THURSDAY, May 31 (HealthDay News) -- Patients with urea-cycle disorders are more likely to survive episodes of hyperammonemia if they are treated with an intravenous infusion of sodium phenylacetate and sodium benzoate, researchers report in the May 31 issue of the New England Journal of Medicine. The overall survival rate was 84 percent in patients with the historically lethal metabolic condition.
Gregory Enns, M.B., Ch.B., of Stanford University in Stanford, Calif., and colleagues report the results of a 25-year, open-label study of sodium phenylacetate and sodium benzoate therapy (Ammonul) for treatment of 299 patients with urea-cycle disorders. Although no control group was included, a similar study showed survival rates as low as 16 percent for neonates who did not receive alternative-pathway therapy.
Overall, patients survived 1,132 of 1,181 episodes of hyperammonemia with survival rates ranging from 73 percent for neonates to 98 percent for those older than 30 days. Patients younger than 30 days with peak ammonium levels of 1000 μmol per liter had the lowest chance of surviving a hyperammonemic episode, at 38 percent.
"The survival rate in our cohort is remarkable and confirms the promise of initial reports showing improved survival after alternative-pathway therapy," the authors write. The report "is an important first step in the development of improved treatment for patients with urea-cycle disorders," adds Vivian Shih, M.D., of Massachusetts General Hospital, Boston, in an accompanying editorial.
Some of the authors have received lecture or consulting fees from Ucyclyd Pharma, makers of Ammonul.
Abstract
Full Text (subscription or payment may be required)
Editorial
