Use of SGLT2 Inhibitors in Dystrophin Deficient Cardiomyopathy: A Multi-center Study Using the ACTION Registry

Area of Science:

• Cardiology
• Genetics
• Pharmacology

Background:

• Cardiac disease is the leading cause of mortality in Duchenne Muscular Dystrophy (DMD).
• No prior data exists on sodium-glucose cotransporter-2 inhibitors (SGLT2i) use in dystrophin-deficient cardiomyopathy.
• This study addresses this gap by examining SGLT2i in DMD patients.

Purpose of the Study:

• To analyze the utilization of SGLT2i in patients with dystrophin-deficient cardiomyopathy.
• To describe the indications for SGLT2i therapy.
• To evaluate the effects of SGLT2i on cardiac function and identify adverse events.

Main Methods:

• Retrospective review of patients initiated on SGLT2i using the ACTION dystrophinopathy registry.
• Data collection on patient demographics, pre-existing cardiac medications, ejection fraction (EF) via echocardiogram and cardiac magnetic resonance imaging (cMRI).
• Analysis of follow-up data for cardiac function changes and adverse events.

Main Results:

• Eighty patients were initiated on SGLT2i, with a median age of 19.6 years.
• Baseline median EF was 37% (echocardiogram) and 37% (cMRI).
• At a median follow-up of 1.4 years, median EF improved to 40% (echocardiogram) and 39% (cMRI).
• 14 adverse events occurred (e.g., bone fracture, acute kidney injury), with 8.8% requiring discontinuation.
• SGLT2i were generally well-tolerated with a low incidence of adverse effects.

Conclusions:

• SGLT2i appear to be well-tolerated in patients with dystrophin-deficient cardiomyopathy.
• Preliminary data suggests SGLT2i may improve cardiac function and slow disease progression.
• Further long-term studies are needed to confirm these findings and establish optimal use.