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<p>Cardiac disease has become the primary cause of death among individuals with Duchenne Muscular Dystrophy. There are currently no published data on the use of sodium-glucose cotransporter-2 inhibitors (SGLT2i) in patients with dystrophin deficient cardiomyopathy. The purpose of this study was to analyze the use of SGLT2i in patients with dystrophin deficient cardiomyopathy and describe indications for therapy, effects on cardiac function, and adverse effects. We utilized the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) dystrophinopathy registry to review patients on SGLT2i. 80 patients were initiated on an SGLT2i. The median age at initiation was 19.6 years and median weight was 66.8&#xa0;kg. Most patients were already prescribed three other cardiac medications classes. At initiation, the median ejection fraction (EF) was 37% (IQR 30.0-42.3%) by echocardiogram and 37% (IQR 31.5–41.0%) by cardiac magnetic resonance imaging (cMRI). Follow-up data was collected for 55 patients with a median follow-up time of 1.4 years. At follow-up, the median EF was 40% (IQR 32-47.8%) by echocardiogram and 39% (IQR 33-40.2%) by cMRI. There were 14 adverse events which included bone fracture (5%), acute kidney injury (5%), urinary tract infection (2.5%), genitourinary infection (1.3%) and others (3.8%). Seven patients (8.8%) required permanent discontinuation. This medication appears well tolerated with a low incidence of adverse effects. Longer term follow-up data is needed. However, SGLT2i may represent an additional therapy for slowing the progression of cardiac disease.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年8月1日

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  • Rachel E. Harris,
  • Carol A. Wittlieb-Weber,
  • Chet Villa,
  • Paul Esteso,
  • Svetlana Shugh,
  • Emily Hayes,
  • Christopher Knoll,
  • Ashwin K. Lal,
  • Deepa Mokshagundam,
  • Renata Shih,
  • Laura Radel,
  • Elizabeth L. Profita,
  • Aislinn DeSieghardt,
  • Benjamin Kroslowitz,
  • Joseph Spinner

摘要

Cardiac disease has become the primary cause of death among individuals with Duchenne Muscular Dystrophy. There are currently no published data on the use of sodium-glucose cotransporter-2 inhibitors (SGLT2i) in patients with dystrophin deficient cardiomyopathy. The purpose of this study was to analyze the use of SGLT2i in patients with dystrophin deficient cardiomyopathy and describe indications for therapy, effects on cardiac function, and adverse effects. We utilized the Advanced Cardiac Therapies Improving Outcomes Network (ACTION) dystrophinopathy registry to review patients on SGLT2i. 80 patients were initiated on an SGLT2i. The median age at initiation was 19.6 years and median weight was 66.8 kg. Most patients were already prescribed three other cardiac medications classes. At initiation, the median ejection fraction (EF) was 37% (IQR 30.0-42.3%) by echocardiogram and 37% (IQR 31.5–41.0%) by cardiac magnetic resonance imaging (cMRI). Follow-up data was collected for 55 patients with a median follow-up time of 1.4 years. At follow-up, the median EF was 40% (IQR 32-47.8%) by echocardiogram and 39% (IQR 33-40.2%) by cMRI. There were 14 adverse events which included bone fracture (5%), acute kidney injury (5%), urinary tract infection (2.5%), genitourinary infection (1.3%) and others (3.8%). Seven patients (8.8%) required permanent discontinuation. This medication appears well tolerated with a low incidence of adverse effects. Longer term follow-up data is needed. However, SGLT2i may represent an additional therapy for slowing the progression of cardiac disease.