Serbin M, Odak S, Macahilig C, Joshi N, Shah A, Ng X, Olson K, Liang GS, Klepitskaya O, Yonan C. Burden of parkinson's disease in a real-world sample of patients with motor fluctuations. Poster presented at the 2020 Virtual AMCP NEXUS; October 2020. [abstract] J Manag Care Spec Pharm. 2020 Oct; 26(10-a):S43. doi: 10.18553/jmcp.2020.26.10-a.s1


BACKGROUND: Levodopa remains the most effective treatment for Parkinson’s disease (PD), but motor fluctuations (MFs) and dyskinesias associated with disease progression continue to be a clinical concern. Even with available adjunctive treatments, patients are burdened by OFF periods, poor-quality ON periods, and reduced health-related quality of life (HRQoL).

OBJECTIVE: To characterize symptoms and HRQoL in a real-world sample of PD patients in the United States who experienced MFs with carbidopa/levodopa (CD/LD) and other adjunctive PD treatments.

METHODS: This retrospective medical chart review study included 310 adult PD patients who began experiencing MFs between 01/2014 and 04/2019 while taking CD/LD. Data were abstracted from patients’ medical records and recorded on a case report form (CRF). Outcomes, as recorded at the most recent office visit, included: waking hours in OFF state; waking hours in ON state with troublesome dyskinesia; and presence of motor and nonmotor symptoms in ON and OFF states. HRQoL was assessed using the EuroQoL-5 Dimension-5 Level (EQ-5D-5L) utility index (range, 0 to 1 [perfect health]) and visual analog scale (VAS: range, 0 to 100 [best possible health]). Outcomes were analyzed descriptively.

RESULTS: Based on CRF patient data, median ages (interquartile range [IQR]) at PD onset and MF onset were 64.8 (58.0-71.3) and 69.1 (63.3-75.6) years, respectively. The median time from start of PD treatment to MF onset was 25.0 (11.0-51.5) months. At patients’ most recent visit, the median number of waking hours (IQR) was 16.0 (14.0-17.0). More than 30% of waking hours were spent in OFF state (mean, 24.5%) and ON state with troublesome dyskinesia (mean, 7.2%). All motor symptoms (except for dyskinesia) were reported in a higher proportion of patients in OFF vs ON state, especially bradykinesia (62.6% vs 42.6%), rigidity (43.2% vs 28.4%), and tremor (55.2% vs 36.8%). Nonmotor symptoms were also more common in the OFF state, with body aches/pain having the greatest difference vs ON state (17.4% vs 14.2%). Based on EQ-5D-5L data from all patients (N=308), mean (±standard deviation) scores suggested poor HRQoL in general (utility, 0.56±0.28; VAS, 66.8±18.7).

CONCLUSIONS:
In this real-world sample of patients with PD and MFs, the presence of fluctuating motor and nonmotor symptoms indicated inadequate symptom control despite treatment with CD/LD and other adjunctive medications, which was reflected in the poor HRQoL. The use of new therapeutic options may help to optimize the management of MFs in patients with PD.

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