Inclusion body myositis functional rating scale: a reliable and valid measure of disease severity

We developed a disease-specific, 10-point functional rating scale for patients with inclusion body myositis (IBMFRS). The IBMFRS was utilized as a secondary outcome measure in a multicenter pilot trial of the clinical safety and tolerability of high-dose beta interferon-1a. In this trial, 28 IBM patients completed the IBMFRS at baseline and monthly for 6 months. The IBMFRS showed statistically significant correlations (P < 0.001) with maximal voluntary isometric contraction, manual muscle testing, handgrip dynamometry, and the amyotrophic lateral sclerosis (ALS) functional rating scale (ALSPRS). Compared to these other outcome measures, the IBMFRS was also the most sensitive measure of change over the course of the study.     

Exploring hand and upper limb function in patients with inclusion body myositis (IBM)

Inclusion body myositis (IBM) is an inflammatory myopathy characterized by progressive weakness of knee extensors and finger flexors. Many patients lose independence with fine motor tasks; however, a gap remains as to how these deficits correlate with performance on functional outcome measures. We describe functional hand impairments as measured by performance-based outcome measures in a cross-sectional sample of 74 patients with IBM. Subjects completed a series of outcome measures (Functional Dexterity Test (FDT), Performance of the Upper Limb (PUL), and Sollerman Hand Function Test (SHFT)) alongside a collection of patient reported outcomes (PROs). Assessments were compared to standard IBM measurements, including grip strength and IBM Functional Rating Scale (IBMFRS). FDT and SHFT demonstrated significant correlations to grip (p<0.001; Spearman correlations r=0.48–0.70). Significant correlation was found between all functional outcome measures and IBMFRS (p<0.001; Spearman correlations r=0.51–0.77), as well as PRO Upper Extremity Scale for IBM (IBM-PRO) (p<0.05; Spearman correlations r=0.55–0.73). Non-ambulatory patients demonstrated significantly weaker grip (p<0.001), resulting in lower PUL scores and increased FDT completion times (p<0.001). Collectively, these assessments may provide insight to understanding functional limitations of the hands and potentially allow for more inclusive clinical trials with future validation of hand assessments in IBM.     

Prognostic significance of blood brain barrier permeability in acute hemorrhagic stroke

BACKGROUND: Blood brain barrier (BBB) disruption is accompanied by edema in the surrounding areas of the intracerebral hemorrhage (ICH). The aim of the study was to clarify the correlation between BBB breakdown and outcome in ICH. PATIENTS: Twenty-seven patients with primary ICH were included in the study. Each patient underwent CT and DTPA-SPECT, and the National Institutes of Health (NIH) and modified Rankin score were performed as well. RESULTS: DTPA-SPECT had a significant correlation with the modified Rankin score after 3 months (p = 0.008) and 6 months (p = 0.01). The CT scan was directly correlated with the NIH score on days 1, 7 and 30 (p = 0.01, p = 0.01 and p = 0.04, respectively). No correlation was found between DTPA-SPECT and CT scan data. CONCLUSIONS: The degree of BBB breakdown, as imaged by the DTPA-SPECT technique, was directly correlated with the late functional outcome. The CT scan has an inverse correlation with the NIH score. These findings may have broad clinical implications.     

Correlation between whole body muscle MRI and functional measures in paediatric patients with facioscapulohumeral muscular dystrophy

Symptoms and severity of facioscapulohumeral muscular dystrophy (FSHD) can vary greatly, even within the same family. Clinical trial readiness requires accurate and reliable methods of assessing disease stage and progression. MRI has not previously been assessed as a disease biomarker in paediatric FSHD. Eleven patients with FSHD1 underwent whole body muscle MRI. Pre-selected muscles were analysed by a paediatric radiologist using the semi-quantitative Mercuri/Kim method. Within each domain (oedema, fat replacement, atrophy) scores for each muscle were then summated to give each participant three cumulative domain scores. The same participants had functional measures scored: FSHD-CSS (Ricci), FSHD-CS (Lamperti), FSHD-COM, PUL2.0, MFM-32, 6MWT, myometry and manual muscle testing. Pearson coefficient was calculated to determine strength of correlation. The scores for atrophy and fat replacement showed strong correlation with functional outcome measures, particularly FSHD-CSS, FSHD-CS and FSHD-COM. In contrast, muscle oedema correlated poorly with all functional outcome measures, with no relationship seen to the 6MWT. This study of eleven children suggests that semi-quantitative visual Mercuri score utilising fat replacement or atrophy on whole body muscle MRI correlates strongly with disease-specific functional measures and may be a useful measure of disease severity in paediatric FSHD.     

El pico flujo espiratorio y la cuenta máxima son marcadores del compromiso respiratorio en la miastenia gravis

IntroductionMyasthenia gravis (MG) is an antibody-mediated autoimmune disease characterised by fluctuating, fatigable muscle weakness, frequently involving bulbar and respiratory muscles. Considering the severity of respiratory involvement in MG, routine evaluation of respiratory function is essential.The aim of this study was to identify a useful clinical marker of respiratory involvement in patients with MG.MethodsWe performed an observational study of patients with MG. All cases were evaluated with the single-breath counting test, peak expiratory flow (PEF), a modified Medical Research Council dyspnoea scale (mMRC), and a neck strength assessment. The results of these parameters were correlated with forced vital capacity (FVC), maximal inspiratory pressure (MIP), and maximal expiratory pressure (MEP).ResultsThe study included 45 patients with MG: 2 patients classified as grade I on the Myasthenia Gravis Foundation of America classification at the time of evaluation, 35 classified as grade II, 7 classified as grade III, and one classified as grade IV. Positive correlations were found between single-breath counting test scores and FVC values (r = 0.57, p = .000), and between PEF and FVC values (r = 0.76, p = .000). Severity of dyspnoea according to the mMRC scale showed a negative correlation with FVC values (r = -0.31, p = .03). PEF also showed a significant correlation with MEP (r = 0.51, p = .002).ConclusionsPEF, the single-breath counting test, and the mMRC scale are useful measures for evaluating respiratory function in patients with MG.     

Longitudinal observational study of sporadic inclusion body myositis: Implications for clinical trials

Sporadic inclusion body myositis (IBM) is the most common acquired myopathy occurring in adults aged over 50 years. The aim of the study was to assess prospectively the clinical features and functional impact of sporadic inclusion body myositis (IBM). Clinical data, manual muscle testing (MMT), quantitative muscle testing (QMT) of quadriceps muscle and IBM functional rating scale (IBM-FRS) were collected according to a standardised protocol at baseline (n = 51) and one-year follow-up (n = 23). MMT, quadriceps QMT and IBM-FRS significantly declined after one year (by 5.2%, 27.9%, and 13.8%, respectively). QMT of the quadriceps muscle and IBM-FRS were the most sensitive measures of disease progression. After a median time of seven years of disease duration, 63% of patients had lost independent walking. Disease onset after 55 years of age, but not sex or treatment, is predictive of a shorter time to requirement of a walking stick. We detected no differences in disease presentation and progression between clinically and pathologically defined IBM patients. The study provides evidence that quadriceps QMT and IBM-FRS could prove helpful as outcome measures in future therapeutic trials in IBM.     

Prestroke Conditions of Acute Ischemic Stroke Patients are Associated with Functional Outcome after Mechanical Thrombectomy

Background and AimMechanical thrombectomy was demonstrated to be useful for acute ischemic stroke. However, whether it is beneficial for patients with poor prestroke conditions, such as older adults and those with low activity of daily living, is unclear.MethodsA total of 134 patients who underwent mechanical thrombectomy in our hospital between April 2015 and January 2019 were retrospectively evaluated. Good outcome was defined as modified Rankin scale score of 0-2 at 90 days after stroke onset. Several factors were analyzed to assess their effects on clinical outcomes.ResultsAt 90 days after stroke onset, 37.3% (50 of 134) of patients had a good outcome. Prestroke modified Rankin scale score was independently associated with a good outcome (odds ratio .39, 95% confidence interval .22-.67, P < .001). In patients with prestroke modified Rankin scale score 0-1, 55.4% (46 of 83) had a good outcome, and no significant difference in prognosis was found between patients aged less than 80 years and those aged greater than or equal to 80 years (P = .64). More than half the patients with prestroke modified Rankin scale score greater than or equal to 2 were graded as modified Rankin scale score 5-6 at 90 days regardless of age, which was significantly higher than those with prestroke modified Rankin scale score 0-1 (P < .001).ConclusionsPatients with prestroke modified Rankin scale score 0-1 are expected to have a good prognosis after mechanical thrombectomy even if aged greater than or equal to 80 years. Patients with prestroke modified Rankin scale score greater than or equal to 2 might have an extremely poor prognosis, and we should be more careful in selecting candidates for mechanical thrombectomy.     

肌萎缩侧索硬化患者复合肌肉动作电位特点分析及其与临床关系的研究

目的分析肌萎缩侧索硬化患者复合肌肉动作电位之特点,探讨波幅改变与肌力、病程以及神经功能等级评分等方面的关系。方法收集2001年5月-2004年11月肌电图检查表现为广泛神经源性损害的肌萎缩侧索硬化患者127例,根据ElEscorial诊断标准,确诊级43例、拟诊级39例、可能级13例、可疑级32例。选择其中确诊级和拟诊级患者82例进行神经功能评分和Appel肌萎缩侧索硬化量表评分,然后对其复合肌肉动作电位波幅值与肌力、病程以及神经功能评分变化间的关系进行比较分析。结果(1)82例患者复合肌肉动作电位波幅下降,与肌力改变呈明显指数拟合关系(r=0.969,P=0.001)。(2)同一患者波幅水平随病程而逐渐降低,波幅下降速度较慢者病程较长;不同患者的波幅与病程无显著相关(r=!0.077,P=0.502)。(3)复合肌肉动作电位波幅水平降低与神经功能等级评分呈显著正相关(r=0.412,P=0.001),与Appel评分呈显著负相关(r=!0.549,P=0.001)。(4)患者复合肌肉动作电位波幅水平的降低与运动神经传导速度无明显相关(r=!0.087,P=0.545),但若将波幅与传导速度转换为正常下限的百分比取其平方根后,二者间则呈线性相关(r=0.382,P<0.001)。结论复合肌肉动作电位波幅改变是肌萎缩侧索硬化原发性与继发性病理改变共同作用的结果,随着患者肌无力和肌萎缩症状的逐渐加重,复合肌肉动作电位波幅亦明显下降,但由于此时髓鞘仍保留完整,传导速度仍可保持正常,提示肌萎缩侧索硬化患者肌力下降的机制主要系下运动神经元损害所致。在同一患者,复查时显示复合肌肉动作电位波幅随病程进展而逐渐下降,但尚难建立反映二者平行关系的时间-波幅曲线,提示复合肌肉动作电位波幅改变与病程关系较为复杂。此外,复合肌肉动作电位波幅与肌萎缩侧索硬化患者的神经功能等级评分和Appel量表评分明显相关,提示早期波幅下降较慢者病情相对较轻。     

Intravenous immunoglobulin therapy in amyotrophic lateral sclerosis

Seven consecutive patients with amyotrophic lateral sclerosis (ALS) were treated with intravenous immunoglobulins (IVIg; 0.4 g/kg per day for 5 consecutive days followed by monthly 2-day infusions at the same daily dosage) continued with oral cyclophosphamide (1–2 mg/kg per day), for 4–13 months (mean 8.1). Response to treatment was assessed by means of the Medical Research Council (MRC) rating scale for muscle strength on 40 muscles (10 per limb), a clinical scale for bulbar function and a modified Rankin disability scale. All patients continued to deteriorate during treatment on as regards both their MRC score and either their bulbar or Rankin score or both. The progression of the disease during treatment, expressed as the monthly variation in MRC score (mean=−2.71; SD=1.36), was no slower than that estimated before therapy (mean=−1.81; SD=0.93). Even if the results of this small, uncontrolled study do not permit the exclusion of an effect of IVIg on the progression of ALS, they also do not provide any evidence that this expensive form of therapy consistently slows the course of the disease.     

Evaluation of serum S100B as a surrogate marker for long-term outcome and infarct volume in acute middle cerebral artery infarction

BACKGROUND: An easily accessible and valid surrogate marker for interventional stroke trials is needed. OBJECTIVE: To investigate the usefulness of various S100B serum measures to predict long-term outcome and infarct volume in patients with acute stroke. DESIGN: Inception cohort study. SETTING: Tertiary care university hospital. PATIENTS: Thirty-nine patients (mean +/- SD age, 69.1 +/- 11.5 years) with acute nonlacunar middle cerebral artery infarction presenting less than 6 hours after symptom onset. MAIN OUTCOME MEASURES: Functional outcome 6 months after stroke (modified Rankin scale score) and final infarct volume on day 7 by means of standardized volumetry of brain images. Serum S100B level was determined at hospital admission and 24, 48, 72, 96, 120, and 144 hours after symptom onset. RESULTS: Single S100B measures obtained 48 and 72 hours after stroke onset demonstrated the highest Spearman rank correlations with modified Rankin scale scores (rho = 0.68 and rho = 0.67, respectively; P<.001) and infarct volume (rho = 0.95 and rho = 0.94, respectively; P<.001). A 48-hour S100B value of 0.37 microg/L or less revealed a sensitivity of 0.87 and a specificity of 0.78 in predicting an independent functional outcome. In a multivariate model, S100B emerged as an outcome predictor that was independent of age, sex, stroke severity, etiology, lesion side, and risk factors. CONCLUSIONS: Single S100B values obtained 48 and 72 hours after stroke onset provide the highest predictive values with respect to functional outcome and infarct volume in nonlacunar middle cerebral artery infarction. More complex measures of the S100B kinetic (ie, area under the curve or peak value) were not superior. Therefore, these single S100B measures appear to be useful surrogate end points in acute interventional stroke trials.     

脑梗死急性期超敏C反应蛋白水平与神经功能恢复的关系

目的探讨脑梗死急性期超敏C反应蛋白（hs-CRP）水平与脑梗死神经功能恢复的关系。方法选取发病72h内的急性脑梗死患者269例,入院第2天测定hs-CRP,按hs-CRP水平分为低hs-CRP组（hs-CRP≤3mg/L）及高hs-CRP组（hs-CRP〉3mg/L）,比较2组患者的临床资料,分析影响hs-CRP水平的因素;入选患者进行90d随访,比较2组患者90d时神经功能恢复情况（改良Rankin量表评分3～6分为恢复不良）,分析影响神经功能恢复的因素。结果 共266例患者完成随访,hs-CRP中位数3.15mg/L（1.12～8.89mg/L）。高hs-CRP组（138例）年龄较大（P〈0.001）,糖尿病发病率较高（P=0.001）,房颤发病率较高（P=0.004）,入院时脑梗死较严重（P=0.003）;高龄、合并糖尿病及房颤、入院时脑梗死严重程度与hs-CRP水平高独立相关（P均〈0.05）;高hs-CRP组神经功能恢复不良比例高于低hs-CRP组（P〈0.001）;hs-CRP升高与神经功能恢复不良独立相关（OR1.213,P=0.001,Logistic分析）。结论 高龄、糖尿病史、房颤病史、脑梗死严重程度重可能是hs-CRP升高的独立危险因素;hs-CRP升高可能是神经功能恢复不良的独立危险因素。     

Combined clinical and radiological prognostic model in acute ischemic stroke

We sought to propose and test the validity ofa comprehensive prognostic model in middle cerebral artery-stroke treated with intravenous thrombolysis. A total of 127 consecutive patients (aged 70 +/- 12 years; 54% males) were included in this retrospective study. Variables included in our prognostic model were: NIHSS on admission (1-3 points), occurrence of hyperdense middle cerebral artery sign and early ischemic signs on baseline CT (1 point each), NIHSS at 24 hours (0-3 points), posttreatment hemorrhage (1 point), and infarct volume (0-4 points). The score range was 1-13, with higher values suggest unfavorable prognosis. Our prognostic score was correlated with the modified Rankin scale (mRS) at 3 months after stroke [correlation coefficient of 0.62, P < 0.001] and can thus help early prediction of the functional outcome. Logistic regression showed that NIHSS at 24 hours and EICs on baseline CT were independent predictor of our prognostic score (adjusted odds ratio of 4.1 and 5). Adopting a cut-off value of prognostic score < or = 3 for favorable prognosis and > or = 7for unfavorable prognosis helped to predict the need for institutionalization and the functional outcome with higher accuracy and predictive values compared with using scores only based on NIHSS.     

Connecting impairment,disability, and handicap in immune mediated polyneuropathies

BACKGROUND: In the World Health Organisation (WHO) International Classification of Impairments, Disabilities, and Handicaps (ICIDH), it is suggested that various levels of outcome are associated with one another. However, the ICIDH has been criticised on the grounds that it only represents a general, non-specific relation between its entities. OBJECTIVE: To examine the significance of the ICIDH in immune mediated polyneuropathies. METHODS: Four impairment measures (fatigue severity scale, MRC sum score, "INCAT" sensory sum score, grip strength with the Vigorimeter), five disability scales (nine hole peg test, 10 metres walking test, an overall disability sum score (ODSS), Hughes functional grading scale, Rankin scale), and a handicap scale (Rotterdam nine items handicap scale (RIHS9)) were assessed in 113 clinically stable patients (83 with Guillain-Barré syndrome, 22 with chronic inflammatory demyelinating polyneuropathy, eight with a gammopathy related polyneuropathy). Regression analyses with backward and forward stepwise strategies were undertaken to determine the correlation between the various levels of outcome (impairment on disability, impairment on handicap, disability leading to handicap, and impairment plus disability on handicap). RESULTS: Impairment measures explained a substantial part of disability (R(2) = 0.64) and about half of the variance in handicap (R(2) = 0.52). Disability measures showed a stronger association with handicap (R(2) = 0.76). Combining impairment and disability scales accounted for 77% of the variance in handicap (RIHS9) scores. CONCLUSIONS: In contrast to some suggestions, support for the ICIDH model is found in the current study because significant associations were shown between its various levels in patients with immune mediated polyneuropathies. Further studies are required to examine other possible contributors to deficits in daily life and social functioning in these conditions.     

Randomized controlled trial of IVIg in untreated chronic inflammatory demyelinating polyradiculoneuropathy

OBJECTIVE: To determine the efficacy of IV immunoglobulin (IVIg) given patients with untreated chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). METHODS: A randomized, double-blind, multicenter, investigator-initiated study compared IVIg (Aventis Behring LLC, King of Prussia, PA) with placebo (5% albumin). On days 1, 2, and 21, IVIg (1 g/kg) or placebo was given. The primary outcome measure was the change in muscle strength from baseline to day 42, using the average muscle score (AMS). Secondary outcome measures included change from baseline AMS at days 10 and 21, the Hughes' functional disability scale, forced vital capacity (FVC), and nerve conduction studies (NCS) of four motor nerves (median, ulnar, peroneal, and tibial). RESULTS: The patients (n = 33) were randomized. Of these, 30 (14 women, 16 men, aged 54 +/- 20 years, range 13 to 82) received IVIg and 23 were given placebo (12 women, 11 men, aged 50 +/- 18 years, range 23 to 73). Baseline AMS values of the groups were similar (IVIg 7.06 +/- 1.31 versus placebo 7.28 +/- 1.18, p = 0.53). There were two dropouts in placebo group and one in the IVIg group. Mean AMS improved at day 42 comparing IVIg with placebo (0.63 versus -0.1, p = 0.006). Improved strength was seen by day 10. The placebo group lost strength over this same interval. In the IVIg, 11 subjects improved by the functional disability scale; none worsened. This differed (p = 0.019) from those in the placebo-treated group (two improved, two got worse, remainder unchanged). Forced vital capacity did not improve with IVIg treatment. IVIg improved ulnar motor distal latency (p = 0.005), tibial distal compound muscle amplitude (p = 0.003), and peroneal nerve conduction velocity (p = 0.03). CONCLUSIONS: IVIg improves strength in patients with untreated CIDP by day 10 with continued benefit through day 42; more than one third improve by at least a functional grade on a disability scale. This study provides data supporting IVIg as the initial treatment for CIDP.     

Correlation of nerve ultrasound,electrophysiological, and clinical findings in post Guillain‐Barré syndrome

We aimed to correlate functional disability, electrophysiology, and nerve ultrasound in patients after Guillain‐Barré syndrome (GBS). Seventy‐five healthy controls and 41 post‐GBS patients (mean 3.4 years, SD ± 2.91 years after onset) underwent clinical, sonographic, and electrophysiological evaluation. Compared to healthy controls, the post‐GBS patients showed: (1) a mean Rasch‐built Overall Disability Scale score of 31.8 (SD ± 11.6), modified Rasch‐built fatigue severity scale score of 15.6 (SD ± 3.2), Medical Research Council sum score of 22 (SD ± 5.6); (2) electrophysiological signs of permanent axonal loss in the majority of the peripheral nerves; (3) sonographical evidence of higher cross‐sectional area values (CSA) of the ulnar (elbow, p < 0.001), radial (spiral groove, p < 0.001), tibial nerve (popliteal fossa, p < 0.001) and brachial plexus (supraclavicular space, p < 0.001). No correlation between sonographic and electrophysiological findings was found. Neither nerve ultrasound nor electrophysiology correlated with muscle strength, overall disability, and fatigue scale. Compared to healthy controls, post‐GBS patients had significant functional disability. Despite significant abnormalities in both electrophysiology and ultrasound compared to healthy controls, neither electrophysiology nor nerve ultrasound correlated with functional disability of these patients.     

Neuron-specific enolase and tau protein as neurobiochemical markers of neuronal damage are related to early clinical course and long-term outcome in acute ischemic stroke

OBJECTIVES: Analyses of neuron-specific enolase (NSE) and tau protein in patients with hyperacute ischemic stroke, their association with infarct volume, severity of the neurological deficit, the neurovascular status and functional outcome. PATIENTS AND METHODS: In 66 consecutive patients, serial venous blood samples were taken at 3, 6, 12, 18, 24, 48, 72, 96, and 120 h after stroke onset. The neurovascular status was assessed by repetitive extra- and transcranial duplex sonography. Neurological deficits were quantified by the NIH stroke scale, and functional outcome was assessed with the modified Rankin scale (mRS). RESULTS: After a first rise within 3 h, NSE decreased followed by a secondary increase until Day 5. Tau protein concentrations showed a continuous increase from admission onward. NSE and tau release were highly correlated with severity of neurological deficits and infarct volume (P = 0.001). NSE, but not tau protein, release was associated to the neurovascular status on admission. NSE and tau protein values were significantly correlated with the functional outcome at 3 months (P < 0.001). CONCLUSION: Release kinetics of NSE and tau protein are associated with patients' clinical deficits and infarct volume, and may be used as an additional predictor of the early course and functional outcome.     

Pre‐stroke use of beta‐blockers does not affect ischaemic stroke severity and outcome

Background and purpose: It is unclear whether pre‐stroke beta‐blockers use may influence stroke outcome. This study evaluates the independent effect of pre‐stroke use of beta‐blockers on ischaemic stroke severity and 3 months functional outcome. Methods: Pre‐stroke use of beta‐blockers was investigated in 1375 ischaemic stroke patients who had been included in two placebo‐controlled trials with lubeluzole. Stroke severity was assessed by either the National Institute of Health Stroke Scale (NIHSS) or the European Stroke Scale (ESS). A modified Rankin scale (mRS) score of >3 at 3 months was used as measure for the poor functional outcome. Results: Two hundred and sixty four patients were on beta‐blockers prior to stroke onset, and 105 patients continued treatment after their stroke. Pretreatment with beta‐blockers did not influence baseline stroke severity. There was no difference in stroke severity between nonusers and those on either a selective beta₁‐blocker or a non‐selective beta‐blocker. The likelihood of a poor outcome at 3 months was not influenced by pre‐stroke beta‐blocker use or beta‐blocker use before and continued after stroke onset. Conclusions: Pre‐stroke use of beta‐blockers does not appear to influence stroke severity and functional outcome at 3 months.     

Clinimetric evaluation of a new overall disability scale in immune mediated polyneuropathies

OBJECTIVES: To determine the validity, reliability, and responsiveness of a new overall disability sum score in immune mediated polyneuropathies. METHODS: Three impairment measures (MRC sum score, sensory sum score, grip strength (Vigorimeter)) and three disability scales (an overall disability sum score (ODSS), Hughes' functional scale (f score), Rankin scale) were assessed in a cross sectional group of 113 clinically stable patients (83 with Guillain-Barré syndrome, 22 with chronic inflammatory demyelinating polyneuropathy (CIDP), eight with a gammopathy related polyneuropathy). The ODSS was also used serially in 20 patients with recently diagnosed Guillain-Barré syndrome (n = 7) or CIDP (n = 13) and changing clinical conditions. Multiple regression studies were performed to compare the impact of impairment disturbances (independent variables) on the various disability scales (dependent variable). RESULTS: Moderate to good construct validity (stable group: Spearman's rank test (absolute values), r = 0.41-0.79; longitudinal group: multiple correlation coefficient, R = 0.69-0.89; p < 0.006 for all associations) and reliability (intraclass correlation coefficient, R = 0.90-0.95; p < 0.0001) were demonstrated for the ODSS. Its SRM values were high (> 0.8), indicating good responsiveness. Impairment measures accounted for a higher variance proportion of the ODSS compared with the f score and Rankin (R = 0.64 v 0.56 and 0.45, respectively). CONCLUSIONS: All clinimetric requirements were met by the overall (arm and leg) disability sum score in immune mediated polyneuropathies. Its use is therefore suggested in evaluating immune mediated polyneuropathies.     

Advantages of a modified scoring method for the Rush Video-Based Tic Rating Scale.

Previously, we published a video-based objective rating scale of tics that met reliability and validity criteria for measurement of five domains of tic disability. In the original form, the scale's metric properties did not permit internal comparison of each of the five domains of impairment and did not provide a total score for use as a primary outcome measure. In this study, we retained the original scale and videotape protocol but tested whether a modified scoring system corrected these limitations. The new scoring method rated assigned tic data to ratings of 0-4 on five disability categories: number of body areas, frequency of motor tics, frequency of phonic tics, severity of motor tics, and severity of phonic tics. The sums of these ratings yielded a total score of overall tic disability (0-20). In a series of 31 patients with Gilles de la Tourette syndrome, we assessed Spearman correlation coefficients for the old and new scoring systems as well as the correlation of the new ratings with the objectively derived sections of the Yale Global Tic Severity Scale (YGTSS), another valid and reliable scale used in clinical practice and research. For each domain, the rank order for the scores on the original scale was well retained in the new scores. Likewise, for each domain, ranking with the new scoring system correlated well with scores on the comparable objective item from the YGTSS. The new total score accurately captured the rank order of the combined five domains from the original scale and correlated well with the total objective motor plus phonic tic score from the YGTSS and the YGTSS Tourette Syndrome Overall Impairment Rating. These data demonstrate that the modified videotape-based scoring system retains the essential information gathered in the original Rush scale. The modification provides comparisons among the five assessed domains and a total objectively based disability score that can be used as a single outcome measure for assessing tic disability.     

Examining outcome measures in a clinical study of stroke

We investigated the relation between outcome and sample size for six selected stroke outcome measures to assist investigators in selecting end points for stroke studies. Data from a clinical trial of 167 stroke patients assessed shortly after admission to the hospital and 5 weeks later provided information on clinical, motor, and functional outcomes measured using a neurologic status scale, a stroke severity scale, the Fugl-Meyer Scale, the Barthel Index, and the activities of daily living and cognition subscales of the Level of Rehabilitation Scale. Data were examined using Pearson correlation coefficients and power analyses. All measures were significantly correlated. There was also substantial congruency between the subscales of a measure and its total score. The measures had variable efficiencies; the Barthel Index was the most efficient and therefore required the fewest subjects to identify a significant effect. These data suggest that careful consideration must be given to the choice of stroke outcome measures in terms of their numbers, interrelationships, and statistical properties, as these factors have important implications for the design, analysis, and conduct of clinical stroke studies.