Vitamin A May Reduce CVHD Risk After Stem Cell Transplantation
Research By: Pooja Khandelwal, MD | Stella Davies, MBBS, PhD, MRCP
Post Date: March 21, 2024 | Publish Date: March 21, 2024
CBDI: Bone Marrow Transplant and Immune Deficiency | Top Scientific Achievement
A randomized phase 2 clinical trial led by stem cell transplant experts at Cincinnati Children’s reports that high-dose oral vitamin A reduces the incidence of acute and chronic graft-versus-host disease (GVHD) and does so with low risks of toxicity.
Developing GVHD is the most serious risk for patients receiving hematopoietic stem cell transplantation (HSCT). Acute GVHD can occur in up to 30% of cases involving related donors and up to 70% for unrelated donors, according to the Leukemia & Lymphoma Society.
This study involved 80 children and young adults. At 100 days post-transplant the incidence of acute GVHD was 12.5% in the vitamin A group compared to 20% in the placebo group, but that difference was not statistically significant.
However, the study found significant reductions in chronic GVHD, which occurred in just 5% of the patients in the vitamin A group versus 15% in the placebo group at one year after treatment. In addition, acute gastrointestinal GVHD was 0% in the vitamin A group compared to 12.5% in the placebo group.
“The vitamin A group exhibited lower levels of proinflammatory markers and T-cell trafficking indicators, suggesting a more favorable immune response and gut microbiome,” says corresponding author Pooja Khandelwal, MD.
“These results are encouraging because high-dose vitamin A costs $1.25/patient, making it an affordable and safe intervention to reduce GVHD and could be adopted universally, especially in low-resource countries,” says senior co-author Stella Davies, MBBS, PhD, MRCP.
Next, Khandelwal plans to use funding from the Leukemia & Lymphoma Society to determine if the results can be replicated at three collaborating adult bone marrow transplant centers.
Cincinnati Children’s co-authors included Lucille Langenberg, BS, Nathan Luebbering, MS, Kelly Lake, BS, Abigail Butcher, Kylie Bota, RD, Kristie Ramos, MD, Cynthia Taggart,RD, Ashley Teusink-Cross, PharmD, MBA , Jane Koo, MD, Gregory Wallace, MD, Lindsey Romick-Rosendale, PhD, Miki Watanabe-Chailland, PhD, David Haslam, MD, and Adam Lane, PhD. Contributors also included experts at The Ohio State Comprehensive Cancer Center and support from the Cincinnati Children’s NMR–based metabolomics facility.
Explore the 2024 Research Annual Report
| Original title: | A randomized phase 2 trial of oral vitamin A for graft-versus-host disease in children and young adults |
| Published in: | Blood |
| Publish date: | March 21, 2024 |
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The Research Horizons blog features news and insights about the latest discoveries and innovations developed by the scientists of Cincinnati Children's. This blog does not provide medical advice, diagnosis, or treatment.
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