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Pediatric Patients with Von Hippel-Lindau and Hemangioblastomas Treated Successfully with Belzutifan
  • +7
  • Emily Duan,
  • Michael Robinson,
  • Charles Davis,
  • Sumit Pruthi,
  • Marisa Lewis,
  • Julian Martinez-Agosto,
  • Michael B. Gorin,
  • Brian M. Shuch,
  • Debra Friedman,
  • Vivian Chang
Emily Duan
University of California Los Angeles Department of Pediatrics
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Michael Robinson
Vanderbilt University Medical Center Vanderbilt O'Brien Kidney Center
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Charles Davis
Ronald Reagan UCLA Medical Center
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Sumit Pruthi
Vanderbilt University Medical Center Vanderbilt O'Brien Kidney Center
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Marisa Lewis
University of California Los Angeles Department of Pediatrics
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Julian Martinez-Agosto
University of California Los Angeles Department of Human Genetics
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Michael B. Gorin
UCLA Jules Stein Eye Institute
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Brian M. Shuch
University of California Los Angeles Jonsson Comprehensive Cancer Center
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Debra Friedman
Vanderbilt University Medical Center Vanderbilt O'Brien Kidney Center
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Vivian Chang
University of California Los Angeles Department of Pediatrics

Corresponding Author:[email protected]

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Abstract

Hemangioblastomas are the most common tumor associated with Von Hippel-Lindau (VHL) and a leading cause of mortality. We present five pediatric patients with VHL-associated hemangioblastomas treated with belzutifan, a HIF2a inhibitor. Three patients were started on belzutifan due to vision loss from progressive retinal hemangioblastomas. Within 1 year of treatment, all three patients had improvement in hemangioblastoma size and visual acuity. For patients with intracranial lesions, belzutifan resulted in an improvement in neurologic symptoms and hemangioblastoma size. Four patients experienced grade 1-2 anemia and two patients required a dose reduction. Our report suggests that belzutifan can be an effective therapy for pediatric patients with VHL-associated hemangioblastomas.
29 Apr 2024Submission Checks Completed
29 Apr 2024Assigned to Editor
29 Apr 2024Submitted to Pediatric Blood & Cancer
08 May 2024Reviewer(s) Assigned