Juan W Valle1, Martin Eatock2, Ben Clueit3, Zahava Gabriel4, Roxanne Ferdinand5, Stephen Mitchell6. 1. Department of Medical Oncology, Christie Hospital NHS Foundation Trust, Manchester, UK. Electronic address: Juan.valle@christie.nhs.uk. 2. Department of Medical Oncology, Northern Ireland Cancer Centre, Belfast City Hospital, Belfast, UK. Electronic address: MartinEatock@bch.n-i.nhs.uk. 3. Pfizer UK, Tadworth, Surrey, UK. Electronic address: Benjamin.Clueit@Pfizer.com. 4. Pfizer UK, Tadworth, Surrey, UK. Electronic address: Zahava.gabriel@pfizer.com. 5. Pfizer UK, Tadworth, Surrey, UK. Electronic address: roxanne.ferdinand@pfizer.com. 6. Abacus International, 6 Talisman Business Centre, Talisman Road, Bicester, Oxfordshire OX26 6HR, UK. Electronic address: Stephen.mitchell@abacusint.com.
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.
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 humanendostatin (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: Laura Prakash; Jeffrey E Lee; James Yao; Priya Bhosale; Aparna Balachandran; Huamin Wang; Jason B Fleming; Matthew H G Katz Journal: J Gastrointest Surg Date: 2015-08-18 Impact factor: 3.452
Authors: Kota Sahara; Diamantis I Tsilimigras; Rittal Mehta; Amika Moro; Anghela Z Paredes; Alexandra G Lopez-Aguiar; Flavio Rocha; Zaheer Kanji; Sharon Weber; Alexander Fisher; Ryan Fields; Bradley A Krasnick; Kamran Idrees; Paula M Smith; George A Poultsides; Eleftherios Makris; Cliff Cho; Megan Beems; Mary Dillhoff; Shishir K Maithel; Itaru Endo; Timothy M Pawlik Journal: Ann Surg Oncol Date: 2019-12-14 Impact factor: 5.344