Key result
Growth hormone therapy increases left ventricular mass up to ~29% in muscular dystrophy cardiomyopathy.
Why the study?
Growth hormone has shown benefits in experimental heart failure, and DMD and BMD often develop dilated cardiomyopathy, motivating investigation of GH effects in these patients.
Does recombinant human growth hormone improve cardiac structure and function in patients with Duchenne and Becker muscular dystrophies complicated by cardiomyopathy?
RCT (n=16)
Double-blind
Randomized
Does recombinant human growth hormone improve cardiac structure and function in patients with Duchenne and Becker muscular dystrophies complicated by cardiomyopathy?
p-value: p=<0.01
A 3-month course of recombinant human growth hormone in patients with Duchenne and Becker muscular dystrophies induces a compensatory hypertrophic response and reduces brain natriuretic peptide levels without adverse effects.
GH may induce compensatory LV hypertrophy in DMD/BMD cardiomyopathy; extends small RCT evidence but requires longer outcome trials.
AIM: Since growth hormone (GH) has proven beneficial in experimental heart failure, and the natural history of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is frequently complicated by the development of dilated cardiomyopathy, we administered GH to six patients with DMD and 10 with BMD, with the evidence of cardiac involvement. METHODS AND RESULTS: Patients were randomized to receive for 3 months either placebo or recombinant human GH, in a double-blind fashion. In GH-treated patients, left ventricular (LV) mass increased by 16% in BMD and by 29% in DMD (both p<0.01), with a significant increase of relative wall thickness (+19%). Systemic blood pressure remained unchanged, while LV end-systolic stress fell significantly by 13% in BMD and by 33% in DMD, with a slight increase of systolic function indexes. No changes were observed related to cardiac arrhythmias and skeletal muscle function in the patient groups during the treatment period, nor any side effects were observed. Brain natriuretic peptide, interleukin-6, and tumor necrosis factor-alpha circulating levels were elevated at baseline. While brain natriuretic peptide decreased by 40%, cytokine levels did not exhibit significant variations during the treatment period. CONCLUSIONS: The 3-month GH therapy in patients with DMD and BMD induces a hypertrophic response associated with a significant reduction of brain natriuretic peptide plasma levels and a slight improvement of systolic function, no changes in skeletal muscle function, and no side effects.
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Antonio Cittadini (2003) conducted an RCT in Duchenne and Becker muscular dystrophies with cardiac involvement (n=16). Recombinant human growth hormone vs. Placebo was evaluated on Left ventricular mass (p=<0.01). Recombinant human growth hormone therapy for 3 months in patients with DMD and BMD increased left ventricular mass by 16% in BMD and 29% in DMD (both p<0.01) and reduced end-systolic stress.