Early start of growth hormone is associated with positive effects on auxology and metabolism in Prader-Willi-syndrome

• Published in: Orphanet journal of rare diseases Vol. 15; no. 1; pp. 1 - 283
• Main Authors: Magill, Lucy, Laemmer, Constanze, Woelfle, Joachim, Fimmers, Rolf, Gohlke, Bettina
• Format: Journal Article
• Language: English
• Published: London BioMed Central Ltd 12.10.2020; BioMed Central; BMC
• Subjects: Age; Analysis; Blood glucose; Body composition; Body fat; Body mass index; Carbohydrate and lipid metabolism; Carbohydrate metabolism; Carbohydrates; Children; Cholesterol; Fasting; Glycosylated hemoglobin; Growth hormone therapy; Growth hormones; Health care; Homeostasis; Hypothalamus; Independent sample; Insulin; Insulin resistance; Insulin-like growth factor-I; Lean body mass; Lipid metabolism; Lipids; Low density lipoprotein; Low density lipoproteins; Males; Medical research; Metabolism; Physiological aspects; Pituitary; Prader-Willi syndrome; Rare diseases; Reagents; Somatotropin; Triglycerides; Germany
• ISSN: 1750-1172; 1750-1172
• DOI: 10.1186/s13023-020-01527-0

Prader-Willi-Syndrome (PWS) is characterized by hypothalamic-pituitary dysfunction. Recent research suggests starting growth hormone-treatment (GHT) as soon as possible. The aim of this study is to analyze possible differences in auxological parameters, carbohydrate and lipid metabolism between two groups of children with PWS that started GHT either during or after their first year of life. Retrospective longitudinal study of 62 children (31 males) with genetically confirmed PWS. Upon diagnosis all children were offered GHT, some started immediately, others commenced later. Cohort A (n = 21; 11 males) started GHT at 0.3-0.99 yrs. (mean 0.72 yrs) and Cohort B (n = 41; 20 males) commenced GHT at 1.02-2.54 yrs. (mean 1.42 yrs) of age. Fasting morning blood samples and auxological parameters were obtained before the start of therapy and semi-annually thereafter. Differences between the two cohorts were estimated with a linear mixed-effect model. Mean length/height-SDS.sub.PWS differed significantly between the groups [1 yr: A: 0.37 ([+ or -]0.83) vs B: 0.05 ([+ or -]0.56); 5 yrs.: A: 0.81 ([+ or -]0.67) vs B: 0.54 ([+ or -]0.64); p = 0.012]. No significant differences were found in BMI, lean body mass or body fat. An early start of GHT during the first year of life seems to have a favorable effect on height-SDS and metabolic parameters.