Sunitinib malate for the treatment of pancreatic neuroendocrine tumors
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Eric Raymond1, Laetitia Dahan, Jean-Luc Raoul
1Service Inter-Hospitalier de Cancérologie et Service de Gastroenteropancréatologie, Hôpital Beaujon, Clichy, France. eric.raymond@bjn.aphp.fr
Background:
The multitargeted tyrosine kinase inhibitor sunitinib has shown activity against pancreatic neuroendocrine tumors in preclinical models and phase 1 and 2 trials.
Methods:
We conducted a multinational, randomized, double-blind, placebo-controlled phase 3 trial of sunitinib in patients with advanced, well-differentiated pancreatic neuroendocrine tumors. All patients had Response Evaluation Criteria in Solid Tumors-defined disease progression documented within 12 months before baseline. A total of 171 patients were randomly assigned (in a 1:1 ratio) to receive best supportive care with either sunitinib at a dose of 37.5 mg per day or placebo. The primary end point was progression-free survival; secondary end points included the objective response rate, overall survival, and safety.
Results:
The study was discontinued early, after the independent data and safety monitoring committee observed more serious adverse events and deaths in the placebo group as well as a difference in progression-free survival favoring sunitinib. Median progression-free survival was 11.4 months in the sunitinib group as compared with 5.5 months in the placebo group (hazard ratio for progression or death, 0.42; 95% confidence interval [CI], 0.26 to 0.66; P<0.001). A Cox proportional-hazards analysis of progression-free survival according to baseline characteristics favored sunitinib in all subgroups studied. The objective response rate was 9.3% in the sunitinib group versus 0% in the placebo group. At the data cutoff point, 9 deaths were reported in the sunitinib group (10%) versus 21 deaths in the placebo group (25%) (hazard ratio for death, 0.41; 95% CI, 0.19 to 0.89; P=0.02). The most frequent adverse events in the sunitinib group were diarrhea, nausea, vomiting, asthenia, and fatigue.
Conclusions:
Continuous daily administration of sunitinib at a dose of 37.5 mg improved progression-free survival, overall survival, and the objective response rate as compared with placebo among patients with advanced pancreatic neuroendocrine tumors. (Funded by Pfizer; ClinicalTrials.gov number, NCT00428597.).
Insights
Sunitinib significantly improved progression-free survival and overall survival in patients with advanced pancreatic neuroendocrine tumors. This targeted therapy demonstrated a higher response rate and better safety profile compared to placebo.
Area of Science:
- Oncology
- Pharmacology
- Clinical Trials
Background:
- Pancreatic neuroendocrine tumors (PNETs) are a group of rare neoplasms with varying clinical behavior.
- Sunitinib, a multitargeted tyrosine kinase inhibitor, has demonstrated preclinical and early-phase clinical activity in PNETs.
- Optimizing treatment strategies for advanced PNETs remains a critical area of research.
Purpose of the Study:
- To evaluate the efficacy and safety of sunitinib compared to placebo in patients with advanced, well-differentiated pancreatic neuroendocrine tumors.
- To determine the impact of sunitinib on progression-free survival (PFS), overall survival (OS), objective response rate (ORR), and safety.
Main Methods:
- A multinational, randomized, double-blind, placebo-controlled phase 3 trial involving 171 patients with advanced, well-differentiated PNETs who had documented disease progression.
- Patients were assigned 1:1 to receive best supportive care with either sunitinib (37.5 mg/day) or placebo.
- Primary endpoint was progression-free survival; secondary endpoints included objective response rate, overall survival, and safety.
Main Results:
- The trial was discontinued early due to observed benefits of sunitinib and safety concerns in the placebo arm.
- Median progression-free survival was significantly longer with sunitinib (11.4 months) versus placebo (5.5 months) (HR 0.42, P<0.001).
- Sunitinib demonstrated a higher objective response rate (9.3% vs 0%) and improved overall survival (HR 0.41, P=0.02), with fewer deaths in the sunitinib group.
Conclusions:
- Continuous daily administration of sunitinib at 37.5 mg significantly improves progression-free survival, overall survival, and objective response rate in patients with advanced pancreatic neuroendocrine tumors.
- Sunitinib represents a valuable therapeutic option for this patient population.
- The study was funded by Pfizer and registered under ClinicalTrials.gov number NCT00428597.
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