In Utero Hematopoietic Stem Cell Transplantation For The Treatment Of Fetuses With Alpha Thalassemia Major
Untreated alpha thalassemia major is almost universally fatal in utero. Current treatment requires in utero blood transfusions and monthly blood transfusions for life or a bone marrow transplant if a suitable donor is identified. The proposed treatment is a maternal bone marrow transplant in utero that takes advantage of maternal-fetal immune tolerance, and may provide a definitive cure.
Single arm, non-randomised study.
Safety and feasibility, efficacy.
Patient recruitment initiating.