A preliminary randomized study of growth hormone administration in Becker and Duchenne muscular dystrophies.
OBJECTIVE
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.


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.
Cittadini
Antonio
A
Department of Internal Medicine and Cardiovascular Sciences, University Federico II, Naples, Italy. cittadin@unina.it
Ines Comi
Lucia
L
Longobardi
Salvatore
S
Rocco Petretta
Vito
V
Casaburi
Cosma
C
Passamano
Luigia
L
Merola
Bartolomeo
B
Durante-Mangoni
Emanuele
E
Saccà
Luigi
L
Politano
Luisa
L
D000293:Adolescent; D000328:Adult; D000704:Analysis of Variance; D006332:Cardiomegaly; D002648:Child; D004311:Double-Blind Method; D004562:Electrocardiography; D006331:Heart Diseases / Q000150:complications / Q000188:drug therapy / Q000503:physiopathology; D019382:Human Growth Hormone / Q000627:therapeutic use*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000032:analysis; D015850:Interleukin-6 / Q000097:blood; D008168:Lung / Q000503:physiopathology; D008297:Male; D008875:Middle Aged; D018482:Muscle, Skeletal / Q000503:physiopathology; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy* / Q000503:physiopathology; D020097:Natriuretic Peptide, Brain / Q000097:blood; D012044:Regression Analysis; D014409:Tumor Necrosis Factor-alpha / Q000032:analysis
D015850:Interleukin-6; D014409:Tumor Necrosis Factor-alpha; D020097:Natriuretic Peptide, Brain; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I