Recombinant human insulin-like growth factor-1 therapy for 6 months improves growth but not motor function in boys with Duchenne muscular dystrophy.
Muscle Nerve
; 61(5): 623-631, 2020 05.
Article
en En
| MEDLINE
| ID: mdl-32108355
ABSTRACT
INTRODUCTION:
Recombinant human insulin-like growth factor-1 (rhIGF-1) is a growth factor and has anabolic effects on muscle. We investigated whether rhIGF-1 therapy 1) improves or preserves muscle function; and 2) improves growth in boys with Duchenne muscular dystrophy (DMD).METHODS:
In this study we compared prepubescent, ambulatory, glucocorticoid-treated boys with DMD (n = 17) vs controls (glucocorticoid therapy only, n = 21) in a 6-month-long, prospective, randomized, controlled trial of subcutaneous rhIGF-1 therapy. The primary outcome was 6-minute walk distance (6MWD). Secondary outcomes included height velocity (HV), change in height standard deviation score (ΔHtSDS), motor function, cardiopulmonary function, body composition, insulin sensitivity, quality of life, and safety.RESULTS:
Change in 6MWD was similar between groups (rhIGF-1 vs controls [mean ± SD] 3.4 ± 32.4 vs -5.1 ± 50.2 meters, P = .53). Treated subjects grew more than controls (HV 6.5 ± 1.7 vs 3.3 ± 1.3 cm/year, P < .0001; 6-month ΔHtSDS 0.25, P < .0001). Lean mass and insulin sensitivity increased in treated subjects.DISCUSSION:
In boys with DMD, 6 months of rhIGF-1 therapy did not change motor function, but it improved linear growth.Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Estatura
/
Proteínas Recombinantes
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Resistencia a la Insulina
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Factor I del Crecimiento Similar a la Insulina
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Sustancias de Crecimiento
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Distrofia Muscular de Duchenne
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Fuerza Muscular
Tipo de estudio:
Clinical_trials
Límite:
Child
/
Humans
/
Male
Idioma:
En
Revista:
Muscle Nerve
Año:
2020
Tipo del documento:
Article
País de afiliación:
Estados Unidos