Timely referral for advanced therapy in Parkinson’s disease. Development of a screening tool

Abstract

OBJECTIVES: To develop a screening tool for timely referral for advanced therapy (AT) in patients with Parkinson disease (PD), and to compare the newly-developed tool with the published 5-2-1 criteria. DESIGN: Cross-sectional, diagnostic, observational study and multivariable logistic regression analysis for item selection. SETTING: 8 hospitals in the Northern part of the Netherlands situated in the catchment area of a specialized movement disorder centre. PARTICIPANTS: 259 consecutive PD patients not yet on AT visiting the outpatient clinic of participating hospitals from February 2017 to July 2018. PREDICTORS: 24 patient and disease characteristics as assessed by the treating neurologists, and scores on the NMS questionnaire. OUTCOME: Apparent eligibility for referral for AT based on consensus by a panel of 5 experts in the field of AT. RESULTS: 17 patients were deemed eligible for referral for AT (point prevalence: 6.6%). Presence of response fluctuations and troublesome dyskinesias were the strongest independent predictors of being eligible for referral. Both variables were included in the final model, as well as levodopa equivalent daily dose. Decision curve analysis showed that the new model outperformed the 5-2-1 criteria. A simple chart was constructed to provide guidance for referral. Discrimination of this simplified scoring system was good (AUC after bootstrapping: 0.97). CONCLUSION: The screening tool may improve efficiency of referral and subsequent treatment with AT in patients with PD. External validation is required prior to application in daily practice.

Does the 5–2-1 criteria identify patients with advanced Parkinson's disease? Real-world screening accuracy and burden of 5–2-1-positive patients in 7 countries

“…Based on their findings, the authors constructed a three-factor screening tool based on LED, response fluctuations, and troublesome dyskinesias, to screen for patients eligible for DAT referral. This study reported a sensitivity and a specificity of 88 and 98% compared with 94 and 73%, respectively, for the 5-2-1 criteria [18]. The authors note that their tool corroborates the concepts of the 5-2-1 criteria, and the study appears to confirm the validity of the overall approach.…”

“…In a similar way anticipated for the 5-2-1 criteria, MANAGE-PD section 1 groups patients into controlled or inadequately controlled on current therapy. A recently-reported study compared the 5-2-1 criteria to a newly developed screening tool designed to predict eligibility for referral for DAT evaluation [18]. In this study, a panel of experts assessed 259 consecutive DAT-naive PD patients, of whom 17 were considered eligible for DAT.…”

“…Previous studies have suggested the potential utility of the 5-2-1 criteria as a screening tool for APD [17,18]. In one study, individuals meeting that 5-2-1 criteria were found to have significantly poorer health outcomes, including worse quality of life (QoL), greater NMS burden, and greater caregiver burden [16].…”

“…However, the few studies evaluating the 5-2-1 screening criteria have tended to be small, single-centered investigations offering limited evidence on real-world screening accuracy of the 5-2-1 criteria and little information on the incremental burden experienced by 5-2-1-positive patients [18]. To improve our understanding of the utility of the 5-2-1 screening criteria in every day clinical practice, the current real-world analysis was undertaken.…”