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Quality of clinical trials in gastroenteropancreatic neuroendocrine tumours
Thomas Walter1, Monika K Krzyzanowska
1Department of Medical Oncology and Hematology, Princess Margaret Hospital, Toronto, ON, Canada.
Background:
The heterogeneity of neuroendocrine tumours (NETs) makes interpretation of clinical trials in this disease challenging. Our aim was to review the quality of treatment trials in NETs in order to inform the design and reporting of future studies.
Methods:
We identified studies by searching MEDLINE. We considered all phase II and III trials of systemic antineoplastic treatments published between 2000 and 2011. Information on trial design, study population, end points, statistical considerations and results was abstracted from each article using a standardized form.
Results:
Seven phase III and 39 phase II trials were identified. The make-up of the study population was variable: only 24% of trials included patients with one type of tumour (pancreatic NET or carcinoid tumour), 41% included patients with both tumour types, and 35% of trials included other endocrine cancers. Disease progression at baseline was often not reported and was documented for all patients in 22% of the trials. The functional status of the tumour, tumour differentiation, and Ki67 index were reported in 35, 43, and 15% of trials, respectively. The primary end point was clearly defined in 72% of trials. Identifiable statistical design, and predefined sample size were reported in 74 and 61% of trials, respectively. Conflicts of interest and study sponsorship were reported in 46 and 85% of trials.
Conclusions:
The quality of the design and reporting of phase II/III NET trials, as described in other cancers, is poor. Future trials should include more homogenous patient populations while adhering to rigorous selection, reporting and interpretation of population and trial parameters.
Insights
The quality of neuroendocrine tumor (NET) clinical trials is poor due to patient heterogeneity and inconsistent reporting. Future studies need more homogenous populations and rigorous parameter selection for better results.
Area of Science:
- Oncology
- Clinical Trial Design
- Translational Research
Background:
- Neuroendocrine tumors (NETs) present significant heterogeneity, complicating clinical trial interpretation.
- A review of NET treatment trials is essential to guide future study design and reporting standards.
Purpose of the Study:
- To assess the quality of phase II and III clinical trials for neuroendocrine tumors (NETs).
- To identify areas for improvement in the design and reporting of future NET studies.
Main Methods:
- Systematic search of MEDLINE for phase II and III systemic antineoplastic treatment trials in NETs published between 2000 and 2011.
- Standardized data abstraction of trial design, patient populations, endpoints, statistical considerations, and results.
Main Results:
- 39 phase II and 7 phase III trials were reviewed.
- Significant variability in patient populations (e.g., inclusion of pancreatic NET, carcinoid tumors, other endocrine cancers).
- Inconsistent reporting of baseline disease progression (22%), tumor differentiation (43%), and Ki67 index (15%).
- Primary endpoints clearly defined in 72% of trials; statistical design and sample size reported in 74% and 61%, respectively.
Conclusions:
- The design and reporting quality of NET phase II/III trials are suboptimal.
- Future NET trials require more homogenous patient populations.
- Adherence to rigorous selection, reporting, and interpretation of population and trial parameters is crucial.
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