MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
This is a treatment guideline for allogeneic hemapoetic stem cell transplant (HSCT) in patients with primary immune deficiencies. Endpoints include time to engraftment, incidence of graft failure, incidence of chimerism at 100 days, 6 months, and 1 year; incidence of acute GVHD at 100 days and chronic GVHD at 6 months and 1 year; incidence of transplant related mortality at 6 months and incidence of disease free survival and overall survival at 6 months. All endpoints will be abstracted from routine data collected by the BMT Database.
• up to 50 years old
• diagnosis of immunodeficiency or histiocytic disorder
• see link to clinicaltrials.gov for complete inclusion criteria
• pregnant or breastfeeding
• active, uncontrolled infection and/or HIV positive
• acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Drug: Alemtuzumab 0.2 mg, Drug: Alemtuzumab 0.3 mg, Drug: Busulfan, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Fludarabine phosphate 30 mg, Drug: Fludarabine phosphate 40 mg, Drug: MESNA, Drug: Melphalan, Biological: Stem Cell Transplantation
Immune Diseases, Rare Diseases
Clinics and Surgery Center (CSC), Allogeneic Hematopoietic Stem Cell Transplant, Primary Immune Deficiencies