Stute Lara, Krüger Rejko
Parkinson Research Clinic, Centre Hospitalier de Luxembourg (CHL), Luxembourg, Luxembourg.
Luxembourg Centre for Systems Biomedicine (LCSB), University of Luxembourg, Esch-Sur-Alzette, Luxembourg.
Fortschr Neurol Psychiatr. 2020 Sep;88(9):558-566. doi: 10.1055/a-1149-2204. Epub 2020 Jun 2.
The diverse and highly individual presentations of Parkinson's disease (PD) as a complex combination of motor and non-motor symptoms are being increasingly well characterised not least through large patient cohorts applying deep phenotyping. However, in terms of treatment of PD, the approach is uniform and purely symptomatic. Better stratification strategies with better precision medicine approaches offer opportunities to improve symptomatic treatment, define first causative therapies and provide more patient-centred care. Insight from targeted therapies for monogenic forms of PD aiming at neuroprotection may pave the way for new mechanism-based interventions also for the more common idiopathic PD. Improved stratification of patients may support symptomatic treatments by predicting treatment efficacy and long-term benefit of current pharmacological or neuromodulatory therapies, e.g. in the context of emerging pharmacogenomic knowledge. Based on asymptomatic carriers with monogenic PD or patients with REM sleep behaviour disorder (RBD), first options for applying preventive treatments emerge. The implications of these treatment strategies in relation to disease progression, and the prospects of their implementation in clinical practice need to be addressed.
帕金森病（PD）表现多样且高度个体化，是运动和非运动症状的复杂组合，通过应用深度表型分析的大型患者队列，其特征越来越清晰。然而，在PD的治疗方面，方法是统一的且纯粹是对症治疗。更好的分层策略和更精准的医学方法为改善对症治疗、确定首批病因治疗方法以及提供更以患者为中心的护理提供了机会。针对单基因形式PD的旨在神经保护的靶向治疗所获得的见解，可能也为更常见的特发性PD开辟基于新机制的干预途径。改善患者分层可通过预测当前药物或神经调节疗法的治疗效果和长期益处来支持对症治疗，例如在新兴的药物基因组学知识背景下。基于单基因PD的无症状携带者或快速眼球运动睡眠行为障碍（RBD）患者，出现了应用预防性治疗的首批选择。需要探讨这些治疗策略对疾病进展的影响以及在临床实践中实施的前景。
