Department of Pediatric Endocrinology, Emma Children's Hospital, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands.
Medical Library, Amsterdam UMC, University of Amsterdam, Amsterdam, Netherlands.
Eur J Endocrinol. 2020 Mar;182(3):351-361. doi: 10.1530/EJE-19-0874.
To provide an overview of cognitive and motor outcome, and quality of life (QoL) in patients with congenital central hypothyroidism (CH-C).
Systematic review with individual patient data (IPD) meta-analysis.
OVID MEDLINE, EMBASE and PsycInfo were searched from inception to June 11th, 2019. Studies in patients with CH-C, either isolated or with multiple pituitary hormone deficiency (MPHD), were included if CH-C patients could be separated from any additional patient groups. Primary outcomes were full-scale intelligence quotient (FSIQ) and motor outcome; secondary outcome was QoL. Following data-extraction, one-stage IPD meta-analysis was performed, fitting a linear mixed model with FSIQ as dependent variable. Random intercepts were fitted for each study.
Six studies measuring FSIQ were eligible for meta-analysis, comprising 30 CH-C patients (20 males; 27 MPHD patients). FSIQ range was wide (64-123). Mean weighted FSIQ was 97 (95% CI: 88-105). Twenty-seven percent had an FSIQ below 85 (≥1 s.d. below norm score), and 10% below 70 (≥2 s.d. below norm score). There was no significant association between FSIQ and sex or age. Age at treatment initiation was available from three studies only, thus impeding a reliable analysis of this parameter. Motor outcome and QoL were each studied in one study; no quantitative analyses could be performed for these outcomes.
A wide range in FSIQ scores was observed in CH-C patients. Results should be interpreted with caution, because included patients mainly had MPHD and age at treatment initiation was unknown for the majority of patients.
概述先天性中枢性甲状腺功能减退症（CH-C）患者的认知和运动结果以及生活质量（QoL）。
系统评价与个体患者数据（IPD）荟萃分析。
从 OVID MEDLINE、EMBASE 和 PsycInfo 中搜索，检索时间截至 2019 年 6 月 11 日。如果 CH-C 患者可以与任何其他患者群体分开，则纳入患有 CH-C 的患者，无论其是否患有多种垂体激素缺乏症（MPHD）。主要结局为全面智商（FSIQ）和运动结果；次要结局为 QoL。在提取数据后，进行了 IPD 一阶段荟萃分析，将 FSIQ 作为因变量拟合线性混合模型。为每个研究拟合随机截距。
有 6 项研究测量了 FSIQ，符合纳入标准，共纳入 30 例 CH-C 患者（20 例男性；27 例 MPHD 患者）。FSIQ 范围很广（64-123）。加权平均 FSIQ 为 97（95%CI：88-105）。27%的患者 FSIQ 低于 85（低于正常分数 1 个标准差），10%的患者 FSIQ 低于 70（低于正常分数 2 个标准差）。FSIQ 与性别或年龄之间无显著相关性。仅从 3 项研究中获得了治疗开始时的年龄，因此无法对该参数进行可靠的分析。有 1 项研究研究了运动结果，有 1 项研究研究了 QoL，对于这些结果无法进行定量分析。
在 CH-C 患者中观察到 FSIQ 评分范围广泛。由于纳入的患者主要患有 MPHD，且大多数患者的治疗开始年龄未知，因此结果应谨慎解释。
