Pilot Study of Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients With Life Threatening Hemophagocytic Disorders
OBJECTIVES: I. Determine the efficacy of unrelated donor hematopoietic stem cell transplantation in the treatment of patients with life threatening hemophagocytic disorders.
II. Determine the rate of disease free survival, incidence of graft failure, and incidence of graft versus host disease in these patients after undergoing this treatment regimen.
Graft Versus Host Disease
X-Linked Lymphoproliferative Syndrome
Familial Erythrophagocytic Lymphohistiocytosis
Virus-Associated Hemophagocytic Syndrome
Drug: anti-thymocyte globulin
Procedure: allogeneic hematopoietic stem cell transplantation
|Study Design:||Primary Purpose: Treatment|
|Study Start Date:||March 2000|
PROTOCOL OUTLINE: Patients receive oral busulfan twice a day on days -9 to -6; cyclophosphamide IV over 1 hour on days -5 to -2; etoposide IV over 4 hours on days -5 to -3; and anti-thymocyte globulin IV twice a day on days -2 and -1 and days 1 and 2. Patients undergo allogeneic hematopoietic stem cell transplantation on day 0. Filgrastim (G-CSF) is administered subcutaneously beginning on day 1 and continuing until blood counts recover. Patients receive graft versus host disease prophylaxis with methotrexate IV on days 1, 3, 6, and 11 and cyclosporine IV over 1-4 hours (orally once the patients resumes eating) every 12 hours (every 8 hours for pediatric patients) starting on or prior to day -3 and continuing up to 1 year.
Patients are followed at days 28 and 100, at 6 months and 1 year, and then annually for 5 years.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00006056
|United States, Minnesota|
|Fairview University Medical Center|
|Minneapolis, Minnesota, United States, 55455|
|Study Chair:||K. Scott Baker||Fairview University Medical Center|