Post-Transplant Cyclophosphamide and JAK Inhibitors Are Subjects of Most GVHD Trials

By Dustin Samples - Last Updated: September 13, 2023

Graft versus host disease (GVHD) is a significant concern following allogeneic hematopoietic stem-cell transplantation (allo-HSCT). The disease can lead to non-relapse mortality and considerable patient morbidity. Its incidence varies and is influenced by recipient characteristics, graft source, and other variables. The current standard GVHD prevention regimens typically involve methotrexate with calcineurin inhibitors, and post-transplant cyclophosphamide (PTCy) is commonly used in haploidentical transplants, often following the John Hopkins protocol. Numerous clinical trials are actively exploring ways to improve GVHD prevention strategies.

In an abstract presented at the 11th Annual Meeting of the Society of Hematologic Oncology (SOHO) in Houston, Texas, researchers from Buffalo, New York, presented the findings of an investigation into relevant ongoing trials on GVHD.

The team conducted extensive searches on clinical trial registration platforms, including, the European clinical trial union, and the Australia-New Zealand clinical trial network for “graft-vs-host-disease” and “prevention.” Trials expected to conclude after May 1, 2023, were included, while those already completed, withdrawn, or terminated prematurely were excluded. In addition, trials with no updates over the past 5 years or those set to finish before May 1, 2023, were excluded.

The research team identified 317 trials pertaining to GVHD prevention. After eliminating duplicates and trials focused on specific treatments, data from 53 trials were analyzed. Most of these trials were in Phase 2 (27 trials), with only 5 in Phase 3.

The median number of patients across all studies was 70, ranging from 12-540. The most frequently studied GVHD prevention regimens included PTCy (25 trials), JAK inhibitors (8 trials), and abatacept (5 trials). The primary endpoint in most trials was the incidence of GVHD (20 trials), while 8 trials focused on GVHD-free, relapse-free survival (GRFS) as their primary outcome.

PTCy is the most prominent subject of study for GVHD prophylaxis across various clinical contexts, emphasizing its clinical importance and ongoing research attention. In addition, JAK inhibitors are actively investigated as potential GVHD prevention agents.

These findings emphasize the ongoing need for innovative and enhanced GVHD prophylactic strategies to improve outcomes for patients undergoing allo-HSCT. Staying up to date on these evolving approaches is critical for oncology nurses to provide the best care to transplant recipients.


Kumarasamy VM, Kashour Z, Faisal MS, et al. The Future Landscape of Graft vs Host Disease Prevention Trials. Abstract CT-646. Presented at the 11th Annual Meeting of the Society of Hematologic Oncology; September 6-9, 2023; Houston, Texas.

Post Tags:SOHO 2023: GVHD