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Published on: July 13, 2010
Early-phase clinical trial eligibility and response evaluation criteria for refractory, relapsed, or progressive
Julie R Park1,2, Judith G Villablanca3,4, Barbara Hero5
1Seattle Children's Hospital, Seattle, Washington, USA.
Background:
International standardized criteria for eligibility, evaluable disease sites, and disease response assessment in patients with refractory, progressive, or relapsed high-risk neuroblastoma enrolled in early-phase clinical trials are lacking.
Methods:
A National Cancer Institute-sponsored Clinical Trials Planning Meeting was convened to develop an international consensus to refine the tumor site eligibility criteria and evaluation of disease response for early-phase clinical trials in children with high-risk neuroblastoma.
Results:
Standardized data collection of patient and disease characteristics (including specified genomic data), eligibility criteria, a definition of evaluable disease, and response evaluations for primary and metastatic sites of disease were developed. Eligibility included two distinct patient groups: progressive disease and refractory disease. The refractory disease group was subdivided into responding persistent disease and stable persistent disease to better capture the clinical heterogeneity of refractory neuroblastoma. Requirements for defining disease evaluable for a response assessment were provided; they included requirements for biopsy to confirm viable neuroblastoma and/or ganglioneuroblastoma in those patients with soft tissue or bone disease not avid for iodine-123 meta-iodobenzylguanidine. Standardized evaluations for response components and time intervals for response evaluations were established.
Conclusions:
The use of international consensus eligibility, evaluability, and response criteria for early-phase clinical studies will facilitate the collection of comparable data across international trials and promote more rapid identification of effective treatment regimens for high-risk neuroblastoma.
Insights
Standardized criteria for high-risk neuroblastoma clinical trials were developed to improve data collection and treatment identification. These international consensus guidelines enhance early-phase trial comparability for progressive or refractory disease.
Area of Science:
- Pediatric Oncology
- Clinical Trial Design
- Neuroblastoma Research
Background:
- Lack of standardized criteria for eligibility, disease assessment, and response evaluation in early-phase high-risk neuroblastoma trials.
- Need for international consensus to address heterogeneity in refractory and relapsed neuroblastoma patient populations.
Purpose of the Study:
- To develop international consensus criteria for eligibility and response assessment in early-phase clinical trials for high-risk neuroblastoma.
- To refine tumor site eligibility and evaluation methods for pediatric neuroblastoma trials.
Main Methods:
- Convened a National Cancer Institute-sponsored Clinical Trials Planning Meeting.
- Developed standardized criteria for patient/disease characteristics, eligibility, evaluable disease definition, and response evaluations.
- Defined distinct eligibility groups for progressive and refractory disease, with further subdivision for refractory disease.
Main Results:
- Established standardized data collection, including genomic data, eligibility criteria, and response assessments for primary and metastatic sites.
- Provided requirements for defining evaluable disease, including biopsy confirmation for specific cases.
- Set standardized response evaluation components and time intervals.
Conclusions:
- International consensus criteria will enable comparable data collection across global trials.
- Facilitates more rapid identification of effective treatment regimens for high-risk neuroblastoma.
- Promotes consistency in early-phase clinical studies for improved research outcomes.
