Author + information
- Michelle Ploutza,b,
- Ryan Moorea,b,
- Masatoshi Ashikia,b,
- Bethany Wisotzkeya,b,
- BreAnn Taylora,b,
- Christopher Spurneya,b,
- Michael Taylora,b and
- John Jefferiesa,b
Background: Cardiomyopathy is a leading cause of death in Duchenne muscular dystrophy (DMD). Eplerenone can prevent progressive left ventricular (LV) dysfunction when added to baseline therapies; however, it is not readily available and has a higher cost point. We, therefore, tested the hypothesis that spironolactone would be a safe alternative to eplerenone which may provide similar myocardial protective effects.
Methods: This is a prospective-retrospective observational cohort study conducted between May 2012 and October 2015. Boys with DMD greater than 8 years of age with evidence of myocardial fibrosis on cardiac MRI were eligible for participation. Patients < 25 kg were started on spironolactone 12.5 mg daily and up-titrated to 25 mg daily after 4 weeks. Patients > 25 kg were started on spironolactone 25 mg daily. Patients could also be on clinician-directed baseline therapies. Serial blood work was obtained and cardiac MRI was performed annually.
Results: Ninety-nine boys were enrolled with a mean follow-up of 23 months. The mean age was 14.7 years (SD 3.4 years) and the mean weight was 54.2 kg (SD 18.2 kg). A majority of patients were on ACE-I/ARB (90%), beta blockers (78.8%), and steroids (96%). Serum potassium was normal in all but 2 patients. One patient had acute kidney injury from medication non-adherence requiring admission for hyperkalemia. The other patient developed chronic kidney disease and spironolactone was stopped electively. Cystatin C remained normal in all but 11 patients. Nine were able to continue therapy. Of the 3 remaining patients, one had decompensated heart failure and died shortly thereafter, one had decompensated heart failure and went on to ventricular assist device placement, and the other had elevated cystatin C prior to starting therapy which remained stable. The mean LV ejection fraction at spironolactone initiation was 54.6% (SD 8.4%) and at study end was 52.2% (SD 8.4%).
Conclusions: Spironolactone is a safe therapy for boys with DMD. The mean LV ejection fraction was well-preserved over 23 month follow-up period. Further studies are necessary to determine whether spironolactone will have protective effects on myocardial fibrosis and ventricular remodeling.
Poster Hall, Hall C
Saturday, March 18, 2017, 3:45 p.m.-4:30 p.m.
Session Title: Heart Failure and Cardiomyopathies: What Next When All Else Is Failing?
Abstract Category: 14. Heart Failure and Cardiomyopathies: Therapy
Presentation Number: 1248-256
- 2017 American College of Cardiology Foundation