Why the study?
Does volanesorsen reduce triglyceride levels and prevent pancreatitis in patients with severe hypertriglyceridemia?
Does volanesorsen reduce triglyceride levels and prevent pancreatitis in patients with severe hypertriglyceridemia?
Volanesorsen effectively reduces extreme triglyceride levels and prevents pancreatitis in patients with familial chylomicronemia syndrome.
Should not yet change practice in severe hypertriglyceridemia; leaves open efficacy and safety in controlled trials.
INTRODUCTION: mutation. Volanesorsen was well-tolerated and decreased patient's TG levels (from >5000 mg/dL (56 mmol/L) to 350-500 mg/dL (4-5.6 mmol/L)) at 12 months. Lipoprotein apheresis (LA) was stopped and there were no episodes of pancreatitis or abdominal pain. Expert opinion: Severe hypertriglyceridemia can potentially be fatal. Until recently, there was no specific treatment for FCS, apart from hypotriglyceridemic diet, fibrates, omega-3 fatty acids, and LA sessions. Therefore, volanesorsen represents a promising therapeutic solution for these patients. The main side effect of volanesorsen therapy is thrombocytopenia, which should be monitored and treated accordingly. Increasing evidence will further elucidate the clinical implications of volanesorsen use in daily practice.
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Kolovou et al. (2022) studied this question.
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