NIH
Expanded umbilical cord blood transplant shows success in NIH study
NIH researchers report that an expanded umbilical cord blood transplant achieved a 94% two-year survival rate in patients with severe aplastic anemia. The technique expands donated cord blood stem cells in the lab using nicotinamide, producing a higher-dose graft to accelerate hematopoietic and immune recovery. All patients engrafted successfully, with no graft failures and low rates of graft-versus-host disease. FDA has approved Gamida Cell Ltd.’s omidubicel (Omisirge) therapy for refractory severe aplastic anemia based on these results. The ongoing trial (NCT03173937) will publish full findings next year, but early data suggest this strategy could transform care for patients lacking matched donors.
Source:
(2025, December 12). NIH. Expanded umbilical cord blood transplant achieves 94% survival in severe aplastic anemia patients. https://www.nih.gov/news-events/news-releases/expanded-umbilical-cord-blood-transplant-achieves-94-survival-severe-aplastic-anemia-patients