Verdeguer A, Pardo N, Madero L, Martinez A, Bureo E, Fernández J M, Muñoz A, Olivé T, Fernández-Delgado R, Cubells J, Diaz M A, Sastre A
Hospital Infantil La Fe, Valencia, Spain.
Bone Marrow Transplant. 2000 Jan;25(1):31-4. doi: 10.1038/sj.bmt.1702094.
This study evaluates the outcome of myeloablative chemo-radiotherapy and autologous stem cell transplantation (ASCT) in children with Hodgkin's disease (HD). Twenty children aged 5 to 18 years (median 10.8 years) at diagnosis, with relapsed, refractory or very poor prognosis HD, underwent ASCT in eight hospitals of our country. Status at transplant was: second complete remission (CR2): n = 12; further CR (CR >2): n = 3, partial remission (PR): n = 2, relapse: n = 2 and first CR (CR1): n = 1. Eighteen patients received chemotherapy-based conditioning regimens: cyclophosphamide, carmustine and etoposide (CBV): 11 (55%), carmustine, etoposide, cytarabine and melphalan (BEAM): 5, other: 2; and two patients were conditioned with TBI/Cy. Peripheral blood (PB) was the source of progenitor cells in 12 patients, BM in seven, and BM plus PB, in one. All patients engrafted. One patient died of sepsis and multiorgan failure at day 28 after transplantation. All four patients with measurable disease (PR or relapse) at transplantation attained complete remission. Five patients relapsed 5-34 months after transplant (median: 11 months). Eighteen children remain alive with a median survival time of 40 months. The projected 5-year overall survival and event-free survival (EFS) rates were 0.95 and 0.62. High-dose therapy with stem cell rescue can lead to durable remissions in children with advanced HD. Bone Marrow Transplantation (2000) 25, 31-34.
本研究评估了霍奇金病（HD）患儿接受清髓性放化疗及自体干细胞移植（ASCT）的疗效。20例诊断时年龄为5至18岁（中位年龄10.8岁）、患有复发性、难治性或预后极差的HD患儿，在我国8家医院接受了ASCT。移植时的状态为：第二次完全缓解（CR2）：n = 12；进一步完全缓解（CR>2）：n = 3，部分缓解（PR）：n = 2，复发：n = 2，首次完全缓解（CR1）：n = 1。18例患者接受了以化疗为基础的预处理方案：环磷酰胺、卡莫司汀和依托泊苷（CBV）：11例（55%），卡莫司汀、依托泊苷、阿糖胞苷和美法仑（BEAM）：5例，其他：2例；2例患者接受了全身照射/环磷酰胺预处理。12例患者的祖细胞来源为外周血（PB），7例为骨髓，1例为骨髓加PB。所有患者均成功植入。1例患者在移植后第28天死于败血症和多器官功能衰竭。移植时所有4例有可测量疾病（PR或复发）的患者均获得完全缓解。5例患者在移植后5至34个月复发（中位时间：11个月）。18例儿童存活，中位生存时间为40个月。预计5年总生存率和无事件生存率（EFS）分别为0.95和0.62。高剂量治疗联合干细胞救援可使晚期HD患儿获得持久缓解。《骨髓移植》（2000年）25卷，31 - 34页。
