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2024-08-01T16:11:12.000Z

PTCy-based GvHD prophylaxis following MMUD peripheral blood stem cell transplantation: Results from the phase II ACCESS trial

Aug 1, 2024
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Learning objective: After reading this article, learners will be able to cite a new clinical development in graft-versus-host disease.


The ongoing phase II ACCESS trial (NCT04904588) is assessing the impact of post-transplant cyclophosphamide (PTCy)-based graft-versus-host disease (GvHD) prophylaxis on overall survival (OS) in adult and pediatric patients with hematological malignancies undergoing a mismatched unrelated donor (MMUD) transplantation.1 Results from the first 70 adult patients who received reduced-intensity condition (RIC) MMUD peripheral blood stem cell (PBSC) transplantation were presented during the European Hematology Association (EHA) 2024 Hybrid Congress by Jimenez.1 Results were compared with the phase II 15-MMUD trial (NCT02793544) of RIC MMUD bone marrow transplantation with PTCy prophylaxis.1


Key learnings:

In the phase II ACCESS trial, with a median follow-up of 12.1 months, the 1-year OS rate was 79% and was not impacted by match grade (7/8 vs <7/8; p = 0.580); this was similar to the 1-year OS rates in the 15-MMUD trial (RIC cohort, 79%; overall, 76%).

The 1-year GvHD-free, relapse-free survival and non-relapse mortality rates were 51% and 13%, respectively. 

The rates of acute GvHD Grade III–IV and moderate/severe chronic GvHD were 9% for both. 

With comparable OS rates to bone marrow transplantation and comparable GvHD rates to matched donor transplantation, these results suggest that RIC MMUD PBSC transplantation with PTCy-based GvHD prophylaxis could expand access to HSCT for patients with hematological malignancies without a matched donor available.


  1. AJ Jimenez. Post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis following mismatched unrelated donor peripheral blood stem cell (PBSC) transplantation. Abstract #S260. Presented at: European Hematology Association 2024 Hybrid Congress; Jun 13–16, 2024; Madrid, ES. 

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