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Aims <p>Fabry disease (FD) is a multisystemic disease affecting the heart and the kidneys of affected patients. In addition to FD-specific treatment, patients require concomitant medication for cardio- and nephroprotection. Sodium-dependent glucose transporter 2 inhibitors (SGLT2i) are recommended for patients with heart failure and/or kidney disease, but efficacy data for FD are scarce.</p> Methods and Results <p>In this multicenter study (<i>n</i> = 8), the effects of SGLT2i therapy after 12&#xa0;months of treatment in 48 patients (12 females) on FD-specific therapy were examined. Patients were retrospectively analyzed at three time points (before SGLT2i: <i>T</i><sub>-1</sub>; SGLT2i start: <i>T</i><sub>0</sub>; and end of observation: <i>T</i><sub>+1</sub>). Patients showed advanced cardiac manifestations with a high frequency of left ventricular hypertrophy (LVH) (females: 81.8% and males: 90.0%) at <i>T</i><sub>0</sub>. Males presented with a significantly lower left ventricular ejection fraction LVEF (56 [29–73]% versus 65 [38–78]%; <i>p</i> = 0.0113). There were no treatment-related adverse events or Fabry-associated clinical events (FACEs) between <i>T</i><sub>0</sub> and <i>T</i><sub>+1</sub>.</p> <p>Females showed a stable disease course independent of the concomitant treatment with SGLT2i. Males showed an eGFR decrease of 3.7&#xa0;ml/min/1.73 m<sup>2</sup> per year (<i>p</i> = 0.0018) before and an ameliorated decrease of 2.7&#xa0;ml/min/1.73 m<sup>2</sup> per year (<i>p</i> = 0.0182) after SGLT2i initiation. Importantly, a slight but significant improvement of LVEF by 0.6% per year (<i>p</i> = 0.0319) was observed, which was more prominent in males with a reduced LVEF (&lt; 50%) at baseline.</p> Conclusion <p>Treatment with SGLT2i of FD patients was safe and patients presented with stable disease courses. Especially males with reduced LVEF might benefit from SGLT2i treatment.</p> Graphical abstract <p></p>

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Impact of SGLT2 inhibitors in patients with Fabry disease

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年3月9日

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  • Malte Lenders,
  • Sima Canaan-Kühl,
  • Christine Kurschat,
  • Albina Nowak,
  • Constantin Gatterer,
  • Victoria Sokalski,
  • Fabian Braun,
  • Markus Cybulla,
  • Gere Sunder-Plassmann,
  • Peter Nordbeck,
  • Christoph Wanner,
  • Eva Brand

摘要

Aims

Fabry disease (FD) is a multisystemic disease affecting the heart and the kidneys of affected patients. In addition to FD-specific treatment, patients require concomitant medication for cardio- and nephroprotection. Sodium-dependent glucose transporter 2 inhibitors (SGLT2i) are recommended for patients with heart failure and/or kidney disease, but efficacy data for FD are scarce.

Methods and Results

In this multicenter study (n = 8), the effects of SGLT2i therapy after 12 months of treatment in 48 patients (12 females) on FD-specific therapy were examined. Patients were retrospectively analyzed at three time points (before SGLT2i: T-1; SGLT2i start: T0; and end of observation: T+1). Patients showed advanced cardiac manifestations with a high frequency of left ventricular hypertrophy (LVH) (females: 81.8% and males: 90.0%) at T0. Males presented with a significantly lower left ventricular ejection fraction LVEF (56 [29–73]% versus 65 [38–78]%; p = 0.0113). There were no treatment-related adverse events or Fabry-associated clinical events (FACEs) between T0 and T+1.

Females showed a stable disease course independent of the concomitant treatment with SGLT2i. Males showed an eGFR decrease of 3.7 ml/min/1.73 m2 per year (p = 0.0018) before and an ameliorated decrease of 2.7 ml/min/1.73 m2 per year (p = 0.0182) after SGLT2i initiation. Importantly, a slight but significant improvement of LVEF by 0.6% per year (p = 0.0319) was observed, which was more prominent in males with a reduced LVEF (< 50%) at baseline.

Conclusion

Treatment with SGLT2i of FD patients was safe and patients presented with stable disease courses. Especially males with reduced LVEF might benefit from SGLT2i treatment.

Graphical abstract