Advances in the care of lipodystrophies

Abstract

Purpose of reviewLipodystrophies are a group of rare, heterogeneous disorders characterized by a lack or maldistribution of adipose tissue. Treatment focusses on the management of complications, including hypertriglyceridemia, which can be severe. Patients are predisposed to early atherosclerotic cardiovascular disease and acute pancreatitis. This review summarizes the recent advances in the treatment of lipodystrophies, with a particular focus on the treatment of hypertriglyceridemia in familial partial lipodystrophy (FPLD).Recent findingsTreatment of dyslipidemia in FPLD requires management of secondary exacerbating factors, particularly insulin resistance and diabetes, together with modification of atherosclerotic cardiovascular disease risk factors. In addition, specific lipid-lowering therapies are usually needed, starting with statins and fibrates. Leptin therapy improves triglycerides. Several emerging treatments for hypertriglyceridemia include apo C-III antagonists (volanesorsen, AKCEA-APOCIII-LRx and ARO-APOC3) and angiopoietin-like 3 antagonists (evinacumab, vupanorsen and ARO-ANG3); efficacy observed in clinical trials of these agents in nonlipodystrophic patients with severe hypertriglyceridemia suggests that they may also be helpful in lipodystrophy.SummaryEmerging therapies for dyslipidemia show promise in advancing the care of patients with lipodystrophy. However, these treatments are not yet approved for use in lipodystrophy. Further study of their efficacy and safety in this patient population is needed.

Clinical features of generalized lipodystrophy in Turkey: A cohort analysis

“…14,15 Also, there are several new drugs currently being studied for lipodystrophy treatment. 16 None of these lipodystrophy-specific treatments were available in Turkey until recently, which provided a unique opportunity to study the natural disease progression in those who were not treated with lipodystrophy-specific approaches. Previously, we reported on our nationwide data from 33 patients with CGL.…”

“…Metreleptin, a recombinant analogue of human leptin, has been approved as an adjunct to diet as replacement therapy to treat the complications of leptin deficiency in patients with GL 14,15 . Also, there are several new drugs currently being studied for lipodystrophy treatment 16 . None of these lipodystrophy‐specific treatments were available in Turkey until recently, which provided a unique opportunity to study the natural disease progression in those who were not treated with lipodystrophy‐specific approaches.…”

A rapid action plan to improve diagnosis and management of lipodystrophy syndromes

“…Maintaining a controlled diet is key for individuals with lipodystrophy syndromes, although this can be challenging for some patients given a lack of fat and insatiable appetite due to leptin deficiency. Standard medications to treat metabolic disease (e.g., diabetes, hyperlipidemia) can be used with limited efficacy ( 56 , 61 , 64 , 87 ). Recombinant leptin, metreleptin, is used as an adjunct to diet as a replacement therapy to treat metabolic complications of lipodystrophy syndromes.…”