A Single-Center Experience Comparing Alemtuzumab, Fludarabine, and Melphalan Reduced-Intensity Conditioning with Myeloablative Busulfan, Cyclophosphamide, and Antithymocyte Globulin for Chronic Granulomatous Disease

摘要: Abstract Myeloablative conditioning (MAC) regimens are commonly used in transplantation for chronic granulomatous disease (CGD) but associated with toxicity. Reduced-intensity (RIC) have lower toxicity may fail to achieve stable donor chimerism. We report a comparison between 4 patients who received RIC regimen consisting of alemtuzumab (1 mg/kg), fludarabine (150 mg/m2), and melphalan (140 mg/m2) 14 MAC busulfan (area under the curve, 1800 2000 µMol/min twice daily × 4 days), cyclophosphamide (50 mg/kg/day × 4), antithymocyte globulin (15 mg/kg daily on days −2 −1, then +1 +2). Seventy-five percent (n = 3) developed mixed chimerism needed either withdrawal immune suppression (n = 1) or additional stem cell products (n = 2) Ninety-two (n = 13) group maintained >95% Complications included acute graft-versus-host (MAC 64%, 0%), 28%, sinusoidal obstructive syndrome 7%, bacteremia 42%, fungemia 14%, viral 25%), death 21%, 0%). A has frequently requires interventions maintain compared CGD.