{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T15:07:29Z","timestamp":1790089649016,"version":"4.0.1"},"reference-count":19,"publisher":"Oxford University Press (OUP)","issue":"10","license":[{"start":{"date-parts":[[2026,8,18]],"date-time":"2026-08-18T00:00:00Z","timestamp":1787011200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2026,9,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background and Objective<\/jats:title>\n                    <jats:p>Management of von Hippel\u2013Lindau (VHL) disease is challenging due to multisystem involvement and limited systemic treatment options. Belzutifan has shown promising efficacy in clinical trials; however, real-world data remain limited. We aimed to evaluate its effectiveness and safety in a real-world cohort.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We conducted a retrospective study of 59 patients with VHL disease treated with belzutifan between August 2021 and January 2026. Objective response rates (ORR) for renal cell carcinoma (RCC), central nervous system hemangioblastomas (CNS HB), and pancreatic neuroendocrine tumors (pNET) were assessed by an independent radiologist. Retinal hemangioblastoma outcomes were evaluated through ophthalmology records by chart review.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Median follow-up was 29\u2009months. ORR was 27.6%, 39.6%, and 85.7% for RCC, CNS HB, and pNET, respectively. Among 13 patients with retinal hemangioblastomas (20 eyes), 17 eyes (85%) showed improvement. At data cutoff, 47 patients (79.6%) remained on treatment, while 12 (20.3%) discontinued (7 due to adverse events, 3 to disease progression, and 2 to other reasons). The most common adverse event was anemia (86.4%). Grade \u22653 adverse events occurred in 10 patients (16.9%). The procedure rate per patient-year decreased from 0.35 in the 3\u2009years prior to treatment to 0.08 during follow-up (Incidence Rate Ratio, 0.23; 95% CI, 0.12-0.47; P\u2009&amp;lt;\u2009.001)<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Belzutifan demonstrated efficacy across VHL-associated lesions, with a favorable safety profile. Most adverse events were grade 1-2. A reduction in procedure rate was observed after treatment initiation.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1093\/oncolo\/oyag331","type":"journal-article","created":{"date-parts":[[2026,8,16]],"date-time":"2026-08-16T11:25:35Z","timestamp":1786879535000},"source":"Crossref","is-referenced-by-count":0,"title":["Clinical outcomes of HIF-2\n                    <b>\u03b1<\/b>\n                    inhibition in Von Hippel\u2013Lindau disease: a multicenter real-world study"],"prefix":"10.1093","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0009-0009-6892-8650","authenticated-orcid":false,"given":"Mousab","family":"Al Abbas","sequence":"first","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United States"]},{"name":"Division of Internal Medicine, University of South Alabama , Mobile, AL 36617,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jabra","family":"Zarka","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Rochester , Rochester, MN 55905,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alvin C","family":"Silva","sequence":"additional","affiliation":[{"name":"Division of Radiology, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0006-3639-0858","authenticated-orcid":false,"given":"Filippo","family":"Cingolani","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Muhammed Uzair","family":"Sarfraz","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0461-6174","authenticated-orcid":false,"given":"Albert","family":"Jang","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Rochester , Rochester, MN 55905,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Muhammad Ali","family":"Khan","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fouad","family":"Nahhat","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Winston","family":"Tan","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Florida , Jacksonville, FL 32224,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian A","family":"Costello","sequence":"additional","affiliation":[{"name":"Division of Hematology and Oncology, Department of Medicine, Mayo Clinic Rochester , Rochester, MN 55905,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Haidar","family":"Abdul-Muhsin","sequence":"additional","affiliation":[{"name":"Division of Urology, Mayo Clinic Arizona , Phoenix, AZ 85054,","place":["United 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