Characterizing advanced Parkinson’s disease: OBSERVE-PD observational study results of 2615 patients

Abstract

Background There are currently no standard diagnostic criteria for characterizing advanced Parkinson’s disease (APD) in clinical practice, a critical component in determining ongoing clinical care and therapeutic strategies, including transitioning to device-aided treatment. The goal of this analysis was to determine the proportion of APD vs. non-advanced PD (non-APD) patients attending specialist PD clinics and to demonstrate the clinical burden of APD. Methods OBSERVE-PD, a cross-sectional, international, observational study, was conducted with 2615 PD patients at 128 movement disorder centers in 18 countries. Motor and non-motor symptoms, activities of daily living, and quality-of-life end points were assessed. The correlation between physician’s global assessment of advanced PD and the advanced PD criteria from a consensus of an international group of experts (Delphi criteria for APD) were evaluated. Results According to physician’s judgment, 51% of patients were considered to have APD. There was a moderate correlation between physician’s judgment and Delphi criteria for APD (K = 0.430; 95% CI 0.406–0.473). Activities of daily living, motor symptom severity, dyskinesia duration/disability, “Off” time duration, non-motor symptoms, and quality-of-life scores were worse among APD vs. non-APD patients ( p < 0.0001 for all). APD patients (assessed by physicians) had higher disease burden by motor and non-motor symptoms compared with non-APD patients and a negative impact on activities of daily living and quality of life. Conclusions These findings aid in identifying standard APD classification parameters for use in practicing physicians. Improvements in identification of APD patients may be particularly relevant for optimizing treatment strategies including transitioning to device-aided treatment. Electronic supplementary material The online version of this article (10.1186/s12883-019-1276-8) contains supplementary material, which is available to authorized users.

<p>Profile Of Patients With Advanced Parkinson’s disease Suitable For Device-Aided Therapies: Restrospective Data Of A Large Cohort Of Romanian Patients</p>

“…Only 44% (125/286) of our patients with APD were deemed suitable for DAT, which is significantly lower than the ratio reported in the recently published OBSERVE PD observational study (66%). 12 It must be noted, however, that although we assessed APD patients for all DATs, realistically only LCIG is available in Romania: apomorphine pump infusion is not available and DBS has very limited availability (in a single center and with an average of 5-6 cases/year due to nationwide financial limitations). Of the 125 patients considered for DAT, actually only 107 were offered LCIG the rest needed unavailable DATs (or refused DAT).…”

“…In many cases, we can only rely on expert opinions; nevertheless, changes to the treatment recommendations may be expected in the near future. [2][3][4][5]12 At this stage, due to frequent side effects, the attending physician may be forced to reduce the dosage of dopaminergic agents. In these cases, the dilemma is whether the impairment of motor performance can be attributable to disease progression (and the consequent decline of levodopa responsiveness) or dose reduction.…”

“…1,4,5 These characteristics were present in patients with APD (and potentially eligible for DAT) in the OBSERVE-PD study. 12 However, in clinical practice, the duration and severity of the motor complications at the time of the decision for DAT may be even worse. In our study, the patients that we considered not to have exhausted the limits of conservative treatment had on average 2.8±0.8 hrs off periods, whereas those considered eligible for DAT had 4.7±1.1 hrs off (with an average of 3.62±1.3 hrs for the whole group of completely evaluated patients).…”

<p>Levodopa-Carbidopa Intestinal Gel Infusion Therapy Discontinuation: A Ten-Year Retrospective Analysis of 204 Treated Patients</p>

“…1,32,33 These characteristics were present in patients with APD (and potentially eligible for DAT) in the OBSERVE-PD study (crosssectional, observational study, conducted with 2615 PD patients at 128 movement disorder centers in 18 countries). 34 However, in real-life practice, the duration and severity of the motor complications at the time of the decision for DAT may be even worse. In our previously presented study (311 APD patients), the patients that we considered not to have exhausted the limits of conservative treatment had on average 2.8±0.8 hours off periods, whereas those considered suitable for DAT had 4.7±1.1 hours off.…”

Access and Use of Device‐Aided Therapies for Parkinson’s Disease in Denmark

“…Infusion therapy with LCIG or SCAI is available in all five regions. Timely identification of the patients who could benefit from DAT, including referral from PPNs to MDSs, can be difficult, given that there is no formal consensus on the definition of advanced PD 12,13 . Furthermore, there are differences in the number of both MDSs and PPNs in each region, ranging from 5.6 to 2.7 MDSs per 100 PD patients and 0.76 to 0.15 PPNs per 100 PD patients (Table 1).…”