Clinical significance in the change of decline in ALSFRS-R

Castrillo‐Viguera, Carmen; Grasso, D.; Simpson, Elizabeth; Shefner, Jeremy M.; Cudkowicz, Merit

doi:10.1080/17482960903093710

Cited by 10 publications

(12 citation statements)

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“…Bulbar onset patients comprised 35% of our population, and the average clinical deterioration of the ALSFRS-R at a rate of 0.92 points per month is in concert with the reported average functional decline in ALS patients 20 21. Therefore, our study population seems to be representative.…”

Section: Resultsmentioning

confidence: 90%

Tracking motor neuron loss in a set of six muscles in amyotrophic lateral sclerosis using the Motor Unit Number Index (MUNIX): a 15-month longitudinal multicentre trial

Neuwirth

Barkhaus

Burkhardt

et al. 2015

J Neurol Neurosurg Psychiatry

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Section: Resultsmentioning

confidence: 90%

Tracking motor neuron loss in a set of six muscles in amyotrophic lateral sclerosis using the Motor Unit Number Index (MUNIX): a 15-month longitudinal multicentre trial

Neuwirth

Barkhaus

Burkhardt

et al. 2015

J Neurol Neurosurg Psychiatry

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“…Understanding the development of ALSFRS over time is relevant for understanding disease progression. Reports vary regarding the linearity of ALSFRS-R measure over time [34,43,54,55], and these reports likely depend on number of patients, distribution of disease progression, and frequency of measuring ALSFRS-R.…”

Section: Understanding Als Symptom Profilementioning

confidence: 99%

“…For example, ALSFRS was, and ALSFRS-R is, the most common ALS end point or outcome measure currently used in clinical trials [52,54], and the most well-accepted tests of disease progression used in the clinic. Understanding the development of ALSFRS over time is relevant for understanding disease progression.…”

Section: Understanding Als Symptom Profilementioning

confidence: 99%

Being PRO-ACTive: What can a Clinical Trial Database Reveal About ALS?

et al. 2015

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Advancing research and clinical care, and conducting successful and cost-effective clinical trials requires characterizing a given patient population. To gather a sufficiently large cohort of patients in rare diseases such as amyotrophic lateral sclerosis (ALS), we developed the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) platform. The PRO-ACT database currently consists of >8600 ALS patient records from 17 completed clinical trials, and more trials are being incorporated. The database was launched in an open-access mode in December 2012; since then, >400 researchers from >40 countries have requested the data. This review gives an overview on the research enabled by this resource, through several examples of research already carried out with the goal of improving patient care and understanding the disease. These examples include predicting ALS progression, the simulation of future ALS clinical trials, the verification of previously proposed predictive features, the discovery of novel predictors of ALS progression and survival, the newly identified stratification of patients based on their disease progression profiles, and the development of tools for better clinical trial recruitment and monitoring. Results from these approaches clearly demonstrate the value of large datasets for developing a better understanding of ALS natural history, prognostic factors, patient stratification, and more. The increasing use by the community suggests that further analyses of the PRO-ACT database will continue to reveal more information about this disease that has for so long defied our understanding.

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“…Rating scales developed and validated in ALS include the Norris Scale, the Appel Scale, and the ALS Functional Rating Scale [220,221]. Decline in the ALSFRS-R score is generally linear, and consensus data on the clinical significance of change of decline in the ALSFRS-R have been published [224][225][226]. Decline in the ALSFRS-R score is generally linear, and consensus data on the clinical significance of change of decline in the ALSFRS-R have been published [224][225][226].…”

Section: Global Functional Assessmentmentioning

confidence: 99%