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. 2025 May;60(5):625-631.
doi: 10.1038/s41409-025-02523-3. Epub 2025 Feb 27.

Melphalan-based conditioning with post-transplant cyclophosphamide for peripheral blood stem cell transplantation: donor effect

Paul B Koller #  1 , Tamer Othman #  1 , Dongyun Yang  2 , Sally Mokhtari  3 , Yazeed Samara  1 , Amanda Blackmon  1 , Vaibhav Agrawal  1 , Hoda Pourhassan  1 , Brian J Ball  1 , Idoroenyi Amanam  1 , Shukaib Arslan  1 , Salman Otoukesh  1 , Karamjeet S Sandhu  1 , Ibrahim Aldoss  1 , Haris Ali  1 , Amandeep Salhotra  1 , Ahmed Aribi  1 , Andrew Artz  1 , Pamela S Becker  1 , Vinod Pullarkat  1 , Forest Marc Stewart  1 , Eileen P Smith  1 , Anthony Stein  1 , Guido Marcucci  1 , Stephen J Forman  1 , Ryotaro Nakamura  1 , Monzr M Al Malki  4
Affiliations

Melphalan-based conditioning with post-transplant cyclophosphamide for peripheral blood stem cell transplantation: donor effect

Paul B Koller et al. Bone Marrow Transplant. 2025 May.

Abstract

Fludarabine and melphalan (FM) conditioning offers effective disease control with an acceptable toxicity profile. Post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis has improved transplant outcomes. We retrospectively reviewed patients receiving FM-based transplants with PTCy at City of Hope. Of 248 patients included, 89 (35.9%) received hematopoietic cell transplant (HCT) from a matched related/unrelated donor (MRD/MUD), 118 (47.6%) from a haploidentical (HID) donor, and 49 (19.8%) from a mismatched unrelated donor (MMUD). There were no differences in acute and chronic GVHD based on donor type. The 2-year overall survival (OS) for patients receiving HID, MMUD, and MRD/MUD was 58%, 55%, and 70%; disease-free survival (DFS) was 52%, 48%, and 66%; and graft-versus-host/relapse-free survival (GRFS) were 48%, 40%, and 59%, respectively. OS, DFS, and GRFS were similar regardless of donor type on multivariable analysis. However, donor age ≥35 years was associated with lower OS and GRFS and higher 2-year non-relapse mortality (NRM) on multivariable analysis across all patients, regardless of donor type. FM with PTCy appears to produce similar outcomes between MRD/MUD, MMUD, and HID when adjusting for donors <35 years, and donor age seems to be the most important factor when selecting a donor with this regimen.

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Conflict of interest statement

Competing interests: PK - Ascentage: Ad board, Daiichi Sankyo: Consulting/Ad Board; BMS: Consulting/Ad Board; Novartis: Consulting/Ad Board; Speakers Bureaul, Travel; Takeda: Ad Board, Speakers Bureau. KS - Autolous: consultancy. IA - Takeda, Adaptive, Syndax, Wugen: consultancy. AA - KiTE and SeaGen: Consultancy. VP - Abbvie: Consultancy, Honoraria, Speakers Bureau; Novartis: Consultancy, Honoraria; Amgen: Speakers Bureau; Sobi: Speakers Bureau; Jazz: Speakers Bureau; Alexion: Honoraria; Rigel: Consultancy, Honoraria. MMA - Tscan: Consultancy; NexImmune: Consultancy, Research Funding; CareDx: Consultancy; Incyte: Research Funding; Tr1X: Consultancy. The rest of the authors have no conflicts to disclose.

Figures

Fig. 1
Fig. 1. Adjusted survival figures based on donor type.
Adjusted overall survival based on donor type (a), disease-free survival based on donor type (b), graft-vs-host/relapse-free survival based on donor type (c).
Fig. 2
Fig. 2. Adjusted competing risk curves based on donor type.
Adjusted non-relapse mortality based on donor type (a), and cumulative incidence of relapse based on donor type (b).
Fig. 3
Fig. 3. Outcomes based on donor type and age.
Overall survival based on donor type and age (a). Non-relapse mortality based on donor type and age (b).
See this image and copyright information in PMC

References

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