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Donor impact on allogeneic transplant outcomes with PTCy for severe aplastic anemia: a study of the SAAWP EBMT

Bone Marrow Transplantation volume 60, pages 1152–1159 (2025)Cite this article

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Abstract

The use of post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis in severe aplastic anemia (SAA) remains understudied, particularly beyond haploidentical transplants. We analyzed outcomes of SAA patients who underwent stem cell transplantation (SCT) with PTCy from haploidentical donors (n = 209), HLA-matched sibling donors (MSD, n = 70), and unrelated donors (UD, n = 69) using EBMT data from 2010 to 2022. Median age was 22 years, and median time to transplantation was 8.6 months. For haploidentical, MSD, and UD cohorts, the 100-day cumulative incidence of grade II-IV acute GVHD was 19%, 11%, and 14% (p = 0.15), while grade III-IV was 6%, 3%, and 2% (p = 0.1). Two-year chronic and extensive chronic GVHD were 14%, 13%, and 14% (p = 0.1) and 5%, 6%, and 2% (p = 0.5), respectively. Non-relapse mortality at two years was 24% for haploidentical, 7% for MSD, and 10% for UD (p = 0.003). Two-year overall survival (OS) and GVHD- and relapse-free survival were 66% and 54% for haploidentical, 92% and 70% for MSD, and 81% and 66% for UD (p < 0.001, p = 0.06). In multivariable analysis, MSD and UD were associated with superior OS and GRFS compared to haploidentical. PTCy is safe and effective in SAA patients, though haploidentical SCT had higher NRM, leading to lower survival.

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Fig. 1: Overall survival stratified by donor type.
Fig. 2: Graft versus host relapse free survival stratified by Donor Type.

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All data can be obtained from corresponding author for appropriated reasons.

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Acknowledgements

The authors are particularly thankful to all centers from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation, who kindly agreed to participate in this study.

Author information

Authors and Affiliations

  1. Department of Hematology, Hospital Universitario y Politécnico La Fe, Valencia, Spain

    Juan Montoro & Jaime Sanz

  2. Universidad Católica de Valencia, Valencia, Spain

    Juan Montoro

  3. EBMT Leiden Study Unit, Leiden, Netherlands

    Dirk-Jan Eikema, Brian Piepenbroek & Joe Tuffnell

  4. King Hussein Cancer Centre Adult BMT Program, Amman, Jordan

    Khalid Halahleh

  5. RM Gorbacheva Research Institute, Pavlov University, St. Petersburg, Russia

    Alexander Kulagin

  6. King Faisal Specialist Hospital and Research Centre, Department of Pediatric Hematology/Oncology, Riyadh, Saudi Arabia

    Ali AlAhmari

  7. Altinbas University, Faculty of Medicine, Bahcelievler Medicalpark Hospital, Pediatic BMT Unit, Istanbul, Turkey

    Basak Adakli Aksoy

  8. Dél-pesti Centrumkórház, Budapest, Hungary

    Péter Reményi

  9. Turku University Hospital, Turku, Finland

    Maija Itäla-Remes

  10. Anadolu Medical Cancer Hospital, Kocaeli, Turkey

    Zafer Gulbas

  11. Alberts Cellular Therapy, Pretoria, South Africa

    Andrew McDonald

  12. Sahyadri Specialty Hospital, Pune, India

    Shashikant Apte

  13. Hospital Gregorio Marañón, Madrid, Spain

    Mi Kwon

  14. Hospital Clinic, Barcelona, Spain

    Montserrat Rovira

  15. Indraprastha Apollo Hospitals, New Delhi, India

    Gaurav Kharya

  16. Kings College Hospital, London, UK

    Victoria Potter

  17. IRCCS Ospedale Policlinico San Martino, Genova, Italy

    Massimiliano Gambella

  18. University Hospital Essen, Dusseldorf, Germany

    Thomas Schroeder

  19. Fondazione Policlinico Universitario A. Gemelli-IRCCS, Rome, Italy

    Sabrina Giammarco

  20. Department of Internal Medicine. American University of Beirut, Beirut, Lebanon

    Ali Bazarbachi

  21. King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

    Mahmoud Aljurf

  22. Singapore General Hospital, Singapore, Singapore

    Aloysius Ho

  23. Hopital Robert Debre Pediatric, Paris, France

    Jean-Hugues Dalle

  24. Institute of Hematology and Blood Transfusion, Prague, Czech Republic

    Jan Vydra

  25. Department of Hematology. Hospital Universitario Virgen del Rocío, Instituto de Biomedicina de Sevilla (IBIS), CISC, Universidad de Sevilla, Sevilla, Spain

    José Antonio Pérez-Simon

  26. Fundeni Clinical Institute, Bucarest, Romania

    Anca Colita & Alina Tanase

  27. Northern Centre for Bone Marrow Transplantation, Newcastle, UK

    Matthew Collin

  28. Leiden University Hospital, Leiden, The Netherlands

    Constantijn Halkes

  29. Kings College Hospital London, London, UK

    Austin Kulasekararaj

  30. University of Naples, Avellino, Italy

    Antonio Risitano

  31. BMT Unit, Hôpital Saint-Louis, Paris Cité Université, Paris, France

    Régis Peffault de Latour

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  1. Juan Montoro
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  2. Dirk-Jan Eikema
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Contributions

Conception and design: JM, AR, RPDL. Data analysis and interpretation: JM, DJE, AR, RPDL. Manuscript writing: JM. Final approval of manuscript: JM, DJE, BP, JT, KH, AK, AA, BAA, PR, MIR, ZG, AM, SA, MI, MR, GK, VP, MG, TS, SG, AB, MA, AH, JHD, JV, JS, JAPS, AC, MC, AT, CH, AK, AR, RPDL.

Corresponding author

Correspondence to Juan Montoro.

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Patients prospectively provided signed informed consent for both data collection through the ProMISe system and any posteriori analysis. The study was conducted in accordance with the Declaration of Helsinki and was approved by the scientific committee of the SAAWP of the EBMT.

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The authors declare no competing interests.

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Montoro, J., Eikema, DJ., Piepenbroek, B. et al. Donor impact on allogeneic transplant outcomes with PTCy for severe aplastic anemia: a study of the SAAWP EBMT. Bone Marrow Transplant 60, 1152–1159 (2025). https://doi.org/10.1038/s41409-025-02633-y

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