Background:Treatment of patients with late-stage parkinsonism is often sub-optimal. Objective:To test the effectiveness of recommendations by a movement disorder specialist with expertise in late-stage parkinsonism. Methods:Ninety-one patients with late-stage parkinsonism considered undertreated were included in apragmatic multi-center randomized-controlled trial with six-month follow-up. The intervention group received a letter with treatment recommendations to their primary clinician based on an extensive clinical assessment. Controls received care as usual. The primary outcome was the Unified Parkinson Disease Rating Scale (UPDRS)part-II (Activities of Daily Living).Other outcomes included quality-of-life (PDQ-8), mental health (UPDRS-I), motor function (UPDRS-III), treatment complications (UPDRS-IV), cognition (Mini-mental-state-examination), non-motor symptoms (Non-Motor-Symptoms-scale), health status (EQ-5D-5L) and levodopa-equivalent-daily-dose (LEDD). We also assessed adherence to recommendations. In addition to intention-to-treat analyses, aper-protocol analysis was conducted. Results:Sample size calculation required 288 patients, but only 91 patients could be included. Treating physicians followed recommendations at least partially in 37(64%) patients. The intention-to-treat analysis showed no difference in primary outcome (between-group difference = –1.2, p = 0.45), but there was greater improvement for PDQ-8 in the intervention group (between-group difference = –3.7, p = 0.02). The per-protocol analysis confirmed these findings, and showed less deterioration in UPDRS-part I, greater improvement on UPDRS-total score and greater increase in LEDD in the intervention group. Conclusions:The findings suggest that therapeutic gains may be reached even in this vulnerable group of patients with late-stage parkinsonism, but also emphasize the need for better strategies to implement specialist recommendations to further improve outcomes.

中文翻译：

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优化治疗不足的晚期帕金森病：一项务实的随机试验。

背景：晚期帕金森病患者的治疗往往不是最佳的。目的：测试具有晚期帕金森病专业知识的运动障碍专家的建议的有效性。方法：将 91 例被认为治疗不足的晚期帕金森综合征患者纳入实用多中心随机对照试验，随访 6 个月。基于广泛的临床评估，干预组收到了一封信，其中包含给他们的主要临床医生的治疗建议。对照照常得到护理。主要结果是统一帕金森病评定量表 (UPDRS) 第二部分（日常生活活动）。其他结果包括生活质量 (PDQ-8)、心理健康 (UPDRS-I)、运动功能 (UPDRS- III）、治疗并发症（UPDRS-IV）、认知（Mini-mental-state-examination）、非运动症状（非运动症状量表）、健康状况（EQ-5D-5L）和左旋多巴等效每日剂量（LEDD）。我们还评估了对建议的遵守情况。除了意向治疗分析外，还进行了符合方案分析。结果：样本量计算需要288例患者，但只能包括91例患者。37 名（64%）患者的治疗医师至少部分遵循了建议。意向治疗分析显示主要结局没有差异（组间差异 = –1.2，p = 0.45），但干预组的 PDQ-8 改善更大（组间差异 = –3.7，p = 0.02）。符合方案的分析证实了这些发现，并显示干预组的 UPDRS-part I 恶化较少，UPDRS 总分有较大改善，LEDD 增加较多。