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. 2026 Jan 13;10(1):233-245.
doi: 10.1182/bloodadvances.2025017194.

Matched unrelated vs haploidentical donor hematopoietic cell transplantation using posttransplant cyclophosphamide

Dipenkumar Modi  1 , Yosra M Aljawai  2 , Todd E DeFor  3 , Caitrin Bupp  3 , Monzr M Al Malki  4 , Javier Bolaños-Meade  5 , Mahasweta Gooptu  6 , Antonio M Jimenez Jimenez  7 , Hongtao Liu  8 , Felix A Mensah  9 , Marco Mielcarek  10 , Brian C Shaffer  11 , Bronwen E Shaw  12 , Stephen R Spellman  3 , Heather E Stefanski  3 , Jeffery J Auletta  3 , Steven M Devine  3 , Farhad Khimani  13 , Ramzi Abboud  14
Affiliations

Matched unrelated vs haploidentical donor hematopoietic cell transplantation using posttransplant cyclophosphamide

Dipenkumar Modi et al. Blood Adv. .

Abstract

Posttransplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis is now standard for matched unrelated donor (MUD) hematopoietic cell transplantation (HCT). Previous studies comparing MUD and haploidentical donor HCT using PTCy were limited in size and follow-up. We therefore performed a registry-based analysis examining the impact of donor type on HCT with PTCy. Adult patients (n = 5873) receiving MUD (n = 1973) or haploidentical (n = 3900) HCT with PTCy for acute leukemia (74.2%) or myelodysplastic syndrome (MDS; 25.8%) reported to the Center for International Blood and Marrow Transplant Research between 2017 and 2021 were included. Primary end points were 3-year overall survival (OS) and GVHD-free, relapse-free survival (GRFS). Cox regression and sensitivity analyses were performed through adjustment of propensity scores. Haploidentical HCT had worse OS (hazard ratio [HR], 1.15; 95% confidence interval [CI], 1.04-1.27; P = .005) and GRFS (HR, 1.19; 95% CI, 1.10-1.29; P < .001) versus MUD HCT. Donor age was the only other donor factor associated with survival. Results were confirmed in sensitivity analysis. When restricted to reduced intensity conditioning or donors <30 years, OS did not differ between groups. Haploidentical HCT was associated with higher primary graft failure (HR, 1.67; P = .002), increased grade 3/4 acute GVHD (HR, 1.28; P = .039), higher moderate/severe chronic GVHD (HR, 1.47; P < .001), and nonrelapse mortality (HR, 1.34; P < .001). Grade 2 to 4 acute GVHD and relapse risk did not differ. This large analysis showed that in adults with acute leukemia or MDS, MUD HCT was associated with improved outcomes versus haploidentical HCT with PTCy-based GVHD prophylaxis.

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Conflict of interest statement

Conflict-of-interest disclosure: D.M. reports consulting role with ADC Therapeutics, Genmab, Bristol Myers Squibb (spouse), AstraZeneca (spouse), and Daiichi Sankyo/Lilly (spouse); and research funding from Karyopharm Therapeutics (to institution), Genentech (to institution), and AstraZeneca (to institution). T.E.D. reports a role with HealthPartners Institute (spouse). M.M.A.M. reports consulting role with CareDX, T Scan, TR1X, MaaT Pharma, and Ossium Health; and received research funding from Incyte, Stemline Therapeutics, and Takeda. J.B.-M. reports consulting role with MJH Healthcare Holdings, LLC and Avoro Capital Advisors; honoraria from Banner MD Anderson Colorado; and travel funds from Dictaforum Servicios. B.C.S. reports research funding from Genentech. B.E.S. reports consulting role with Orca Bio (to institution). J.J.A. reports consulting role with, and honoraria from, Ascella Health. S.M.D. reports a leadership role with the National Marrow Donor Program. F.K. reports research funding from Bristol Myers Squibb (to institution) and Incyte (to institution). The remaining authors declare no competing financial interests.

Figures

None
Graphical abstract
Figure 1.
Figure 1.
Impact of donor type on overall and GVHD-free, relapse-free survival. Adjusted Kaplan-Meier estimates of OS (A) and GRFS (B) in recipients of MUD HCT or haplo-HCT.
Figure 2.
Figure 2.
Impact of donor type on acute and chronic GVHD. Cumulative incidence of (A) grade 2 to 4 aGVHD, (B) grade 3/4 aGVHD, and (C) moderate/severe cGVHD in recipients of MUD HCT or haplo-HCT.
Figure 3.
Figure 3.
Impact of donor type on disease-free survival, nonrelapse mortality and relapse. Cumulative incidence of DFS (A), NRM (B), and relapse (C) in recipients of MUD HCT or haplo-HCT.
Figure 4.
Figure 4.
Forest plot results of the multivariable adjusted risk of primary and secondary end points comparing MUD HCT (reference) with haplo-HCT. URD, unrelated donor.
See this image and copyright information in PMC

References

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