Allogeneic stem cell transplantation for sickle cell
Posted Apr 15 2011 1:56pm
Allogeneic hematopoietic stem-cell transplantation (HSCT) is a potentially curative treatment option for selected individuals with sickle cell disease or thalassemia major.In practice, only children are candidates before they accumulate too much end organ damage to be transplant candidates.There have not and are not likely to be randomized studies but the NMDP (2008) lists hemoglobinopathies, including sickle cell disease (SCD) and thalassemia major, as diseases which are treatable by allogeneic HSCT. The NHLBI (2007) notes that bone marrow transplantation can be a very effective treatment for sickle cell anemia, but because of its risks, only some individuals can or should have this procedure. The NHLBI says that HSCT for SCD should be considered for children who experience significant, noninfectious complications caused by vaso-occlusion. For severely affected children who have human leukocyte antigen- (HLA) identical sibling donors, families should be informed about benefits, risks and treatment alternatives. Regarding thalassemia, the NHLBI (2008) notes that stem cell transplantation is the only treatment that can cure thalassemia; it is most successful in children.
The published peer-reviewed scientific literature supports the safety and effectiveness of myeloablative allogeneic hematopoietic stem-cell transplantation (HSCT) for the treatment of selected children and young adults with sickle cell disease (SCD) and thalassemia major.
Bernaudin F, Socie G, Kuentz M, Chevret S, Duvall M, Bertrand Y, et al. Long-term results of related myeloablative stem-cell transplantation to cure sickle cell disease. Blood. 2007 Oct 1;110(7):2749-56. Epub 2007 Jul 2.
Bhatia M, Walters MC. Hematopoietic stem cell transplantation for thalassemia and sickle cell disease: past, present and future. Bone Marrow Transplant. 2008 Jan;42(2):109-17.