Pharmacological interventions in corticobasal degeneration: a review

ABSTRACT. Corticobasal degeneration (CBD) is a sporadic tauopathy that presents with a varied combination of motor, cognitive, and behavioral features, making its diagnosis difficult. CBD has high morbidity and poor prognosis, with no effective therapy at present. We searched the PubMed/MEDLINE database for articles published from 1990 to 2019, using the keywords “corticobasal degeneration” AND “treatment.” The PRISMA method was adopted. Retrieved articles were characterized as having one of two methodological approaches: (1) studies aimed at primary tauopathy treatment and (2) symptomatic management. Review articles (based on CBD expert groups), case reports, case series, and pilot clinical trials were selected. Few attempts have been made to study drug options and drug efficacy in CBD systematically, and an effective treatment is not yet available. Treatment is symptomatic and based on similarity with other diseases due to the scarcity of studies specifically addressing CBD. CBD seems not to spark interest in more clinical trials for its low prevalence and reliability in clinical diagnosis.摘要。皮质基底节变性（Corticobasal degeneration, CBD）是一种散发性tau蛋白病，临床表现为运动、认知与行为特征的多样组合，导致诊断难度较大。该病具有高发病率与不良预后，目前尚无有效治疗方案。本研究以“皮质基底节变性”与“治疗”为检索关键词，检索了1990年至2019年PubMed/MEDLINE数据库收录的文献，并采用PRISMA法进行文献筛选。纳入的文献可分为两类方法学方向：（1）针对原发性tau蛋白病治疗的研究；（2）症状管理类研究。同时筛选纳入了基于CBD专家小组的综述文献、病例报告、病例系列研究及试点临床试验。目前针对CBD的药物选择与疗效的系统性研究仍较为匮乏，尚未有获批的有效治疗手段。由于针对该病的专项研究稀缺，当前治疗仅能参考其他相似疾病的方案实施对症支持。鉴于CBD患病率较低且临床诊断可靠性欠佳，该领域尚未能吸引足够多的临床试验开展。