Grootjen Lionne N, Trueba-Timmermans Demi J, Damen Layla, Mahabier Eva F, Kerkhof Gerthe F, Hokken-Koelega Anita C S
Dutch Reference Center for Prader-Willi Syndrome, 3015 CN Rotterdam, The Netherlands.
Department of Pediatrics, Subdivision of Endocrinology, Erasmus University Medical Center-Sophia Children's Hospital, 3015 CN Rotterdam, The Netherlands.
J Clin Med. 2022 Apr 29;11(9):2496. doi: 10.3390/jcm11092496.
Long-term effects of growth hormone (GH) treatment in young children with Prader-Willi syndrome (PWS) have never been compared with untreated age-matched controls with PWS, and it is unclear if starting GH in the first year of life is safe and more effective than starting GH in early childhood. We investigated the effects of long-term GH on body composition, anthropometrics and cognition in young children with PWS compared to untreated controls and assessed whether starting GH in the first year of life is optimal and safe. An open-label, prospective study was performed, comparing GH-treated children with untreated controls, and comparing children who started GH in the first year of life (subgroup A) with children who started between 2-5 years (subgroup C). A total of 82 GH-treated children with PWS and 22 age-matched controls with PWS were included. The main outcome measures were body composition, anthropometrics, IQ, and safety parameters. After 8 years, GH-treated children had significantly better body composition and were taller than age-matched controls. Subgroup A had a lower FM% trajectory during treatment than subgroup C and showed a greater and longer-term increase in the LBM index. After 8 years, subgroup A had a lower trunk/peripheral fat ratio ( = 0.043) and higher IQ ( = 0.043). No adverse effects of starting GH in the first year were found. Children with PWS who received long-term GH had a better body composition and growth than untreated age-matched controls and starting GH in the first year of life was optimal and safe.
生长激素（GH）治疗对普拉德-威利综合征（PWS）幼儿的长期影响从未与未接受治疗的年龄匹配的PWS对照儿童进行过比较，并且尚不清楚在生命的第一年开始使用GH是否比在幼儿期开始使用更安全、更有效。我们研究了长期使用GH对PWS幼儿身体成分、人体测量学和认知的影响，并与未治疗的对照组进行比较，同时评估在生命的第一年开始使用GH是否最佳且安全。我们进行了一项开放标签的前瞻性研究，将接受GH治疗的儿童与未治疗的对照组进行比较，并将在生命的第一年开始使用GH的儿童（A组）与在2至5岁之间开始使用GH的儿童（C组）进行比较。总共纳入了82名接受GH治疗的PWS儿童和22名年龄匹配的PWS对照儿童。主要结局指标为身体成分、人体测量学、智商和安全参数。8年后，接受GH治疗的儿童身体成分明显更好，且比年龄匹配的对照儿童更高。A组在治疗期间的脂肪量百分比轨迹低于C组，并且去脂体重指数有更大且更长期的增加。8年后，A组的躯干/外周脂肪比率更低（P = 0.043），智商更高（P = 0.043）。未发现生命第一年开始使用GH有不良影响。接受长期GH治疗的PWS儿童比未治疗的年龄匹配对照儿童有更好的身体成分和生长情况，并且在生命的第一年开始使用GH是最佳且安全的。
