Growth Hormone Normalises Height, Prediction of Final Height and Hand Length in Children with Prader-Willi Syndrome after 4 Years of Therapy

Abstract

Background: Based on the reported favourable effects of growth hormone (GH) treatment on growth and body composition in Prader-Labhart-Willi syndrome, we studied age dependency and the long-term effects on growth dynamics to elucidate the assumed hypothalamic GH deficiency. Methods: We examined 23 children treated with hGH (24 U/m²/week) during a median of 4 (range 1.5–5.5) years; group 1: 10 young underweight (age 0.3–4.1 years), group 2: 8 prepubertal overweight (age 3.7–9.5 years) and group 3: 5 pubertal overweight children (age 9.0–14.6 years). Results: After 4 years of therapy, height gain amounted to 1.8 SD; height (0.0 SD) and hand length (–0.2 SD) were normalised in the 2 prepubertal groups; in children above 6 years, height prediction approached parental target height. Weight for height rose in group 1 (to 0.64 SD) and decreased in group 2 (to 0.71 SD) to normal levels. Bone maturation of the pubertal children was too advanced to show a clear growth response to GH (height gain 0.42 SD). Even in this group, weight for height was reduced, but remained supernormal. Conclusion: Under exogenous GH, growth and body proportions are normalised in prepubertal children. With early institution of treatment, final height prediction reaches the parental target height range after 3 years. Such a growth-promoting effect of exogenous GH has so far only been described in children with GH deficiency.

Clinical Effects of Treatment for Hypogonadism in Male Adolescents with Prader-Labhart-Willi Syndrome

“…During long-term therapy with GH, stature normalized (growth data shown elsewhere [18]), but muscle mass remained reduced in boys with PWS. Irrespective of the DEXA method used, the deficit of LM even during GH therapy was found in several studies [20, 41].…”

“…The hypothalamic dysfunction causes growth hormone (GH) deficiency [3], hypogonadism [4,5,6] and a dysregulation of energy balance with hypoactivity [7, 8] and insatiable hunger [9,10,11], resulting in an increased fat and decreased lean body mass [12, 13]. In addition to control of nutrient intake and enhancement of activity, beneficial effects of early therapy of GH deficiency in PWS have been demonstrated [14,15,16]: short stature is normalized and muscle mass enhanced [8, 12,17,18,19,20]. …”

Increased Adrenal Androgen Levels in Patients with Prader-Willi Syndrome Are Associated with Insulin, IGF-I, and Leptin, but Not with Measures of Obesity

“…In a prospective study, we examined a group of 23 children, 11 girls and 12 boys, with genetically proven PWS, in whom growth and body composition before and during long-term GH therapy (GHT) were previously described [21, 22]. At baseline, the mean age was 5.6 (range 0.5–14.6) years and the bone age 6.15 (range 0.6–14.6) years.…”

Prader-Willi syndrome: new possibilities for its treatment in the children

“…Однако они не были контро-лируемыми и включали незначительное число боль-ных. В дальнейшем стабильный ростовой эффект и улучшение прогнозируемого конечного роста на фоне длительного применения р-ГР (2-5 лет) отме-чались у большинства пациентов [36,39,40].…”

“…В этом же году ис-следователи из США [42] представили результаты длительного лечения р-ГР (7,9 года) 21 пациента с СПВ (13 мальчиков, 8 девочек), конечный рост ко-торых составил -0,3 SDS. Ряд работ свидетельствует и о положительном влиянии р-ГР на параметры акромикрии, вплоть до нормализации размеров ки-стей и стоп [43][44][45][46]. В контролируемом рандомизированном 5-лет-нем исследовании у маленьких пациентов с СПВ (возраст от 3 до 7 лет) A. Lindgren и соавт.…”