Bradley M B, Satwani P, Baldinger L, Morris E, van de Ven C, Del Toro G, Garvin J, George D, Bhatia M, Roman E, Baxter-Lowe L A, Schwartz J, Qualter E, Hawks R, Wolownik K, Foley S, Militano O, Leclere J, Cheung Y-K, Cairo M S
Department of Pediatrics, Columbia University, New York, NY 10032, USA.
Bone Marrow Transplant. 2007 Oct;40(7):621-31. doi: 10.1038/sj.bmt.1705785. Epub 2007 Jul 30.
There is a significant amount of morbidity and mortality following myeloablative umbilical cord blood transplantation (UCBT). Reduced intensity (RI) conditioning offers an alternative to myeloablative conditioning before UCBT. We investigated RI-UCBT in 21 children and adolescents with malignant (n=14), and non-malignant diseases (n=7). RI conditioning consisted of fludarabine (150-180 mg/m2) with either busulfan (< or = 8 mg/kg)+rabbit antithymocyte globulin (R-ATG; n=16) or cyclophosphamide+R-ATG+/-etoposide (n=5). Human leukocyte antigen match: 4/6 (n=13), 5/6 (n=5) and 6/6 (n=3). The median total nucleated cell and CD34+ cell dose per kilogram were 3.58 x 10(7) and 2.54 x 10(5), respectively. The median time for neutrophil and platelet engraftment was 17.5 and 52 days, respectively. There were six primary graft failures (chronic myelogenous leukemia (CML), beta-thalassemia, hemophagocytic lymphohistiocytosis (HLH) and myelodysplastic syndrome (MDS)). The probability of developing grade II to grade IV acute graft-versus-host disease (GVHD) and chronic GVHD was 28.6 and 16.7%, respectively. Incidence of transplant-related mortality (TRM) was 14%. The 5 years overall survival (OS) in all patients was 59.8%. The 5 years OS for patients with average versus poor-risk malignancy was 77.8 versus 22.2% (P=0.03). RI-UCBT may result in graft failure in specific high-risk chemo-naïve patients (CML, beta-thalassemia, HLH and MDS), but in more heavily pretreated pediatric and adolescent recipients results in rapid engraftment and may be associated with decreased severe GVHD and TRM.
清髓性脐带血移植（UCBT）后存在大量发病和死亡情况。降低强度（RI）预处理为UCBT前的清髓性预处理提供了一种替代方案。我们对21例患有恶性疾病（n = 14）和非恶性疾病（n = 7）的儿童及青少年进行了RI - UCBT研究。RI预处理包括氟达拉滨（150 - 180 mg/m²）联合白消安（≤8 mg/kg）+兔抗胸腺细胞球蛋白（R - ATG；n = 16）或环磷酰胺+R - ATG±依托泊苷（n = 5）。人类白细胞抗原匹配情况：4/6（n = 13）、5/6（n = 5）和6/6（n = 3）。每千克的中位总核细胞和CD34⁺细胞剂量分别为3.58×10⁷和2.54×10⁵。中性粒细胞和血小板植入的中位时间分别为17.5天和52天。有6例原发性移植物失败（慢性粒细胞白血病（CML）、β地中海贫血、噬血细胞性淋巴组织细胞增生症（HLH）和骨髓增生异常综合征（MDS））。发生II至IV级急性移植物抗宿主病（GVHD）和慢性GVHD的概率分别为28.6%和16.7%。移植相关死亡率（TRM）为14%。所有患者的5年总生存率（OS）为59.8%。中危与高危恶性肿瘤患者的5年OS分别为77.8%和22.2%（P = 0.03）。RI - UCBT可能会导致特定高危初治患者（CML、β地中海贫血、HLH和MDS）出现移植物失败，但在预处理更严重的儿科和青少年受者中会导致快速植入，并且可能与严重GVHD和TRM的减少有关。
