Del Signore Susanna, Roubenoff Ronenn
Bluecompanion Ltd, 6 London Street, London, W2 1HR, UK.
Global Translational Medicine, Novartis Institutes for Biomedical Research, Basel, Switzerland.
Aging Clin Exp Res. 2017 Feb;29(1):69-74. doi: 10.1007/s40520-016-0710-7. Epub 2017 Feb 3.
We have observed over the last 15 years a wide debate both in the medical scientific community and in the public health arena on the definition and operationalization of frailty, typically a geriatric condition, and in particular of physical frailty linked to sarcopenia. Because physical frailty in its initial phase can still be reversed, fighting sarcopenia in elderly persons has the potential to slow or halt progressive decline towards disability and dependency. Quite recently, regulators focused attention on frailty as an indicator of biological age to be measured to characterize elderly patients before their inclusion in clinical trials. A European guidance regarding most adapted evaluation instruments of frailty is currently under public consultation. Does the regulatory initiative imply we should now consider frailty, and particularly physical frailty, primarily as an important risk factor for adverse events and poor response, or mainly as a clinical tool helping the physician to opt for one therapeutic pathway or another? Or is physical frailty above all a specific geriatric condition deserving an effective and innovative therapeutic approach with the objective to curb the incidence of its most common result, e.g., mobility disability? Pharmaceutical industry developers consider both faces of the coin very relevant. We agree with regulators that better characterization of subpopulations, not only in elderly patients, can improve the benefit risk ratio of medicines. At the same time, we believe it is in the public health interest to develop novel drugs indicated for specific geriatric conditions, like osteoporosis in the 1990s and sarcopenia today. We consider it an important therapeutic goal to effectively delay mobility disability and to extend the active, independent, and healthy life years of aging people. The "Sarcopenia and Physical fRailty IN older people: multi-componenT Treatment strategies" (SPRINTT) collaborative project under IMI is paving the way for adapted methodologies to study the change of physical frailty and sarcopenia in at-risk older persons and to adequately characterize the population that needs to be treated.
在过去15年里，我们观察到医学科学界和公共卫生领域围绕虚弱的定义与实施展开了广泛讨论，虚弱通常是一种老年疾病，尤其是与肌肉减少症相关的身体虚弱。由于身体虚弱在初始阶段仍可逆转，对抗老年人的肌肉减少症有可能减缓或阻止向残疾和依赖状态的渐进性衰退。最近，监管机构将注意力集中在虚弱作为生物年龄指标上，以便在老年患者纳入临床试验之前对其进行测量和特征描述。一项关于最适合虚弱评估工具的欧洲指南目前正在公开征求意见。监管举措是否意味着我们现在应主要将虚弱，尤其是身体虚弱，视为不良事件和不良反应的重要风险因素，还是主要将其视为帮助医生选择治疗途径的临床工具？或者身体虚弱首先是一种特定的老年疾病，值得采用有效且创新的治疗方法，以降低其最常见后果（如行动不便）的发生率？制药行业开发者认为这两个方面都非常重要。我们同意监管机构的观点，即更好地对亚人群进行特征描述，不仅针对老年患者，能够提高药物的效益风险比。同时，我们认为开发针对特定老年疾病的新药符合公共卫生利益，就像20世纪90年代的骨质疏松症和如今的肌肉减少症。我们认为有效延缓行动不便并延长老年人积极、独立和健康的生活年限是一个重要的治疗目标。IMI旗下的“Sarcopenia and Physical fRailty IN older people: multi-componenT Treatment strategies”（SPRINTT）合作项目正在为研究高危老年人身体虚弱和肌肉减少症变化的适用方法以及充分描述需要治疗的人群特征铺平道路。
