Dutch Growth Research Foundation, Rotterdam, Netherlands.
Erasmus University Medical Center-Sophia Children's Hospital, Department of Pediatrics, Subdivision of Endocrinology, Rotterdam, Netherlands.
J Clin Endocrinol Metab. 2018 Oct 1;103(10):3714-3719. doi: 10.1210/jc.2018-00687.
Infants and toddlers with Prader-Willi syndrome (PWS) have mental and motor developmental delay. Short-term data suggest a positive effect of GH on mental and motor development in infants and children with PWS. There are, however, no longer-term results about the effects of GH treatment on mental and motor development.
To investigate the longer-term effects of GH on psychomotor development in infants and toddlers with PWS and the effect of age at start of GH treatment on psychomotor development.
Prospective cohort study during 3 years of GH treatment.
The PWS Reference Center in the Netherlands.
All children were treated with GH 1 mg/m2/d (≈0.035 mg/kg/d).
Mental and motor developmental age assessed with Bayleys Scales of Infant Development II and expressed as percentage of the expected development (100%).
During 3 years of GH, mean (SEM) mental development increased from 58.1% (2.8) at baseline to 79.6% (3.7), and motor development increased from 41.9% (2.9) to 78.2% (3.9; both P < 0.01). A lower baseline psychomotor development and a younger age at start of GH treatment were associated with a higher increase in mental and motor development (P < 0.01).
Mental and motor development increased significantly during 3 years of GH treatment, reducing the gap between infants with PWS and healthy peers. A younger age at start of GH treatment leads to greater improvement in psychomotor development.
患有普拉德-威利综合征（PWS）的婴儿和幼儿存在智力和运动发育迟缓。短期数据表明，GH 对 PWS 婴儿和儿童的智力和运动发育有积极影响。然而，目前尚无关于 GH 治疗对精神运动发育的长期影响的结果。
调查 GH 对 PWS 婴儿和幼儿精神运动发育的长期影响，以及 GH 治疗开始时的年龄对精神运动发育的影响。
在 GH 治疗的 3 年内进行的前瞻性队列研究。
荷兰的 PWS 参考中心。
所有儿童均接受 GH 1 mg/m2/d（≈0.035 mg/kg/d）治疗。
采用贝利婴幼儿发展量表第二版评估智力和运动发育年龄，并以预期发育的百分比（100%）表示。
在 GH 治疗的 3 年内，平均（SEM）精神发育从基线时的 58.1%（2.8）增加到 79.6%（3.7），运动发育从 41.9%（2.9）增加到 78.2%（3.9；均 P < 0.01）。较低的基线精神运动发育和较年轻的 GH 治疗起始年龄与精神和运动发育的更高增加相关（P < 0.01）。
GH 治疗 3 年内，精神和运动发育显著增加，缩小了 PWS 婴儿与健康同龄人的差距。GH 治疗开始时的年龄越小，精神运动发育的改善越大。
