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

Abstract

Background 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. Study design 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. Results Mean length/height-SDSPWS differed significantly between the groups [1 yr: A: 0.37 (±0.83) vs B: 0.05 (±0.56); 5 yrs.: A: 0.81 (±0.67) vs B: 0.54 (±0.64); p = 0.012]. No significant differences were found in BMI, lean body mass or body fat. Low-density cholesterol was significantly lower in A than in B [LDL: 1 yr: A: 79 (±20) mg/dl vs B: 90 (±19) mg/dl; 5 yrs.: A: 91(±18) mg/dl vs 104 (±26) mg/dl; p = 0.024]. We found significant differences in the glucose homeostasis between the groups [fasting insulin: p = 0.012; HOMA-IR: p = 0.006; HbA1c: p < 0.001; blood glucose: p = 0.022]. Conclusions An early start of GHT during the first year of life seems to have a favorable effect on height-SDS and metabolic parameters.

Effects of early recombinant human growth hormone treatment in young Chinese children with Prader–Willi syndrome

“…Moreover, FG was not different between the two groups, and no cases of diabetes were observed, although transient hyperglycemia was noted in 5 patients. This was similar to most previous studies about adult or older children [ 41 – 43 ]. However, different from those studies in which rhGH treatment had no effects on glucose homeostasis in PWS patients [ 37 , 44 – 46 ].…”

Early recombinant human growth hormone treatment improves mental development and alleviates deterioration of motor function in infants and young children with Prader–Willi syndrome

“…The merit of our study was the simultaneous use of PDMS-2 and the GDS-C to comprehensively evaluate, monitor, and accurately capture the development of different motor skills and the overall development of pediatric patients with PWS, and this study enrolled younger patients with a median age of 7.0 months, in line with increasing evidence supporting the benefit of early intervention with rhGH [ 1 , 2 , 6 , 9 , 14 , 16 , 36 ]. However, the short duration and small number of patients may not be able to reveal the full spectrum of rhGH potency, so future studies should recruit larger sample sizes and have longer study periods to elucidate the effect of rhGH treatment on motor and mental outcomes.…”

Correlation of Genotype and Perinatal Period, Time of Diagnosis and Anthropometric Data before Commencement of Recombinant Human Growth Hormone Treatment in Polish Patients with Prader–Willi Syndrome

“…Interestingly, the height SDS still did not show any differences at the rhGH start. In the view of the positive effects of rhGH therapy in modifying not only the anthropometric characteristics but hypothetically also the satiety and appetite behaviour, it seems to be a crucial step to diagnose PWS in the first months of life and therefore start the treatment early [ 38 , 39 ]. Commencement of the rhGH therapy, which has a potential beneficial influence on metabolic state, as well as on psychomotor development, before the nutritional phase of increased appetite may modify the subsequent nutritional phenotype of patients with PWS.…”