Triheptanoin
Fuentes regulatorias consultadas
Indicaciones aprobadas
- Fuente de calorías y ácidos grasos para adultos y pacientes pediátricos con trastornos de oxidación de ácidos grasos de cadena larga confirmados molecularmente.
Advertencias clínicas
- Advertencia mayor · No administrar en insuficiencia pancreática: la falta de enzimas pancreáticas puede reducir la absorción y el efecto clínico. — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
- Advertencia mayor · Monitorizar el funcionamiento y la integridad de la sonda; no usar sondas ni componentes de PVC y no administrar el líquido solo. — https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
Interacciones medicamentosas
- SeveraInhibidores de la lipasa pancreática, como orlistatA08AB01
Mecanismo: Pueden reducir la exposición al metabolito heptanoato y el efecto clínico de triheptanoína.
Recomendación: Evitar la administración concomitante.
https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5644d49f-08b9-e806-e063-6294a90a80f9
Eventos adversos
Comunes (≥1%)
Dolor abdominal · Diarrea · Vómitos · Náuseas
Embarazo y lactancia
Categoría FDA: Datos humanos insuficientes
No hay datos suficientes para evaluar el riesgo durante el embarazo; existe un registro de exposición. Se desconoce si pasa a la leche; valorar los beneficios de la lactancia y la necesidad clínica materna.
Bibliografía reciente (PubMed)
Paroxysmal movement disorders are common in Glut1 deficiency syndrome (Glut1DS). Not all patients respond to or tolerate ketogenic diets. The objective was to evaluate the effectiveness and safety of triheptanoin in reducing the frequency of disabling movement disorders in patients with Glut1DS not receiving a ketogenic diet. UX007G-CL301 was a randomized, double-blind, placebo-controlled, phase 3 crossover study. After a 6-week run-in, eligible patients were randomized 1:1 to the first sequence (triheptanoin/placebo or placebo/triheptanoin) titration plus maintenance, followed by washout and the opposite sequence titration plus maintenance. The placebo (safflower oil) matched the appearance, taste, and smell of triheptanoin. Open-label triheptanoin was administered in the extension. The frequency of disabling paroxysmal movement disorder events per 4 weeks (recorded by diary during maintenance; primary endpoint) was assessed by Wilcoxon rank-sum test. Forty-three patients (children, n = 16; adults, n = 27) were randomized and treated. There was no difference between triheptanoin and placebo in the mean (interquartile range) number of disabling paroxysmal movement disorder events (14.3 [4.7-38.3] vs. 11.8; [3.2-28.7]; Hodges-Lehmann estimated median difference: 1.46; 95% confidence interval, -1.12 to 4.36; P = 0.2684). Treatment-emergent adverse events were mild/moderate in severity and included diarrhea, vomiting, upper abdominal pain, headache, and nausea. Two patients discontinued the study because of non-serious adverse events that were predominantly gastrointestinal. The study was closed early during the open-label extension because of lack of effectiveness. Seven patients continued to receive triheptanoin compassionately. There were no significant differences between the triheptanoin and placebo groups in the frequency of disabling movement disorder events during the double-blind maintenance period. © 2024 The Authors. Movement Disorders published by Wiley Per