Analysis and validation of a Parkinson's disease register as a recruitment tool for clinical studies.

Promotion of research is a key strategy of the National Health Service (NHS). Currently, many patients are not afforded the opportunity to participate in clinical studies. A register of research-interested patients has the potential to maximise inclusivity. We have established a register of research-interested patients with Parkinson's disease within the South West of England, with pragmatic inclusion criteria and multiple recruitment routes. We undertook an analysis of the register, investigation of its utility as a recruitment tool and a survey of recruiters. There were 529 active participants; 30% were self-referred and 70% were recruited by a healthcare practitioner. Response rate to annual questionnaires was 86.5%. Staff time required for pack preparation, recruitment and data entry was 15 min per new recruit and 5 min per follow-up questionnaire. In total, 85% of recruiters viewed the register positively. A single mailing to participants resulted in a recruitment rate that significantly exceeded that achieved by traditional recruitment methods

Early factors for predicting discontinuation to subcutaneous Apomorphine infusion in Parkinson's disease:A prospective analysis of the Thai Apomorphine Registry

INTRODUCTION: Although continuous subcutaneous apomorphine infusion (CSAI) is an effective therapy for Parkinson's disease (PD) with motor fluctuations, data from Asian cohorts is limited. The therapy is often discontinued due to the complexity of its delivery. METHODS: Fifty-one PD patients undergoing CSAI as an add-on therapy were enrolled in the Thai Apomorphine Registry, an electronic database that recorded clinical characteristics and parameters during the 14-consecutive-day titration and long-term follow-up. Factors at the time of titration were documented in order to identify predictors of long-term discontinuation. RESULTS: Following initiation, PD patients were administered a mean CSAI dose of 5.89 mg/h (SD 1.36) over a mean time of 12.28 h (SD 1.90) each day. The mean follow-up period was 626.2 days (SD 619.17). Significant reductions in UPDRS-I, II, III, and IV scores, total NMSQ score, PDQ-8 score, daily off and dyskinesia hours, Timed Up and Go test, walking step test, levodopa-equivalent daily dose, number of times a day the levodopa was taken versus pre-CSAI values were observed (p < 0.05, each). Thirty-five (68.6%) patients discontinued during the follow-up period. Relative risks of variables recorded at the time of titration that determined discontinuation of CSAI therapy were an absence of full-time caregivers, achieving a daily off hours reduction <3.5 h, and NMSQ scores at the time of CSAI titration ≥9.5 points. CONCLUSION: Identifying factors that predict discontinuation of CSAI at the time of its initiation may help physicians to better understand the patient's drug response and how to manage them long-term

Management of Spastic Paresis and Cervical Dystonia: access to Therapeutic Innovations Through an International Program of Practical Courses

Purpose: Our purpose was to determine satisfaction and confidence of the Ixcellence Network training program on health care practitioners using botulinum toxin A (BoNT-A) for neurologic disorders, including spastic paresis and cervical dystonia. / Methods: The Ixcellence Network training program was designed by a scientific committee of 6 experts and then tested at centers in Europe, and Latin America. The training, provided by 16 experienced neurologists and rehabilitation specialists, consisted of theoretic and practical sessions that covered the different stages of the patient's journey from diagnosis to tailored treatment and rehabilitation. Trainees' feedback and the impact on participants' practice were evaluated by 2 individual questionnaires, at the end of the session (T0) and at 6 months (T6). Trainers' feedback was also collected through an individual questionnaire. / Findings: Between September 2012 and December 2017, 728 trained physicians participated in training programs with 48%, 23%, and 29% of attendees participating in training sessions dedicated to adult spastic paresis, child spastic paresis, and cervical dystonia, respectively. At T0, 93% of attendees thought that they had been given new information and 90% thought that the training would change their daily practice. This was confirmed at T6 by 93% of respondents. Trainees were highly satisfied with the program, in particular with the practical sessions. Trainers expectations were met for attendees' level of expertise, motivation, language, and participation. / Implications: In this descriptive study, we show that the Ixcellence Network program represents a new educational approach to promote consistency in care practices and dissemination of expertise on the use of BoNT-A for neurologic disorders.

Sustained response in early responders to safinamide in patients with Parkinson's disease and motor fluctuations: A post hoc analysis of the SETTLE study

Safinamide is a selective, reversible, monoamine oxidase B inhibitor for the treatment of patients with Parkinson's disease (PD) and motor fluctuations. This was a post hoc analysis of the SETTLE study, in which patients with PD and motor fluctuations were randomly assigned to 24-week treatment with safinamide (50 mg/day for 2 weeks, increased to 100 mg/day if tolerated) or placebo. In the present analysis, responders were defined according to their treatment responses at Week 2 and Week 24 based on changes in ON-time without troublesome dyskinesia from baseline with cutoffs of 1 hour. It was found that 81% (103/127) of the responders at Week 2 maintained the response through Week 24 in the safinamide group. Other outcomes did not necessarily coincide with the ON-time response; however, “Early” responders who showed a treatment response at both Week 2 and Week 24 had substantial improvements from baseline in OFF-time, UPDRS Part II and III scores, and PDQ-39 summary index scores through Week 24. The safinamide group had a higher proportion of early responders than the placebo group (39% vs 20%, p &lt; 0.0001). At baseline, early responders in the safinamide group had significantly higher UPDRS Part II and III scores, shorter ON-time, and longer OFF-time than the other responder populations. In conclusion, the results of the present post hoc analysis suggest that patients with a short ON-time, severe motor symptoms, and highly compromised activities of daily living can benefit from safinamide early in treatment and over the long term

Evaluation of Psychometric Properties of Thai Version Telehealth Usability Questionnaire (T-TUQ)

This cross-sectional validation study aimed to translate, cross-culturally adapt, and investigate the psychometric properties of a Thai version of the Telehealth Usability Questionnaire (T-TUQ). Two hundred and ten Thai participants, mean age of 61.2±15.2 years, were recruited from three specialty clinics: 50 (23.8%) hematology, 70 (33.3%) movement disorders, and 90 (42.9%) general neurology. The T-TUQ was translated from the original English version to produce a Thai language version. Back translation and pilot cognitive interviews were completed. All five subscales (usefulness, ease of use, effectiveness, reliability, and satisfaction) showed excellent internal consistency (alpha &gt;0.80), displayed by Cronbach's alpha coefficient of 0.83, 0.94, 0.86, 0.83, and 0.92, respectively. For construct validity, exploratory factor analysis revealed two dimensions from eigenvalues and scree plot, defined as utility and accessibility subscales. In conclusion, the T-TUQ could be a reliable and valid instrument to evaluate the usability of telehealth with a Thai population

Task force consensus on nosology and cut-off values for axial postural abnormalities in parkinsonism

Background: There is no consensus with regard to the nosology and cut-off values for postural abnormalities in parkinsonism. Objective: To reach a consensus regarding the nosology and cut-off values. Methods: Using a modified Delphi panel method, multiple rounds of questionnaires were conducted by movement disorder experts to define nosology and cut-offs of postural abnormalities. Results: After separating axial from appendicular postural deformities, a full agreement was found for the following terms and cut-offs: camptocormia, with thoracic fulcrum (&gt;45°) or lumbar fulcrum (&gt;30°), Pisa syndrome (&gt;10°), and antecollis (&gt;45°). "Anterior trunk flexion," with thoracic (≥25° to ≤45°) or lumbar fulcrum (&gt;15° to ≤30°), "lateral trunk flexion" (≥5° to ≤10°), and "anterior neck flexion" (&gt;35° to ≤45°) were chosen for milder postural abnormalities. Conclusions: For axial postural abnormalities, we recommend the use of proposed cut-offs and six unique terms, namely camptocormia, Pisa syndrome, antecollis, anterior trunk flexion, lateral trunk flexion, anterior neck flexion, to harmonize clinical practice and future research. Keywords: Parkinson's disease; Pisa syndrome; antecollis; atypical parkinsonisms; camptocormia; diagnostic criteria.; postural abnormalities