Pemafibrate Improves Lipid and Liver Metabolism in Adult GH Deficiency: A Prospective Observational Study

The outcomes of pemafibrate administration on hypertriglyceridemia and metabolic dysfunction-associated steatotic liver disease associated with adult growth hormone deficiency (AGHD) are unknown.

To evaluate the effects of pemafibrate on hypertriglyceridemia and liver parameters in AGHD in a real-world setting.

A prospective observational study (December 2019-August 2023).

Two referral hospitals in Japan.

Thirty-four consecutive Japanese patients with AGHD complicated with hypertriglyceridemia. Hypertriglyceridemia was defined using a fasting serum triglyceride (TG) concentration of ≥1.7 mmol/L or treated with lipid-lowering medication.

Patients were prescribed pemafibrate or continued conventional therapy for 24 weeks.

The percentage reduction in fasting serum TG level between baseline and 24 weeks was evaluated. Hepatic parameters, including hepatic steatosis index (HSI) derived from aspartate aminotransferase, alanine aminotransferase, and body mass index, were also evaluated, along with other metabolic parameters. The Mann-Whitney U-test and Fisher's exact test were used to compare the change between groups. A multiple regression was performed to identify predictors of TG change.

The change in serum TG level was significantly larger in the pemafibrate group than that in the conventional group (median: -51.0% [interquartile range (IQR): -69.0% to -21.0%] vs -13.0% [IQR: -34.0% to 9.0%], P = .0138). HSI decreased after 24 weeks of pemafibrate. Relative TG change correlated with baseline body mass index [regression coefficient (β) .0463, P < .0001] and HSI (β .0645, P = .0107).

Pemafibrate had beneficial effects on hypertriglyceridemia as well as liver metabolism of patients with AGHD. However, its efficacy was attenuated by obesity.