BACKGROUND: In a phase III study, sunitinib led to a significant increase in progression-free survival (PFS) versus placebo in patients with pancreatic neuroendocrine tumours (panNETs). This study was a post-marketing commitment to support the phase III data. METHODS: In this ongoing, open-label, phase IV trial (NCT01525550), patients with progressive, advanced unresectable/metastatic, well-differentiated panNETs received continuous sunitinib 37.5 mg once daily. Eligibility criteria were similar to those of the phase III study. The primary endpoint was investigator-assessed PFS per Response Evaluation Criteria in Solid Tumours v1.0 (RECIST). Other endpoints included PFS per Choi criteria, overall survival (OS), objective response rate (ORR), and adverse events (AEs). RESULTS: Sixty-one treatment-naive and 45 previously treated patients received sunitinib. By March 19, 2016, 82 (77%) patients had discontinued treatment, mainly due to disease progression. Median treatment duration was 11.7 months. Investigator-assessed median PFS per RECIST (95% confidence interval [CI]) was 13.2 months (10.9-16.7): 13.2 (7.4-16.8) and 13.0 (9.2-20.4) in treatment-naive and previously treated patients, respectively. ORR (95% CI) per RECIST was 24.5% (16.7-33.8) in the total population: 21.3% (11.9-33.7) in treatment-naive and 28.9% (16.4-44.3) in previously treated patients. Median OS, although not yet mature, was 37.8 months (95% CI, 33.0-not estimable). The most common treatment-related AEs were neutropenia (53.8%), diarrhoea (46.2%), and leukopenia (43.4%). CONCLUSIONS: This phase IV trial confirms sunitinib as an efficacious and safe treatment option in patients with advanced/metastatic, well-differentiated, unresectable panNETs, and supports the phase III study outcomes. AEs were consistent with the known safety profile of sunitinib.

2nd Department of Oncology Faculty of Medicine Comenius University Bratislava Slovakia

Andrew Love Cancer Center Geelong Hospital Victoria Victoria Australia

Centrul de Oncologie Sf Nectarie Oncologie Medicala Craiova Romania

China Japan Friendship Hospital Beijing China

Department of Gastroenterology Oslo University Hospital Rikshospitalet Oslo Norway

Department of Medical Oncology Fundeni Clinical Institute Bucharest Romania

Department of Medical Oncology Paris Saint Joseph Hospital Group Paris France

Department of Medicine and Bioregulatory Science Graduate School of Medical Sciences Kyushu University Fukuoka Japan

Division of Gastrointestinal Medical Oncology and Neuroendocrine Tumors European Institute of Oncology IEO Milan

Division of Gastrointestinal Medical Oncology and Neuroendocrine Tumors European Institute of Oncology IEO Milan Italy

Division of Hematology Oncology Columbia University Medical Center New York New York USA

Faculty of Health Sciences University of the Witwatersrand Johannesburg South Africa

Faculty of Medicine Masaryk Memorial Cancer Institute Masaryk University Brno Czech Republic

GI and HPB Surgical Oncology Tata Memorial Hospital Mumbai India

Henry Ford Health System Detroit Michigan USA

Hepato Gastroenterology Department Cliniques Universitaires Saint Luc Brussels Belgium

Hospital Universitario Madrid Sanchinarro Centro Integral Oncológico Clara Campal Madrid Spain

Key Laboratory of Carcinogenesis and Translational Research Department of GI Oncology Peking University Cancer Hospital and Institute Beijing China

National Cancer Center Tokyo Japan

No 307 Hospital Academy of Military Medical Sciences Beijing China

Pancreatic and Hepatobiliary Surgery Fudan University Shanghai Cancer Center Shanghai China

Pfizer Oncology Pfizer Inc Cambridge Massachusetts USA

Pfizer Oncology Pfizer Inc San Diego California USA

PLA Cancer Center Nanjing Bayi Hospital Nanjing China

Program in Neuroendocrine and Carcinoid Tumors Dana Farber Cancer Institute Boston Massachusetts USA

Service d'Oncologie Digestive Hôpital Beaujon Clichy France

Všeobecné Fakultní Nemocnice Praha Onkologická Klinika Prague Czech Republic

West China Hospital of Sichuan University Chengdu China

Zhongshan Hospital Fudan University Shanghai China

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