Contextualizing Historic Unmet Need in FCS in a Contemporary Landscape

For decades, management of familial chylomicronemia syndrome has been marked by profound therapeutic limitations and a persistently high burden of disease. In the conversation between Nihar R. Desai, MD, and Anthony Giamo, MD, the faculty examine how reliance on extreme dietary modification and modestly effective pharmacologic options has left many patients at risk for recurrent acute pancreatitis. Even with meticulous adherence to a very low‑fat diet, clinicians often anticipate the next hospitalization, underscoring the inadequacy of historical approaches.

Desai reviews the traditional therapeutic toolkit—fibrates, omega‑3 fatty acids, and lifestyle modification—and explains that these agents commonly fail to produce adequate triglyceride reductions in FCS because they do not correct the underlying defect in chylomicron clearance. This limited efficacy forces clinicians to persist with therapies of marginal benefit simply because no better options were available. Both physicians stress that this therapeutic insufficiency has direct implications for patient outcomes and creates anxiety for patients and providers alike.