Çetinkaya Semra, Poyrazoğlu Şükran, Baş Firdevs, Ercan Oya, Yıldız Metin, Adal Erdal, Bereket Abdullah, Abalı Saygın, Aycan Zehra, Erdeve Şenay Savaş, Berberoğlu Merih, Şıklar Zeynep, Tayfun Meltem, Darcan Şükran, Mengen Eda, Bircan İffet, Jones Filiz Mine Çizmecioğlu, Şimşek Enver, Papatya Esra Deniz, Özbek Mehmet Nuri, Bolu Semih, Abacı Ayhan, Büyükinan Muammer, Darendeliler Feyza
Health Sciences University, Dr Sami Ulus Obstetrics and Gynecology, Children's Health and Disease, Health Implementation and Research Center, Department of Pediatric Endocrinology, Ankara, Turkey.
Istanbul University, Faculty of Medicine, Institute of Child Health, Department of Pediatric Endocrinology, Istanbul, Turkey.
J Pediatr Endocrinol Metab. 2018 Jan 26;31(2):175-184. doi: 10.1515/jpem-2017-0123.
The aim of the study was to assess the response to growth hormone (GH) treatment in very young patients with GH deficiency (GHD) through a national, multi-center study. Possible factors affecting growth response were assessed (especially mini-puberty).
Medical reports of GHD patients in whom treatment was initiated between 0 and 3 years of age were retrospectively evaluated.
The cohort numbered 67. The diagnosis age was 12.4±8.6 months, peak GH stimulation test response (at diagnosis) as 1.0±1.4 ng/mL. The first and second years length gain was 15.0±4.3 and 10.4±3.4 cm. Weight gain had the largest effect on first year growth response; whereas weight gain and GH dose were both important factors affecting second year growth response. In the multiple pituitary hormone deficiency (MPHD) group (n=50), first year GH response was significantly greater than in the isolated GH deficiency (IGHD) group (n=17) (p=0.030). In addition first year growth response of infants starting GH between 0 and 12 months of age (n=24) was significantly greater than those who started treatment between 12 and 36 months of age (n=43) (p<0.001). These differences were not seen in the second year. Δ Length/height standard deviation score (SDS), Δ body weight SDS, length/height SDS, weight SDS in MPHD without hypogonadism for the first year of the GH treatment were found as significantly better than MPHD with hypogonadism.
Early onsets of GH treatment, good weight gain in the first year of the treatment and good weight gain-GH dose in the second year of the treatment are the factors that have the greatest effect on length gain in early onset GHD. The presence of the sex steroid hormones during minipubertal period influence growth pattern positively under GH treatment (closer to the normal percentage according to age and gender).
本研究旨在通过一项全国性多中心研究，评估极年幼的生长激素缺乏症（GHD）患者对生长激素（GH）治疗的反应。评估了影响生长反应的可能因素（尤其是小青春期）。
对0至3岁开始治疗的GHD患者的医学报告进行回顾性评估。
该队列共有67例患者。诊断年龄为12.4±8.6个月，生长激素刺激试验峰值反应（诊断时）为1.0±1.4 ng/mL。第一年和第二年的身长增加分别为15.0±4.3厘米和10.4±3.4厘米。体重增加对第一年的生长反应影响最大；而体重增加和GH剂量都是影响第二年生长反应的重要因素。在多重垂体激素缺乏（MPHD）组（n = 50）中，第一年的GH反应明显大于孤立性生长激素缺乏（IGHD）组（n = 17）（p = 0.030）。此外，0至12个月开始使用GH的婴儿（n = 24）的第一年生长反应明显大于12至36个月开始治疗的婴儿（n = 43）（p < 0.001）。第二年未观察到这些差异。发现无性腺功能减退的MPHD患者在GH治疗第一年的身长/身高标准差评分（SDS）变化、体重SDS变化、身长/身高SDS、体重SDS明显优于有性腺功能减退的MPHD患者。
早期开始GH治疗、治疗第一年良好的体重增加以及治疗第二年良好的体重增加-GH剂量是对早发性GHD身长增加影响最大的因素。小青春期期间性类固醇激素的存在对GH治疗下的生长模式有积极影响（根据年龄和性别更接近正常百分比）。
