Hospital for Children and Adolescents, Paediatrics, University of Helsinki, Helsinki, Finland.
Acta Paediatr. 2010 Nov;99(11):1712-8. doi: 10.1111/j.1651-2227.2010.01904.x. Epub 2010 Jul 5.
To assess the effects of recombinant human growth hormone (rhGH) treatment in children with Prader-Willi syndrome.
A 1-year study and an observational follow-up visit 10 years later.
In 20 patients with Prader-Willi syndrome (PWS): clinical assessment, laboratory tests, body composition analysis by dual energy X-ray absorptiometry, sleep polygraphy, health-related quality of life assessed by 16D.
Only two patients had normal growth hormone secretion at baseline. All patients were significantly shorter than their expected heights, but experienced catch-up growth during growth hormone treatment. At follow-up, 13 patients had reached adult heights and were markedly taller than historical controls. The cumulative dose of rhGH over 10 years correlated inversely with the total body fat percentage (p = 0.033). However, patients remained severely obese at 10 years. Sleep polygraphy was abnormal in more than half of the patients. Health-related quality of life of the patients remained substantially below that of normal population.
Growth hormone markedly improved adult height in subjects with PWS when compared to historical data. The cumulative dose of growth hormone correlated with reduction in body fat; nevertheless, patients remained severely obese.
评估重组人生长激素（rhGH）治疗普拉德-威利综合征（PWS）儿童的效果。
为期 1 年的研究及 10 年后的观察随访。
对 20 例 PWS 患者：进行临床评估、实验室检查、双能 X 线吸收法进行身体成分分析、睡眠描记术、采用 16D 评估健康相关生活质量。
仅 2 例患者基础状态时生长激素分泌正常。所有患者身高均明显低于预期值，但在生长激素治疗期间实现追赶生长。随访时，13 例患者已达到成人身高，且明显高于历史对照。10 年内 rhGH 的累积剂量与总体脂肪百分比呈负相关（p = 0.033）。然而，患者在 10 年后仍严重肥胖。超过一半的患者睡眠描记术异常。患者的健康相关生活质量仍明显低于正常人群。
与历史数据相比，生长激素可显著改善 PWS 患者的成年身高。生长激素的累积剂量与体脂减少相关；然而，患者仍严重肥胖。
