Golbe Lawrence I, Ohman-Strickland Pamela, Beisser Emily B, Elghoul Francesca T
Department of Neurology Rutgers Robert Wood Johnson Medical School New Brunswick New Jersey USA.
Department of Biostatistics and Epidemiology Rutgers School of Public Health Piscataway New Jersey USA.
Mov Disord Clin Pract. 2020 Jul 19;7(6):664-671. doi: 10.1002/mdc3.13010. eCollection 2020 Aug.
Progressive supranuclear palsy (PSP) causes major disability, shortens life, and as yet has no disease-modifying and little symptomatic treatment. A convenient prognostic tool is needed to assist patients, families, and clinicians in planning care.
We calculated times to acquisition of certain disease milestones and death.
We followed a cohort of 417 patients with PSP-Richardson syndrome from 1995 to 2016, applying the Progressive Supranuclear Palsy Rating Scale (PSPRS) at each visit. We generated median times to acquisition of 13 milestones using the input variables of sex, onset age, rate of disease progression from motor symptom onset to initial visit, and PSPRS score at the baseline. Of the outcome milestones, 5 were stages of a new, provisional PSP staging system. The other 8 milestones comprised death and disabling levels of cognitive loss, gaze palsy, dysarthria, dysphagia, and gait/balance impairment.
We derived median times to milestones, with 25th and 75th percentiles and 95% confidence intervals of the median for baseline PSPRS scores from 25 to 65 (scale range, 0-100). Sex and initial progression velocity significantly influenced the death milestone, but not most of the others. Median time to death ranged from 4.8 years for a man with PSPRS score of 25 and a slow progression velocity from onset to initial visit of 0.51 PSPRS points/month to 1.8 years for a woman with PSPRS 65 and rapid initial velocity of 2.25 points/month.
We have created a convenient, inexpensive, noninvasive reference for counseling patients with PSP-Richardson syndrome on approximate time to encountering 13 life-altering disease milestones.
进行性核上性麻痹(PSP)会导致严重残疾,缩短寿命,且目前尚无改善病情的治疗方法,对症治疗也很少。需要一种便捷的预后工具来帮助患者、家属和临床医生规划护理。
我们计算了达到某些疾病里程碑和死亡的时间。
我们对1995年至2016年间的417例PSP-理查森综合征患者进行了随访,每次就诊时应用进行性核上性麻痹评定量表(PSPRS)。我们使用性别、发病年龄、从运动症状发作到初次就诊的疾病进展速度以及基线时的PSPRS评分作为输入变量,生成了达到13个里程碑的中位时间。在结局里程碑中,5个是新的临时PSP分期系统的阶段。其他8个里程碑包括死亡以及认知丧失、凝视麻痹、构音障碍、吞咽困难和步态/平衡障碍的致残水平。
我们得出了达到里程碑的中位时间,以及基线PSPRS评分为25至65(量表范围为0-100)时的第25和第75百分位数以及中位值的95%置信区间。性别和初始进展速度对死亡里程碑有显著影响,但对其他大多数里程碑没有影响。死亡的中位时间范围为:PSPRS评分为25且从发病到初次就诊进展速度缓慢(每月0.51个PSPRS点)的男性为4.8年,到PSPRS评分为65且初始进展速度较快(每月2.25个点)的女性为1.8年。
我们创建了一个便捷、廉价、非侵入性的参考资料,用于为PSP-理查森综合征患者提供咨询,告知他们达到13个改变生活的疾病里程碑的大致时间。