Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

Fanconi Anemia Clinical Trial

Official title: Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia MT2002-02

Status Completed

Clinical Trial Summary

RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell transplant may make the transplant more likely to work. This may be an effective treatment for patients with high risk Fanconi's anemia.

PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.

Clinical Trial Description

OBJECTIVES:

Primary

• Determine whether the incidence of neutrophil engraftment is acceptable in high-risk patients with Fanconi's anemia treated with busulfan, cyclophosphamide, fludarabine, and antithymocyte globulin followed by allogeneic hematopoietic stem cell transplantation.

Secondary

• Determine the tolerability of mycophenolate mofetil in these patients.

• Determine the incidence of acute and chronic graft-vs-host disease in patients treated with this regimen.

• Determine the incidence of major infections in patients with a history of major infections treated with this regimen.

• Determine the incidence of relapse in patients with refractory anemia with excess blasts, refractory anemia with excess blasts in transformation, or acute myeloid leukemia treated with this regimen

• Determine the probability of 1-year survival of patients treated with this regimen.

OUTLINE: Patients are stratified according to donor/recipient HLA type (identical vs other).

• Cytoreductive combination chemotherapy: Patients receive busulfan intravenously (IV) over 2 hours twice daily on days -7 and -6 and cyclophosphamide IV over 2 hours and fludarabine IV over 30 minutes once daily on days -5 to -2.

• Graft failure prophylaxis: Patients receive methylprednisolone IV twice daily on days -5 to 30 and anti-thymocyte globulin IV over 4-6 hours twice daily on days -5 to -1.

• Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours twice daily on days -3 to 100 (if patient has a matched sibling donor) or days -3 to 180 (if patient has another donor type). Patients also receive mycophenolate mofetil orally or IV twice daily on days -3 to 45.

• Allogeneic hematopoietic stem cell transplantation (HSCT): Patients undergo allogeneic HSCT (using bone marrow or umbilical cord blood) on day 0. Patients receive filgrastim (G-CSF) subcutaneously beginning on day 1 and continuing until blood counts recover.

After completion of study treatment, patients are followed periodically for 3 years. ;

Related Conditions & MeSH terms

• Anemia
• Fanconi Anemia
• Fanconi Syndrome

• NCT number: NCT00258427
• Study type: Interventional
• Source: Masonic Cancer Center, University of Minnesota
• Status: Completed
• Phase: Phase 2
• Start date: March 26, 2002
• Completion date: October 10, 2020