a Pfizer Ltd , Surrey , United Kingdom.
b Pfizer Inc. , New York , NY, USA.
Curr Med Res Opin. 2019 Jun;35(6):963-973. doi: 10.1080/03007995.2018.1546682. Epub 2018 Dec 10.
Quality of life (QoL) and health economic data are becoming increasingly important factors in healthcare decision making. While there is a wealth of information establishing the benefit of growth hormone (GH) replacement therapy in adults with growth hormone deficiency (aGHD), recent reviews on the QoL and health economic impact of aGHD and the effect of treatment on these factors is limited.
The aim of this article is to summarize the impact of early and sustained treatment on the QoL and economic burden of aGHD by conducting a targeted literature review.
Standard electronic databases, including PubMed and the Cochrane collaboration website, were searched for publications between January 2006 and July 2016 for evidence of the humanistic and economic burden of aGHD. Search terms included growth hormone deficiency, health-related quality of life, HRQoL, patient-reported outcomes, outcome assessment, well-being and adherence.
The literature search identified 732 initial hits and a final 14 publications were included. The analysis showed that the economic burden of aGHD is largely driven by the productivity losses associated with the disease. This is because most patients with aGHD are of working age and the QoL domains (memory & concentration and energy & vitality) most commonly affected by aGHD severely impair a person's ability to work and may limit their contribution to society.
Untreated aGHD can seriously affect patients' functioning. Early and continued treatment with GH replacement therapy could potentially improve the QoL and reduce the economic burden associated with aGHD. This review has limitations: only English language articles published since January 2006 were included and many of the studies were conducted in the Nordic countries; it is unclear how representative these studies are of the population as a whole. This was a literature review and not a systematic review, as it was thought to be unlikely that, in this rare disease, any additional publications would have been identified. Overall, this review reveals a paucity of data in this underserved population and points to research gaps which could be addressed with new studies.
生活质量（QoL）和健康经济学数据在医疗保健决策中变得越来越重要。虽然有大量信息表明生长激素（GH）替代疗法对生长激素缺乏症（aGHD）成人有益，但最近关于 aGHD 的 QoL 和健康经济学影响以及治疗对这些因素的影响的综述有限。
本文旨在通过进行针对性文献综述，总结早期和持续治疗对 aGHD 的 QoL 和经济负担的影响。
标准电子数据库，包括 PubMed 和 Cochrane 合作网站，搜索了 2006 年 1 月至 2016 年 7 月期间关于 aGHD 人文和经济负担的证据的出版物。搜索词包括生长激素缺乏症、健康相关生活质量、HRQoL、患者报告的结果、结果评估、幸福感和依从性。
文献检索共确定了 732 个初始结果，最终纳入了 14 篇出版物。分析表明，aGHD 的经济负担主要是由与疾病相关的生产力损失驱动的。这是因为大多数 aGHD 患者处于工作年龄，受 aGHD 影响最大的 QoL 领域（记忆力和注意力以及能量和活力）严重影响一个人的工作能力，并可能限制他们对社会的贡献。
未经治疗的 aGHD 会严重影响患者的功能。早期和持续使用 GH 替代疗法治疗可能会改善 QoL 并降低与 aGHD 相关的经济负担。本综述存在局限性：仅纳入了自 2006 年 1 月以来发表的英语文章，并且许多研究都是在北欧国家进行的；尚不清楚这些研究对整个人群的代表性如何。这是一篇文献综述，而不是系统综述，因为在这种罕见疾病中，不太可能发现任何额外的出版物。总的来说，这篇综述揭示了在这个服务不足的人群中数据的缺乏，并指出了可能需要新研究来解决的研究空白。
