A1 Refereed original research article in a scientific journal
Unrelated donor transplantation with posttransplant cyclophosphamide vs ATG for myelodysplastic neoplasms
Authors: Chalandon, Yves; Eikema, Diderik-Jan; Moiseev, Ivan Sergeevich; Ciceri, Fabio; Koster, Linda; Vydra, Jan; Passweg, Jakob R; Rovira, Montserrat; Ozcelik, Tulay; Gedde-Dahl, Tobias; Kröger, Nicolaus; Potter, Victoria; Yakoub-Agha, Ibrahim; Rambaldi, Alessandro; Itälä-Remes, Maija; Tanase, Alina D; Onida, Francesco; Gurnari, Carmelo; Scheid, Christof; Drozd-Sokolowska, Joanna; Raj, Kavita; McLornan, Donal P; Robin, Marie
Publisher: Elsevier
Publication year: 2024
Journal: Blood Advances
Journal name in source: Blood advances
Journal acronym: Blood Adv
Volume: 8
Issue: 18
First page : 4792
Last page: 4802
ISSN: 2473-9529
eISSN: 2473-9537
DOI: https://doi.org/10.1182/bloodadvances.2024013468
Web address : https://ashpublications.org/bloodadvances/article/doi/10.1182/bloodadvances.2024013468/517033/Unrelated-donor-transplantation-with-post
Self-archived copy’s web address: https://research.utu.fi/converis/portal/detail/Publication/457243588
Prospective randomized trials have reported a benefit for anti-thymocyte globulin (ATG)-based graft-versus-host disease (GvHD) prophylaxis in the setting of allogeneic hematopoietic stem cell transplantation (Allo-HSCT) with unrelated donors (UD). However, the optimal GvHD prophylaxis strategy has been recently challenged by the increasing use of post-transplant cyclophosphamide (PTCY). We report from the EBMT registry the outcomes of 960 patients with myelodysplastic neoplasms (MDS) undergoing allo-HSCT from UD with PTCY or ATG as GvHD prophylaxis. Primary outcomes were overall survival (OS) and progression-free survival (PFS). Disease characteristics were similar in both groups. Day 28 neutrophil engraftment was significantly better with ATG (93% vs 85%, p<0.001). With a median follow-up of 4.4 years (95% confidence interval [CI] 4.2 - 4.8), 5-year OS was 58% (95% CI 50-65) with PTCY and 49% (95% CI 46-53%) in the ATG group, p=0.07. 5-year PFS was higher for PTCY with 53% (95% CI 45-60) vs 44% (95% CI 40-48) for ATG, p=0.043. Grade II-IV aGvHD incidence was lower using PTCY (23% [95% CI 17-29%] vs 30% [95% CI 27-33%]), p=0.044 while there was no difference in incidence of cGvHD at 5 years. Multivariable analyses confirmed better OS and PFS with PTCY, with a HR for ATG of 1.32 (1 - 1.74), p=0.05, and a better PFS for PTCY with a HR for ATG of 1.33 (1.03 - 1.73), p=0.03. This study suggests that GvHD prophylaxis using PTCY instead of ATG in this setting remains a valid option. Further prospective randomized studies would be essential to confirm these results.
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