FIRSTMAPPP: sunitinib for metastatic progressive pheochromocytomas and paragangliomas
The first randomised trial ever completed in metastatic pheochromocytoma and paraganglioma found that the kinase inhibitor sunitinib roughly doubled the proportion of patients whose disease had not progressed at one year compared with placebo.
Overview
Academic international randomised double-blind placebo-controlled phase 2 trial of 78 patients with progressive metastatic pheochromocytoma or paraganglioma assigned to sunitinib 37.5 mg daily or placebo.
Progression-free survival at twelve months was 36 percent with sunitinib against 19 percent with placebo, meeting the primary endpoint; responses were seen including in SDHB carriers, and toxicity was that of sunitinib (fatigue, hypertension, diarrhoea). Accrual took more than a decade.
- Twelve-month progression-free survival 36 percent with sunitinib versus 19 percent with placebo.
- Objective responses in a minority, including patients with SDHB germline mutations.
Sunitinib is an evidence-based option for progressive metastatic pheochromocytoma and paraganglioma, alongside radionuclide therapy and, for VHL-related disease, belzutifan.
- Small trial that took more than ten years to accrue, illustrating the difficulty of trials in this disease.
- Overall survival was not improved and the placebo arm could cross over.
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