Related Experiment Video
Updated: Jun 23, 2026
Assessing Functional Performance in the Mdx Mouse Model
Published on: March 28, 2014
A preliminary randomized study of growth hormone administration in Becker and Duchenne muscular dystrophies
Antonio Cittadini1, Lucia Ines Comi, Salvatore Longobardi
1Department of Internal Medicine and Cardiovascular Sciences, University Federico II, Naples, Italy. cittadin@unina.it
Insights
Growth hormone (GH) therapy improved cardiac function in patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). The 3-month treatment showed cardiac hypertrophy and reduced brain natriuretic peptide levels without side effects.
Area of Science:
- Cardiology
- Endocrinology
- Genetics
Background:
- Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) often lead to dilated cardiomyopathy.
- Growth hormone (GH) has demonstrated benefits in experimental heart failure models.
Purpose of the Study:
- To investigate the effects of GH administration on cardiac function in patients with DMD and BMD.
- To assess cardiac remodeling and functional changes in response to GH therapy.
Main Methods:
- A double-blind, randomized study involving 6 DMD and 10 BMD patients with cardiac involvement.
- Patients received either placebo or recombinant human GH for 3 months.
- Evaluated left ventricular (LV) mass, relative wall thickness, LV end-systolic stress, systolic function, and cardiac biomarkers.
Main Results:
- GH treatment significantly increased LV mass (16% in BMD, 29% in DMD) and relative wall thickness (+19%).
- LV end-systolic stress decreased significantly (13% in BMD, 33% in DMD), with slight improvements in systolic function.
- Brain natriuretic peptide levels decreased by 40%, while cytokine levels remained unchanged. No side effects or changes in skeletal muscle function were observed.
Conclusions:
- Three-month GH therapy in DMD and BMD patients promotes cardiac hypertrophy.
- GH treatment leads to reduced brain natriuretic peptide levels and improved systolic function.
- The therapy demonstrated a favorable safety profile with no observed side effects or impact on skeletal muscle function.
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.
More Related Videos
06:52Behavioral and Locomotor Measurements Using an Open Field Activity Monitoring System for Skeletal Muscle Diseases
Published on: September 29, 2014
09:18Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy
Published on: January 12, 2019