BioCentury
ARTICLE | Clinical News

Triheptanoin: Compassionate use program data

October 7, 2013 7:00 AM UTC

A retrospective medical record review of a compassionate use program in 20 evaluable patients with long-chain fatty acid oxidation disorders showed that triheptanoin reduced overall mean hospitalizations per year by 36% (1.26 vs. 1.94 events per year, p=0.113) and mean hospital days per year by 69% (5.4 vs. 17.55 days per year, p=0.024) compared to pretreatment. Triheptanoin also reduced the mean number of major events related to hypoglycemic episodes per year by 96% (0.04 vs. 0.92 events per year, p=0.009) and mean hospital days related to hypoglycemic episodes per year by 98% (0.18 vs. 8.42 days per year, p=0.026) compared to pretreatment. The review, which compared the incident rates of major medical events before and after treatment, included patients who received triheptanoin for up to 13 years as part of a compassionate use program and had at least 6 months of pretreatment data. Data were presented at the Inborn Errors of Metabolism meeting in Barcelona. ...