The treatment also produced responses in 77% of participants with pancreatic tumors, 30% with central nervous system hemangioblastomas, and all 12 patients who had developed retinal hemangioblastomas.
Most patients experienced side effects, but they were primarily low grade, researchers report in The New England Journal of Medicine.
Von Hippel-Lindau (VHL) is an inherited condition that prompts the growth of many types of tumors and cysts. The usual treatment is surgical removal of the growths.
But the drug eliminated or delayed a lot of those operations.
"The number of procedures patients underwent decreased from 327 local interventions before treatment to 3 after treatment started," said Dr. Manuela Schmidinger of the Medical University of Vienna in a linked editorial.
"Considering both the efficacy of the treatment in all VHL-associated tumors and the acceptable adverse-event profile of this HIF-2alpha inhibitor, early and possibly even intermittent use of belzutifan may spare patients with VHL disease multiple surgeries, decrease their risk of loss of organ function (such as renal failure and blindness), and reduce their risk of death from metastatic renal cell carcinoma or CNS hemangioblastomas," Dr. Schmidinger said.
The treatment costs about $27,000 a month, according to prices published on the website goodrx.com. The volunteers in the new open-label, single-group trial were taking the drug for a median of nearly 22 months.
The results were originally released when the U.S. Food and Drug Administration approved the drug, which goes by the brand name Welireg. Merck Sharp and Dohme funded the study.
The journal also contains a case report of using belzutifan to successfully treat a 17-year-old girl with Pacak-Zhuang syndrome, an extremely rare condition where patients are also predisposed to developing tumors. Fewer than 25 cases of the syndrome have been reported.
"Treatment with belzutifan led to a rapid and sustained tumor response along with resolution of hypertension, headaches, and long-standing polycythemia," Dr. Junne Kamihara of the Dana-Farber Cancer Institute in Boston and colleagues report.
"Rapid tumor shrinkage was already evident by day 17, and the response has been sustained for 24 months, with treatment ongoing at the data-cutoff date. No further tumors have developed," the team writes. "Although belzutifan was given to this patient after a substantial tumor burden was already present, the use of such a targeted agent for earlier tumor 'interception' (i.e., intervention at an early step in neoplasm development) in patients with a genetic predisposition is appealing to consider."
SOURCE: https://bit.ly/3oO3A1w, https://bit.ly/3kP4FVK and https://bit.ly/3DvystX The New England Journal of Medicine, online November 24, 2021.
By Gene Emery
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