基于 IDRF 的手术决策和逐步治疗强化治疗中危神经母细胞瘤患者的 II 期 JN-I-10 疗效研究：研究方案。
A phase II JN-I-10 efficacy study of IDRF-based surgical decisions and stepwise treatment intensification for patients with intermediate-risk neuroblastoma: a study protocol.
机构信息
Department of Pediatrics, Graduate School of Medical Science, Kyoto Prefectural University of Medicine, Kajii-cho, Kawaramachi-Hirokoji, Kamigyo-ku, Kyoto, 602-8566, Japan.
Department of Pediatric Surgery, Osaka City General Hospital, Osaka, Japan.
出版信息
BMC Pediatr. 2020 May 12;20(1):212. doi: 10.1186/s12887-020-02061-5.
BACKGROUND
Few clinical trials have been reported for patients with intermediate-risk neuroblastoma because of the scarcity of the disease and the variety of clinical and biological characteristics. A multidisciplinary treatment that consists of multidrug chemotherapy and surgery is expected to lead to a good prognosis with few complications. Therefore, a clinical trial for patients with intermediate-risk tumors was designed to establish a standard treatment that reduces complications and achieves good outcomes.
METHODS
We planned a prospective phase 2, single-arm study of the efficacy of image-defined risk factors (IDRF)-based surgical decision and stepwise treatment intensification for patients with intermediate-risk neuroblastomas. For the localized tumor group, IDRF evaluations will be performed after each three-course chemotherapy, and surgery will be performed when appropriate. For patients with metastatic tumors, a total of five chemotherapy courses will be performed, and primary lesions will be removed when the IDRF becomes negative. The primary endpoint is 3-year progression-free survival rate, and the secondary endpoints include 3-year progression-free survival rates and overall survival rates of the localized group and the metastasis group and the incidence of adverse events. From international results, 75% is considered an appropriate 3-year progression-free survival rate. If this trial's expected 3-year progression-free survival rate of 85% is statistically greater than 75% in the lower limit of the 95.3% confidence interval, with an accuracy 10% (85 ± 10%), both groups require more than 65 patients.
DISCUSSION
This study is the first clinical trial on the efficacy of IDRF-based surgical decision and stepwise treatment intensification for patients with intermediate-risk neuroblastomas. We expect that this study will contribute to the establishment of a standard treatment for patients with intermediate-risk neuroblastoma.
TRIAL REGISTRATION
UMIN000004700, jRCTs051180203; Registered on December 9, 2010.
背景
由于疾病的稀有性和各种临床及生物学特征，很少有临床试验针对中危神经母细胞瘤患者进行报道。多学科治疗包括多药物化疗和手术，有望在减少并发症的同时取得良好的预后。因此，针对中危肿瘤患者设计了一项临床试验，以制定一种减少并发症并取得良好结果的标准治疗方案。
方法
我们计划进行一项前瞻性 2 期、单臂研究，评估基于图像定义的危险因素（IDRF）的手术决策和逐步治疗强化在中危神经母细胞瘤患者中的疗效。对于局限性肿瘤组，在每三个疗程的化疗后进行 IDRF 评估，并在适当的时候进行手术。对于转移性肿瘤患者，总共进行 5 个化疗疗程，当 IDRF 转为阴性时，切除原发灶。主要终点是 3 年无进展生存率，次要终点包括局限性组和转移性组的 3 年无进展生存率和总生存率，以及不良事件的发生率。根据国际结果，75%被认为是适当的 3 年无进展生存率。如果该试验的预期 3 年无进展生存率为 85%，在 95.3%置信区间下限的 75%以上具有统计学意义，且精度为 10%（85±10%），则两组都需要超过 65 名患者。
讨论
这是第一项针对基于 IDRF 的手术决策和逐步治疗强化治疗中危神经母细胞瘤患者疗效的临床试验。我们预计该研究将有助于为中危神经母细胞瘤患者建立标准治疗方案。
试验注册
UMIN000004700，jRCTs051180203；于 2010 年 12 月 9 日注册。
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