In 1984 an 8 year old girl with sickle cell disease developed acute myeloid leukemia. To treat the cancer, the doctors gave her chemo and a stem cell transplant from her sibling. Not only did this cure her cancer, but it cured her SCD too. She remains disease free nearly three decades later.
Alternative Donor/Unrelated Donor Transplants for the β-Thalassemia and Sickle Cell Disease
Considerable progress with respect to donor source has been achieved in allogeneic stem cell transplant for patients with hemoglobin disorders, with matched sibling donors in the 1980s, matched unrelated donors and cord blood sources in the 1990s, and haploidentical donors in the 2000s. Many studies have solidified hematopoietic progenitors from matched sibling marrow, cord blood, or mobilized peripheral blood as the best source—with the lowest graft rejection and graft versus host disease (GvHD), and highest disease-free survival rates. For patients without HLA-matched sibling donors, but who are otherwise eligible for transplant, fully allelic matched unrelated donor (8/8 HLA-A, B, C, DRB1) appears to be the next b… Continue Reading