Objective To determine whether early and more frequent mobilization after stroke affects health-related quality of life. Methods A Very Early Rehabilitation Trial (AVERT) was an international, multicenter (56 sites), phase 3 randomized controlled trial, spanning 2006–2015. People were included if they were aged ≥18 years, presented within 24 hours of a first or recurrent stroke (ischemic or hemorrhagic), and satisfied preordained physiologic criteria. Participants were randomized to usual care alone or very early and more frequent mobilization in addition to usual care. Quality of life at 12 months was a prespecified secondary outcome, evaluated using the Assessment of Quality of Life 4D (AQoL-4D). This utility-weighted scale has scores ranging from −0.04 (worse than death) to 1 (perfect health). Participants who died were assigned an AQoL-4D score of 0. Results No significant difference in quality of life at 12 months between intervention (median 0.47, interquartile range [IQR] 0.07–0.81) and usual care (median 0.49, IQR 0.08–0.81) groups was identified (p = 0.86), nor were there any group differences across the 4 AQoL-4D domains. The same lack of group difference in quality of life was observed at 3 months. When cohort data were analyzed (both groups together), quality of life was strongly associated with acute length of stay, independence in activities of daily living, cognitive function, depressive symptoms, and anxiety symptoms (all p < 0.001). Quality of life in AVERT participants was substantially lower than population norms, and the gap increased with age. Conclusions Earlier and more frequent mobilization after stroke did not influence quality of life. Clinical trial registration anzctr.org.au; ACTRN12606000185561 Classification of evidence This study provides Class II evidence that for people with stroke, earlier and more frequent mobilization did not influence quality of life over the subsequent year.

Summary Background: Hospital length of stay and discharge destination are important outcome measures in evaluating effectiveness and efficiency of health services. Although hospital administrative data are readily used as a data collection source in health services research, no research has assessed this data collection method against other commonly used methods. Objective: Determine if administrative data from electronic patient management programs are an effective data collection method for key hospital outcome measures when compared with alternative hospital data collection methods. Method: Prospective observational study comparing the completeness of data capture and level of agreement between three data collection methods; manual data collection from ward-based sources, administrative data from an electronic patient management program (i.PM), and inpatient medical record review (gold standard) for hospital length of stay and discharge destination. Results: Manual data collection from ward-based sources captured only 376 (69%) of the 542 in-patient episodes captured from the hospital administrative electronic patient management program. Administrative data from the electronic patient management program had the highest levels of agreement with inpatient medical record review for both length of stay (93.4%) and discharge destination (91%) data. Conclusion: This is the first paper to demonstrate differences between data collection methods for hospital length of stay and discharge destination. Administrative data from an electronic patient management program showed the highest level of completeness of capture and level of agreement with the gold standard of inpatient medical record review for both length of stay and discharge destination, and therefore may be an acceptable data collection method for these measures. Citation: Sarkies MN, Bowles K-A, Skinner EH, Mitchell D, Haas R, Ho M, Salter K, May K, Markham D, O’Brien L, Plumb S, Haines T.P. Data collection methods in health services research – hospital length of stay and discharge destination. Appl Clin Inf 2015; 6: 96–109 http://dx.doi.org/10.4338/ACI-2014-10-RA-0097

BACKGROUND:Hospital length of stay and discharge destination are important outcome measures in evaluating effectiveness and efficiency of health services. Although hospital administrative data are readily used as a data collection source in health services research, no research has assessed this data collection method against other commonly used methods. OBJECTIVE:Determine if administrative data from electronic patient management programs are an effective data collection method for key hospital outcome measures when compared with alternative hospital data collection methods. METHOD:Prospective observational study comparing the completeness of data capture and level of agreement between three data collection methods; manual data collection from ward-based sources, administrative data from an electronic patient management program (i.PM), and inpatient medical record review (gold standard) for hospital length of stay and discharge destination. RESULTS:Manual data collection from ward-based sources captured only 376 (69%) of the 542 inpatient episodes captured from the hospital administrative electronic patient management program. Administrative data from the electronic patient management program had the highest levels of agreement with inpatient medical record review for both length of stay (93.4%) and discharge destination (91%) data. CONCLUSION:This is the first paper to demonstrate differences between data collection methods for hospital length of stay and discharge destination. Administrative data from an electronic patient management program showed the highest level of completeness of capture and level of agreement with the gold standard of inpatient medical record review for both length of stay and discharge destination, and therefore may be an acceptable data collection method for these measures.

Background: Rasagiline (N-propargyl-1[R]-aminoindan) mesylate is a novel irreversible selective monoamine oxidase type B inhibitor, previously demonstrated to improve symptoms in early Parkinson disease (PD).Objective: To determine the safety, tolerability, and efficacy of rasagiline in levodopa-treated patients with PD and motor fluctuations.Design: Multicenter, randomized, placebo-controlled, double-blind, parallel-group study.Patients: Parkinson disease patients (N=472) with at least 2(1)/(2) hours of daily "off' (poor motor function) time, despite optimized treatment with other anti-PD medications.Interventions: Rasagiline, 1.0 or 0.5 mg/d, or matching placebo.Main Outcome Measures: Change from baseline in total daily off time measured by patients' home diaries during 26 weeks of treatment, percentage of patients completing 26 weeks of treatment, and adverse event frequency.Results: During the treatment period, the mean adjusted total daily off time decreased from baseline by 1.85 hours (29%) in patients treated with 1.0 mg/d of rasagiline, 1.41 hours (23%) with 0.5 mg/d rasagiline, and 0.91 hour (15%) with placebo. Compared with placebo, patients treated with 1.0 mg/d rasagiline had 0.94 hour less off time per day, and patients treated with 0.5 mg/d rasagiline had 0.49 hour less off time per day. Prespecified secondary end points also improved during rasagiline treatment, including scores on an investigator-rated clinical global impression scale and the Unified Parkinson's Disease Rating Scale (activities of daily living in the off state and motor performance in the "on" state). Rasagiline was well tolerated.Conclusions: Rasagiline improves motor fluctuations and PD symptoms in levodopa-treated PD patients. In light of recently reported benefits in patients with early illness, rasagiline is a promising new treatment for PD.

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We report on the development and results of preliminary psychometric testing of a disease specific health‐related quality of life (HRQoL) scale intended for use in individuals diagnosed with idiopathic Parkinson's disease (PD). Results from an initial qualitative study provided content for item development and scale construction of the Parkinson's disease quality of life scale (PDQUALIF). The 33‐item instrument includes seven domains: social/role function, self‐image/sexuality, sleep, outlook, physical function, independence, and urinary function, plus one item of Global HRQoL. Initial psychometric testing of the instrument was conducted in 233 outpatient clinic attendees with physician‐confirmed idiopathic PD. Factor structure, reliability and validity of the scale have been established in this cross‐sectional study. Continuing development of the PDQUALIF will be directed at enhancing the psychometric properties, establishing responsiveness and determining appropriateness in culturally diverse samples. © 2003 Movement Disorder Society

Context: Monotherapy with rasagiline mesylate may be useful in early Parkinson disease (PD).Objective: To evaluate the safety and efficacy of the selective monoamine oxidase type B inhibitor rasagiline.Design: Multicenter, 26-week, parallel-group, randomized, double-blind, placebo-controlled clinical trial.Setting: Academically based movement disorders clinics.Patients: Patients with early PD not requiring dopaminergic therapy (n=404).Intervention: Research participants were randomized to rasagiline mesylate at dosages of 1 mg or 2 mg per day or matching placebo. A I-week escalation period was followed by a 25-week maintenance period.Main Outcome Measure: The primary prespecified measure of efficacy was the change in the total Unified Parkinson's Disease Rating Scal score between baseline and 26 weeks of treatment, comparing each active treatment group with the placebo group.Results: Monotherapy with rasagiline was effective in this 26-week study. The adjusted effect size for the total Unified Parkinson's Disease Rating Scale was -4.20 units comparing I mg of rasagiline and placebo (95% confidence interval, -5.66 to -2.73 units; P<.001) and -3.56 units comparing a 2-mg dosage and placebo (95% confidence interval, -5.04 to -2.08 units; P<.001). There were no meaningful differences in the frequency of adverse events or premature withdrawals among the treatment groups.Conclusions: Rasagiline is effective as monotherapy for patients with early PD. The 2 dosages in this trial were both effective relative to placebo. Further study is warranted to evaluate the longer-term effects of rasagiline in PD.

Background: The treatment of children with attention deficit hyperactivity disorder (ADHD) and Tourette syndrome (TS) has been problematic because methylphenidate (MPH)-the most commonly used drug to treat ADHD-has been reported to worsen tics and because clonidine (CLON)-the most commonly prescribed alternative-has unproven efficacy. Methods: The authors conducted a multicenter, randomized, double-blind clinical trial in which 136 children with ADHD and a chronic tic disorder were randomly administered CLON alone, MPH alone, combined CLON + MPH, or placebo (2 X 2 factorial design). Each subject participated for 16 weeks (weeks 1-4 CLON/placebo dose titration, weeks 5-8 added MPH/placebo dose titration, weeks 9-16 maintenance therapy). Results: Thirty-seven children were administered MPH alone, 34 were administered CLON alone, 33 were administered CLON + MPH, and 32 were administered placebo. For our primary outcome measure of ADHD (Conners Abbreviated Symptom Questionnaire-Teacher), significant improvement occurred for subjects assigned to CLON (p < 0.002) and those assigned to MPH (p < 0.003). Compared with placebo, the greatest benefit occurred with combined CLON + MPH (p < 0.0001). CLON appeared to be most helpful for impulsivity and hyperactivity; MPH appeared to be most helpful for inattention. The proportion of individual subjects reporting a worsening of tics as an adverse effect was no higher in those treated with MPH (20%) than those being administered CLON alone (26%) or placebo (22%). Compared with placebo, measured tic severity lessened in all active treatment groups in the following order: CLON + MPH, CLON alone, MPH alone. Sedation was common with CLON treatment (28% reported moderate or severe sedation), but otherwise the drugs were tolerated well, including absence of any evident cardiac toxicity. Conclusions: Methylphenidate and clonidine (particularly in combination) are effective for ADHD in children with comorbid tics. Prior recommendations to avoid methylphenidate in these children because of concerns of worsening tics are unsupported by this trial.

Our objective was to assess the test-retest reliability of the Unified Parkinson's Disease Rating Scale (UPDRS). The UPDRS is the most widely used instrument for measuring severity of parkinsonian symptoms in clinical research and in practice. The validity and inter-rater reliability of this scale have been previously studied. We examined the test-retest (intrarater) reliability of the UPDRS and derived subscales. Four hundred patients with early-stage Parkinson's disease (PD) who were participating in a multicenter clinical trial were evaluated using the UPDRS on two separate occasions (screening and baseline visits) prior to receiving treatment. The same neurologist at each center rated the subjects at both examinations that were, on average, 14.6 +/- 7.6 days apart (range 3-36 days). Test-retest reliability was estimated using the intraclass correlation coefficient (ICC) for the total UPDRS score, the mental, ADL, and motor subscale scores, and other derived subscale scores. Weighted kappa statistics were calculated for individual UPDRS items. The ICCs for the UPDRS scores were as follows: total score, 0.92; mental, 0.74; ADL, 0.85; motor, 0.90. ICCs for derived symptom-based scales ranged from 0.69-0.88. Reliability of specific items was generally lower than for summary scales. Reliability was slightly better in patients for whom the testing interval was within 14 days. Based on conventional standards, the UPDRS scores were found to have excellent test-retest reliability in this sample of patients with early PD rated by academic movement disorder specialists. The findings are in agreement with previous reports Oil interrater reliability. (C) 2002 Movement Disorder Society.

BACKGROUND:Parkinson disease (PD) is a degenerative neurological disorder for which no treatment has been shown to slow the progression.OBJECTIVE:To determine whether a range of dosages of coenzyme Q10 is safe and well tolerated and could slow the functional decline in PD.DESIGN:Multicenter, randomized, parallel-group, placebo-controlled, double-blind, dosage-ranging trial.SETTING:Academic movement disorders clinics.PATIENTS:Eighty subjects with early PD who did not require treatment for their disability.INTERVENTIONS:Random assignment to placebo or coenzyme Q10 at dosages of 300, 600, or 1200 mg/d.MAIN OUTCOME MEASURE:The subjects underwent evaluation with the Unified Parkinson Disease Rating Scale (UPDRS) at the screening, baseline, and 1-, 4-, 8-, 12-, and 16-month visits. They were followed up for 16 months or until disability requiring treatment with levodopa had developed. The primary response variable was the change in the total score on the UPDRS from baseline to the last visit.RESULTS:The adjusted mean total UPDRS changes were +11.99 for the placebo group, +8.81 for the 300-mg/d group, +10.82 for the 600-mg/d group, and +6.69 for the 1200-mg/d group. The P value for the primary analysis, a test for a linear trend between the dosage and the mean change in the total UPDRS score, was.09, which met our prespecified criteria for a positive trend for the trial. A prespecified, secondary analysis was the comparison of each treatment group with the placebo group, and the difference between the 1200-mg/d and placebo groups was significant (P =.04).CONCLUSIONS:Coenzyme Q10 was safe and well tolerated at dosages of up to 1200 mg/d. Less disability developed in subjects assigned to coenzyme Q10 than in those assigned to placebo, and the benefit was greatest in subjects receiving the highest dosage. Coenzyme Q10 appears to slow the progressive deterioration of function in PD, but these results need to be confirmed in a larger study.

Oxidative damage due to free-radical generation in the setting of underlying defects of neuronal energy metabolism has been implicated as a pathogenetic mechanism for Huntington's disease (HD). The authors conducted a randomized, double-blind, placebo-controlled, multicenter trial of the tolerability of OPC-14117, a lipophilic free-radical scavenger that concentrates in the brain. Ambulatory patients with HD received OPC-14117 60 mg/d, 120 mg/d, 240 mg/d, or placebo and were assessed by the Unified Huntington's Disease Rating Scale (UHDRS) for 20 weeks, including 12 or 16 weeks of assigned treatment and 8 or 4 weeks of blinded withdrawal of the study drug. Tolerability was measured by the proportion of patients completing the initial 12-week course of treatment on their originally assigned regimen. Sixty-four patients were enrolled in the study, 56 of whom completed the 12 weeks of treatment. Treatment was discontinued in four patients (1 placebo, 1 60 mg/d, 2 240 mg/d) due to asymptomatic but persistent serum elevations of liver transaminase. Two patients (1 60 mg/d and 1 120 mg/d) withdrew because of increased involuntary movements, one patient (60 mg/d) withdrew due to persistent dry eyes, and one patient (120 mg/d) withdrew because of persistent vomiting. There were no significant differences between treatment arms in the primary measures of tolerability, the frequency and types of clinical adverse events, or the clinical/functional features of HD. OPC-14117 was safe and generally well tolerated; however, elevations of liver transaminase suggested that continued surveillance monitoring is warranted in conducting more long-term studies of this antioxidant therapy.

Cognitive impairment is a frequent manifestation of advanced human immunodeficiency virus (HIV) infection. The response to antiretroviral medication is often partial and poorly sustained. Recent studies suggest that free radical production within the CNS and neuronal apoptosis may play important roles in the pathogenesis of HN dementia. We conducted a randomized double-blind, placebo-controlled trial using a parallel group, 2 x 2 factorial design evaluating deprenyl, a monoamine oxidase B inhibitor and putative anti-apoptotic agent, and thioctic acid, an antioxidant, in 36 patients with HIV-associated cognitive impairment. Both deprenyl and thioctic acid were well tolerated with few adverse events. Deprenyl recipients showed significant improvement on tests of verbal memory compared with patients not taking deprenyl. Thioctic acid treatment did not improve cognitive function. These results suggest that deprenyl treatment is associated with cognitive improvement in subjects with mild HN-associated cognitive impairment, whereas thioctic acid has no benefit. A larger efficacy trial is needed to assess the long-term effect of deprenyl on cognitive performance in patients with HIV infection.

Facioscapulohumeral muscular dystrophy (FSHD) is an autosomal-dominant disorder localized to 4q35. Neither the gene nor the gene product has been identified. There is presently no established treatment for FSHD. Prospective data on the natural history of this disorder are essential for the effective design of therapeutic trials. We systematically followed 81 well defined FSHD patients for up to 3 years using a standardized protocol that included manual muscle testing (MMT), maximum voluntary isometric contraction testing (MVICT), and functional testing. Muscle strength was strongly associated with measures of muscle mass, age at onset, and duration of disease. Decline in strength over time was slow but detectable with both MVICT and MMT. The magnitude of decline was not associated with either age, gender, age at onset, or duration of disease. This study establishes reliable and valid measures of disease state and progression for use as outcome variables in clinical trials in FSHD, and also provides guidelines for determining sample size and duration of follow-up. A two-armed clinical trial involving 160 patients per group and 1 year of follow-up would provide 80% power to detect complete arrest of the progression of the disease. Trials with fewer patients would thus have adequate power to detect only improvements in strength, unless follow-up duration were extended well beyond 1 year.

Motor fluctuations associated with levodopa therapy are common problems encountered in the long-term treatment of Parkinson's disease (PD). Entacapone, a peripherally acting, reversible inhibitor of catechol-O-methyltransferase, slows the elimination of levodopa in humans by reducing the formation of 3-O-methyldopa. We conducted a placebo-controlled, double-blind, parallel-group, multicenter trial of entacapone in PD patients with motor fluctuations. Two hundred five patients were randomized to receive either entacapone 200 mg or matching placebo with each dose of levodopa and were followed for 24 weeks. The primary measure of efficacy was the change in percentage of ''on'' time (relief of parkinsonism) while awake, as recorded by subjects at home in diaries completed at 30-minute intervals. At baseline, patients averaged approximately 10 hours of ''on'' time per day while awake (60.5% ''on'' time), and entacapone treatment increased the percent ''on'' time by 5.0 percentage points. The effect of entacapone was more prominent in patients with a smaller percent ''on'' time (<55%) at baseline, and increased as the day wore on. Entacapone is effective at increasing the duration of response to levodopa and at relieving parkinsonism in patients experiencing motor fluctuations and was well tolerated during the 24 weeks of treatment.

BACKGROUND A retrospective study of 138 children with Tourette's syndrome for associated school problems revealed that at the time of initial evaluation, 64 subjects (46%) experienced a school-related problem. OBJECTIVE To survey a childhood population with Tourette's syndrome to explore the contributions of neurobehavioral concomitants to academic difficulties. RESULTS A diagnosis of a specific learning disorder had previously been made in 30 (22%) of 138 children. Among the 108 without a diagnosis of learning disorder, 36 (33%) experienced school difficulties defined as grade retention (16 [15%]) and/or special education placement (41 [38%]). Regression analysis of subjects without a diagnosis of learning disability revealed that the presence of attention-deficit hyperactivity disorder served as a significant predictor of school problems. CONCLUSIONS Tics represented the primary reason for referral, but did not emerge as a significant predictor of academic problems. Rather, school-related difficulties appeared to be strongly associated with comorbid attention-deficit hyperactivity disorder.

In the controlled trial Deprenyl and Tocopherol Antioxidative Therapy of Parkinsonism (DATATOP), 310 of the 800 enrolled subjects did not reach the primary end point of disability requiring levodopa therapy during 21 +/- 4 (mean +/- SD) months of observation or need early initiation of deprenyl (selegiline) during a 2-month withdrawal of experimental treatments. While maintaining the blindness of their original deprenyl and tocopherol treatment assignments, these 310 subjects were administered deprenyl 10 mg/day (open label) and were monitored systematically at 1- to 3-month intervals for up to 18 months (12 +/- 5 mo). During this extended trial, the 189 subjects who had been assigned originally to active deprenyl tended to reach the end point of disability faster than the 121 subjects who had not been assigned originally to deprenyl (hazard ratio, 1.43; 95% CI, 0.98, 2.09; p = 0.065). However, the differential rates of reaching the end point may have been due in part to the more severe baseline impairment of deprenyl-assigned subjects, who benefited originally from deprenyl but who were more likely to require levodopa during this extended period of observation. Prior treatment with deprenyl did not lead to superior survival with respect to the end point of disability requiring levodopa, suggesting that the initial advantages of deprenyl were not sustained.

To investigate the effect of drugs acting on the endogenous opioid system, we studied 10 adults with Tourette's syndrome who received propoxyphene hydrochloride (260 mg/day), naltrexone hydrochloride (50 mg/day), and placebo in a double‐blinded, randomized clinical trial. Using a self‐report scale (Tourette's Syndrome Symptom List), subjects noted a significant ( p < 0.04) lessening of tics after treatment with naltrexone when compared with placebo. An improvement in performance on the Trail Making B test, a measure of attention and visuomotor sequencing and planning, occurred after receiving naltrexone when compared with placebo ( p < 0.08) or propoxyphene ( p < 0.02). The Trail Making B test best discriminated the treatments ( p < 0.02, analysis of variance). No other treatment effects were observed for several other measures of tic severity, attentional ability, or obsessive‐compulsive symptoms. Our findings indicate that pharmacological manipulation of the endogenous opioid system does influence symptoms of Tourette's syndrome.