Core Outcome Measures in Effectiveness Trials

A systematic review of non-surgical treatments for pancreatic neuroendocrine tumours

General Information

Abstract:
INTRODUCTION: Pancreatic neuroendocrine tumours (pNETs) are rare and the majority of patients present with advanced disease. Such patients have limited treatment options. We conducted a systematic review of published clinical trials of non-surgical interventions in pNET, to understand the efficacy, safety and health related quality of life (HRQoL) outcomes from the current evidence base. METHODS: Electronic databases and manual bibliographic searches were conducted to identify relevant studies. Data were extracted by two independent reviewers. RESULTS: Forty seven clinical studies met the predefined inclusion criteria. The following interventions were included: targeted therapies (two RCTs and six single-arm studies), chemotherapy (two RCTs, one prospective nonrandomised, comparative study and 14 single-arm studies);somatostatin analogues (SSA) and radiolabeled SSA therapies (nine single-arm studies), liver-directed therapies (six single-arm studies), mixed treatment regimens (one RCT, four single-arm studies) and other interventions such as interferon and recombinant human endostatin (one single-arm study for each). The paucity of RCT data and lack of consistency in reporting validated study outcomes and differing patient inclusion criteria between studies made it difficult to compare the relative efficacy of therapies. DISCUSSION: The majority of published studies assessing treatment regimens for the management of pNET are single arm, non-randomised studies, often enrolling a small number of patients and not reporting clinically meaningful outcomes. However data from recently conducted studies assessing targeted therapies indicate that it is possible to conduct adequately powered RCTs reporting standardised oncological endpoints in this rare cancer. Further, similarly robust studies should be conducted to define the optimal treatment algorithm.

Authors:
Valle, J. W. Eatock, M. Clueit, B. Gabriel, Z. Ferdinand, R. Mitchell, S.

Publication

Journal:
Cancer Treat Rev
Volume:
40
Issue:
3
Pages:
376 - 89
Year:
2014
DOI:
Further Study Information

Date:
Funding source(s):

Health Area

Disease Category
Cancer

Disease Name
Pancreatic cancer

Target Population

Age Range
-

Sex
Unknown


Nature / type of Intervention
Unknown

Method(s)

Unknown

Stakeholders Involved

Unknown

Study Type

Systematic review of outcomes measured in trials

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