Parent-To-Child (Haplo) Transplant
- This study uses half-matched Family Donors, also called Familial Haploidentical donors, as bone marrow donors for patients with sickle cell disease.
- Chemotherapy and a single dose of radiation is used to suppress the patient's immune system so that the new donor cells can be given to the patient, ultimately replacing the unhealthy sickle red blood cells with regular red blood cells.
- Donors undergo a two step process- a). stimulation of their bone marrow cells to circulate in their blood and b). collection of their bone marrow cells.
- For more details visit the Haplo Transplant Overview page.
These haploidentical transplants are being offered at several hospitals throughout the country as part of a phase II clinical trial (study) for sickle cell disease. Clinical trials are a part of the FDAs drug approval process to evaluate the safety and effectiveness of new drugs or new combinations of therapies.
You can read more about our trial The Safety and Efficacy of Prophylactic Defibrotide Administration in Children, Adolescents, and Young Adults with Sickle Cell Disease Following Myeloablative Conditioning (MAC) and Haploidentical Stem Cell Transplantation Utilizing CD34 Enrichment and T-Cell (CD3) Addback here or for a printable brochure click here.
While using HLA matched siblings as transplant donors has been successful in curing sickle cell disease, not everyone has a full HLA matched donor available. The purpose of this study is to repeat that same success for children who do not have fully matched donors.
Medical Disclaimer: The pages on this web site are for education and informational purposes only. Do not use this material to diagnose or treat a health problem; it is not a substitute for professional medical care. If you have concerns about a health problem, consult your doctor. This site is not meant as a substitute for the important healthcare discussions that should occur between an individual and their medical team. No liabiity is assumed by the Haplo SCD Consortium for any damages resulting from use or access to information posted on this website.