Haploidentical Bone Marrow Transplantation for Sickle Cell Disease.
Обработка статьи · этапы, ошибки и управление
Ошибки и ограничения
История · было → стало
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Kassim Adetola A, Walters Mark C, Eapen Mary, Smith Madoc, Logan Brent R, Solh Melhem, McKinney Christopher, Nieder Michael, Ross Maureen, Kent Michael, Abusin Ghada A, Mallhi Kanwaldeep, Silva Jorge Galvez, Shaughnessy Paul, Kanter Julie, Haines Hilary, Farah Rafic, Khaled Yasser A, Ritzau Nicole, Mendizabal Adam, Abraham Allistair, Bollard Catherine, Cooke Kenneth, de la Fuente Josu, Hanna Rabi, Horowitz Mary M, Jordan Lori C, Bakshi Nitya, Krishnamurti Lakshmanan, Leifer Eric, Mahadeo Kris Michael, Shenoy Shalini, Jones Richard J, DeBaun Michael R, Brodsky Robert A
Аннотация
METHODS: In a phase 2, open-label, single-arm, multicenter study, 54 eligible participants from 19 U.S. centers were enrolled. Of these, 42 (78%) received transplantation with conditioning including antithymocyte globulin, fludarabine, cyclophosphamide, thiotepa, and total body irradiation. GVHD prophylaxis included posttransplant cyclophosphamide, mycophenolate mofetil, and sirolimus. The primary outcome was event-free survival at 2 years, while secondary outcomes included overall survival and other transplant-related end points.
RESULTS: The median age at enrollment was 22.8 years (range, 15.5 to 43.2), and the median follow-up period was 37.2 months (range, 20.4 to 56.4). The 2-year event-free and overall survival rates were 88.0% (95% confidence interval [CI], 73.5 to 94.8%) and 95.0% (95% CI, 81.5 to 98.7%), respectively. Two participants experienced primary and another secondary graft failure. The incidence of grade-3-to-4 acute GVHD at day 100 was 4.8% (95% CI, 0.9 to 14.4%), while the 2-year chronic GVHD rate was 22.4% (95% CI, 10.9 to 36.4%). Two of the four reported deaths were due to early infectious complications.
CONCLUSIONS: HLA-haploidentical BMT is an accessible and potentially curative therapy for adults with sickle cell disease. Adverse events were those anticipated from this procedure, including GVHD. (Funded by the National Heart, Lung, and Blood Institute and the National Cancer Institute; BMT CTN 1507; ClinicalTrials.gov number, NCT03263559).