Grill J, Le Deley M C, Gambarelli D, Raquin M A, Couanet D, Pierre-Kahn A, Habrand J L, Doz F, Frappaz D, Gentet J C, Edan C, Chastagner P, Kalifa C
Department of Pediatrics, Institut Gustave Roussy, Villejuif, France.
J Clin Oncol. 2001 Mar 1;19(5):1288-96. doi: 10.1200/JCO.2001.19.5.1288.
To evaluate a strategy that avoids radiotherapy in first-line treatment in children under 5 years of age with brain or posterior fossa ependymoma, by exclusively administering 16 months of adjuvant multiagent chemotherapy after surgery.
Between June 1990 and October 1998, 73 children with ependymoma (82% with high-grade tumors) were enrolled onto this multicenter trial. Children received adjuvant conventional chemotherapy after surgery consisting of seven cycles of three courses alternating two drugs at each course (procarbazine and carboplatin, etoposide and cisplatin, vincristine and cyclophosphamide) over a year and a half. Systematic irradiation was not envisaged at the end of chemotherapy. In the event of relapse or progression, salvage treatment consisted of a second surgical procedure followed by local irradiation with or without second-line chemotherapy.
Conventional chemotherapy was well tolerated and could be administered in outpatient clinics. No radiologically documented response to chemotherapy more than 50% was observed. With a median follow-up of 4.7 years (range, 5 months to 8 years), the 4-year progression-free survival rate in this series was 22% (95% confidence interval [CI], 13% to 43%) and the overall survival rate was 59% (95% CI, 47% to 71%). Overall, 40% (95% CI, 29% to 51%) of the patients were alive having never received radiotherapy 2 years after the initiation of chemotherapy and 23% (95% CI, 14% to 35%) were still alive at 4 years without recourse to this modality. In the multivariate analysis, the two factors associated with a favorable outcome were a supratentorial tumor location (P =.0004) and complete surgery (P =.0009). Overall survival at 4 years was 74% (95% CI, 59% to 86%) for the patients in whom resection was radiologically complete and 35% (95% CI, 18% to 56%) for the patients with incomplete resection.
A significant proportion of children with ependymoma can avoid radiotherapy with prolonged adjuvant chemotherapy. Deferring irradiation at the time of relapse did not compromise overall survival of the entire patient population.
评估一种策略，即对于5岁以下患有脑或后颅窝室管膜瘤的儿童，在一线治疗中避免放疗，而是在术后单纯给予16个月的辅助多药化疗。
1990年6月至1998年10月，73例室管膜瘤患儿（82%为高级别肿瘤）纳入该多中心试验。患儿术后接受辅助常规化疗，在一年半的时间里进行七个周期的化疗，每个周期包括三个疗程，每个疗程交替使用两种药物（丙卡巴肼和卡铂、依托泊苷和顺铂、长春新碱和环磷酰胺）。化疗结束时未考虑进行系统性放疗。如果出现复发或进展，挽救性治疗包括再次手术，随后进行局部放疗，可联合或不联合二线化疗。
常规化疗耐受性良好，可在门诊进行。未观察到化疗的影像学记录缓解率超过50%。中位随访4.7年（范围5个月至8年），该系列中4年无进展生存率为22%（95%置信区间[CI]，13%至43%），总生存率为59%（95%CI，47%至71%）。总体而言，40%（95%CI，29%至51%）的患者在化疗开始后2年从未接受放疗仍存活，23%（95%CI，14%至35%）在4年时未采用这种治疗方式仍存活。在多变量分析中，与良好预后相关的两个因素是幕上肿瘤位置（P = 0.0004）和完全手术（P = 0.0009）。对于影像学上切除完全的患者，4年总生存率为74%（95%CI，59%至86%），对于切除不完全的患者为35%（95%CI，18%至56%）。
相当一部分室管膜瘤患儿通过延长辅助化疗可避免放疗。复发时推迟放疗并未影响整个患者群体的总生存。
