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

dc.contributor.authorMontoro, Juan
dc.contributor.authorEikema, Dirk-Jan
dc.contributor.authorPiepenbroek, Brian
dc.contributor.authorTuffnell, Joe
dc.contributor.authorHalahleh, Khalid
dc.contributor.authorKulagin, Alexander
dc.contributor.authorAlAhmari, Ali
dc.contributor.authorAdaklı Aksoy, Başak
dc.contributor.authorRemenyi, Peter
dc.contributor.authorItala-Remes, Maija
dc.contributor.authorGülbaş, Zafer
dc.contributor.authorMcDonald, Andrew
dc.contributor.authorApte, Shashikant
dc.contributor.authorKwon, Mi
dc.contributor.authorRovira, Montserrat
dc.contributor.authorKharya, Gaurav
dc.contributor.authorPotter, Victoria
dc.contributor.authorGambella, Massimilano
dc.contributor.authorSchroeder, Thomas
dc.contributor.authorGiammarco, Sabrina
dc.contributor.authorBazarbachi, Ali
dc.contributor.authorAljurf, Mahmoud
dc.contributor.authorHo, Aloysius
dc.contributor.authorDalle, Jean-Hugues
dc.contributor.authorVydra, Jan
dc.contributor.authorSanz, Jaime
dc.contributor.authorPerez-Simon, Jose Antonio
dc.contributor.authorColita, Anca
dc.contributor.authorCollin, Matthew
dc.contributor.authorTanase, Alina
dc.contributor.authorHalkes, Constantijn
dc.contributor.authorKulasekararaj, Austin
dc.contributor.authorRisitano, Antonio
dc.contributor.authorde Latour, Regis Peffault
dc.date.accessioned2025-08-14T17:53:26Z
dc.date.available2025-08-14T17:53:26Z
dc.date.issued2025
dc.departmentFakülteler, Tıp Fakültesi, Dahili Tıp Bilimleri Bölümü, Çocuk Sağlığı ve Hastalıkları Ana Bilim Dalı
dc.description.abstractThe 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.
dc.identifier.citationMontoro, J., Eikema, D. J., Piepenbroek, B., Tuffnell, J., Halahleh, K., Kulagin, A., ... & Peffault de Latour, R. (2025). Donor impact on allogeneic transplant outcomes with PTCy for severe aplastic anemia: a study of the SAAWP EBMT. Bone Marrow Transplantation. 10.1038/s41409-025-02633-y
dc.identifier.doi10.1038/s41409-025-02633-y
dc.identifier.issn0268-3369
dc.identifier.issn1476-5365
dc.identifier.pmid40425810
dc.identifier.scopus2-s2.0-105006583437
dc.identifier.scopusqualityQ1
dc.identifier.urihttps://hdl.handle.net/20.500.12939/5930
dc.identifier.wosWOS:001497638900001
dc.identifier.wosqualityQ1
dc.indekslendigikaynakPubMed
dc.indekslendigikaynakWeb of Science
dc.indekslendigikaynakScopus
dc.institutionauthorAdaklı Aksoy, Başak
dc.publisherScientific & Medical Division
dc.relation.ispartofBone Marrow Transplantation
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanı
dc.rightsinfo:eu-repo/semantics/closedAccess
dc.subjectReaxys is designed to support chemistry researchers at every stage with the ability to investigated chemistry related research topics in peer-reviewed literature
dc.subjectpatents and substance databases. Reaxys retrieves substances
dc.subjectsubstance properties
dc.subjectreaction and synthesis data
dc.titleDonor impact on allogeneic transplant outcomes with PTCy for severe aplastic anemia: a study of the SAAWP EBMT

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