Treatment of unresectable and metastatic hepatoblastoma: A pediatric oncology group phase II study
Treatment of unresectable and metastatic hepatoblastoma: A pediatric oncology group phase II study
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DOI：
10.1200/jco.2002.07.400
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发表时间：
2002-08-15
影响因子：
45.3
通讯作者：
Bowman, LC
中科院分区：
文献类型：
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作者：
Katzenstein, HM;London, WB;Bowman, LC
目的：目的：评价不可切除或转移性肝母细胞瘤（HB）患儿序贯下列药物治疗后的疾病缓解率、可切除患者比例、无事件生存期（EFS）和毒性反应：（1）卡铂（CARBO）;（2）CARBO、长春新碱和氟尿嘧啶（CARBO-VCR-5-FU）和（3）高剂量顺铂和依托泊苷患者和方法：33例可评估的III期（n = 22）和IV期（n = 11）HB患者依次接受一个疗程的CARBO治疗（700 mg/m2），然后是三个疗程的CARBO（700 mg/m2），第0天，5-FU（1，000 mg/m2/d），连续输注第0 - 2天; VCR（1.5 mg/m2），第0、7和14天。在该治疗后，肿瘤可切除的患者接受手术，然后接受两个额外疗程的CARBO-VCR-5-FU。在CARBO-VCR-5-FU治疗后肿瘤仍不可切除或在该治疗期间未显示缓解或疾病进展的儿童接受两个疗程的HDDP治疗（40毫克/平方米/天），第1至5天;和ETOP（100 mg/m2/d），第2 ~ 4天。III期疾病（n = 22）和IV期疾病（n = 11）的5年EFS估计值分别为59% ± 11%和27% ± 16%（P = 0.037）。33例患者中有27例（82%）对化疗至少有部分缓解; 18例（55%）对CARBO有缓解; 24例（80%）对CARBO和CARBO-VCR-5-FU有缓解; 9例（75%）对HDDP-ETOP有缓解。33例患者中有19例（58%）实现了手术切除，包括22例III期患者中的15例（68%）和4例II期IV期患者（36%）。肿瘤完全切除患者的5年EFS为79% ± 10%.Conclusion：患者序贯接受CARBO、CARBO-VCR-5-FU和HDDP-ETOP治疗的缓解率和EFS与其他治疗方案相当。该方案可有效治疗局部的、不可切除的HB，且毒性可能低于其他方案。对于转移性疾病患者，需要新的方法。(C)2002年，美国临床肿瘤学会。
Purpose: To estimate the disease-response rate, proportion of patients whose tumors can be made resectable, event-free survival (EFS), and toxicity in children with unresectable or metastatic hepatoblastoma (HB) after sequential treatment with the following: (1) carboplatin (CARBO); (2) CARBO, vincristine, and fluorouracil (CARBO-VCR-5-FU), and (3) high-dose cisplatin and etoposide (HDDP-ETOP).Patients and Methods: Thirty-three assessable patients with stage III (n = 22) and stage IV (n = 11) HB were treated sequentially with one course of CARBO (700 mg/m(2)), followed by three courses of CARBO (700 mg/m(2)), day 0, 5-FU (1,000 mg/m(2)/d), by continuous infusion days 0 to 2; and VCR (1.5 mg/m(2)), days 0, 7, and 14. After that therapy, patients whose tumors were resectable underwent surgery and then received two additional courses of CARBO-VCR-5-FU. Children whose tumors remained unresectable after CARBO-VCR-5-FU or who demonstrated no response or progressive disease during this therapy received two courses of HDDP (40 mg/m(2)/d), days 1 to 5; and ETOP (100 mg/m(2)/d), days 2 to 4.Results: Five-year EFS estimates were 59% +/- 11% for stage III disease (n = 22) and 27% +/- 16% for stage IV disease (n = 11), respectively (P = .037). Twenty-seven (82%) of 33 patients had at least a partial response to chemotherapy; 18 (55%) of 33 responded to CARBO; 24 (80%) of 30 responded to CARBO and CARBO-VCR-5-FU; and nine (75%) of 12 responded to HDDP-ETOP. Surgical resection was achieved in 19 (58%) of 33 patients, including 15 (68%) of 22 stage III patients and four (36%) of II stage IV patients. Five-year EFS for patients whose tumors were completely resected was 79% +/- 10%.Conclusion: Patients treated sequentially with CARBO, CARBO-VCR-5-FU, and HDDP-ETOP had response rates and EFS comparable to other therapeutic regimens. This regimen is effective in treating localized, unresectable HB and potentially has less toxicity than other regimens. Novel approaches are needed for patients with metastatic disease. (C) 2002 by American Society of Clinical Oncology.