New drug could make stem cell transplants safer for rare immune disorder

This phase II trial tests whether a new drug called Briquilimab can make stem cell transplants safer for people with GATA2 deficiency, a rare genetic condition that weakens the immune system and raises the risk of infections and blood cancers. The study involves 13 participants aged 6 to 70 who receive Briquilimab before a donor stem cell transplant to help their body accept the new cells. Researchers will check if the transplant is successful by 100 days and one year after the procedure.