Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Issue:  Bone marrow transplantation has been used increasingly for the long-term treatment and cure of SCD and beta thalassemia.  However, severe side effects can occur as a result of the high-intensity chemotherapy that is used to prepare the patient for transplant.  Can use of lower-intensity chemotherapy result in successful bone marrow replacement with less-severe side effects but permanent control of the disease?

Participants:  Patients with SCD or thalassemia, 1 month to 21 years of age, who are highly symptomatic

Comparison:   transplant of stem cells, from either a family-related or cord-bloodBmatched donor, following moderate-intensity chemotherapy (the experimental treatment)

versus

(no control group)

More information:  http://www.clinicaltrials.gov/ct2/show/NCT00408447