Consequences of not treating children with laron syndrome (primary growth hormone insensitivity).
The follow-up of a large cohort of patients with Laron syndrome (LS) from infancy to adult age has enabled us to determine the effects of long-term insulin-like growth factor-I (IGF-I) deficiency on auxological, biochemical, physiological and psychological parameters. We found that early and continuous IGF-I deficiency (the anabolic effector of growth hormone) causes dwarfism, acromicria, organomicria, marked obesity, insulin resistance, retardation of skeletal maturation and osteoporosis, as well as muscular and central nervous tissue underdevelopment, and a series of biochemical changes including hypercholesterolemia. These multiple pathologies impair the quality of life of these patients. It is concluded that patients with LS need IGF-I replacement treatment throughout life.
Laron
Z
Z
Endocrinology and Diabetes Research Unit, Schneider Children's Medical Center, Tel Aviv University, Israel. laronz@clalit.org.il
D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D015331:Cohort Studies; D018450:Disease Progression; D005260:Female; D005500:Follow-Up Studies; D006130:Growth Disorders / Q000209:etiology* / Q000473:pathology / Q000503:physiopathology / Q000523:psychology / Q000628:therapy; D019382:Human Growth Hormone / Q000502:physiology*; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000172:deficiency / Q000627:therapeutic use; D008297:Male; D013577:Syndrome
D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I