Sickle Cell Disease Conditioning for Bone Marrow Transplant
Status:
Completed
Trial end date:
2016-01-11
Target enrollment:
Participant gender:
Summary
Most bone marrow transplants for children with sickle cell disease are performed using high
doses of two chemotherapy agents: busulfan and cyclophosphamide for the pre-transplant
conditioning. This approach produces cure in most cases (approximately 95%). It, however, has
serious side effects, including seizures and infertility. The primary goal of this study is
to determine how much we can lower the dosages of busulfan and cyclophosphamide by
incorporating fludarabine, a safer chemotherapy agent, into conditioning. The secondary goal
is to develop a better understanding of how bone marrow transplants cause neurologic problems
like seizures.