DUOGLOBE. One-Year Outcomes in a Real-World Study of Levodopa Carbidopa Intestinal Gel for Parkinson's Disease

Background: Levodopa-carbidopa intestinal gel (LCIG) is an established treatment for improving motor and some non-motor symptoms (NMS) in patients with advanced Parkinson's disease (PD). Prospective long-term data in routine clinical practice are limited. Objective: Assess LCIG effectiveness and safety in patients with advanced PD after 12 months during real-world routine clinical practice. Methods: Duodopa/Duopa in patients with advanced Parkinson's disease—a global observational study evaluating long-term effectiveness (DUOGLOBE) (NCT02611713) is an ongoing, prospective, multinational, observational study of LCIG-naïve patients treated as part of routine clinical practice; 3 years of follow-up are planned. The primary outcome is the change in patient-reported off time. Other assessments include the Unified Dyskinesia Rating Scale (UDysRS), Non-Motor Symptoms Scale (NMSS), Parkinson's Disease Sleep scale (PDSS-2), Epworth Sleepiness Scale (ESS), health-related quality of life (HR-QoL), caregiver burden, and serious adverse events (SAEs). Outcomes from baseline to month (M) 12 are presented. Results: In this 12-month follow-up, patients (N = 195) had baseline characteristics similar to other LCIG studies. Significant improvements (mean change to M12) were observed in off time (−3.9 ± 3.6 hr/day, P < 0.001), dyskinesia assessed using the UDysRS (−9.6 ± 22.5, P < 0.001), NMSS (−23.1 ± 41.4, P < 0.001), sleep and sleepiness symptoms on the PDSS-2 (−6.5 ± 12.2, P < 0.001) and ESS (−1.0 ± 5.7, P < 0.05), HR-QoL (−9.0 ± 21.6, P < 0.001), and caregiver burden (−1.9 ± 6.7, P = 0.008). Overall, 40.5% (n = 79) of patients experienced SAEs; fall (n = 6; 3.1%) and urinary tract infection (n = 6; 3.1%) were SAEs reported in ≥3% of patients. Conclusions: These 12-month outcome data show sustained, long-term improvements and support the real-world effectiveness of LCIG in patients with advanced PD. Safety was consistent with previous studies

Outcomes Impacting Quality of Life in Advanced Parkinson’s Disease Patients Treated with Levodopa-Carbidopa Intestinal Gel

Background: It is believed that motor symptoms, including dyskinesia, and non-motor symptoms impact health-related quality of life (HRQoL) in patients with Parkinson’s disease (PD), and that improvements in these metrics are correlated. Objective: Investigate the relationship between HRQoL and measures of PD severity and treatment efficacy, including motor and non-motor symptoms. Methods: This was a planned investigation of an international, prospective, single-arm, post-marketing observational study of the long-term effectiveness of levodopa-carbidopa intestinal gel (LCIG) in patients with advanced PD. Pearson correlation coefficients (PCC) were calculated for baseline and change from baseline at 12 months between HRQoL and motor and non-motor symptoms. Results: A total of 195 patients were included. At baseline, HRQoL was moderately positively correlated with Activities of Daily Living (UPDRS II, PCC=0.44), non-motor symptoms (0.48), and measures of sleep (0.50 and 0.40); all p<0.001. After 12 months of treatment with LCIG, improvements in HRQoL were moderately positively correlated with improvement from baseline in non-motor symptoms (PCC=0.42), sleep (0.54), and daytime sleepiness (0.40; all p<0.001), and weakly correlated with improvement in dyskinesia signs and symptoms (PCC=0.23; p=0.011). Improvement in HRQoL was not correlated with improvements in OFF time or dyskinesia time. Conclusion: Both at baseline and for change from baseline at 12 months, HRQoL was correlated with baseline and change from baseline in dyskinesia, Activities of Daily Living, and non-motor symptoms, including sleep; but not with baseline or change in OFF time

Application of the \ue2\u20ac 5-2-1' screening criteria in advanced Parkinson's disease: Interim analysis of DUOGLOBE

Aim: A Delphi expert consensus panel proposed that fulfilling 651 of the \ue2\u20ac 5-2-1 criteria' ( 65five-times daily oral levodopa use, 65two daily hours with \ue2\u20ac Off' symptoms or 65one daily hour with troublesome dyskinesia) suggests advanced Parkinson's disease (PD). Patients & methods: DUOdopa/Duopa in Patients with Advanced PD-a GLobal OBservational Study Evaluating Long-Term Effectiveness (DUOGLOBE)-is a single-arm, postmarketing, observational, long-term effectiveness study of levodopa-carbidopa intestinal gel (LCIG) for advanced PD. Results: This 6-month interim analysis (n = 139) affirms that most (98%) enrolled patients fulfill 651 of the 5-2-1 criteria. These patients responded favorably to LCIG treatment. Safety was consistent with other LCIG studies. Conclusion: In advanced PD patients, the 5-2-1 criteria generally aligns with clinician assessment