ArticleEndocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists2026
Belzutifan for HIF2A-Related Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data.
Article in Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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Who cites it
4 citing papers in PubMed.
- The clinical landscape of HIF2α inhibitors in oncology.Nature reviews. Clinical oncology · 2026Review
- Pheochromocytomas and Paragangliomas.Endocrinology and metabolism clinics of North America · 2026Review
- Expanding the clinical tumor phenotype of the EPAS1-asssociated tumor syndrome.The Journal of clinical endocrinology and metabolism · 2026Article
- Advances in the management of neuroendocrine tumours: Evidence from a systematic review.Biomedical reports · 2026Article
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9 authors.
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Abstract
objectivesPheochromocytoma and paraganglioma (PPGL) are rare neuroendocrine tumors of the adrenal medulla and autonomic ganglia, respectively. Germline and somatic genetic drivers are identified in up to 70% of cases. Hypoxia-inducible factor 2α (HIF-2α), encoded by the EPAS1 (HIF2A) gene, plays a central role in PPGL pathogenesis and can be stabilized directly or indirectly by pathogenic variants of several genes, collectively called pseudohypoxia cluster (or Cluster 1). Belzutifan, a small-molecule HIF-2α inhibitor, has demonstrated efficacy in von Hippel-Lindau-related cancers, renal cell carcinoma, and few case reports of PPGL. We report real-world outcomes of belzutifan therapy in 5 patients with recurrent, multifocal, or metastatic EPAS1 (HIF2A)-related PPGL.
methodsClinical parameters, biochemical markers, tumor burden (RECIST 1.1), and treatment-related adverse events were carefully monitored, and recorded.
resultsFour of 5 patients (80%) achieved a partial response, and one patient had stable disease, with no disease progression to date. The average reduction in the sum of tumor diameters was 36.8%. Chromogranin A, erythropoietin, and hemoglobin declined by 69%, 97%, and 13%, respectively, eliminating the need for therapeutic phlebotomy in patients with prior erythrocytosis. Catecholamine normalization allowed discontinuation or reduction of antihypertensive medications in 2 patients. Adverse events included hypoxia and anemia (1/5), mild transaminase elevation (2/5), and fatigue with weight gain (1/5).
conclusionsBelzutifan is a highly effective and well-tolerated therapeutic option for EPAS1(HIF2A)-related PPGL, producing substantial biochemical and radiographic responses, improved blood pressure control, and resolution of erythrocytosis. These findings support belzutifan as a promising genotype-driven therapy for pseudohypoxia-driven PPGL.
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