A comprehensive systematic review of health-related quality of life measures in short stature paediatric patients.

IF 3 3区 医学 Q2 ENDOCRINOLOGY & METABOLISM
Adekunle Adedeji, Stefanie Witt, Florian Innig, Julia Quitmann
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Abstract

This systematic review investigates Patient-reported Outcome Measures (PROMs) and Observed Reported Outcome Measures (ObsROMs) pertinent to assessing Health-Related Quality of Life (HRQoL) in short-stature paediatric patients, focusing on Achondroplasia (ACH), Growth Hormone Deficiency (GHD), Isolated Growth Hormone Deficiency (IGHD), and Small-for-Gestational-Age (SGA) diagnoses. Utilising rigorous selection criteria, 53 studies published from 1998 to 2023 were analysed, revealing a predominance of European-based research. Notably, the review elucidated the utilisation of disease-specific and generic HRQoL measures, showcasing the multifaceted nature of short-stature conditions and their impact across physical, emotional, and social domains. The Quality of Life in Short Stature Youth (QoLISSY), Paediatric Quality of Life Inventory (PedsQL), and KIDSCREEN emerged as frequently employed instruments, offering nuanced insights into HRQoL perceptions across diverse age demographics. Additionally, the review highlighted the adaptation of adult HRQoL measures for adolescent populations, signalling a need for age-appropriate assessment tools. Furthermore, integrating PROMs and ObsROMs in HRQoL assessment underscored a comprehensive approach, considering both subjective patient perspectives and observed outcomes. Future research directions encompass comprehensive search strategies, longitudinal studies with diverse populations, and the development of age-appropriate HRQoL assessment tools. In conclusion, this review emphasises the importance of comprehensive HRQoL assessment to address the diverse needs of short-stature paediatric patients effectively.

Abstract Image

身材矮小儿科患者健康相关生活质量测量的全面系统回顾。
本系统综述调查了与评估短身材儿科患者与健康相关的生活质量(HRQoL)有关的患者报告结果指标(PROMs)和观察报告结果指标(Observed Reported Outcome Measures,ObsROMs),重点关注软骨发育不全(ACH)、生长激素缺乏症(GHD)、孤立性生长激素缺乏症(IGHD)和妊娠期矮小(SGA)诊断。利用严格的筛选标准,对 1998 年至 2023 年间发表的 53 项研究进行了分析,结果显示以欧洲的研究为主。值得注意的是,综述阐明了疾病特异性和通用 HRQoL 测量方法的使用情况,展示了矮身材疾病的多面性及其对身体、情感和社会领域的影响。短身材青少年生活质量(QoLISSY)、儿科生活质量量表(PedsQL)和 KIDSCREEN 是经常使用的工具,它们提供了不同年龄人口对 HRQoL 感知的细微差别。此外,综述还强调了针对青少年群体对成人 HRQoL 测量方法的调整,这表明需要与年龄相适应的评估工具。此外,将 PROMs 和 ObsROMs 纳入 HRQoL 评估强调了一种综合方法,既要考虑患者的主观观点,又要考虑观察结果。未来的研究方向包括综合搜索策略、不同人群的纵向研究以及开发适合不同年龄的 HRQoL 评估工具。总之,本综述强调了全面的 HRQoL 评估对于有效满足矮身材儿科患者不同需求的重要性。
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来源期刊
Endocrine
Endocrine ENDOCRINOLOGY & METABOLISM-
CiteScore
6.50
自引率
5.40%
发文量
295
审稿时长
1.5 months
期刊介绍: Well-established as a major journal in today’s rapidly advancing experimental and clinical research areas, Endocrine publishes original articles devoted to basic (including molecular, cellular and physiological studies), translational and clinical research in all the different fields of endocrinology and metabolism. Articles will be accepted based on peer-reviews, priority, and editorial decision. Invited reviews, mini-reviews and viewpoints on relevant pathophysiological and clinical topics, as well as Editorials on articles appearing in the Journal, are published. Unsolicited Editorials will be evaluated by the editorial team. Outcomes of scientific meetings, as well as guidelines and position statements, may be submitted. The Journal also considers special feature articles in the field of endocrine genetics and epigenetics, as well as articles devoted to novel methods and techniques in endocrinology. Endocrine covers controversial, clinical endocrine issues. Meta-analyses on endocrine and metabolic topics are also accepted. Descriptions of single clinical cases and/or small patients studies are not published unless of exceptional interest. However, reports of novel imaging studies and endocrine side effects in single patients may be considered. Research letters and letters to the editor related or unrelated to recently published articles can be submitted. Endocrine covers leading topics in endocrinology such as neuroendocrinology, pituitary and hypothalamic peptides, thyroid physiological and clinical aspects, bone and mineral metabolism and osteoporosis, obesity, lipid and energy metabolism and food intake control, insulin, Type 1 and Type 2 diabetes, hormones of male and female reproduction, adrenal diseases pediatric and geriatric endocrinology, endocrine hypertension and endocrine oncology.
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