Quality of Clinical Trials in Gastroenteropancreatic Neuroendocrine TumoursWalter T.a, c · Krzyzanowska M.K.a, b
aDepartment of Medical Oncology and Hematology, Princess Margaret Hospital, and bDepartment of Medicine, University of Toronto, Toronto, Ont., Canada; cHospices Civils de Lyon, Hôpital Edouard Herriot, Fédération des Spécialités Digestives, Lyon, France
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.
© 2012 S. Karger AG, Basel