Gou Peng, Cheng Xinran, Leng Jie, Su Na
Department of Children Genetics and Endocrinology and Metabolism, Chengdu Women and Children Center Hospital, Chengdu, Sichuan, 610074, People's Republic of China.
Ther Clin Risk Manag. 2022 Mar 16;18:113-124. doi: 10.2147/TCRM.S363564. eCollection 2022.
This study aimed to evaluate the clinical efficacy of recombinant human growth hormone (rhGH) in the treatment of children with idiopathic short stature (ISS) and growth hormone deficiency (GHD) and to explore the related factors affecting treatment efficacy.
The current research reflects a real-world study. A total of 79 patients with ISS and 95 patients with GHD (both groups pre-puberty) who had been treated with rhGH for more than one year from January 2010 to September 2019 were included in this study. The patients were divided into two groups, ie, an ISS and a GHD group, respectively. The growth indexes, such as chronological age (CA), bone age (BA), height standard deviation score (HtSDS), insulin-like growth factor-1 (IGF-1) SDS, and body mass index were recorded and compared between the two groups before and after treatment. The treatment efficacy was evaluated according to changes in HtSDS before and after treatment, and the influencing factors of clinical efficacy were analyzed using a multivariate regression model.
At the start of treatment, the differences in CA, BA, height, weight, sexual development stage, HtSDS, mid-parental height SDS, and IGF-1 SDS between the two groups were not statistically significant (P > 0.05). However, the initial dose of rhGH in the GHD group was significantly lower than in the ISS group (P < 0.001). Following rhGH treatment, the differences in CA, BA, BA/CA ratio, and IGF-1 SDS measured at 6, 12, 18, and 24 months between the ISS and GHD groups were not statistically significant, while the difference in HtSDS measured at 6 months was statistically significant. With the extension of rhGH treatment time, the annual growth rate (GV) gradually decreased, and the difference between HtSDS and the baseline gradually increased; however, the differences between the ISS and GHD groups were not statistically significant. The most important factor affecting the treatment efficacy for patients with ISS was age at the start of treatment; the most important factors affecting the treatment efficacy for patients with GHD were age and IGF-1 SDS.
Recombinant human growth hormone treatment can significantly improve the height of patients with ISS and GHD. There was no significant difference in growth rate between patients with ISS and those with GHD at relatively high doses. The common factor affecting the treatment efficacy of the two groups was the age at the start of treatment. During treatment, monitored data indicated that rhGH treatment of GHD and ISS thyroid function showed a clinical phenomenon in the form of increased free triiodothyronine, rather than hypothyroidism, which was rarely reported in existing studies.
本研究旨在评估重组人生长激素（rhGH）治疗特发性矮小（ISS）和生长激素缺乏症（GHD）患儿的临床疗效，并探讨影响治疗效果的相关因素。
本研究为一项真实世界研究。纳入2010年1月至2019年9月期间接受rhGH治疗超过1年的79例ISS患儿和95例GHD患儿（两组均为青春期前）。将患儿分为两组，即ISS组和GHD组。记录并比较两组治疗前后的生长指标，如实际年龄（CA）、骨龄（BA）、身高标准差评分（HtSDS）、胰岛素样生长因子-1（IGF-1）标准差评分及体重指数。根据治疗前后HtSDS的变化评估治疗效果，并采用多因素回归模型分析临床疗效的影响因素。
治疗开始时，两组患儿在CA、BA、身高、体重、性发育阶段、HtSDS、父母平均身高标准差评分及IGF-1标准差评分方面的差异无统计学意义（P>0.05）。然而，GHD组rhGH的初始剂量显著低于ISS组（P<0.001）。rhGH治疗后，ISS组和GHD组在6、12、18和24个月时测量的CA、BA、BA/CA比值及IGF-1标准差评分差异无统计学意义，而6个月时测量的HtSDS差异有统计学意义。随着rhGH治疗时间的延长，年生长速率（GV）逐渐降低，HtSDS与基线的差异逐渐增大；然而，ISS组和GHD组之间的差异无统计学意义。影响ISS患儿治疗效果的最重要因素是治疗开始时的年龄；影响GHD患儿治疗效果的最重要因素是年龄和IGF-1标准差评分。
重组人生长激素治疗可显著提高ISS和GHD患儿的身高。在相对高剂量时，ISS患儿和GHD患儿的生长速率无显著差异。影响两组治疗效果的共同因素是治疗开始时的年龄。治疗期间监测数据表明，rhGH治疗GHD和ISS时甲状腺功能呈现游离三碘甲状腺原氨酸升高的临床现象，而非甲状腺功能减退，这在现有研究中鲜有报道。
