Brod Meryl, Alolga Suzanne Lessard, Beck Jane F, Wilkinson Lars, Højbjerre Lise, Rasmussen Michael Højby
The Brod Group, 219 Julia Avenue, Mill Valley, CA, 94941, USA.
Novo Nordisk A/S, Vandtårnsvej 114, 2860, Søborg, Denmark.
Qual Life Res. 2017 Jul;26(7):1673-1686. doi: 10.1007/s11136-017-1529-1. Epub 2017 Feb 28.
Research demonstrates that children and adolescents with growth hormone deficiency (GHD) are impacted in multiple ways beyond their short stature; however, there are no disease-specific measures to assess these impacts. The purpose of this study was to examine the burden of GHD on children and adolescents, and to conduct concept elicitation to develop a model of the impact of GHD to support a disease-specific outcome measure.
Four focus groups and 52 telephone interviews were conducted with children with GHD and parents/guardians of children with GHD to understand the experience and impacts from the child's perspective, reported by children or parent-observers about the impact on the child. The interviews and focus groups were conducted in Germany, the United Kingdom, and the United States. Interview transcripts were analyzed thematically based on modified grounded theory principles.
There were 73 descriptions of patient's experiences elicited from 70 respondents, as three respondents spoke for two children each. A majority of GHD descriptive narratives refer to boy children (n = 51, 69.9%) and a majority of children had taken GHD treatment (n = 64, 89%). Analysis identified four major areas of GHD impact: Signs and Symptoms (beyond short stature), Physical Aspects of Daily Life, Social Well-Being, and Emotional Well-Being.
The burden of GHD in children and adolescents is considerable and not limited to short stature. The severity of GHD impact on children and adolescents appears to be variable and individualized, but these data indicate that early identification and growth hormone treatment may lead to fewer impacts.
研究表明，生长激素缺乏症（GHD）患儿和青少年受到的影响不仅限于身材矮小；然而，尚无针对该疾病的评估这些影响的特定措施。本研究的目的是调查GHD对儿童和青少年的负担，并进行概念激发以建立GHD影响模型，以支持特定疾病的结局指标。
对GHD患儿及其父母/监护人进行了4个焦点小组讨论和52次电话访谈，以从儿童的角度了解经历和影响，由儿童或父母观察者报告对儿童的影响。访谈和焦点小组讨论在德国、英国和美国进行。访谈记录根据修正的扎根理论原则进行主题分析。
从70名受访者中获得了73条关于患者经历的描述，有3名受访者分别代表两名儿童发言。大多数GHD描述性叙述涉及男童（n = 51，69.9%），大多数儿童接受过GHD治疗（n = 64，89%）。分析确定了GHD影响的四个主要领域：体征和症状（身材矮小之外）、日常生活的身体方面、社会幸福感和情绪幸福感。
GHD对儿童和青少年的负担相当大，且不仅限于身材矮小。GHD对儿童和青少年的影响严重程度似乎因人而异，但这些数据表明，早期识别和生长激素治疗可能会减少影响。
