Measuring Disease Progression in Early Parkinson Disease

Parashos, Sotirios A.; Luo, Sheng; Biglan, Kevin; Bódis-Wollner, Iván; He, Bo; Liang, Grace; Ross, G. Webster; Tilley, Barbara C.; Shulman, Lisa M.

doi:10.1001/jamaneurol.2014.391

Cited by 61 publications

(34 citation statements)

References 15 publications

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“…For example, one study demonstrated that UPDRS-III did not predict poorer HRQOL [1], whereas two other studies reported that UPDRS-III was a predictor of PDQ-39SI [7]. Sotirios et al [25] provided evidence that UPDRS-II and UPDRS-III could be used to measure disease progression in early PD. In our study, UPDRS-III was correlated moderately with PDQ-39SI (r=0.42).…”

Section: Discussionmentioning

confidence: 99%

The Key Determinants to Quality of Life in Parkinson’s Disease Patients: Results from the Parkinson’s Disease Biomarker Program (PDBP)

Lee

Sterling

et al. 2016

JPD

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Background To determine the impact of motor and non-motor symptoms on health-related quality of life (HRQOL) in Parkinson’s disease (PD), the study explored a large cohort of 645 patients from the PD Biomarker Program. Methods The PD Questionnaire-39 (PDQ-39) measured HRQOL, whereas the Unified PD Rating Scale (UPDRS) assessed motor and non-motor symptoms. Determinants of HRQOL in PD patients were identified by stepwise linear regression analysis. The relationship between the PDQ-39 and UPDRS subscale scores then was explored through structural equation modeling. Results The mean disease duration was 6.8 years and the mean PDQ-39 summary index (PDQ-39SI) was 18.4. UPDRS-I (non-motor function) and UPDRS-II (motor questionnaire) scores demonstrated the strongest correlations with PDQ-39SI (r>0.4, P<0.05), whereas UPDRS-III (motor exam) and UPDRS-IV (motor complications) scores were correlated moderately with PDQ-39SI (0.3

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Section: Discussionmentioning

confidence: 99%

The Key Determinants to Quality of Life in Parkinson’s Disease Patients: Results from the Parkinson’s Disease Biomarker Program (PDBP)

Lee

Sterling

et al. 2016

JPD

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“…The Unified Parkinson's Disease Rating Scale (UPDRS) III was used to evaluate the severity of the patients' motor symptoms [4]. The UPDRS III score was assessed on treatment after the first levodopa or a pramipexole dose and then after 12 and 18 months of treatment.…”

Section: Methodsmentioning

confidence: 99%

The Effect of Creatine and Coenzyme Q10 Combination Therapy on Mild Cognitive Impairment in Parkinson's Disease

Li¹,

Wang²,

Zhou³

et al. 2015

Eur Neurol

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Background: To investigate the effect of creatine and coenzyme Q10 (CoQ10) combination therapy on mild cognitive impairment (MCI) in Parkinson's disease (PD; PD-MCI) and its influences on plasma phospholipid (PL) levels in PD-MCI. Methods: The demographic data of 75 PD-MCI patients who enrolled in this collaborative PD study were collected. These patients were evaluated using the Unified Parkinson's Disease Rating Scale (UPDRS) III and the Montreal Cognitive Assessment (MoCA). These 75 PD-MCI patients were randomly treated with creatine monohydrate 5 g b.i.d. and CoQ10 100 mg t.i.d. orally or placebo. MoCA evaluation and PL level measurements were performed after 12 and 18 months of treatment. Results: After 12 and 18 months of treatment, the differences in the MoCA scores of the combination therapy and control groups were statistically significant (p < 0.05 at 12 months and p < 0.01 at 18 months), and the plasma PL levels of the combination therapy group were significantly lower than those of the control group (p < 0.01 at 12 months and p < 0.001 at 18 months). Conclusions: Combination therapy with creatine and CoQ10 could delay the decline of cognitive function in PD-MCI patients and could lower their plasma PL levels; therefore, this combination therapy may have a neuroprotective function.

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“…Newly available, high quality evidence can be incorporated into the study design allowing for alteration of sample size based on observed variability, modifying ineffective doses, moving patients among various trial phases, or simply ending a trial early due to failure of efficacy (such as in the case of NET-PD and PRECEPT studies [47,48]). Although these designs can improve efficiency and reduce drug development time, they may introduce bias, require complex operational support from statisticians and safety monitoring boards in order to make real time decisions based on current data [2,49].…”

Section: Clinical Trial Designmentioning

confidence: 99%

“…Stage of disease can also influence the usefulness of the questionnaires. A study suggested that UPDRS scores correlates well with disease progression, but questionnaires assessing overall disability such as the PDQ-39 and Total Functional Capacity scale were not sensitive to change in early disease [47], whereas in moderate stage disease, the PDQ-39 summary index was found to be most sensitive to change [74].…”

Section: Mds-updrsmentioning

confidence: 99%

Challenges in detecting disease modification in Parkinson's disease clinical trials

Athauda

Foltynie

2016

Parkinsonism & Related Disorders

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Despite the wealth of encouraging data from numerous compounds that demonstrate "neuroprotection" in pre-clinical studies of Parkinson's disease, and despite numerous clinical trials, to date, no intervention has been demonstrated to able to modify the course of disease progression.While this "failure to translate" is likely due to numerous factors including our incomplete understanding of the pathogenic mechanisms underlying PD together with excessive reliance on data from the toxin-based animal models of PD, here we will discuss the "structural issues" pertaining to inadequate clinical trial design, selection of inappropriate endpoints and poor patient selection which are often not addressed following failed disease modification trials. Future directions to overcome these challenges such as reducing the heterogeneity of patient cohorts,

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Measuring Disease Progression in Early Parkinson Disease

Cited by 61 publications

References 15 publications

The Key Determinants to Quality of Life in Parkinson’s Disease Patients: Results from the Parkinson’s Disease Biomarker Program (PDBP)

The Key Determinants to Quality of Life in Parkinson’s Disease Patients: Results from the Parkinson’s Disease Biomarker Program (PDBP)

The Effect of Creatine and Coenzyme Q10 Combination Therapy on Mild Cognitive Impairment in Parkinson's Disease

Challenges in detecting disease modification in Parkinson's disease clinical trials

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