Abstract
Haploidentical hematopoietic cell transplantation (HCT) is widely used in high-risk acute lymphoblastic leukemia (ALL), yet optimal donor selection remains unclear. We retrospectively analyzed 933 ALL patients receiving post-transplant cyclophosphamide (PTCy)-based GVHD prophylaxis to evaluate the impact of donor characteristics on outcomes. Donors had a median age of 36 years; 38% were female, including 23% who donated to male recipients. Peripheral blood (PB) was the graft source in 69%, and CMV-seronegative donors were used in 16% of CMV-negative recipients. Multivariate analysis showed that PB grafts were associated with inferior overall survival (OS) and GVHD-free/relapse-free survival. CMV-matched seronegative pairs had reduced non-relapse mortality and improved OS. Chronic GVHD was more frequent with older donors and in male recipients of female donor grafts, the latter also increasing extensive cGVHD risk. In conclusion, donor and graft selection significantly influence outcomes after haplo-HCT with PTCy. When feasible, bone marrow should be favored over PB grafts, and CMV-seronegative donors prioritized for CMV-seronegative recipients. Older donor age and female-to-male sex mismatch were associated with increased chronic GVHD risk.
This is a preview of subscription content, access via your institution
Access options
Subscribe to this journal
Receive 12 print issues and online access
$259.00 per year
only $21.58 per issue
Buy this article
- Purchase on SpringerLink
- Instant access to the full article PDF.
USD 39.95
Prices may be subject to local taxes which are calculated during checkout


Similar content being viewed by others
Data availability
The dataset supporting the conclusions of this article is available from the ALWP of the EBMT in Paris, Saint Antoine Hospital: allainthibeault.ferhatberland-ext@aphp.fr.
References
Marcoux C, Kebriaei P. Transplant in ALL: Who, when, and how? Hematology. 2024;2024:93–101.
Giebel S. How I treat newly diagnosed acute lymphoblastic leukemia. Clin Hematol Int. 2024;6:51–61.
Bazarbachi A, Labopin M, Angelucci E, Gülbas Z, Ozdogu H, Arat M, et al. Haploidentical transplantation with post-transplantation cyclophosphamide for T cell acute lymphoblastic leukemia: a report from the European Society for Blood and Marrow Transplantation Acute Leukemia Working Party. Biol Blood Marrow Transpl. 2020;26:936–42.
Sanz J, Galimard J-E, Labopin M, Afanasyev B, Sergeevich MI, Angelucci E, et al. Post-transplant cyclophosphamide containing regimens after matched sibling, matched unrelated and haploidentical donor transplants in patients with acute lymphoblastic leukemia in first complete remission, a comparative study of the ALWP of the EBMT. J Hematol Oncol. 2021;14:84.
Passweg JR, Baldomero H, Alexander T, Angelucci E, Averbuch D, Bazarbachi A, et al. Utilization of hematopoietic cell transplantation and cellular therapy technology in Europe and associated Countries. Using the 2022 activity survey data to correlate with economic and demographic factors. A report from the EBMT. Bone Marrow Transplant. 2025;60:227–36.
McCurdy SR, Zhang M-J, St. Martin A, Al Malki MM, Bashey A, Gaballa S, et al. Effect of donor characteristics on haploidentical transplantation with posttransplantation cyclophosphamide. Blood Adv. 2018;2:299–307.
Pruitt A, Gao F, De Togni E, Cochran H, Godbole S, Slade M, et al. Impact of donor age and relationship on outcomes of peripheral blood haploidentical hematopoietic cell transplantation. Bone Marrow Transplant. 2023;58:855–62.
Sanz J, Labopin M, Blaise D, Raiola AM, Busca A, Vydra J, et al. Haploidentical stem cell donor choice for patients with acute myeloid leukemia: a study from the ALWP of the EBMT. Blood Adv. 2024;8:2332–41.
Fuchs EJ, McCurdy SR, Solomon SR, Wang T, Herr MR, Modi D, et al. HLA informs risk predictions after haploidentical stem cell transplantation with posttransplantation cyclophosphamide. Blood. 2022;139:1452–68.
Mariotti J, Raiola AM, Evangelista A, Carella AM, Martino M, Patriarca F, et al. Impact of donor age and kinship on clinical outcomes after T-cell–replete haploidentical transplantation with PT-Cy. Blood Adv. 2020;4:3900–12.
Mehta RS, Marin D, Alousi A, Kanakry CG, Champlin RE, Rezvani K, et al. Haploidentical vs matched unrelated donors for patients with ALL: donor age matters more than donor type. Blood Adv. 2023;7:1594–603.
Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc. 1958;53:457–81.
Fine JP, Gray RJ. A proportional hazards model for the subdistribution of a competing risk. J Am Stat Assoc. 1999;94:496–509.
Gooley TA, Leisenring W, Crowley J, Storer BE. Estimation of failure probabilities in the presence of competing risks: New representations of old estimators. Stat Med. 1999;18:695–706.
Cox DR. Regression models and life tables. J R Stat Soc B. 1972;34:187–220.
Sorror ML, Giralt S, Sandmaier BM, De Lima M, Shahjahan M, Maloney DG, et al. Hematopoietic cell transplantation-specific comorbidity index as an outcome predictor for patients with acute myeloid leukemia in first remission: Combined FHCRC and MDACC experiences. Blood. 2007;110:4606–13.
Biard L, Labopin M, Chevret S, Resche-Rigon M. Investigating covariate-by-centre interaction in survival data. Stat Methods Med Res. 2018;27:920–32.
Friedrichs B, Tichelli A, Bacigalupo A, Russell NH, Ruutu T, Shapira MY, et al. Long-term outcome and late effects in patients transplanted with mobilised blood or bone marrow: a randomised trial. Lancet Oncol. 2010;11:331–8.
Anasetti C, Logan BR, Lee SJ, Waller EK, Weisdorf DJ, Wingard JR, et al. Peripheral-blood stem cells versus bone marrow from unrelated donors. N Engl J Med. 2012;367:1487–96.
Shahzad M, Mohsin A, Amin MK, Ur Rehman A, Chaudhary SG, Anwar I, et al. Outcomes after bone marrow versus peripheral blood matched unrelated donor allogeneic hematopoietic cell transplantation using posttransplant cyclophosphamide-based graft versus host disease prophylaxis. Blood. 2024;144:2118.
Mehta RS, Saliba RM, Alsfeld LC, Jorgensen JL, Wang SA, Anderlini P, et al. Bone marrow versus peripheral blood grafts for haploidentical hematopoietic cell transplantation with post-transplantation cyclophosphamide. Transpl Cell Ther. 2021;27:1003.e1.
Ruggeri A, Labopin M, Bacigalupo A, Gülbas Z, Koc Y, Blaise D, et al. Bone marrow versus mobilized peripheral blood stem cells in haploidentical transplants using posttransplantation cyclophosphamide. Cancer. 2018;124:1428–37.
Bashey A, Zhang M-J, McCurdy SR, St. Martin A, Argall T, Anasetti C, et al. Mobilized peripheral blood stem cells versus unstimulated bone marrow as a graft source for t-cell–replete haploidentical donor transplantation using post-transplant cyclophosphamide. J Clin Oncol. 2017;35:3002–9.
Nagler A, Dholaria B, Labopin M, Savani BN, Angelucci E, Koc Y, et al. Bone marrow versus mobilized peripheral blood stem cell graft in T-cell-replete haploidentical transplantation in acute lymphoblastic leukemia. Leukemia. 2020;34:2766–75.
Santoro N, Ruggeri A, Labopin M, Bacigalupo A, Ciceri F, Gülbaş Z, et al. Unmanipulated haploidentical stem cell transplantation in adults with acute lymphoblastic leukemia: a study on behalf of the Acute Leukemia Working Party of the EBMT. J Hematol Oncol. 2017;10:113.
Eapen M, Horowitz MM, Klein JP, Champlin RE, Loberiza FR, Ringdén O, et al. Higher mortality after allogeneic peripheral-blood transplantation compared with bone marrow in children and adolescents: The histocompatibility and alternate stem cell source working committee of the International Bone Marrow Transplant Registry. J Clin Oncol. 2004;22:4872–80.
Sanz J, Labopin M, Choi G, Kulagin A, Peccatori J, Vydra J, et al. Younger unrelated donors may be preferable over HLA match in the PTCy era: a study from the ALWP of the EBMT. Blood. 2024;143:2534–43.
Sanz J, Ferhat A-T, Kulagin A, Kröger N, Rovira M, Lazzari L, et al. Ptcy overcomes HLA mismatch in unrelated donor transplant for acute lymphoblastic leukemia: a study from the ALWP of the EBMT. Blood. 2024;144:3570.
Ljungman P. The role of cytomegalovirus serostatus on outcome of hematopoietic stem cell transplantation. Curr Opin Hematol. 2014;21:466–9.
Shaw BE, Mayor NP, Szydlo RM, Bultitude WP, Anthias C, Kirkland K, et al. Recipient/donor HLA and CMV matching in recipients of T-cell-depleted unrelated donor haematopoietic cell transplants. Bone Marrow Transplant. 2017;52:717–25.
Mehta RS, Choe H, Saultz J, Gong Z, Sharma P, Al-Juhaishi T, et al. Impact of donor age and donor cytomegalovirus serostatus on outcomes after related donor allogeneic hematopoietic stem cell transplantation. Am J Hematol. 2025;100:987–97.
Marty FM, Ljungman P, Chemaly RF, Maertens J, Dadwal SS, Duarte RF, et al. Letermovir prophylaxis for cytomegalovirus in hematopoietic-cell transplantation. N Engl J Med. 2017;377:2433–44.
DeFilipp Z, Advani AS, Bachanova V, Cassaday RD, Deangelo DJ, Kebriaei P, et al. Hematopoietic cell transplantation in the treatment of adult acute lymphoblastic leukemia: updated 2019 evidence-based review from the American Society for Transplantation and Cellular Therapy. Biol Blood Marrow Transpl. 2019;25:2113–23.
Giebel S, Labopin M, Socié G, Beelen D, Browne P, Volin L, et al. Improving results of allogeneic hematopoietic cell transplantation for adults with acute lymphoblastic leukemia in first complete remission: an analysis from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation. Haematologica. 2017;102:139–49.
Nishiwaki S, Akahoshi Y, Morita-Fujita M, Shimizu H, Uchida N, Ozawa Y, et al. Improvements in allogeneic hematopoietic cell transplantation outcomes for adults with ALL over the past 3 decades. Blood Adv. 2022;6:4558–69.
Author information
Authors and Affiliations
Contributions
JS, BS, JM, SG, ZP, AN, SP, MM and FC: designed the study. ATF: performed statistical analysis and helped with the interpretation of the results. JS: wrote the manuscript. EA, DB, MA, YK, MB, MK, AK, MP, SS, JT, JA, RPL, GG, NK: provided patient data for the study. All authors reviewed and approved the manuscript.
Corresponding author
Ethics declarations
Competing interests
The authors declare no competing interests.
Ethical approval
The scientific board of the ALWP of EBMT approved this study. All patients gave written informed consent for the use of their data. All methods were performed in accordance with the relevant guidelines and regulations.
Additional information
Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.
Supplementary information
Rights and permissions
Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.
About this article
Cite this article
Sanz, J., Ferhat, AT., Raiola, A.M. et al. Donor selection in PTCy haploidentical transplant for acute lymphoblastic leukemia: a study from the ALWP of the EBMT. Bone Marrow Transplant 61, 166–171 (2026). https://doi.org/10.1038/s41409-025-02763-3
Received:
Revised:
Accepted:
Published:
Version of record:
Issue date:
DOI: https://doi.org/10.1038/s41409-025-02763-3


