A Case of Refractory Heart Failure in Becker Muscular Dystrophy Improved With Corticosteroid Therapy

Abstract

SummaryThe patient was a 26 year-old man who was referred to our hospital in June 2011 because of severe heart failure. At age 24 years, he was found to have Becker muscular dystrophy. He received enalapril for cardiac dysfunction; however, he had worsening heart failure and was thus referred to our hospital. Echocardiography showed enlargement of the left ventricle, with a diastolic dimension of 77 mm and ejection fraction of 19%. His condition improved temporarily after an infusion of dobutamine and milrinone. He was then administered amiodarone for ventricular tachycardia; however, he subsequently developed hemoptysis. Amiodarone was discontinued and corticosteroid pulse therapy was administered followed by oral prednisolone (PSL). His creatinine phosphokinase (CPK) level and cardiomegaly improved after the corticosteroid therapy. The PSL dose was reduced gradually, bisoprolol was introduced, and the catecholamine infusion was tapered. A cardiac resynchronization device was implanted; however, the patient's condition gradually worsened, which necessitated dobutamine infusion for heart failure. We readministered 30 mg PSL, which decreased the CPK level and improved the cardiomegaly. The dobutamine infusion was discontinued, and the patient was discharged. He was given 7.5 mg PSL as an outpatient, and he returned to normal life without exacerbation of the heart failure. There are similar reports showing that corticosteroids are effective for skeletal muscle improvement in Duchenne muscular dystrophy; however, their effectiveness for heart failure has been rarely reported. We experienced a case of Becker muscular dystrophy in which corticosteroid therapy was effective for refractory heart failure. (Int Heart J 2016; 57: 640-644) Key words: Cardiomyopathy, Prednisolone (PSL), Creatinine phosphokinase (CPK) M uscular dystrophy is a hereditary disease that causes degeneration and necrosis of the muscle fibers, and results in progressive muscle weakness. Myocardial disorder is known to be a complication. Muscular dystrophy is classified into several types according to etiology and clinical manifestation. The etiology of Duchenne muscular dystrophy (DMD) or Becker muscular dystrophy (BMD) is the abnormal production of dystrophin. BMD results from a partial defect of dystrophin, whereas DMD is caused by a complete loss of the protein.1) Heart failure may be one of the main clinical manifestations of DMD and BMD, and sometimes appears to be the only initial manifestation without skeletal muscle weakness in BMD.The combination of an angiotensin-converting enzyme (ACE) inhibitor and a β-blocker is known to be effective for cardiac dysfunction in patients with DMD or BMD.2,3) However, ACE inhibitors and β-blockers would not influence the muscle degeneration caused by DMD or BMD of an inherited etiology. Editorial p.527Corticosteroid therapy for DMD has shown potential for improving the symptoms mainly by slowing the rate of muscle weakness progression, or stabilizing muscle strength and function.4) Recently, it was report...

Variability and trends in corticosteroid use by male United States participants with Duchenne muscular dystrophy in the Duchenne Registry

“…Among participants with Becker, a considerable minority (25.3%) had used corticosteroids. Although there are case reports suggesting that some patients with Becker may benefit from corticosteroid therapy [28, 29], risks of long-term corticosteroid use must be weighed against uncertainties of benefit in this population. It is also possible that some patients diagnosed with Becker that were prescribed corticosteroids may have been thought to have Duchenne or intermediate dystrophinopathy.…”

Beta-blocker initiation under dobutamine infusion in acute advanced heart failure: a target trial emulation with observational data

“…Currently, no evidence, guidelines, or physiological rationale seem to provide strong support for this practice. As mentioned in some case reports, 9 , 12 physicians may expect beta-blockers to provide antiarrhythmic or cardioprotective effects even under dobutamine infusion. Additionally, physicians may want to initiate beta-blockers early to secure their long-term use, as suggested in previous studies.…”

Cardiac Involvement in Emery-Dreifuss Muscular Dystrophy and Related Management Strategies

“…1) Compared with Duchenne and Becker muscular dystrophy (DMD and BMD), the clinical course for its treatment is benign. [2][3][4] The syndrome was named as Emery-Dreifuss muscular dystrophy (EDMD), which is characterized by the following triad. First, a gradual progressive wasting and weakness in the scapulohumeroperoneal muscles, for which symptoms in most cases can appear in early childhood (< 15 years); however, the progress in muscle wasting is slow with loss of ambulation being reported only for exceptional cases.…”