CCG7942/POG9354方案治疗儿童外周型原始神经外胚层肿瘤的临床研究

目的 研究儿童外周型原始神经外胚层肿瘤(pPNET)的临床表现、诊断、治疗及生存情况.方法 回顾性分析2008 年10 月至2013 年10 月收治的10 例pPNET 患儿完整的临床资料,其中3 例存在转移,7 例无转移.7 例无转移患儿采用美国儿童癌症协作组CCG7942 治疗方案,3 例有转移患儿采用美国儿童肿瘤协作组POG9354 治疗方案.治疗后采用WHO 实体瘤疗效评定标准和通用不良反应术语标准对相关毒副反应进行评价.结果 采用CCG7942 方案治疗的7 例患儿中,完全缓解4 例,稳定1 例,进展2 例,复发2 例;采用POG9354 方案治疗的3 例患儿中,完全缓解1 例,进展2 例,复发2 例,死亡2 例.目前7 例无转移患儿的生存期为5~60 个月,无事件生存率为71%;3 例有转移患儿的生存期为13~25 个月,无事件生存率为33%.所有患儿除均有4 级骨髓抑制的毒性损害外,其他毒性反应如消化道、肝肾功能多为1~2 级损害.结论 CCG7942方案对治疗没有转移的pPNET 患儿有效、安全;存在转移的pPNET 患儿即使采用POG9354 方案疗效仍然欠佳,需要研究新的治疗方案以提高疗效.

Clinical efficacy of CCG7942/POG9354 protocol in treatment of peripheral primitive neuroectodermal tumor in children

Objective To investigate the clinical manifestations, diagnosis, and treatment of peripheral primitiveneuroectodermal tumor (pPNET) in children and the survival of patients treated with the CCG7942/POG9354 protocol.Methods A retrospective analysis was performed on the clinical data of 10 patients with pPNET admitted from October 2008 to October 2013. Of the 10 patients, 3 had metastasis, while others had no metastasis. The 7 patients withoutmetastasis were treated with the Children''s Cancer Study Group 7942 (CCG7942) protocol, and the other 3 patients with metastasis with the Pediatric Oncology Group 9354 (POG9354) protocol. The therapeutic response and chemotherapy related toxicities were evaluated by WHO criteria and Common Terminology Criteria for Adverse Events (version 4.0).Results In the 7 patients treated with the CCG7942 protocol, 4 achieved a complete remission (CR), 1 had stabledisease, 2 developed progressive disease (PD), and 2 had recurrence. In the 3 patients treated with the POG9354 protocol, 1achieved a CR, 2 developed PD, 2 had recurrence, and 2 died. For the 7 patients without metastasis, the survival timewas 5-60 months, and the event-free survival rate was 71%. For the 3 patients with metastasis, the survival time was 13-25 months, and the event-free survival rate was 33%. All patients developed grade 4 bone marrow suppression, and othergrade 1-2 toxicities, including gastrointestinal reactions, liver function impairment, and renal function impairment, werealso seen. Conclusions CCG7942 protocol is effective and safe for children with non-metastatic pPNET. However,POG9354 protocol has unsatisfactory efficacy in children with metastatic pPNET, so further studies are needed toimprove the therapy for this disease.