Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
About this trial
This is an interventional treatment trial for Sickle Cell Anemia
Eligibility Criteria
Inclusion: Patients with a haploidentical related HLA donor and hemoglobin SS, hemoglobin SC, or hemoglobin Sb0/+ thalassemia and at least one of the following conditions: previous central nervous system vaso-occlusive episode with or without residual neurologic findings; frequent painful vaso-occlusive episodes which significantly interfere with normal life activities and which necessitate chronic transfusion therapy; recurrent SCD chest syndrome events, which necessitate chronic transfusion therapy; severe anemia, which prevents acceptable quality of life and necessitates chronic transfusion therapy. Patients with a haploidentical related HLA donor and homozygous b0/+ thalassemia or severe variants of b0/+ thalassemia and require chronic transfusion therapy. Women of childbearing potential must have a negative pregnancy test. Between the ages of birth and 65 years. Exclusion: HLA identical or 5/6 HLA matched sibling donor Biopsy proven chronic active hepatitis or portal fibrosis. SCD chronic lung disease > stage 3 Severe renal dysfunction defined as creatinine clearance <40 ml/min/1.73 M2. Severe cardiac dysfunction defined as shortening fraction <25%. HIV infection. Unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate Stem Cell Transplant. Patient or guardian(s) unable to understand the nature and risks inherent in the stem cell transplant process. Pregnant or lactating females and those unwilling to use acceptable contraception.
Sites / Locations
- Texas Children's Hospital
- The Methodist Hospital