Progressive supranuclear palsy: Advances in diagnosis and management
Progressive supranuclear palsy: Advances in diagnosis and management
复制标题
DOI：
10.1016/j.parkreldis.2020.04.014
复制
发表时间：
2020-04-01
影响因子：
4.1
通讯作者：
Litvan, Irene
中科院分区：
文献类型：
--
作者：
Coughlin, David G.;Litvan, Irene
进行性核上性麻痹(PSP)是一种复杂的临床病理疾病，目前尚无治愈方法或疾病调节疗法，只有在尸检时才能明确证实。对PSP表型多样性的日益了解导致了对发病机制的临床标准和新见解的扩大，再加上体内生物标记物的改进，使更多地获得目前的临床试验成为可能。目前PSP的标准治疗是多学科的、支持性的和对症的，一些潜在的疾病调节剂的试验已经完成，但结果令人失望。目前正在进行的临床试验通过包括免疫治疗和基因治疗在内的各种机制针对tau的异常聚集提供了一种更直接的治疗方法。在这里，我们回顾了PSP的临床病理相关性，体内生物标记物包括MRI、PET和脑脊液生物标记物。此外，我们还回顾了目前治疗PSP的药理学和非药理学方法，以及之前和正在进行的临床试验。新扩大的临床标准和改进的特定生物标记物将有助于更早和更准确地识别PSP患者，并扩大这些潜在有益的临床试验的机会。
Progressive supranuclear palsy (PSP) is a complex clinicopathologic disease with no current cure or disease modulating therapies that can only be definitively confirmed at autopsy. Growing understanding of the phenotypic diversity of PSP has led to expanded clinical criteria and new insights into etiopathogenesis that coupled with improved in vivo biomarkers makes increased access to current clinical trials possible. Current standard-ofcare treatment of PSP is multidisciplinary, supportive and symptomatic, and several trials of potentially disease modulating agents have already been completed with disappointing results. Current ongoing clinical trials target the abnormal aggregation of tau through a variety of mechanisms including immunotherapy and gene therapy offer a more direct method of treatment. Here we review PSP clinicopathologic correlations, in vivo biomarkers including MRI, PET, and CSF biomarkers. We additionally review current pharmacologic and non-pharmacologic methods of treatment, prior and ongoing clinical trials in PSP. Newly expanded clinical criteria and improved specific biomarkers will aid in identifying patients with PSP earlier and more accurately and expand access to these potentially beneficial clinical trials.