This study is designed to estimate the efficacy and toxicity of familial HLA mismatched bone marrow transplants in patients with non-malignant disease who are less than 21 years of age and could benefit from the procedure.
Nonmalignant disorder requiring bone marrow transplant including bone marrow failure syndromes, metabolic disorders, immunologic disorders, or hemoglobinopathy
Overt or silent stroke or persistently elevated transcranial doppler velocities despite transfusion therapy
Recurrent acute chest syndrome with significant respiratory compromise each time
Sickle nephropathy
Patients who have an HLA-identical sibling who is able and willing to donate bone marrow
Patients with cirrhosis or established bridging fibrosis of the liver or active hepatitis
Uncontrolled bacterial, viral, or fungal infection within 6 weeks prior to enrollment
Evidence of HIV infection or known HIV positive serology
Washington University School of Medicine
Lead sponsor