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. 2024 Sep 24;8(18):4792-4802.
doi: 10.1182/bloodadvances.2024013468.

Unrelated donor transplantation with posttransplant cyclophosphamide vs ATG for myelodysplastic neoplasms

Yves Chalandon  1 , Diderik-Jan Eikema  2 , Ivan Moiseev  3 , Fabio Ciceri  4 , Linda Koster  5 , Jan Vydra  6 , Jakob Passweg  7 , Montserrat Rovira  8 , Tulay Ozcelik  9 , Tobias Gedde-Dahl  10 , Nicolaus Kröger  11 , Victoria Potter  12 , Ibrahim Yakoub-Agha  13 , Alessandro Rambaldi  14 , Maija Itälä-Remes  15 , Alina Tanase  16 , Francesco Onida  17 , Carmelo Gurnari  18 , Christof Scheid  19 , Joanna Drozd-Sokolowska  20 , Kavita Raj  21 , Donal P McLornan  21 , Marie Robin  22
Affiliations

Unrelated donor transplantation with posttransplant cyclophosphamide vs ATG for myelodysplastic neoplasms

Yves Chalandon et al. Blood Adv. .

Abstract

It has been reported in prospective randomized trials that antithymocyte globulin (ATG)-based graft-versus-host disease (GVHD) prophylaxis has benefits in the setting of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with unrelated donors (UDs). However, the optimal GVHD prophylaxis strategy has been challenged recently by the increasing use of posttransplant cyclophosphamide (PTCY). We report from the European Society for Blood and Marrow Transplantation registry the outcomes of 960 patients with myelodysplastic neoplasms who underwent allo-HSCT from UD with PTCY or ATG as GVHD prophylaxis. The primary outcomes were overall survival (OS) and progression-free survival (PFS). The disease characteristics were similar in both groups. Day 28 neutrophil engraftment was significantly better with ATG (93% vs 85%). Over a median follow-up of 4.4 years, the 5-year OS was 58% with PTCY, and 49% in the ATG group. The 5-year PFS was higher for PTCY at 53% vs 44% for ATG. Grade 2 to 4 acute GVHD incidence was lower when PTCY was used (23%), whereas there was no difference in the incidence of chronic GVHD at 5 years. Multivariable analyses confirmed better OS and PFS with PTCY with a hazard ratio (HR) for ATG of 1.32 (1-1.74) and a better PFS for PTCY with a HR for ATG of 1.33. 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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Conflict of interest statement

Conflict-of-interest disclosure: Y.C reports receiving consulting fees for serving on the advisory board from Merck Sharp & Dohne, Novartis, Incyte, Bristol Myers Squibb (BMS), Pfizer, AbbVie, Roche, Jazz, Gilead, Amgen, AstraZeneca, Servier, and Pierre Fabre; and travel support from MSD, Roche, Gilead, Amgen, Incyte, AbbVie, Janssen, AstraZeneca, Jazz, Sanofi, and Pierre Fabre all via the institution. I.Y.-A. reports receiving honoraria from BMS, Novartis, Kite, and Miltenyi Biomedecine. M. Robin reports receiving research support from AbbVie, Astex, Novartis, Neovii, and Medac. The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Incidence of GVHD. (A) Cumulative incidence at day 100 of aGVHD grade 2 to 4. (B) Cumulative incidence at day 100 of aGVHD grade 3 to 4. (C) Cumulative incidence of cGVHD at 5 years among patients with MDS who received PTCY or ATG as GVHD prophylaxis for UD allo-HSCT. Numbers below the graph show the number of patients at risk.
Figure 2.
Figure 2.
Outcomes of patients with MDS transplanted with PTCY vs ATG. (A) OS, (B) PFS, (C) GRFS, (D) RI, and (E) NRM of patients with MDS who received PTCY or ATG as GVHD prophylaxis for UD allo-HSCT and stratified by donor type (MUD vs MMUD). Numbers below the graph show the number of patients at risk.
Figure 3.
Figure 3.
Outcomes of patients with MDS transplanted with PTCY vs ATG. (A) OS was 60% (95% CI, 52-72) for patients who underwent a MUD transplant and received PTCY, 53% (95% CI, 42-63) for patients who underwent a MMUD transplant and received PTCY, 52% (95% CI, 48-57) for patients who underwent a MUD transplant and received ATG, and 41% (95% CI, 34-49) for patients who underwent a MMUD transplant and received ATG (P = .004). (B) PFS was 56% (95% CI, 45-67) for patients who underwent a MUD transplant and received PTCY, 49% (95% CI, 38-60) for patients who underwent a MMUD transplant and received PTCY, 46% (95% CI, 42-51) for patients who underwent a MUD transplant and received ATG, and 37% (95% CI, 30-44) for patients who underwent a MMUD transplant and received ATG (P = .007). (C) GRFS was 35% (95% CI, 25-45) for patients who underwent a MUD transplant and received PTCY, 36% (95% CI, 25-46) for patients who underwent a MMUD transplant and received PTCY, 32% (95% CI, 28-37) for patients who underwent a MRD transplant and received ATG, and 26% (95% CI, 19-33) for patients who underwent a MMUD transplant and received ATG (P = .09). (D) RI was 22% (95% CI, 12-31) for patients who underwent a MUD transplant and received PTCY, 23% (95% CI, 14-32) for patients who underwent a MMUD transplant and received PTCY, 26% (95% CI, 22-29) for patients who underwent a MUD transplant and received ATG, and 24% (95% CI, 18-31) for patients who underwent a MMUD transplant and received ATG (P = .7). (E) NRM was 22% (95% CI, 14-30) for patients who underwent a MUD transplant and received PTCY, 28% (95% CI, 19-37) for patients who underwent a MMUD transplant and received PTCY, 28% (95% CI, 24-32) for patients who underwent a MUD transplant and received ATG, and 39% (95% CI, 32-46) for patients who underwent a MMUD transplant and received ATG (P = .009) when stratified by donor type (MUD vs MMUD). Numbers below the graph show the number of patients at risk.
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