They are usually considered only for children younger than 16 who have: footnote 1 Severe sickle cell disease complications, including repeat strokes , episodes of acute chest syndrome, and painful events . The person with sickle cell disease is exposed to chemotherapy. Stem cell transplants offer a potential cure for a child's sickle cell disease. Sickle cell disease recurred in approximately 10% of patients after transplantation.
Interestingly, this study used a non-chemotherapy treatment to prevent rejection: low-dose radiation combined with two immunosuppressive drugs (alemtuzumab and sirolimus). A Blood Stem Cell Transplant for Sickle Cell Disease The safety and scientific validity of this study is the responsibility of the study sponsor and investigators.
Stem cell transplants offer a potential cure for a child's sickle cell disease. A Phase 2 study (NCT00061568) evaluated the safety and efficacy of stem cell transplantation from a matched-related donor in 30 patients, ages 16-65, with severe sickle cell anemia. The combined results of 3 level III studies have demonstrated that more than 90% of patients survive and approximately 85% survive free from sickle cell disease after transplantation with a period of followup that extends to 11 years. This destroys the stem cells present in the bone marrow, the cells that later go on to become red blood cells (and other types of blood cells). Some adults will also need to take antibiotics, including those who’ve had their spleen removed. If the transplant is successful, the patient is cured from sickle cell disease. They are usually considered only for children younger than 16 who have: footnote 1 Severe sickle cell disease complications, including repeat strokes , episodes of acute chest syndrome, and painful events . Children with sickle cell disease should take penicillin from ages 2 months to 5 years. Currently, the only treatment that can cure sickle cell disease is a bone marrow transplant. People with sickle cell disease interested in joining NIH blood stem-cell transplant studies may call 1-800-411-1222 or visit the NIH clinical trials registry at https://clinicaltrials.gov for more information. † St. Jude Children’s Research Hospital was the fi rst organization to fi nd the cure for sickle cell disease through bone marrow transplant. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Bone marrow (stem cell) transplant is the only treatment available today that can cure sickle cell disease.
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