Abstract
BackgroundFew 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.MethodsWe 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.DiscussionThis 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 registrationUMIN000004700, jRCTs051180203; Registered on December 9, 2010.
Highlights
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
The concept of image-defined risk factors (IDRF) has been proposed internationally as a criterion for estimating the risk of surgery from imaging findings and determining whether removal or biopsy is to be performed as initial surgery [7]
Even if residual tumors are observed at the end of chemotherapy, treatment will be permitted to end if the risk of surgery was considered high according to the IDRF-based assessment
Summary
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 clinical trial for patients with intermediate-risk tumors was designed to establish a standard treatment that reduces complications and achieves good outcomes. The concept of image-defined risk factors (IDRF) has been proposed internationally as a criterion for estimating the risk of surgery from imaging findings and determining whether removal or biopsy is to be performed as initial surgery [7]. In this clinical trial, one of the goals is to reduce surgical complications while ensuring that the prognosis is not impaired. Chemotherapy will be performed for the intermediate risk group as a primary treatment after biopsy
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