Oral vitamin A and GVHD prevention in children and young adults
Khandelwal P, Langenberg L, Luebbering N, et al. A Randomized Phase 2 Trial of Oral Vitamin A for Graft-Versus-Host Disease in Children and Young Adults. Blood. 2024; (doi: 10.1182/blood.2023022865).
Evidence suggests vitamin A, which promotes gastrointestinal (GI) homeostasis, may reduce graft-versus-host disease (GVHD) in young hematopoietic stem cell transplantation (HSCT) recipients if administered pre-procedure. The Phase II trial involved 80 children and young adults randomized into two treatment arms. Half of the participants received 4,000 IU/kg, maximum 250,000 IU of vitamin A in a single oral dose before HSCT conditioning. The other half received placebo. Per an intent-to-treat analysis, the primary outcome — GVHD incidence at day+100 — was 12.5% among vitamin A recipients and 20% among controls. Researchers also calculated the incidence of acute GI GVHD at day+180 and chronic GVHD at 1 year post-transplant, which measured at 2.5% in the vitamin A group vs. 12.5% in the placebo group and 5% in the vitamin A group vs. 15% in the placebo group, respectively. An "as treated" analysis also supported the use of affordable, high-dose vitamin A prior to HSCT, with the cumulative incidence of acute GI GVHD at day+180 holding at 0% vs. 12.5% and the incidence of chronic GVHD reaching just 2.7% vs. 15%. Only one patient experienced potential treatment-related toxicity: grade 3 hyperbilirubinemia, which resolved on its own.