Journal: The New England journal of medicine
This phase 2 international trial evaluated belzutifan, a HIF-2α inhibitor, in 72 patients with locally advanced or metastatic pheochromocytoma or paraganglioma not amenable to surgery or curative treatment.
At a median follow-up of 30.2 months:
- Confirmed objective response rate: 26%
- Disease control achieved in: 85% of patients
- Median duration of response: 20.4 months
- Median progression-free survival: 22.3 months
- Overall survival at 24 months: 76%
Additionally, 32% of patients on antihypertensive medications experienced at least a 50% dose reduction lasting six months or more.
Treatment-related adverse events were common (99%), including:
- Grade 3 anemia: 22%
- Serious adverse events: 11%
Belzutifan demonstrated durable antitumor activity in this patient population.