Non-Myeloablative Conditioning and Bone Marrow Transplantation
Status:
Active, not recruiting
Trial end date:
2022-03-01
Target enrollment:
Participant gender:
Summary
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of
hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear
that alloBMT can give to these patients an improvement in their disease, myeloablative
transplants have important toxicities and mortalities associated. The lack of suitable donors
continues to be a limit to access to transplantation. Substantial progress has been made
recently in the development of pre-treatment regimens that facilitate the sustained
engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly
immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or
total body irradiation without endangering the sustained engraftment of HLA-identical
allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell
transplantation suggest that the procedure can be performed in patients who are ineligible
for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies
can be obtained.