Trial Information

Summary: A Research Study to Test the Safety of Reinfusing Autologous Bone Marrow, Cord Blood or Peripheral Blood Cells in Patients With Fanconi Anemia

What is the purpose of this study?

The primary purpose of this research study is to test the safety of reinfusing (giving back) autologous (one's own) bone marrow, cord blood, or peripheral blood cells. The research will also help determine if the reinfusion will help your or your child's blood counts.

Who can participate?

The study is open to patients with Fanconi anemia who have already undergone a bone marrow harvest, cord blood collection, or peripheral stem cell harvest and have the cells cryopreserved (frozen).

Patients must have peripheral blood cell counts that meet the following criteria:

• Absolute neutrophil (granulocyte) count of less than 500/mm3 or
• Platelet count less than 20,000/mm3 or
• Hemoglobin less than 8 gm/dl.

Patients may be receiving androgen therapy only if they have received these drugs for at least three months prior to study enrollment.

What is involved?

If accepted for enrollment in the study, a history, physical exam, and laboratory work consisting of a complete (CBC) and chemistries are required prior to the reinfusion of cells as well as a bone marrow aspirate and biopsy. The actual reinfusion of cells is required to be done at Cincinnati Children's Hospital General Clinical Research Center in Cincinnati, Ohio. Following the reinfusion of cells, a CBC will be required twice weekly for one month; weekly for three months; then every two weeks until one year post-infusion. A bone marrow aspirate will be required at 1, 3, and 6 months post-infusion. The CBC's and bone marrow studies may be done by your local physician.

What are the benefits?

By giving back stored blood cells, it may be possible to restore some marrow function, which would produce adequate levels of red cells, white cells, and platelets; however, it is possible that there will be no direct benefit from participating in this study.

What are the risks?

The most common reaction to stem cell infusions are fever, chills, and allergic reaction. Other possible side effects include increased blood pressure, infection, seizures, bad breath, kidney failure and destruction of red blood cells. Risks associated with the blood draws and bone marrow aspiration/biopsy include pain, bleeding and infection. There may also be risks and discomforts that are not yet known.

Will I be paid for participating in this study?

No. All care that is provided at Cincinnati Children's Hospital Medical Center General Clinical Research Center in Cincinnati, Ohio will be paid for by research funds. Any care not provided at this facility may be your or your insurance carriers responsibility.

If you would like to learn more about participating in this study, please send an e-mail message using the form below.