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We conducted a multicenter pilot investigation of the safety and feasibility of bone marrow transplantation (BMT) in adults with severe sickle cell disease (SCD) (NCT 01565616) using a reduced toxicity preparative regimen of busulfan (13.2 mg/kg), fludarabine (175 mg/m2) and thymoglobulin (6 mg/kg) and cyclosporine or tacrolimus and methotrexate for graft‐vs‐host disease (GVHD) prophylaxis. Twenty‐two...
Neonatal alloimmune neutropenia (NAIN) is a rare cause of congenital neutropenia seen in <1% of births. Significant morbidity, usually infections, may result from this disease. The pathophysiology of NAIN, mediated by maternal antibodies crossing the placenta to destroy fetal cells expressing paternal antigens, is similar to that of neonatal alloimmune thrombocytopenia, as well as Rh/ABO hemolytic...
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