Bone marrow transplant has been shown to be curative for sickle cell disease in the majority of children and young adults treated, whereas standard treatment options are non-curative. Patients with persistent sickle cell disease have diminished health and quality of life as they age and typically die from their disease in mid-adulthood. Bone marrow transplant for sickle cell disease in young adults has been done in limited fashion and as such the benefit of doing so is not yet fully known.
This clinical trial is for men and women age 18 to 40 years of age with severe sickle cell disease and for whom hematopoietic cell transplantation is a treatment option. It will compare patients who have had bone marrow transplant for sickle cell disease with patients who are treated with the standard treatment for sickle cell disease.
Patients with sickle cell disease who may potentially benefit from bone marrow transplant will be enrolled into the study before searching for an eligible donor. Once enrolled into the study a donor search will be conducted and patients who identify an eligible donor will proceed to bone marrow transplant. Patients who are unable to identify a donor will be followed with standard treatment options. Patients will be closely monitored for 2 years after study entry and/or bone marrow transplant, but will be observed for up to 10 years to assess for long term health benefits of their treatments. Patients who have a bone marrow transplant will be treated with a variety of medications designed to help the patient tolerate the transplant procedure and allow for ‘acceptance’ of the transplanted cells. Patients who do not receive a bone marrow transplant will be treated with the standard treatment options for sickle cell disease.
- Men and women ages 18 – 40 years of age
- Severe sickle cell disease (ie: 3 or more pain crisis in the last year, history of stroke or other neurologic event, regular blood transfusions, etc)
- Patients who are not severely physically impaired due to poor health
- HIV negative
- Have not had a bone marrow transplant previously
- Able to tolerate a bone marrow transplant – as determined by your physician
- Detailed eligibility reviewed when you contact the study team