Paillard C, Rochette E, Lutz P, Bertrand Y, Michel G, Bordigoni P, Dalle J H, Rohrlich P, Vannier J P, Perel Y, Plantaz D, Leverger G, Sirvent A, Dore E, Isfan F, Merlin E, Pereira B, Halle P, Rabiau N, Kanold J, Deméocq F
CHU Clermont-Ferrand, Centre Régional de Cancérologie et Thérapie Cellulaire Pédiatrique, Clermont-Ferrand, France.
Bone Marrow Transplant. 2013 Nov;48(11):1401-8. doi: 10.1038/bmt.2013.82. Epub 2013 Jun 10.
We report French prospective experience with reduced-intensity conditioning-allo-SCT in 46 patients (median age: 15.5 years, 4.8-20.2) presenting high-risk AL (n=11), Hodgkin's lymphoma (n=15) or solid tumors (n=20). Graft sources were BM (n=21), PBSC (n=20) and cord blood (CB; n=5) from related (n=20) or unrelated (n=26) donors. For CB grafts, only one patient out of five achieved sustained engraftment. For PBSC/BM grafts, engraftment rate was 95%, hematopoietic recovery times were not significantly different between BM, PBSC, sibling or unrelated grafts, day+100. Full donor chimerism was achieved in 94% of patients, and incidences of primary acute GVHD and chronic GVHD were 49% and 14%, respectively. Underlying disease was fatal in 39% of patients. TRM was 6.9%. Three-year OS was 49.15%. OS and EFS were not significantly different between patients transplanted with different grafts and with or without primary GVHD. Patients with solid tumor or measurable disease at transplant had poorer outcomes. Three-year EFS: 33.3% for ALL, 75.0% for AML, 51.8% for Hodgkin's lymphoma, 28.6% for neuroblastoma and 22.2% for sarcoma patients. This multicentre study concluded that Bu/fludarabine/anti-thymocyte globulin conditioning with PB or BM, related or unrelated grafts in patients with various malignancies at high-risk for transplantation toxicity results in high engraftment rates, low TRM and acceptable survival.
我们报告了46例患者（中位年龄：15.5岁，4.8 - 20.2岁）接受低强度预处理异基因造血干细胞移植（allo - SCT）的法国前瞻性经验，这些患者患有高危急性白血病（n = 11）、霍奇金淋巴瘤（n = 15）或实体瘤（n = 20）。移植物来源为骨髓（n = 21）、外周血干细胞（PBSC；n = 20）和脐带血（CB；n = 5），供者为相关供者（n = 20）或无关供者（n = 26）。对于脐带血移植物，5例患者中只有1例实现了持续植入。对于外周血干细胞/骨髓移植物，植入率为95%，骨髓、外周血干细胞、同胞或无关供者移植物之间的造血恢复时间在第100天无显著差异。94%的患者实现了完全供者嵌合，原发性急性移植物抗宿主病（GVHD）和慢性GVHD的发生率分别为49%和14%。39%的患者潜在疾病导致死亡。移植相关死亡率（TRM）为6.9%。三年总生存率（OS）为49.15%。接受不同移植物以及有或无原发性GVHD的患者之间，OS和无事件生存率（EFS）无显著差异。移植时患有实体瘤或可测量疾病的患者预后较差。三年EFS：急性淋巴细胞白血病（ALL）为33.3%，急性髓细胞白血病（AML）为75.0%，霍奇金淋巴瘤为51.8%，神经母细胞瘤为28.6%，肉瘤患者为22.2%。这项多中心研究得出结论，对于有移植毒性高风险的各种恶性肿瘤患者，采用白消安/氟达拉滨/抗胸腺细胞球蛋白预处理联合外周血或骨髓、相关或无关供者移植物，可实现高植入率、低TRM和可接受的生存率。
