali D, Secondino S, Barni S, et al. Critical issues on high-dose chemotherapy with autologous hematopoietic progenitor cell transplantation in breast cancer patients. Expert Opin Biol Ther. 2012;12:1505–15.

    Article  CAS  PubMed  Google Scholar 

  152. Berry DA, Ueno NT, Johnson MM, Lei X, Caputo J, Rodenhuis S, et al. High dose chemotherapy with autologous stem cell support versus standard-dose chemotherapy: overview of individual patient data from 15 randomized adjuvant therapy breast cancer trials. J Clin Oncol. 2011;29:3214–23.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  153. Pedrazzoli P, Martino M, Delfanti S, Generali D, Bregni M, Lanza F, et al. High-dose chemotherapy with autologous hematopoietic stem cell transplantation in high-risk breast cancer patients. J Nat Cancer Inst. 2015;51:70–75.

    Article  CAS  Google Scholar 

  154. Boudin L, Gonçalves A, Sabatier R, Moretta J, Sfumato P, Asseeva P, et al. Highly favorable outcome in BRCA-mutated metastatic breast cancer patients receiving high-dose chemotherapy and autologous hematopoietic stem cell transplantation. Bone Marrow Transplant. 2016;51:1082–6.

    Article  CAS  PubMed  Google Scholar 

  155. Boudin L, Chabannon C, Sfumato P, Sabatier R, Bertucci F, Tarpin C, et al. Immunohistochemical subtypes predict survival in metastatic breast cancer receiving high-dose chemotherapy with autologous haematopoietic stem cell transplantation. Eur J Cancer. 2016;57:118–26.

    Article  CAS  PubMed  Google Scholar 

  156. Necchi A, Miceli R, Bregni M, Bokemeyer C, Berger LA, Oechsle K, et al. Prognostic impact of progression to induction chemotherapy and prior paclitaxel therapy in patients with germ cell tumors receiving salvage high-dose chemotherapy in the last 10 years: a study of the European Society for Blood and Marrow Transplantation Solid Tumors Working Party. Bone Marrow Transplant. 2016;51:384–90.

    Article  CAS  PubMed  Google Scholar 

  157. Einhorn LH, Williams SD, Chamness A, Brames MJ, Perkins SM, Abonour R. High-dose chemotherapy and stem-cell rescue for metastatic germ-cell tumors. N Engl J Med. 2007;357:340–8.

    Article  CAS  PubMed  Google Scholar 

  158. Beyer J, Albers P, Altena R, Aparicio J, Bokemeyer C, Busch J, et al. Maintaining success, reducing treatment burden, focusing on survivorship: highlights from the third European consensus conference on diagnosis and treatment of germ-cell cancer. Ann Oncol. 2013;24:878–88.

    Article  CAS  PubMed  Google Scholar 

  159. Necchi A, Lanza F, Rosti G, Martino M, Farè E, Pedrazzoli P, European Society for Blood and Marrow Transplantation, Solid Tumors Working Party (EBMT-STWP) and the Italian Germ Cell Cancer Group (IGG). High-dose chemotherapy for germ cell tumors: do we have a model? Expert Opin Biol Ther. 2015;15:33–44.

    Article  CAS  PubMed  Google Scholar 

  160. Lorch A, Bascoul-Mollevi C, Kramar A, Einhorn L, Necchi A, Massard C, et al. Conventional-dose versus high-dose chemotherapy as first salvage treatment in male patients with metastatic germ cell tumors: evidence from a large international database. J Clin Oncol. 2011;29:2178–84.

    Article  PubMed  Google Scholar 

  161. Ladenstein R, Pötschger U, Le Deley MC, Whelan J, Paulussen M, Oberlin O, et al. Primary disseminated multifocal Ewing sarcoma: results of the Euro-EWING 99 trial. J Clin Oncol. 2010;28:3284–91.

    Article  CAS  PubMed  Google Scholar 

  162. Spreafico F, Massimino M, Gandola L, Cefalo G, Mazza E, Landonio G, et al. Survival of adults treated for medulloblastoma using paediatric protocols. Eur J Cancer. 2005;41:1304–10.

    Article  PubMed  Google Scholar 

  163. Kelsey PJ, Oliveira MC, Badoglio M, Sharrack B, Farge D, Snowden JA. Haematopoietic stem cell transplantation in autoimmune diseases: from basic science to clinical practice. Curr Res Transl Med. 2016;64:71–82.

    Article  CAS  PubMed  Google Scholar 

  164. Snowden JA, Badoglio M, Labopin M, Giebel S, McGrath E, Marjanovic Z, et al. Evolution, trends, outcomes, and economics of hematopoietic stem cell transplantation in severe autoimmune diseases. Blood Adv. 2017;1:2742–55.

    Article  PubMed  PubMed Central  Google Scholar 

  165. Alexander T, Farge D, Badoglio M, Lindsay JO, Muraro PA, Snowden JA. Hematopoietic stem cell therapy for autoimmune diseases - clinical experience and mechanisms. J Autoimmun. 2018;92:35–46.

    Article  CAS  PubMed  Google Scholar 

  166. Snowden JA, Saccardi R, Allez M, Ardizzone S, Arnold R, Cervera R, et al. Haematopoietic SCT in severe autoimmune diseases: updated guidelines of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 2012;47:770–90.

    Article  CAS  PubMed  Google Scholar 

  167. Mancardi GL, Sormani MP, Gualandi F, Saiz A, Carreras E, Merelli E, et al. Autologous hematopoietic stem cell transplantation in multiple sclerosis: a phase II trial. Neurology. 2015;84:981–8.

    Article  CAS  PubMed  Google Scholar 

  168. Muraro PA, Pasquini M, Atkins HL, Bowen JD, Farge D, Fassas A, et al. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurol. 2017;74:459–69.

    Article  PubMed  PubMed Central  Google Scholar 

  169. Mancardi G, Sormani MP, Muraro PA, Boffa G, Saccardi R. Intense immunosuppression followed by autologous haematopoietic stem cell transplantation as a therapeutic strategy in aggressive forms of multiple sclerosis. Mult Scler. 2018;24:245–55.

    Article  PubMed  Google Scholar 

  170. Das J, Snowden J, Burman J, Freedman M, Atkins H, Bowman M, et al. The use of autologous haematopoietic stem cell transplantation as a first line disease modifying therapy in patients with ‘aggressive' multiple sclerosis. Mult Scler. 2018;24:87–88.

    Google Scholar 

  171. Burt RK, Balabanov R, Burman J, Sharrack B, Snowden JA, Oliveira MC, et al. Effect of nonmyeloablative hematopoietic stem cell transplantation vs continued disease-modifying therapy on disease progression in patients with relapsing-remitting multiple sclerosis: a randomized clinical trial. J Am Med Assoc. 2019;321:165–74.

    Article  Google Scholar 

  172. Burt RK, Shah SJ, Dill K, Grant T, Gheorghiade M, Schroeder J, et al. Autologous non-myeloablative haemopoietic stem-cell transplantation compared with pulse cyclophosphamide once per month for systemic sclerosis (ASSIST): an open-label, randomised phase 2 trial. Lancet. 2011;378:498–506.

    Article  CAS  PubMed  Google Scholar 

  173. Burt RK, Oliveira MC, Shah SJ, Moraes DA, Simoes B, Gheorghiade M, et al. Cardiac involvement and treatment-related mortality after non-myeloablative haemopoietic stem-cell transplantation with unselected autologous peripheral blood for patients with systemic sclerosis: a retrospective analysis. Lancet. 2013;381:1116–24.

    Article  PubMed  Google Scholar 

  174. Van Laar J, Farge D, Sont JK, Naraghi K, Marjanovic Z, Larghero J, et al. Autologous hematopoietic stem cell transplantation versus intravenous pulse cyclophosphamide in diffuse cutaneous systemic sclerosis: a randomized clinical trial. J Am Med Assoc. 2014;311:2490–8.

    Article  CAS  Google Scholar 

  175. Sullivan KM, Goldmuntz EA, Keyes-Elstein L, McSweeney PA, Pinckney A, Welch B, et al. Myeloablative autologous stem-cell transplantation for severe scleroderma. N Engl J Med. 2018;378:35–47.

    Article  PubMed  PubMed Central  Google Scholar 

  176. Hawkey CJ, Allez M, Clark MM, Labopin M, Lindsay JO, Ricart E, et al. Autologous hematopoetic stem cell transplantation for refractory Crohn disease: a randomized clinical trial. J Am Med Assoc. 2015;314:2524–34.

    Article  CAS  Google Scholar 

  177. Lindsay JO, Allez M, Clark M, Labopin M, Ricart E, Rogler G, et al. Autologous stem-cell transplantation in treatment-refractory Crohn’s disease: an analysis of pooled data from the ASTIC trial. Lancet Gastroenterol Hepatol. 2017;2:399–406.

    Article  PubMed  Google Scholar 

  178. Brierley CK, Castilla-Llorente C, Labopin M, Badoglio M, Rovira M, Ricart E, et al. Autologous haematopoietic stem cell transplantation for Crohn's disease: a retrospective survey of long-term outcomes from the European Society for Blood and Marrow Transplantation. J Crohns Colitis. 2018. https://doi.org/10.1093/ecco-jcc/jjy069.

  179. Burt RK, Han X, Gozdziak P, Yaung K, Morgan A, Clendenan AM, et al. Five year follow-up after autologous peripheral blood hematopoietic stem cell transplantation for refractory, chronic, corticosteroid-dependent systemic lupus erythematosus: effect of conditioning regimen on outcome. Bone Marrow Transplant. 2018;53:692–700.

    Article  CAS  PubMed  Google Scholar 

  180. Farge D, Burt RK, Oliviera M-C, Mousseaux E, Rovira M, Marjanovic Z, et al. Cardiopulmonary assessment of patients with systemic sclerosis for haematopoietic stem cell transplantation (HSCT): recommendations from the European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP) and collaborating partners. Bone Marrow Transplant. 2017;52:1495–503.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  181. Kowal-Bielecka O, Fransen J, Avouac J, Becker M, Kulak A, Allanore Y, et al. Update of EULAR recommendations for the treatment of systemic sclerosis. Ann Rheum Dis. 2017;76:1327–39.

    Article  PubMed  Google Scholar 

  182. Snowden JA, Panés J, Alexander T, Allez M, Ardizzone S, Dierickx D, et al. Autologous haematopoietic stem cell transplantation (AHSCT) in severe Crohn's disease: a review on behalf of ECCO and EBMT. J Crohns Colitis. 2018;12:476–88.

    Article  PubMed  Google Scholar 

  183. Sullivan KM, Majhail NS, Bredeson C, Carpenter PA, Chatterjee S, Crofford LJ, et al. Systemic sclerosis as an indication for autologous hematopoietic cell transplantation: Position Statement from the American Society for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2018;24:1961–4.

    Article  PubMed  PubMed Central  Google Scholar 

  184. Rabusin M, Snowden JA, Veys P, Quartier P, Dalle JH, Dhooge C, et al. Long term outcomes of hematopoietic stem cell transplantation (HSCT) for severe treatment resistant autoimmune cytopenia in children. Biol Blood Marrow Transplant. 2013;19:666–9.

    Article  PubMed  Google Scholar 

  185. Silva J, Ladomenou F, Carpenter B, Chandra S, Sedlacek P, Formankova R, et al. Allogeneic hematopoietic stem cell transplantation for severe, refractory juvenile idiopathic arthritis. Blood Adv. 2018;2:777–86.

    Article  CAS  Google Scholar 

  186. Niewerth D, Creutzig U, Bierings MB, Kaspers GJ. A review on allogeneic stem cell transplantation for newly diagnosed pediatric acute myeloid leukemia. Blood. 2010;116:2205–14.

    Article  CAS  PubMed  Google Scholar 

  187. Burke MJ, Wagner JE, Cao Q, Ustun C, Verneris MR. Allogeneic hematopoietic cell transplantation in first remission abrogates poor outcomes associated with high-risk pediatric acute myeloid leukemia. Biol Blood Marrow Transplant. 2013;19:1021–5.

    Article  PubMed  PubMed Central  Google Scholar 

  188. Klusmann JH, Reinhardt D, Zimmermann M, Kremens B, Vormoor J, Dworzak M, et al. The role of matched sibling donor allogeneic stem cell transplantation in pediatric high-risk acute myeloid leukemia: results from the AML-BFM 98 study. Haematologica. 2012;97:21–29.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  189. Creutzig U, Zimmermann M, Bourquin JP, Dworzak MN, Kremens B, Lehrnbecher T, et al. Favorable outcome in infants with AML after intensive first- and second-line treatment: an AML-BFM study group report. Leukemia. 2012;26:654–61.

    Article  CAS  PubMed  Google Scholar 

  190. Marks D, Khattry N, Cummins M, Goulden N, Green A, Harvey J, et al. Haploidentical stem cell transplantation for children with acute leukaemia. Br J Haematol. 2006;134:196–201.

    Article  PubMed  Google Scholar 

  191. Locatelli F, Pende D, Maccario R, Mingari MC, Moretta A, Moretta L. Haploidentical hemopoietic stem cell transplantation for the treatment of high-risk leukemias: how NK cells make the difference. Clin Immunol. 2009;133:171–8.

    Article  CAS  PubMed  Google Scholar 

  192. Hasle H. A critical review of which children with acute myeloid leukaemia need stem cell procedures. Br J Haematol. 2014;166:23–33.

    Article  PubMed  Google Scholar 

  193. Mann G, Attarbaschi A, Schrappe M, De Lorenzo P, Peters C, Hann I, et al. Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study. Blood. 2010;116:2644–50.

    Article  CAS  PubMed  Google Scholar 

  194. von Stackelberg A, Volzke E, Kuhl JS, Seeger K, Schrauder A, Escherich G, et al. Outcome of children and adolescents with relapsed acute lymphoblastic leukaemia and non-response to salvage protocol therapy: a retrospective analysis of the ALL-REZ BFM Study Group. Eur J Cancer. 2011;47:90–97.

    Article  Google Scholar 

  195. Peters C, Cornish JM, Parikh SH, Kurtzberg J. Stem cell source and outcome after hematopoietic stem cell transplantation (HSCT) in children and adolescents with acute leukemia. Pedia Clin North Am. 2010;57:27–46.

    Article  Google Scholar 

  196. Schrauder A, von Stackelberg A, Schrappe M, Cornish J, Peters C. Allogeneic hematopoietic SCT in children with ALL: current concepts of ongoing prospective SCT trials. Bone Marrow Transplant. 2008;41(Suppl 2):S71–74.

    Article  PubMed  Google Scholar 

  197. Peters C, Schrappe M, von Stackelberg A, Schrauder A, Bader P, Ebell W, et al. Stem-cell transplantation in children with acute lymphoblastic leukemia: a prospective international multicenter trial comparing sibling donors with matched unrelated donors-The ALL-SCT-BFM-2003 trial. J Clin Oncol. 2015;33:1265–74.

    Article  CAS  PubMed  Google Scholar 

  198. Pulsipher MA, Peters C, Pui CH. High-risk pediatric acute lymphoblastic leukemia: to transplant or not to transplant? Biol Blood Marrow Transplant. 2011;17(1 Suppl):S137–148.

    Article  PubMed  PubMed Central  Google Scholar 

  199. Bader P, Kreyenberg H, Henze GH, Eckert C, Reising M, Willasch A, et al. Prognostic value of minimal residual disease quantification before allogeneic stem-cell transplantation in relapsed childhood acute lymphoblastic leukemia: the ALL-REZ BFM Study Group. J Clin Oncol. 2009;27:377–84.

    Article  PubMed  Google Scholar 

  200. Eckert C, Henze G, Seeger K, Hagedorn N, Mann G, Panzer-Grumayer R, et al. Use of allogeneic hematopoietic stem-cell transplantation based on minimal residual disease response improves outcomes for children with relapsed acute lymphoblastic leukemia in the intermediate-risk group. J Clin Oncol. 2013;31:2736–42.

    Article  PubMed  Google Scholar 

  201. Bader P, Kreyenberg H, von Stackelberg A, Eckert C, Salzmann-Manrique E, Meisel R, et al. Monitoring of minimal residual disease after allogeneic stem-cell transplantation in relapsed childhood acute lymphoblastic leukemia allows for the identification of impending relapse: results of the ALL-BFM-SCT 2003 trial. J Clin Oncol. 2015;33:1275–84.

    Article  CAS  PubMed  Google Scholar 

  202. Beck JC, Cao Q, Trotz B, Smith AR, Weigel BJ, Verneris MR, et al. Allogeneic hematopoietic cell transplantation outcomes for children with B-precursor acute lymphoblastic leukemia and early or late BM relapse. Bone Marrow Transplant. 2011;46:950–5.

    Article  CAS  PubMed  Google Scholar 

  203. Meisel R, Klingebiel T, Dilloo D. German/Austrian Pediatric Registry for Stem Cell Transplantation. Peripheral blood stem cells versus bone marrow in pediatric unrelated donor stem cell transplantation. Blood. 2013;121:863–5.

    Article  CAS  PubMed  Google Scholar 

  204. Suttorp M, Eckardt L, Tauer JT, Millot F. Management of chronic myeloid leukemia in childhood. Curr Hematol Malig Rep. 2012;7:116–24.

    Article  PubMed  Google Scholar 

  205. Suttorp M, Yaniv I, Schultz KR. Controversies in the treatment of CML in children and adolescents: TKIs versus BMT? Biol Blood Marrow Transplant. 2011;17(1 Suppl):S115–122.

    Article  PubMed  Google Scholar 

  206. de la Fuente J, Baruchel A, Biondi A, de Bont E, Dresse MF, Suttorp M, Millot F. Managing children with chronic myeloid leukaemia (CML): recommendations for the management of CML in children and young people up to the age of 18 years. Br J Haematol. 2014;167:33–47.

    Article  PubMed  Google Scholar 

  207. Jaeger BA, Tauer JT, Ulmer A, Kuhlisch E, Roth HJ, Suttorp M. Changes in bone metabolic parameters in children with chronic myeloid leukemia on imatinib treatment. Med Sci Monit. 2012;18:721–8.

    Article  Google Scholar 

  208. Ulmer A, Tabea Tauer J, Glauche I, Jung R, Suttorp M. TK inhibitor treatment disrupts growth hormone axis: clinical observations in children with CML and experimental data from a juvenile animal model. Klin Padiatr. 2013;225:120–6.

    Article  CAS  PubMed  Google Scholar 

  209. Suttorp M, Claviez A, Bader P, Peters C, Gadner H, Ebell W, et al. Allogeneic stem cell transplantation for pediatric and adolescent patients with CML: results from the prospective trial CML-paed I. Klin Padiatr. 2009;221:351–7.

    Article  CAS  PubMed  Google Scholar 

  210. Millot F, Claviez A, Leverger G, Corbaciglu S, Groll AH, Suttorp M. Imatinib cessation in children and adolescents with chronic myeloid leukemia in chronic phase. Pediatr Blood Cancer. 2014;61:355–7.

    Article  PubMed  Google Scholar 

  211. Locatelli F, Crotta A, Ruggeri A, Eapen M, Wagner JE, Macmillan ML, et al. Analysis of risk factors influencing outcomes after cord blood transplantation in children with juvenile myelomonocytic leukemia: a EUROCORD, EBMT, EWOG-MDS, CIBMTR study. Blood. 2013;122:2135–41.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  212. Madureira AB, Eapen M, Locatelli F, Teira P, Zhang MJ, Davies SM, et al. Analysis of risk factors influencing outcome in children with myelodysplastic syndrome after unrelated cord blood transplantation. Leukemia. 2011;25:449–54.

    Article  CAS  PubMed  Google Scholar 

  213. Strahm B, Nollke P, Zecca M, Korthof ET, Bierings M, Furlan I, et al. Hematopoietic stem cell transplantation for advanced myelodysplastic syndrome in children: results of the EWOG-MDS 98 study. Leukemia. 2011;25:455–62.

    Article  CAS  PubMed  Google Scholar 

  214. Burkhardt B, Reiter A, Landmann E, Lang P, Lassay L, Dickerhoff R, et al. Poor outcome for children and adolescents with progressive disease or relapse of lymphoblastic lymphoma: a report from the Berlin-Frankfurt-Muenster Group. J Clin Oncol. 2009;27:3363–9.

    Article  PubMed  Google Scholar 

  215. Gross TG, Hale GA, He W, Camitta BM, Sanders JE, Cairo MS, et al. Hematopoietic stem cell transplantation for refractory or recurrent non-Hodgkin lymphoma in children and adolescents. Biol Blood Marrow Transplant. 2010;16:223–30.

    Article  PubMed  Google Scholar 

  216. Woessmann W, Peters C, Lenhard M, Burkhardt B, Sykora KW, Dilloo D, et al. Allogeneic haematopoietic stem cell transplantation in relapsed or refractory anaplastic large cell lymphoma of children and adolescents--a Berlin-Frankfurt-Munster Group report. Br J Haematol. 2006;133:176–82.

    Article  PubMed  Google Scholar 

  217. Kelly KM. Hodgkin lymphoma in children and adolescents: improving the therapeutic index. Blood. 2015;126:2452–8.

    Article  CAS  PubMed  Google Scholar 

  218. Kahn JM, Kelly KM. Adolescent and young adult Hodgkin lymphoma: raising the bar through collaborative science and multidisciplinary care. Pediatr Blood Cancer. 2018;65:e27033.

    Article  PubMed  PubMed Central  Google Scholar 

  219. Fox TA, Chakraverty R, Burns S, Carpenter B, Thomson K, Lowe D, et al. Successful outcome following allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency. Blood. 2018;131:917–31.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  220. Albert MH, Hauck F, Wiebking V, Aydin S, Notheis G, Koletzko S, et al. Allogeneic stem cell transplantation in adolescents and young adults with primary immunodeficiencies. J Allergy Clin Immunol Pract. 2018;6:298.e2–301.e2.

    Article  Google Scholar 

  221. Brown L, Xu-Bayford J, Allwood Z, Slatter M, Cant A, Davies EG, et al. Neonatal diagnosis of Severe combined immunodeficiency leads to significantly improved survival outcome: the case for newborn screening. Blood. 2011;117:3243–6.

    Article  CAS  PubMed  Google Scholar 

  222. Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, et al. Transplantation outcomes for severe combined immunodeficiency, 2000-9. N Engl J Med. 2014;371:434–46.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  223. Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE, et al. SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. Blood. 2018;132:1737–49.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  224. Gennery AR, Slatter MA, Grandin L, Taupin P, Cant AJ, Veys P, et al. Transplantation of haematopoietic stem cells and long term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? J Allergy Clin Immunol. 2010;126:602–10.

    Article  PubMed  Google Scholar 

  225. Cicalese MP, Ferrua F, Castagnaro L, Pajno R, Barzaghi F, Giannelli S, et al. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency. Blood. 2016;128:45–54.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  226. Cicalese MP, Ferrua F, Castagnaro L, Rolfe K, De Boever E, Reinhardt RR, et al. Gene therapy for adenosine deaminase deficiency: a comprehensive evaluation of short- and medium-term safety. Mol Ther. 2018;26:917–31.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  227. Ferrua F, Galimberti S, Courteille V, Slatter MA, Booth C, Moshous D, et al. Hematopoietic stem cell transplantation for CD40 ligand deficiency: results from an EBMT/ESID-IEWP-SCETIDE-PIDTC Study. J Allergy Clin Immunol. 2019. https://doi.org/10.1016/j.jaci.2018.12.1010.

  228. Moratto D, Giliani S, Bonfim C, Mazzolari E, Fischer A, Ochs HD, et al. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study. Blood. 2011;118:1675–84.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  229. Ngwube A, Hanson IC, Orange J, Rider NL, Seeborg F, Shearer W, et al. Outcomes after allogeneic transplant in patients with Wiskott-Aldrich syndrome. Biol Blood Marrow Transplant. 2018;24:537–41.

    Article  PubMed  Google Scholar 

  230. Güngör T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet. 2014;383:436–48.

    Article  CAS  PubMed  Google Scholar 

  231. Connelly JA, Marsh R, Parikh S, Talano JA. Allogeneic hematopoietic cell transplantation for chronic granulomatous disease: controversies and state of the art. J Pediatr Infect Dis Soc. 2018;7(suppl_1):S31–S39.

    Article  Google Scholar 

  232. Hacein-Bey Abina S, Gaspar HB, Blondeau J, Caccavelli L, Charrier S, Buckland K, et al. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. J Am Med Assoc. 2015;313:1550–63.

    Article  CAS  Google Scholar 

  233. Boelens JJ, Orchard PJ, Wynn RF. Transplantation in inborn errors of metabolism: current considerations and future perspectives. Br J Haematol. 2014;167:293–303.

    Article  PubMed  Google Scholar 

  234. Aldenhoven M, Jones SA, Bonney D, Borrill RE, Coussons M, Mercer J, et al. Hematopoietic cell transplantation for mucopolysaccharidosis patients is safe and effective: results after implementation of international guidelines. Biol Blood Marrow Transplant. 2015;21:1106–9.

    Article  CAS  PubMed  Google Scholar 

  235. Aldenhoven M, van den Broek BTA, Wynn RF, O'Meara A, Veys P, Rovelli A, et al. Quality of life of Hurler syndrome patients after successful hematopoietic stem cell transplantation. Blood Adv. 2017;1:2236–42.

    Article  PubMed  PubMed Central  Google Scholar 

  236. Lum SH, Miller WP, Jones S, Poulton K, Ogden W, Lee H, et al. Changes in the incidence, patterns and outcomes of graft failure following hematopoietic stem cell transplantation for Hurler syndrome. Bone Marrow Transplant. 2017;52:846–53.

    Article  CAS  PubMed  Google Scholar 

  237. Sessa M, Lorioli L, Fumagalli F, Acquati S, Redaelli D, Baldoli C, et al. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial. Lancet. 2016;388:476–87.

    Article  CAS  PubMed  Google Scholar 

  238. Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, et al. Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy. N Engl J Med. 2017;377:1630–8.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  239. Locatelli F, Kabbara N, Ruggeri A, Ghavamzadeh A, Roberts I, Li CK, et al. Outcome of patients with hemoglobinopathies given either cord blood or bone marrow transplantation from an HLA-identical sibling. Blood. 2013;122:1072–8.

    Article  CAS  PubMed  Google Scholar 

  240. Gaziev J, Marziali M, Isgro A, Sodani P, Paciaroni K, Gallucci C, et al. Bone marrow transplantation for thalassemia from alternative related donors: improved outcomes with a new approach. Blood. 2013;122:2751–6.

    Article  CAS  PubMed  Google Scholar 

  241. Lucarelli G, Isgro A, Sodani P, Gaziev J. Hematopoietic stem cell transplantation in thalassemia and sickle cell anemia. Cold Spring Harb Perspect Med. 2012;2:a011825.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  242. Galambrun C, Pondarre C, Bertrand Y, Loundou A, Bordigoni P, Frange P, et al. French multicenter 22 year-experience of stem cell transplantation for beta-thalassemia major: lessons and future directions. Biol Blood Marrow Transplant. 2013;19:62–68.

    Article  PubMed  Google Scholar 

  243. Angelucci E, Baronciani D. Allogeneic stem cell transplantation for thalassemia major. Haematologica. 2008;93:1780–4.

    Article  PubMed  Google Scholar 

  244. Fitzhugh CD, Hsieh MM, Taylor T, Coles W, Roskom K, Wilson D, et al. Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT. Blood Adv. 2017;1:652–61.

    Article  PubMed  PubMed Central  Google Scholar 

  245. Gaziev J, Isgrò A, Sodani P, Paciaroni K, De Angelis G, Marziali M, et al. Haploidentical HSCT for hemoglobinopathies: improved outcomes with TCRαβ+/CD19+-depleted grafts. Blood Adv. 2018;2:263–70.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  246. Sun Q, Wu B, Lan H, Meng F, Ma X, Chen X, et al. Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen. Br J Haematol. 2018;182:554–8.

    Article  PubMed  PubMed Central  Google Scholar 

  247. Anurathapan U, Hongeng S, Pakakasama S, Sirachainan N, Songdej D, Chuansumrit A, et al. Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors. Bone Marrow Transplant. 2016;51:813–8.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  248. Matthes-Martin S, Lawitschka A, Fritsch G, Lion T, Grimm B, Breuer S, et al. Stem cell transplantation after reduced-intensity conditioning for sickle cell disease. Eur J Haematol. 2013;90:308–12.

    Article  PubMed  Google Scholar 

  249. Lucarelli G, Gaziev J, Isgro A, Sodani P, Paciaroni K, Alfieri C, et al. Allogeneic cellular gene therapy in hemoglobinopathies--evaluation of hematopoietic SCT in sickle cell anemia. Bone Marrow Transplant. 2012;47:227–30.

    Article  CAS  PubMed  Google Scholar 

  250. Kamani NR, Walters MC, Carter S, Aquino V, Brochstein JA, Chaudhury S, et al. Unrelated donor cord blood transplantation for children with severe sickle cell disease: results of one cohort from the phase II study from the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). Biol Blood Marrow Transplant. 2012;18:1265–72.

    Article  PubMed  PubMed Central  Google Scholar 

  251. Foell J, Pfirstinger B, Rehe K, Wolff D, Holler E, Corbacioglu S. Haploidentical stem cell transplantation with CD3+-/CD19+- depleted peripheral stem cells for patients with advanced stage sickle cell disease and no alternative donor: results of a pilot study. Bone Marrow Transplant. 2017;52:938–40.

    Article  CAS  PubMed  Google Scholar 

  252. de la Fuente J, Dhedin N, Koyama T, Bernaudin F, Kuentz M, Karnik L, et al. Haploidentical bone marrow transplantation with post-transplantation cyclophosphamide plus thiotepa improves donor engraftment in patients with sickle cell anemia: results of an International Learning Collaborative. Biol Blood Marrow Transplant. 2018. https://doi.org/10.1016/j.bbmt.2018.11.027.

  253. Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, et al. Gene therapy in patients with transfusion-dependent β-thalassemia. N Engl J Med. 2018;378:1479–93.

    Article  CAS  PubMed  Google Scholar 

  254. Orchard PJ, Fasth AL, Le Rademacher J, He W, Boelens JJ, Horwitz EM, et al. Hematopoietic stem cell transplantation for infantile osteopetrosis. Blood. 2015;126:270–6.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  255. Hashemi Taheri AP, Radmard AR, Kooraki S, Behfar M, Pak N, Hamidieh AA, et al. Radiologic resolution of malignant infantile osteopetrosis skeletal changes following hematopoietic stem cell transplantation. Pediatr Blood Cancer. 2015;62:1645–9.

    Article  PubMed  Google Scholar 

  256. Natsheh J, Drozdinsky G, Simanovsky N, Lamdan R, Erlich O, Gorelik N, et al. Improved outcomes of hematopoietic stem cell transplantation in patients with infantile malignant osteopetrosis using fludarabine-based conditioning. Pediatr Blood Cancer. 2016;63:535–40.

    Article  CAS  PubMed  Google Scholar 

  257. Chiesa R, Ruggeri A, Paviglianiti A, Zecca M, Gónzalez-Vicent M, Bordon V, et al. Outcomes after unrelated umbilical cord blood transplantation for children with osteopetrosis. Biol Blood Marrow Transplant. 2016;22:1997–2002.

    Article  PubMed  Google Scholar 

  258. Samarasinghe A, Webb DK. How I manage aplastic anaemia in children. Br J Haematol. 2012;157:26–40.

    Article  CAS  PubMed  Google Scholar 

  259. Samarasinghe S, Steward C, Hiwarkar P, Saif MA, Hough R, Webb D, et al. Excellent outcome of matched unrelated donor transplantation in paediatric aplastic anaemia following failure with immunosuppressive therapy: a United Kingdom multicentre retrospective experience. Br J Haematol. 2012;157:339–436.

    Article  CAS  PubMed  Google Scholar 

  260. Samarasinghe S, Marsh J, Dufour C. Immune suppression for childhood acquired aplastic anemia and myelodysplastic syndrome: where next? Haematologica. 2014;99:597–9.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  261. Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol. 2015;171:585–94.

    Article  CAS  PubMed  Google Scholar 

  262. Devillier R, Dalle JH, Kulasekararaj A, D’aveni M, Clément L, Chybicka A, et al. Unrelated alternative donor transplantation for severe acquired aplastic anemia: a study from the French Society of Bone Marrow Transplantation and Cell Therapies and the EBMT Severe Aplastic Anemia Working Party. Haematologica. 2016;101:884–90.

    Article  PubMed  PubMed Central  Google Scholar 

  263. Fagioli F, Quarello P, Zecca M, Lanino E, Corti P, Favre C, et al. Haematopoietic stem cell transplantation for Diamond Blackfan anaemia: a report from the Italian Association of Paediatric Haematology and Oncology Registry. Br J Haematol. 2014;165:673–81.

    Article  PubMed  Google Scholar 

  264. Ladenstein R, Potschger U, Hartman O, Pearson AD, Klingebiel T, Castel V, et al. 28 years of high-dose therapy and SCT for neuroblastoma in Europe: lessons from more than 4000 procedures. Bone Marrow Transplant. 2008;41(Suppl 2):S118–127.

    Article  CAS  PubMed  Google Scholar 

  265. Matthay KK, Reynolds CP, Seeger RC, Shimada H, Adkins ES, Haas-Kogan D, et al. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: a Children's Oncology Group Study. J Clin Oncol. 2009;27:1007–13.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  266. Burman J, Kirgizov K, Carlson K, Badoglio M, Mancardi GL, De Luca G, et al. Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2017;52:1133–7.

    Article  CAS  PubMed  Google Scholar 

  267. Snowden JA, McGrath E, Duarte RF, Saccardi R, Orchard K, Worel N, et al. JACIE accreditation for blood and marrow transplantation: past, present and future directions of an international model for healthcare quality improvement. Bone Marrow Transplant. 2017;52:1367–71.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  268. Jessop H, Farge D, Saccardi R, Alexander T, Rovira M, Sharrack B, et al. General information for patients and carers considering haematopoietic stem cell transplantation (HSCT) for severe autoimmune diseases (ADs): a Position Statement from the EBMT Autoimmune Diseases Working Party (ADWP), the EBMT Nurses Group, the EBMT Patient, Family and Donor Committee and the Joint Accreditation Committee of ISCT and EBMT (JACIE). Bone Marrow Transplant. 2019. https://doi.org/10.1038/s41409-019-0430-7.

  269. Gratwohl A, Brand R, Niederwieser D, Baldomero H, Chabannon C, Cornelissen J, et al. Introduction of a quality management system and outcome after hematopoietic stem-cell transplantation. J Clin Oncol. 2011;29:1980–6.

    Article  PubMed  Google Scholar 

  270. Gratwohl A, Brand R, McGrath E, van Biezen A, Sureda A, Ljungman P, et al. Use of the quality management system "JACIE" and outcome after hematopoietic stem cell transplantation. Haematologica. 2014;99:908–15.

    Article  PubMed  PubMed Central  Google Scholar 

  271. Gratwohl A, Sureda A, Baldomero H, Gratwohl M, Dreger P, Kröger N, et al. Macroeconomics and outcome after hematopoietic stem cell transplantation. E-Biomed. 2015;2:2101–9.

    Google Scholar 

  272. Schetelig J, de Wreede LC, Andersen NS, Moreno C, van Gelder M, Vitek A, et al. Centre characteristics and procedure-related factors have an impact on outcomes of allogeneic transplantation for patients with CLL: a retrospective analysis from the European Society for Blood and Marrow Transplantation (EBMT). Br J Haematol. 2017;178:521–33.

    Article  PubMed  Google Scholar 

  273. Anthias C, Ethell ME, Potter MN, Madrigal A, Shaw BE. The impact of improved JACIE standards on the care of related BM and PBSC donors. Bone Marrow Transplant. 2015;50:244–7.

    Article  CAS  PubMed  Google Scholar 

  274. Anthias C, O'Donnell PV, Kiefer DM, Yared J, Norkin M, Anderlini P, et al. European Group for Blood and Marrow Transplantation Centers with FACT-JACIE accreditation have significantly better compliance with related donor care standards. Biol Blood Marrow Transplant. 2016;22:514–9.

    Article  PubMed  Google Scholar 

  275. Menon S, Shirani A, Zhao Y, Oger J, Traboulsee A, Freedman MS, et al. Characterising aggressive multiple sclerosis. J Neurol Neurosurg Psychiatry. 2013;84:1192–98.

  276. Iacobelli S. Suggestions on the use of statistical methodologies in studies of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant. 2013;48:S1–37.

Download references

Acknowledgements

The authors are grateful for the advice and helpful comments received from a number of EBMT fellow specialists in HSCT including Michael Albert, Tobias Alexander, Dina Averbuch, Frederic Baron, Eli Bazarbachi, Meral Beksac, Eolia Brissot, Gesine Bug, Simone Cesaro, Yves Chalandon, Fabo Ciceri, Tomasz Czerw, Francesco Dazzi, Jordi Esteve, Katarina Fleischhauer, Laurent Garderet, Sebastian Giebel, Lidia Gil, Maria Gilleece, Norbert-Claude Gorin, Patrick Hayden, Jorg Hälter, Juan C. Hernández Boluda, Michael Hudecek, Katharina Kleinschmidt, Michelle Kenyon, Christian Koenecke, Franco Locatelli, Florent Malard, Donal McLornan, Malgorzata Mikulska, John Murray, Francesco Onida, Paolo Pedrazzoli, Olaf Penack, Zinaida Perić, Antonio Risitano, Marie Robin, Steve Robinson, Annalisa Ruggeri, Jaime Sanz, Bipin Savani, Johannes Schetelig, Christof Schied, Christoph Schmid, Hélène Schoemans, Stefan Schönland, Basil Sharrack, Roni Shouval, Alexandros Spyridonidis, Antoine Toubert, Olivier Tourniac, Alvaro Urbano-Ispizua, Luca Vago, Michel van Gelder, Bregje Verhoeven, Jurjen Versluis, Lotte Wietten and André Willasch.

Author information

Author notes

  1. These authors contributed equally: Nicolaus Kröger, Mohamad Mohty

Authors and Affiliations

  1. Hospital Universitario Puerta de Hierro Majadahonda – Universidad Autónoma de Madrid, Madrid, Spain

    Rafael F. Duarte

  2. EBMT Paris Study Office, Hopital Saint Antoine, Paris, France

    Myriam Labopin

  3. Goethe University Hospital, Frankfurt/Main, Germany

    Peter Bader

  4. Medical University of Warsaw, Warsaw, Poland

    Grzegorz W. Basak

  5. Vita-Salute San Raffaele University & Ospedale San Raffaele Scientific Institute, Milan, Italy

    Chiara Bonini

  6. Institut Paoli Calmettes & Centre d’Investigations Cliniques en Biothérapies, Marseille, France

    Christian Chabannon

  7. University of Regensburg, Regensburg, Germany

    Selim Corbacioglu

  8. Medizinische Klinik V, Universität Heidelberg, Heidelberg, Germany

    Peter Dreger

  9. Giannina Gaslini Children’s Hospital, Genoa, Italy

    Carlo Dufour

  10. Great North Children’s Hospital, Newcastle-Upon-Tyne, UK

    Andrew R. Gennery

  11. University Medical Center Utrecht, Utrecht, The Netherlands

    Jürgen Kuball

  12. Willem-Alexander Children’s Hospital, Leiden University Medical Center, Leiden, The Netherlands

    Arjan C. Lankester

  13. Romagna Transplant Network, Ravenna, Italy

    Francesco Lanza

  14. Barts Health NHS Trust, London, UK

    Silvia Montoto

  15. Chaim Sheva Medical Center, Tel-Hashomer, Israel

    Arnon Nagler

  16. Saint-Louis Hospital, Paris Diderot University, Paris, France

    Régis Peffault de Latour

  17. Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, United Kingdom

    John A. Snowden

  18. Collegium Medicum, Nicolaus Copernicus University, Bydgoszcz, Poland

    Jan Styczynski

  19. CHU de Lille, LIRIC, INSERM U995, Université de Lille, 59000, Lille, France

    Ibrahim Yakoub-Agha

  20. University Hospital Eppendorf, Hamburg, Germany

    Nicolaus Kröger

  21. Hopital Saint Antoine, Sorbonne Université, Paris, France

    Mohamad Mohty

Authors
  1. Rafael F. Duarte
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Myriam Labopin
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Peter Bader
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. Grzegorz W. Basak
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. Chiara Bonini
    View author publications

    You can also search for this author in PubMed Google Scholar

  6. Christian Chabannon
    View author publications

    You can also search for this author in PubMed Google Scholar

  7. Selim Corbacioglu
    View author publications

    You can also search for this author in PubMed Google Scholar

  8. Peter Dreger
    View author publications

    You can also search for this author in PubMed Google Scholar

  9. Carlo Dufour
    View author publications

    You can also search for this author in PubMed Google Scholar

  10. Andrew R. Gennery
    View author publications

    You can also search for this author in PubMed Google Scholar

  11. Jürgen Kuball
    View author publications

    You can also search for this author in PubMed Google Scholar

  12. Arjan C. Lankester
    View author publications

    You can also search for this author in PubMed Google Scholar

  13. Francesco Lanza
    View author publications

    You can also search for this author in PubMed Google Scholar

  14. Silvia Montoto
    View author publications

    You can also search for this author in PubMed Google Scholar

  15. Arnon Nagler
    View author publications

    You can also search for this author in PubMed Google Scholar

  16. Régis Peffault de Latour
    View author publications

    You can also search for this author in PubMed Google Scholar

  17. John A. Snowden
    View author publications

    You can also search for this author in PubMed Google Scholar

  18. Jan Styczynski
    View author publications

    You can also search for this author in PubMed Google Scholar

  19. Ibrahim Yakoub-Agha
    View author publications

    You can also search for this author in PubMed Google Scholar

  20. Nicolaus Kröger
    View author publications

    You can also search for this author in PubMed Google Scholar

  21. Mohamad Mohty
    View author publications

    You can also search for this author in PubMed Google Scholar

Consortia

for the European Society for Blood and Marrow Transplantation (EBMT)

Corresponding author

Correspondence to Rafael F. Duarte.

Ethics declarations

Conflict of interest

The authors declare that they have no conflict of interest for this manuscript.

Additional information

Publisher’s note: Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Duarte, R.F., Labopin, M., Bader, P. et al. Indications for haematopoietic stem cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2019. Bone Marrow Transplant 54, 1525–1552 (2019). https://doi.org/10.1038/s41409-019-0516-2

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41409-019-0516-2

This article is cited by

Search

Advanced search

Quick links