Koltowska-Häggström Maria, Mattsson Anders F, Shalet Stephen M
KIMS Medical Outcomes, Pfizer Health AB, Endocrine Care, Sollentuna, Sweden.
Eur J Endocrinol. 2009 Nov;161 Suppl 1:S51-64. doi: 10.1530/EJE-09-0266. Epub 2009 Aug 14.
Quality of life (QoL) has emerged as an important construct that has found numerous applications across healthcare-related fields, ranging from research and clinical evaluation of treatment effects to pharmacoeconomic evaluations and global healthcare policy. Impairment of QoL is one of the key clinical characteristics in adult GHD and has been extensively studied in the Pfizer International Metabolic Database (KIMS). We provide summarized evidence on GH treatment effects for both clinical and health economic applications based on the KIMS data. The primary focus is on those aspects of QoL research that cannot be investigated in the traditional clinical trial setting, such as specific patient subgroups, cross-country comparisons and long-term follow-up. First, the impact of age, gender, disease onset, primary aetiology, extent of hypopituitarism, previous radiotherapy and obesity on QoL before and during long-term GH replacement is discussed. Secondly, the studies on QoL in relation to country-specific normative values are reviewed. Finally, health economic data derived from KIMS including both burden of disease and utility assessment are evaluated. We conclude that the wide spectrum of analyses performed on the KIMS data allows for practical application of the results not only to research and clinical practice but also to health policy and global medical decision making.
生活质量（QoL）已成为一个重要概念，在医疗相关领域有着广泛应用，涵盖从治疗效果的研究与临床评估到药物经济学评估及全球医疗政策等各个方面。生活质量受损是成人生长激素缺乏症（GHD）的关键临床特征之一，并且在辉瑞国际代谢数据库（KIMS）中已得到广泛研究。我们基于KIMS数据，为临床和健康经济学应用提供关于生长激素治疗效果的汇总证据。主要关注生活质量研究中那些在传统临床试验环境中无法研究的方面，例如特定患者亚组、跨国比较和长期随访。首先，讨论年龄、性别、疾病发病、原发性病因、垂体功能减退程度、既往放疗和肥胖对长期生长激素替代治疗前及治疗期间生活质量的影响。其次，回顾关于与特定国家规范值相关的生活质量研究。最后，评估来自KIMS的健康经济学数据，包括疾病负担和效用评估。我们得出结论，对KIMS数据进行的广泛分析不仅使研究结果能够实际应用于研究和临床实践，还能应用于卫生政策和全球医疗决策。
