Clinical Division of Endocrinology and Metabolism, Medical University of Vienna, Vienna, Austria.
Horm Res Paediatr. 2011;76 Suppl 1:33-8. doi: 10.1159/000329156. Epub 2011 Jul 21.
Pharmacoepidemiological surveys provide a valuable contribution to the continued monitoring of drug-related effects in patients with rare disorders. One of the earliest examples of this type of survey is KIGS (Pfizer International Growth Study Database), which has monitored the safety and effectiveness of growth hormone (GH) therapy in GH-deficient children since its inception in 1987. Following closely in the footsteps of KIGS is KIMS (Pfizer International Metabolic Database). As of 2009, KIMS has been collecting data on the long-term safety and clinical outcomes of GH replacement in GH-deficient adults for 15 years. Approximately 5 years ago, the ACROSTUDY database was established to monitor the long-term safety and effectiveness of pegvisomant in patients with acromegaly.
By collecting data on the treatment of relatively rare conditions in routine clinical practice, pharmacoepidemiological surveys such as KIMS and ACROSTUDY provide valuable information on the safety and effectiveness of treatment with GH replacement and pegvisomant in the real world.
药物流行病学调查为持续监测罕见疾病患者的药物相关作用提供了有价值的贡献。这类调查最早的例子之一是 KIGS（辉瑞国际生长研究数据库），自 1987 年成立以来，该数据库一直在监测生长激素（GH）治疗在生长激素缺乏儿童中的安全性和有效性。紧随 KIGS 之后的是 KIMS（辉瑞国际代谢数据库）。截至 2009 年，KIMS 已经收集了 15 年来 GH 缺乏症成年人接受 GH 替代治疗的长期安全性和临床结局数据。大约 5 年前，建立了 ACROSTUDY 数据库，以监测培维索孟治疗肢端肥大症患者的长期安全性和有效性。
通过在常规临床实践中收集治疗相对罕见疾病的数据，KIMS 和 ACROSTUDY 等药物流行病学调查为 GH 替代治疗和培维索孟治疗的安全性和有效性提供了宝贵的真实世界信息。
