Long-term complications that may be related to chemotherapy in preparation for transplantation, bone marrow transplant, or autologous gene therapy in sickle cell patients are unknown. The purpose of this study is to answer questions about the long-term impact of having a bone marrow transplant or autologous gene therapy to treat sickle cell disease and to compare the health outcomes to individuals who have sickle cell disease but have never had a bone marrow transplant or autologous gene therapy. The study also want to compare health outcomes of those who have received a bone marrow transplant or autologous gene therapy to a healthy sibling if they had a non-related donor or autologous gene therapy. The study will also compare health to that of donor siblings.
Thank you for your interest, but this study is recruiting by invitation only.
United States (Nationwide)
Kimberly Kasow
Pediatrics - Hematology/Oncology
Clinical or Medical
Observational
Blood Conditions
Chronic Conditions
20-3471