Associations Among Tremor Amplitude, Activities of Daily Living, and Quality of Life in Patients with Essential Tremor.

Background: Essential tremor (ET) is a disabling syndrome consisting of tremor, primarily in the upper limbs. We assessed the correlation of The Essential Tremor Rating Assessment Scale (TETRAS) Performance Item 4 ratings of upper limb tremor with the TETRAS activities of daily living (ADL) subscale and with 2 quality of life (QoL) scales. Methods: This noninterventional, cross-sectional, point-in-time survey of neurologists(n = 60), primary care physicians (n = 38), and their patients with ET (n = 1,003) used real-world data collected through the Adelphi ET Disease Specific Programme™. Physician-reported measures (TETRAS Performance Item 4 and TETRAS ADL total) and patient-reported QoL measures (generic EuroQol-5 Dimension 5 Level [EQ-5D-5 L] and ET-specific Quality of Life in Essential Tremor Questionnaire (QUEST)) were assessed with bivariate and multivariable analyses. Sensitivity analyses were also conducted. Results: The bivariate association between TETRAS Performance Item 4 score and TETRAS ADL total score was high (Pearson r = 0.761, P < 0.001). The bivariate associations between TETRAS Performance Item 4 score and EQ-5D-5 L index score (r = -0.410, P < 0.001) and between TETRAS ADL total score and EQ-5D-5 L index score (r = -0.543, P < 0.001) were moderate. The bivariate associations between TETRAS Performance Item 4 score and QUEST total score (r = 0.457, P < 0.001), and between TETRAS ADL total score and QUEST total score (r = 0.630, P < 0.001) were also moderate. These associations were unaltered by the inclusion of covariates. Discussion: This study showed that greater tremor severity (TETRAS Performance Item 4) was positively correlated with ADL impairment (TETRAS ADL) and negatively associated with QoL (EQ-5D-5 L and QUEST). TETRAS Performance Item 4 score is a robust predictor of TETRAS ADL total score, and TETRAS Performance Item 4 and TETRAS ADL total scores were robust predictors of the 2 QoL scales. The results demonstrate the value of TETRAS scores as valid endpoints for future clinical trials. Highlights: This real-world study assessed TETRAS scores as predictors of impaired QoL in ET. TETRAS Performance Item 4 and ADL were associated with EQ-5D-5 L and QUEST. TETRAS scores may serve as valid endpoints for future clinical trials.

Real-world assessment of "OFF" episode-related healthcare resource utilization among patients with Parkinson's disease in the United States.

AIMS: Within 5 years of initiating carbidopa/levodopa, ∼50% of patients with Parkinson's disease (PD) experience "OFF" episodes; little is known about the cost burden. We investigated the association of "OFF" episodes with patient characteristics, healthcare resource utilization (HCRU), and healthcare costs. METHODS: Analyses used neurologist-provided data from the US-specific 2017 and 2019 Adelphi Real World Disease Specific Programme for PD, including duration of "OFF" episodes and HCRU for 10-12 consecutive patients. Patients were grouped by presence/absence of "OFF" episodes and by average hours of daily "OFF" time. Between-group differences were assessed for demographics, personal circumstances, and clinical characteristics. Regression analyses modeled the relationship of "OFF" episodes with HCRU and costs. RESULTS: Of 1,309 patients, 41% experienced "OFF" episodes, 25% of whom were "OFF" ≥4 h/day. Patients having "OFF" episodes had more severe PD, were diagnosed for longer, and were younger than those without "OFF" (p < .0001). "OFF" episodes were associated with a greater number of prescribed PD drugs (p < .0001). Patients without "OFF" episodes were more likely to have full-time employment and less likely to be retired or unemployed because of PD (p < .001). Patients with and without "OFF" episodes had different living situations (p < .001): patients experiencing "OFF" were less likely to live alone and more likely to live in a nursing home and have a professional caregiver (p < .001). In the past 12 months, the number of hospitalizations, intensive care admissions, and emergency room visits; nights hospitalized; costs of consultations and hospitalizations; and total direct costs were all higher for patients experiencing "OFF" episodes (p < .05). CONCLUSION: Patients with PD and "OFF" episodes had higher HCRU and costs than those without "OFF," suggesting that "OFF" episodes contribute to the economic burden of PD. Further research is warranted to examine the extent that current PD treatments and treatment patterns impact HCRU and costs.

Real-world assessment of the impact of "OFF" episodes on health-related quality of life among patients with Parkinson's disease in the United States.

BACKGROUND: Many patients with Parkinson's disease (PD) who receive carbidopa/levodopa experience symptom reemergence or worsening, or "OFF" episodes. This study assessed the association of "OFF" episodes with health-related quality of life (HRQoL). METHODS: US-specific data from the 2017 and 2019 Adelphi Real World Disease Specific Programme for PD, a real-world cross-sectional survey, were used. Neurologists provided data for 10-12 consecutive patients with PD who completed the 39-item Parkinson's Disease Questionnaire (PDQ-39) and the EuroQol 5-Dimension (EQ-5D). Data were grouped by patients who experienced "OFF" episodes versus those who did not and by average hours of daily "OFF" time. Differences between patient groups were assessed for demographics and clinical characteristics; regression analyses were used to model the relationship between HRQoL and "OFF" episodes with age, sex, body mass index, current PD stage on the Hoehn and Yahr scale, and number of concomitant conditions related and unrelated to mobility as covariates. RESULTS: Data from 722 patients were analyzed. Overall, 321 patients (44%) had "OFF" episodes (mean of 2.9 h of daily "OFF" time). Patients who experienced "OFF" episodes were less likely to work full-time and more likely to live with family members other than their spouse/partner or reside in a long-term care facility than those without "OFF" episodes. The presence of "OFF" episodes, regardless of the average hours of daily "OFF" time, was significantly associated with high scores (reflecting poor HRQoL) on most PDQ-39 dimensions and the summary index and low scores (reflecting poor health status) on the EQ-5D health utility index, visual analog scale (VAS), and all dimensions. Furthermore, increased average hours of daily "OFF" time was significantly correlated with higher scores for all PDQ-39 dimensions and the summary index, as well as with the EQ-5D health utility index and VAS scores. Patients with "OFF" episodes experienced reduced HRQoL even after correcting for potentially confounding variables. CONCLUSIONS: This study demonstrated that the occurrence of "OFF" episodes in patients with PD is associated with reduced HRQoL and that the impact on HRQoL increased incrementally with increasing average hours of daily "OFF" time.