Can Vitamin A Supplementation Reduce the Incidence of Chronic GVHD After Allogeneic Transplant?

Hematopoietic cell transplant (HCT) can be curative for many patients with hematologic malignancies or other blood disorders. But allogeneic transplants, in which hematopoietic cells are derived from a donor, involve a substantial risk that the donor’s immune cells will perceive the patient’s cells as foreign and attack them, resulting in either chronic or acute graft-versus-host disease (GVHD). Moderate to severe chronic GVHD occurs in about 20-25% of patients who undergo allogeneic HCT, and the mortality rate for those patients is high.

Research has shown that vitamin A, which helps regulate a variety of immune responses, may have the potential to reduce the incidence of chronic GVHD or limit its severity. Roswell Park Comprehensive Cancer Center is one of only three centers nationwide and the only one in New York State offering a phase 2 clinical trial (NCT06450925) to determine whether a single oral high-dose supplementation of vitamin A can reduce the incidence of moderate to severe chronic GVHD vs. placebo in HCT patients.

Theresa Hahn, PhD, Professor of Oncology in the Department of Cancer Prevention and Control at Roswell Park, serves as site principal investigator for the study. To be eligible, patients must:

• Be at least 18 years old
• Be scheduled to undergo an allogeneic hematopoietic cell transplant
• Have a vitamin A level below the upper limit of normal
• Be able to tolerate enteral vitamin administration
• Have a total bilirubin level below 1.5x ULN and an AST and/or ALT below 3xULN for their age
• Receive peripheral blood as the graft

Patients will be randomized to one of two arms. Prior to HCT, participants in Arm A will receive one observed oral dose of vitamin A in either the outpatient clinic or inpatient BMT center. Participants in Arm B will receive placebo pills.

The study’s primary objective is to compare the incidence of moderate to severe GVHD in both groups of patients within one-year post-transplant. Secondarily, it will compare overall survival and the incidence of acute GVHD and relapse within two years post-transplant.

Children's Hospital Medical Center, Cincinnati, is the sponsor of this study, which is made possible by support from the Leukemia & Lymphoma Society.

“We are excited to be one of very few centers in the U.S. to offer this trial to patients receiving an allogeneic HCT at Roswell Park,” Dr. Hahn says. “Based on prior trials in pediatric patients, we expect to reduce the incidence of moderate to severe chronic GVHD to 5%. If successful, this will provide a better survivorship experience for our patients.”