T-Cell Depleted, Alternative Donor Transplant in Pediatric and Adult Patients With Severe Sickle Cell Disease (SCD) and Other Transfusion-Dependent Anemias
Paul Szabolcs
Summary
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.
Description
CD3/CD19 depletion of mismatched donor grafts in the setting of reduced intensity, immune-ablative conditioning for patients with sickle cell disease and other transfusion-dependent anemias should sufficiently achieve engraftment while decreasing the incidence of treatment-related toxicities and achieving an acceptable incidence of graft versus host disease. Utilizing mismatched unrelated volunteer donors and haploidentical related donors will increase the number of patients able to undergo hematopoietic stem cell transplant (HSCT) for these diseases. Additionally, the institutional availabili…
Eligibility
- Age range
- 5–40 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria 1. Patient, parent, or legal guardian must have given written informed consent and/or assent according to FDA guidelines. 2. Ages 5 years to 40 years, at time of consent. 3. Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following: * Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years. * Recurrent acute chest syndrome (ACS) despite supportive care, minimum o…
Interventions
- BiologicalCD3/CD19 depleted leukocytes
Negative selection for CD3+/CD19+ cells will be performed on the CliniMACS® depletion device.
- BiologicalCD45RA depleted leukocytes
Negative selection for CD45RA will be performed on the CliniMACS® depletion device.
- DrugHydroxyurea
Sickle Cell Disease Conditioning
- DrugRituximab
Sickle Cell Disease Conditioning
- DrugAlemtuzumab
Sickle Cell Disease Conditioning
- DrugFludarabine
Sickle Cell Disease Conditioning
- DrugThiotepa
Location
- Children's Hospital of Pittsburgh of UPMCPittsburgh, Pennsylvania