9条目帕金森病筛查问卷的开发与验证

文献信息

中文摘要

目的： 我们旨在开发并验证一份简短的、自填式问卷，整合运动症状与前驱期非运动症状，用于筛查帕金森病（PD）。
方法： 我们开展了一项三阶段研究。第1阶段采用一项前瞻性注册的系统综述（纳入107项研究）生成52条目草案。第2阶段在205名参与者（122例早期PD和83名健康对照）中对草案进行优化，使用条目水平的受试者工作特征曲线下面积（AUC）以及年龄和性别校正的多变量logistic回归。第3阶段在独立队列（n = 246；122例PD、45例特发性震颤[ET]和79名对照）中验证9条目韩国帕金森病筛查问卷（KPDSQ），并包括4周的重测复评。
结果： 最终9条目KPDSQ包括七个运动条目（静止性震颤、拖步或短步态、日常活动缓慢、摆臂减少、声音变轻、面具脸和冻结步态）和两个非运动条目（嗅觉减退和梦境演绎行为）。在≥2的截断值时，区分PD与对照的敏感性为88.5%，特异性为92.4%（AUC = 0.970；95% CI，0.946-0.984；p < 0.001）。在≥3时，KPDSQ可区分PD与ET（AUC = 0.774；95% CI，0.680-0.847；p < 0.001）。内部一致性高（Cronbach's α = 0.800），重测信度极好（ICC = 0.921），且评分与PD患者的MDS-UPDRS Part III评分相关（Spearman's ρ = 0.330；p < 0.001）。
结论： 9条目KPDSQ是一种可靠、临床有效的筛查工具，结合了运动症状和前驱期非运动特征。有必要开展前瞻性社区验证。

英文摘要

OBJECTIVE: We aimed to develop and validate a brief, self-administered questionnaire integrating motor and prodromal non-motor symptoms for screening Parkinson's disease (PD).
METHODS: We conducted a three-phase study. Phase 1 used a prospectively registered systematic review of 107 studies to generate a 52-item draft. In Phase 2, the draft was refined in 205 participants (122 with early PD and 83 healthy controls) using item-level areas under the receiver operating characteristic curves (AUCs) and age- and sex-adjusted multivariable logistic regression. Phase 3 validated the 9-item Korean PD Screening Questionnaire (KPDSQ) in an independent cohort (n = 246; 122 PD, 45 essential tremor [ET], and 79 controls), and included a 4-week test-retest reassessment.
RESULTS: The final 9-item KPDSQ comprises seven motor items (rest tremor, shuffling or short-step gait, slow daily activities, decreased arm swing, soft voice, masked face, and freezing of gait) and two non-motor items (hyposmia and dream-enactment behavior). At a cutoff of ≥2, sensitivity was 88.5% and specificity was 92.4% for distinguishing PD from controls (AUC = 0.970; 95% CI, 0.946-0.984; p < 0.001). At ≥3, the KPDSQ distinguished PD from ET (AUC = 0.774; 95% CI, 0.680-0.847; p < 0.001). Internal consistency was high (Cronbach's α = 0.800), test-retest reliability was excellent (ICC = 0.921), and scores correlated with MDS-UPDRS Part III scores in patients with PD (Spearman's ρ = 0.330; p < 0.001).
CONCLUSIONS: The 9-item KPDSQ is a reliable, clinically valid screening tool combining motor and prodromal non-motor features. Prospective community-based validation is warranted.