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. 2025 Feb 11;9(3):660-669.
doi: 10.1182/bloodadvances.2024014781.

PTCy-based graft-versus-host disease prophylaxis for matched sibling donor allogeneic hematopoietic cell transplantation

Nihar Desai  1   2 , Majed Altareb  1   2 , Mats Remberger  3 , Carol Chen  1 , Tommy Alfaro Moya  1   2 , Eshrak Al-Shaibani  1   2 , Igor Novitzky-Basso  1   2 , Ivan Pasic  1   2 , Wilson Lam  1   2 , Fotios V Michelis  1   2 , Armin Gerbitz  1   2 , Auro Viswabandya  1   2 , Rajat Kumar  1   2 , Dennis Dong Hwan Kim  1   2 , Jeffrey H Lipton  1   2 , Jonas Mattsson  1   2 , Arjun Datt Law  1   2
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PTCy-based graft-versus-host disease prophylaxis for matched sibling donor allogeneic hematopoietic cell transplantation

Nihar Desai et al. Blood Adv. .

Abstract

Posttransplant cyclophosphamide (PTCy) is a promising graft-versus-host disease (GVHD) prophylaxis in haploidentical and matched unrelated donor hematopoietic stem cell transplantation (HSCT), but its role in matched sibling donor (MSD) transplants remains unclear. We conducted a retrospective study of 413 MSD-HSCT patients receiving peripheral blood stem cell (PBSC) grafts from January 2010 to January 2023. Patients were categorized into 4 groups: group I (calcineurin inhibitor [CNI] + methotrexate [MTX] or mycophenolate mofetil [MMF]), group II (CNI + MTX or MMF + antithymocyte globulin [ATG]), group III (PTCy + ATG + CNI), and group IV (PTCy + CNI + MMF). PTCy was associated with a significant reduction in grade 2- 4 and grade 3-4 acute GVHD and moderate-to-severe chronic GVHD compared with CNI + MTX (or MMF)-containing regimens. PTCy did not increase relapse risk; PTCy reduced nonrelapse mortality, leading to improved GVHD-free/relapse-free survival (GRFS; Hazard Ratio, 0.4; P < .001). PTCy was also associated with improved overall survival. Bloodstream infections were increased with PTCy. The addition of ATG to PTCy did not further improve GRFS and was associated with a higher incidence of clinically significant cytomegalovirus (csCMV) and Epstein-Barr virus (csEBV) reactivation and a numerical increase in NRM. PTCy significantly appeared to improve GRFS in the MSD setting using PBSC grafts. The addition of ATG to PTCy increases csCMV and csEBV reactivation without further improving GRFS. Prospective trials and PTCy dose optimization are warranted.

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Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Incidence of varying degrees of acute and chronic GVHD across different forms of GVHD prophylaxis. (A) Cumulative incidence of grade 2 to 4 aGVHD. (B) Cumulative incidence of grade 3 to 4 aGVHD. (C) Cumulative incidence of all-grade cGVHD. (D) Cumulative incidence of moderate-to-severe (mod-sev) cGVHD.
Figure 2.
Figure 2.
Comparison of posttransplant outcomes according to type of GVHD prophylaxis. (A) OS. (B) GRFS. (C) Cumulative incidence of NRM. (D) Cumulative incidence of relapse.
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