Intergroup rhabdomyosarcoma study-IV: Results for patients with nonmetastatic disease
Intergroup rhabdomyosarcoma study-IV: Results for patients with nonmetastatic disease
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DOI：
10.1200/jco.2001.19.12.3091
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发表时间：
2001-06-15
影响因子：
45.3
通讯作者：
Donaldson, SS
中科院分区：
文献类型：
--
作者：
Crist, WM;Anderson, JR;Donaldson, SS
目的：本研究的目的是通过比较手术、放疗（RT）和化疗等基于风险的治疗方案来改善儿童横纹肌肉瘤的预后。883例既往未经治疗的非转移性横纹肌肉瘤合格患者进入组间横纹肌肉瘤研究-IV（IRS-IV）（1991年至1997年）手术后，并随机治疗原发肿瘤部位，组（1至3），和阶段（I至III）。无失败生存期（FFS）率和生存期是随机分组之间以及接受IRS-III和IRS-IV治疗的患者亚组之间比较的终点。大多数患者随机接受长春新碱和更生霉素（VA）和环磷酰胺（VAC，n = 235），或VA和异环磷酰胺（VA 1，n = 222）或长春新碱、异环磷酰胺和依他泊苷（VIE，n = 236），第3组肿瘤患者随机接受常规RT（C-RT）与超分割PT（HF-RT）。总的3年FFS和生存率分别为77%和86%。VAC、VAI和VIE的3年FFS率分别为75%、77%和77%（P = 0.42）。HF-RT与C-RT的结果无显著差异（分别为P = 0.85和P = 0.90）。总体而言，IRS-IV期胚胎性肿瘤患者从强化三药化疗中获益（3年FFS，83%），I期或II/III期1/2组疾病患者观察到改善，其中许多患者在IRS-III期接受VA化疗。2/3期、第3组疾病患者的IRS-III和IRS-IV结局相似。非随机分组患者亚组的3年FFS中，75%的患者出现肾脏异常;睾丸旁，第1组病例为81%;第1/2组眼眶或眼睑肿瘤为91%。如果患者年龄超过10岁，睾丸旁原发灶的预后较差（3年FFS，63% vs 90%）。结论：YAC、VAI或VIE联合手术（有或无RT）治疗局部或区域性横纹肌肉瘤疗效相当，但对胚胎性肿瘤的疗效优于以往的治疗方法。年龄较小的第1组睾丸旁胚胎性肿瘤患者和所有第1/2组眼眶或眼睑肿瘤患者通常可通过VA化疗沿着第2组疾病的术后RT治愈。J Clin Oncol 19：3091-3102. (C)2001年，美国临床肿瘤学会。
Purpose: The study goal was to improve outcome in children with rhabdomyosarcoma by comparing risk-based regimens of surgery, radiotherapy (RT) and chemotherapy.Patients and Methods: Eight hundred eighty-three previously untreated eligible patients with nonmetastatic rhabdomyosarcoma entered the Intergroup Rhabdomyosarcoma Study-IV (IRS-IV) (1991 to 1997) after surgery and were randomized treatment by primary tumor site, group (1 to 3), and stage (I to III). Failure-free survival (FFS) rates and survival were the end points used in comparisons between randomized groups and between patient subgroups treated on IRS-III and IRS-IV. Most patients were randomized to receive vincristine and dactinomycin (VA) and cyclophosphamide (VAC, n = 235), or VA and ifosfamide (VA1, n = 222), or vincristine, ifosfamide, and etaposide (VIE, n = 236), Patients with group 3 tumors were randomized to receive conventional RT (C-RT) versus hyperfractionated PT (HF-RT).Results: Overall 3-year FFS and survival were 77% and 86%, respectively. Three-year FFS rates with VAC, VAI, and VIE were 75%, 77%, and 77%, respectively (P = .42). No significant difference in outcome was noted with HF-RT versus C-RT (P = .85 and P = .90, respectively). Overall, patients with embryonal tumors benefited from intensive three-drug chemotherapy in IRS-IV (3-year FFS, 83%) The improvement was seen for patients with stage I or stage II/III, group 1/2 disease, many of whom received VA chemotherapy on IRS-III. Patients with stage 2/3, group 3 disease had similar outcomes on IRS-III and IRS-IV. Three-year FFS for the nonrandomized patient subsets wets 75% with renal abnormalities; 81% for paratesticular, group 1 cases; and 91% for group 1/2 orbit or eyelid tumors. Patients with paratesticular primaries had poorer outcomes if they were more than 10 years old (3-year FFS, 63% v 90%). Myelosuppression occurred in most patients, but toxic deaths occurred in less than 1%.Conclusion: YAC and VAI or VIE with surgery (with or without RT), are equally effective for patients with local or regional rhabdomyosarcoma and are more effective for embryonal tumors than therapies used previously. Younger patients with group 1 paratesticular embryonal tumors and all patients with group 1/2 orbit or eyelid tumors can usually be cured with VA chemotherapy along with postoperative RT for group 2 disease. J Clin Oncol 19:3091-3102. (C) 2001 by American Society of Clinical Oncology.