Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs

Inclusion Criteria

• •Patients with sickle cell disease (SCD) 1-25 years of age with an HLA-identical, but unrelated, donor or 1 human leukocyte antigen (HLA) allele mismatched bone marrow or up to 2 HLA antigen mismatched umbilical cord blood (UCB) donor with one or more of the following:
• •* Stroke, central nervous system (CNS) hemorrhage or a neurologic event lasting longer than 24 hours.
• •* Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions.
• •* Recurrent vaso-occlusive pain, 3 or more episodes per year for 3 years or more years; or recurrent priapism.
• •* Impaired neuropsychological function and/or abnormal cerebral MRI scan or abnormal transcranial Doppler (TCD).
• •* Stage I or II sickle lung disease.
• •* Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate (GFR) 30-50% of the predicted normal value).
• •* Bilateral proliferative retinopathy and major visual impairment in at least one eye.
• •* Osteonecrosis of multiple joints with documented destructive changes.
• •* Requirement for chronic transfusions but with RBC alloimmunization \>2 antibodies during long term transfusion therapy.
• •* Failure of hydroxyurea (HU) therapy.
• •Patients aged 0-21 years with transfusion dependent alpha- or beta-thalassemia who have an HLA-identical or 1 HLA allele mismatched bone marrow or up to 2 HLA mismatched UCB donor.