A Pilot Study of Pre-transplant Immunosuppressive Therapy for Haploidentical Transplants in Patients With Sickle Cell Disease
City of Hope Medical Center
Summary
This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.
Description
All patients will receive an haploidentical hematopoietic stem cell transplant with the following conditioning and GvHD prevention: Pre-transplant immunosuppressive therapy: 2 cycles of Fludarabine and Dexamethasone x 5 days each cycle Conditioning regimen: rATG daily x 3 days, Fludarabine daily x 6 days and Busulfan daily x 4 days GVHD prophylaxis: Cyclophosphamide day +3 and +4, Tacrolimus and Mycophenolate mofetil
Eligibility
- Age range
- 1–30 years
- Sex
- All
- Healthy volunteers
- No
Inclusion criteria * Diagnosis: Patients with sickle cell anemia (Hgb SS or SB° Thalassemia) with baseline Hgb S more than 60%. * Disease status: * Significant neurologic event (stroke) or any neurological deficit lasting \> 24 hours; or increased transcranial Doppler velocity (\>200 m/s). * History of one or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea). * History of one or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollmen…
Interventions
- BiologicalHematopoietic stem cell transplantation
Haploidentical stem cell transplantation with pre-transplant immunosuppressive therapy
Location
- City of Hope Medical CenterDuarte, California