Efficacy and Safety of
SGLT2
Inhibitors in Patients With Genetic Lipodystrophy: A Real‐Life Experience From a National Reference Network
Léna Robert, Ines Belalem, Corinne Vigouroux, Hippolyte Dupuis, Marie Christine Vantyghem, Mathilde Simonson, Estelle Nobecourt, Héléna Mosbah, Sophie Beliard, Stéphanie Jellimann, Sonja Janmaat, Camille Vatier, Sophie Lamothe ABSTRACT
Aim
Lipodystrophy syndromes are rare diseases with metabolic and cardiovascular consequences . This study explores the metabolic and renal effects of sodium‐glucose cotransporter 2 inhibitors (SGLT2i) in patients with genetic lipodystrophy.
Patients and Methods
Patients with familial partial lipodystrophy ( n = 57) or congenital generalised lipodystrophy ( n = 2) (86% women) were initiated with SGLT2i in the French reference network PRISIS. We retrospectively collected clinical and biological data before initiation, after 6–18 months (short‐term, mean ± SD 12.5 ± 6.4 months) and at the latest follow‐up on SGLT2i (long‐term, 29.0 ± 10.7 months).
Results
Body mass index decreased from 25 [22.9–28] (median [IQR]) to 24.4 [22.1–26.5] then 23.9 [22.1–26.4] kg/m 2 ( p < 0.001) and HbA1c from 7.8 [7.1–8.8] to 7.3 [6.7–7.7] and 7.3% [6.9–8] ( p < 0.01 and p = 0.05, respectively). Systolic blood pressure (130 [120–143.25] to 126 [120–137] mmHg), ALT (33 [23.5–44] to 30 [26–40] IU/L), GGT (44 [28.5–62.5] to 37 [27.85–45.5] IU/L) and liver stiffness (6.8 [5.9–11.8] to 5.8 [5–6.9] kPa) declined significantly at short‐term ( p < 0.05, 0.02, 0.02 and < 0.01, respectively). The dual‐energy X‐ray absorptiometry‐based fat mass percentage (23.6 [18–29.3] to 20.8 [17.8–25.3], p = 0.07) and truncal‐to‐lower limb fat ratio (1.36 [1.18–1.50] to 1.17 [0.98–1.41], p = 0.08) tended to decrease at short‐term. Urinary albumin‐to‐creatinine ratio, when elevated at baseline (> 3 mg/mmol, n = 18 patients), decreased from 10.8 [6.3–24.9] to 8.0 mg/mmol [2.8–14.4] ( p = 0.02). Treatment discontinuation (7%) was mainly due to vaginal mycoses. No serious adverse events were reported.
Conclusion
In a real‐life setting, SGLT2i therapy is associated with improvement in HbA1c, body weight, blood pressure, hepatic parameters and albuminuria in patients with genetic lipodystrophy.