Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine, Cincinnati, OH, USA.
Bambino Gesù Children's Hospital, Rome, Italy.
Growth Horm IGF Res. 2021 Apr-Jun;57-58:101392. doi: 10.1016/j.ghir.2021.101392. Epub 2021 Apr 30.
We sought to obtain a better understanding of the burden of short stature using a systematic literature review.
Studies of the burden of short stature, of any cause in adults and children, were searched using Embase, MEDLINE and Cochrane databases in April 2020, capturing publications from 2008 onwards. Case series and populations with adult-onset growth hormone deficiency (GHD) were excluded.
Of 1684 publications identified, 41 studies (33 in children, 8 in adults) were included. All studies assessed human burden. Most study populations in children included short stature due to GHD, idiopathic short stature (ISS) and short stature after being born small for gestational age (SGA). In these populations, four studies showed that quality of life (QoL) in children with short stature was significantly worse than in children with normal stature. A significant association between QoL and short stature was observed in children with chronic kidney disease (CKD) (3 studies), achondroplasia (1 study) and transfusion-dependent β-thalassaemia (1 study), and in samples with mixed causes of short stature (3 studies). Three studies (one in GHD/ISS/SGA and two in CKD) found no significant association between short stature and QoL, and several studies did not report statistical significance. Approximately half of adult studies showed that QoL was reduced with short stature, and the other half showed no association. Two studies, one in adults with Prader-Willi syndrome and one in children with GHD, suggested a potential association between short stature and poorer cognitive outcomes. Three studies demonstrated an increased caregiver burden in parents of children with short stature.
Evidence suggests that, compared with those with normal stature, children and adults with short stature of any cause may experience poorer QoL. Further research could extend our understanding of the human burden in this field.
通过系统文献回顾，更好地了解身材矮小的负担。
2020 年 4 月，使用 Embase、MEDLINE 和 Cochrane 数据库检索了关于任何病因导致的成人和儿童身材矮小负担的研究，纳入了 2008 年以来发表的研究。排除了病例系列和成人发病生长激素缺乏症（GHD）人群。
在 1684 篇文献中，有 41 项研究（33 项在儿童中，8 项在成人中）被纳入。所有研究均评估了人类负担。大多数儿童研究人群包括由于 GHD、特发性身材矮小（ISS）和出生时小于胎龄（SGA）后身材矮小的儿童。在这些人群中，四项研究表明，身材矮小儿童的生活质量（QoL）显著低于正常身高儿童。在患有慢性肾脏病（CKD）的儿童（3 项研究）、软骨发育不全（1 项研究）和依赖输血的β-地中海贫血（1 项研究）以及混合病因身材矮小的样本中（3 项研究），观察到 QoL 与身材矮小之间存在显著相关性。三项研究（一项在 GHD/ISS/SGA，两项在 CKD）发现身材矮小与 QoL 之间无显著相关性，另有几项研究未报告统计学意义。约一半的成人研究表明，身材矮小会降低 QoL，另一半则没有关联。两项研究，一项在成年 Prader-Willi 综合征患者中，一项在儿童 GHD 患者中，表明身材矮小与认知结局较差之间可能存在关联。三项研究表明，身材矮小儿童的父母照顾者负担加重。
有证据表明，与正常身高者相比，任何病因导致的身材矮小的儿童和成人可能会经历较差的 QoL。进一步的研究可以扩展我们对该领域人类负担的理解。
