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1.
Artigo em Inglês | MEDLINE | ID: mdl-34949141

RESUMO

Objective: Uniform data collection is fundamental for multicentre clinical trials. We aim to determine the variability, between ALS trial centers, in the prevalence of unexpected or implausible improvements in the revised ALS functional rating scale (ALSFRS-R) score, and its associations with individual patient and item characteristics.Methods: We used data from two multicentre studies to estimate the prevalence of an unexpected increase or implausible improvement in the ALSFRS-R score, defined as an increase of 5 points or more between two consecutive, monthly visits. For each patient with a 5-point or more increase, we evaluated the individual contribution of each ALSFRS-R item.Results: Longitudinal ALSFRS-R scores, originating from 114 trial centers enrolling a total of 1,240 patients, were analyzed. A 5-point or more increase in ALSFRS-R total score was found in 151 (12.2%) patients, with prevalence per study center ranging from 0% to 83%. Bulbar onset, faster disease progression at enrollment, and a lower ALSFRS-R score at baseline were associated with a sudden 5-point or more increase in the ALSFRS-R total score. ALSFRS-R items 2 (saliva), 9 (stairs), 10 (dyspnea), and 11 (orthopnea) were the primary drivers when a 5-point or more increase occurred.Conclusions: Sudden 5-point or more increases in ALSFRS-R total scores between two consecutive visits are relatively common. These sudden increases were not found to occur with equal frequency in trial centers; which underscores the need for amending existing standard operating procedures toward a universal version and monitoring of data quality during the study, in multicentre research.

2.
Muscle Nerve ; 63(5): 678-682, 2021 05.
Artigo em Inglês | MEDLINE | ID: mdl-33501670

RESUMO

INTRODUCTION: In multifocal motor neuropathy (MMN), knowledge about the pattern of treatment response in a wide spectrum of muscle groups, distal as well as proximal, after intravenous immunoglobulin (IVIg) initiation is lacking. METHODS: Hand-held dynamometry data of 11 upper and lower limb muscles, from 47 patients with MMN was reviewed. Linear mixed models were used to determine the treatment response after IVIg initiation and its relationship with initial muscle weakness. RESULTS: All muscle groups showed a positive treatment response after IVIg initiation. Changes in SD scores ranged from +0.1 to +0.95. A strong association between weakness at baseline and the magnitude of the treatment response was found. DISCUSSION: Improved muscle strength in response to IVIg appears not only in distal, but to a similar degree also in proximal muscle groups in MMN, with the largest response in muscle groups that show the greatest initial weakness.


Assuntos
Imunoglobulinas Intravenosas/uso terapêutico , Força Muscular/efeitos dos fármacos , Debilidade Muscular/tratamento farmacológico , Polineuropatias/tratamento farmacológico , Adulto , Feminino , Humanos , Imunoglobulinas Intravenosas/administração & dosagem , Masculino , Pessoa de Meia-Idade , Força Muscular/fisiologia , Dinamômetro de Força Muscular , Debilidade Muscular/fisiopatologia , Polineuropatias/fisiopatologia , Resultado do Tratamento
3.
J Neurol ; 268(5): 1738-1746, 2021 May.
Artigo em Inglês | MEDLINE | ID: mdl-33355879

RESUMO

BACKGROUND: We aimed to determine (1) the test-retest reliability of a newly developed portable fixed dynamometer (PFD) as compared to the hand-held dynamometer (HHD) in patients with motor neuron disease (MND) and (2) the PFD's ability to reduce possible examiner-induced ceiling effects. METHODS: Test-retest reliability of isometric muscle strength of the quadriceps was measured in patients with MND and non-neurological controls using the HHD and PFD. Reliability was estimated by the intraclass correlation coefficient (ICC) and standard error of measurement (SEM) using linear mixed effects models, and the Bland-Altman method of agreement. RESULTS: In total, 45 patients with MND and 43 healthy controls were enrolled in this study. The ICC of the PFD was excellent and similar in both patients and controls (ICC Patients 99.5% vs. ICC Controls 98.6%) with a SEM of 6.2%. A strong examiner-induced ceiling effect in HHD was found when the participant's strength exceeded that of examiner. Employing the PFD increased the range of muscle strength measurements across individuals nearly twofold from 414 to 783 N. CONCLUSIONS: Portable fixed dynamometry may significantly reduce examiner-induced ceiling effects, optimize the standardization of muscle strength testing, and maximize reliability. Ultimately, PFD may improve the delivery of care due to its potential for unsupervised, home-based assessments and reduce the burden to the patient of participating in clinical trials for MND or other neuromuscular diseases.


Assuntos
Contração Isométrica , Doença dos Neurônios Motores , Humanos , Força Muscular , Dinamômetro de Força Muscular , Reprodutibilidade dos Testes
5.
J Neurol ; 266(11): 2734-2742, 2019 Nov.
Artigo em Inglês | MEDLINE | ID: mdl-31325017

RESUMO

OBJECTIVE: The primary aim was to determine the safety of treatment with human immune globulin 10% with recombinant human hyaluronidase (fSCIg) compared to intravenous immunoglobulin (IVIg) in a prospective open-label study in patients with multifocal motor neuropathy (MMN). METHODS: Our study consisted of two phases: the IVIg phase (visits 1-3; 12 weeks), in which patients remained on IVIg treatment, and the fSCIg phase (visits 4-7; 36 weeks), in which patients received fSCIg treatment. After visit 3, IVIg was switched to an equivalent dose and frequency of fSCIg. Outcome measures were safety, muscle strength, disability and treatment satisfaction. RESULTS: Eighteen patients were enrolled in this study. Switching to fSCIg reduced the number of systemic adverse events (IVIg 11.6 vs. fSCIg 5.0 adverse events/per person-year, p < 0.02), and increased the number of local reactions at the injection site (IVIg 0 vs. fSCIg 3.3 local reactions/per person-year, p < 0.01). Overall, no significant differences in muscle strength and disability between fSCIg and IVIg were found. Treatment with fSCIg was perceived as optimal treatment option by 8 of the 17 patients (47.1%) and they continued with fSCIg after study closure because of improved independence and flexibility to administer treatment. CONCLUSION: Treatment with fSCIg can be considered a safe alternative for patients with MMN on IVIg treatment. fSCIg could be a favorable option in patients who prefer self-treatment and more independency, and in patients who experience systemic adverse events on IVIg or have difficult intravenous access.


Assuntos
Hialuronoglucosaminidase/uso terapêutico , Imunoglobulinas Intravenosas/uso terapêutico , Polirradiculoneuropatia/tratamento farmacológico , Adulto , Idoso , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Força Muscular/efeitos dos fármacos , Polineuropatias/tratamento farmacológico , Proteínas Recombinantes/uso terapêutico , Resultado do Tratamento
6.
Artigo em Inglês | MEDLINE | ID: mdl-31271047

RESUMO

Objective: Spirometry is commonly used as screening tool for respiratory insufficiency in neuromuscular diseases. Despite the well-known effects of reference standards on spirometric outcomes, its standardization is overlooked in current guidelines. We aim to illustrate the effect of spirometric reference values on prognostication, medical decision-making, and trial eligibility in the applied setting of amyotrophic lateral sclerosis (ALS). Methods: We selected 4,651 patients with 32,022 FVC measurements from the PRO-ACT dataset. The FVC estimates were standardized according to five reference standards: Knudson '76, Knudson '83, ECSC, NHANES III, and GLI-2012. (Generalized) linear mixed-effects and Cox proportional hazard models were used to evaluate longitudinal patterns and time-to-event outcomes. Results: The mean population %predicted FVC varied between 78.5% (95% CI 78.0-79.1) and 88.5% (95% CI 87.9-89.1). The unstandardized liters provided the worst fit on the survival data (AIC 20573, c-index 0.760), whereas the GLI provided the best fit (AIC 20374, c-index 0.780, p < 0.001). The mean population rate of decline in %predicted FVC could vary as much as 11.4% between reference standards. The median time-to-50% predicted FVC differed by 2.9 months between recent (14.5 months, 95% CI 14.4-16.1) and early reference standards (17.4 months, 95% CI 16.1-18.2). Conclusion: Independent of technique, device, or evaluator, spirometric reference values affect the utility of spirometry in ALS. Standardization of reference values is of the utmost importance to optimize clinical decision-making, improve prognostication, enhance between-center comparison and unify patient selection for clinical trials.


Assuntos
Esclerose Amiotrófica Lateral , Seleção de Pacientes , Esclerose Amiotrófica Lateral/complicações , Esclerose Amiotrófica Lateral/diagnóstico , Esclerose Amiotrófica Lateral/terapia , Feminino , Humanos , Masculino , Inquéritos Nutricionais , Valores de Referência , Insuficiência Respiratória/etiologia , Insuficiência Respiratória/fisiopatologia , Espirometria/métodos , Capacidade Vital/fisiologia
7.
J Neurol ; 266(10): 2387-2395, 2019 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-31187191

RESUMO

BACKGROUND: The extensive heterogeneity between patients with amyotrophic lateral sclerosis (ALS) complicates the quantification of disease progression. In this study, we determine the value of remote, accelerometer-based monitoring of physical activity in patients with ALS. METHODS: This longitudinal cohort study was conducted in a home-based setting; all study materials were sent by mail. Patients wore the ActiGraph during waking hours for 7 days every 2-3 months and provided information regarding their daily functioning (ALSFRS-R). We defined four accelerometer-based endpoints that either reflect the average daily activity or quantify the patient's physical capacity. RESULTS: A total of 42 patients participated; the total valid monitoring period was 9288 h with a 93.0% adherence rate. At baseline, patients were active 27.9% (range 11.6-52.4%) of their time; this declined by 0.64% (95% 0.43-0.86, p < 0.001) per month. Accelerometer-based endpoints were strongly associated with the ALSFRS-R (r 0.78, 95% CI 0.63-0.92, p < 0.001), but showed less variability over time than the ALSFRS-R (coefficient of variation 0.64-0.81 vs. 1.06, respectively). Accelerometer-based endpoints could reduce sample size by 30.3% for 12-month trials and 44.6% for 18-month trials; for trials lasting less than 9 months, the ALSFRS-R resulted in smaller sample sizes. CONCLUSION: Accelerometry is an objective method for quantifying disease progression, which could obtain real-world insights in the patient's physical functioning and may personalize the delivery of care. In addition, remote monitoring provides patients with the opportunity to participate in clinical trials from home, paving the way to a patient-centric clinical trial model.


Assuntos
Acelerometria/normas , Esclerose Amiotrófica Lateral/diagnóstico , Esclerose Amiotrófica Lateral/fisiopatologia , Progressão da Doença , Exercício Físico , Monitorização Ambulatorial/normas , Idoso , Exercício Físico/fisiologia , Feminino , Humanos , Estudos Longitudinais , Masculino , Pessoa de Meia-Idade , Reprodutibilidade dos Testes
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