Multinational randomized phase 2 trial of sunitinib for progressive metastatic pheochromocytoma and paraganglioma

The randomized phase 2 FIRSTMAPPP trial, at 14 centers in 4 European countries, evaluated sunitinib versus placebo for progressive metastatic pheochromocytoma and paraganglioma. The primary endpoint was rate of progression-free survival at 12 months. The 12-month progression-free survival rate was 36% (90% CI 23-50) with sunitinib versus 19% (11-31) with placebo. The most frequent grade 3 or 4 adverse events were asthenia (18% of patients with sunitinib versus 3% of patients with placebo), hypertension (13% versus 10%) and back or bone pain (3% versus 8%). One patient in the sunitinib group died from drug-related rectal bleeding. The authors conclude that FIRSTMAPPP results support the use of sunitinib as the medical option with the highest level of evidence for antitumor efficacy in progressive metastatic pheochromocytoma and paraganglioma.