Clinical significance in the change of decline in ALSFRS-R

@article{CastrilloViguera2010ClinicalSI,
  title={Clinical significance in the change of decline in ALSFRS-R},
  author={Carmen Castrillo‐Viguera and Daniela L. Grasso and Elizabeth Simpson and Jeremy M. Shefner and Merit E Cudkowicz},
  journal={Amyotrophic Lateral Sclerosis},
  year={2010},
  volume={11},
  pages={178 - 180}
}
Our objective was to survey ALS clinicians and researchers regarding what percentage reduction in the ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale – Revised) slope they would consider clinically meaningful. A nine-question survey was provided to 65 members of the Northeast ALS Consortium (NEALS). They were asked to rate the clinical relevance of 10–50% changes in decline of the ALSFRS-R slope on a seven-point scale (1–7), where 1=‘not at all clinically meaningful’, 4… 

A retrospective investigation of the relationship between baseline covariates and rate of ALSFRS-R decline in ALS clinical trials

The findings of this retrospective study, which are novel in the clinical trial time frame, contribute to the understanding of disease trajectory in ALS and can be used to guide future design and analysis of clinical trials.

Heterogeneity in ALSFRS-R decline and survival: a population-based study in Italy

It is confirmed that ALSFRS-R decline is not homogeneous among ALS patients and during the disease, and factors influencing this trend may not match with those affecting survival.

Qualitative measures that assess functional disability and quality of life in ALS

A literature search aimed at identifying both established and promising new measures of functional status for potential use in clinical trials found several newer qualitative scales including ALSFRS-EX, ALS-MITOS, CNS-BFS, DALS-15, MND-DS, and ROADS and serves as a reference guide for researchers deciding which qualitative measures to use as endpoints in their ALS clinical trials to assess functional status.

Relationships between slow vital capacity and measures of respiratory function on the ALSFRS-R

The higher correlation between %predicted SVC and specific ALSFRS-R symptom scores in patients with rapidly versus more slowly progressing disease reinforces the importance of continually monitoring respiratory function throughout the disease course.

Trajectories of impairment in amyotrophic lateral sclerosis: Insights from the Pooled Resource Open‐Access ALS Clinical Trials cohort

Variable curvilinearity of ALSFRS‐R trajectories confounds interpretation in clinical studies that assume linear decline and is better used as a multidimensional measure.

A qualitative evaluation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R) by the patient community: a web-based cross-sectional survey.

The revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) may need a revision to address issues, preferably in co-development with people living with ALS and their caregivers, and/or alternate outcome measures should be considered for patients with ALS.

Dexpramipexole effects on functional decline and survival in subjects with amyotrophic lateral sclerosis in a Phase II study: Subgroup analysis of demographic and clinical characteristics

The observed benefit of 300- vs. 50-mg dexpramipexole on functional decline and survival was generally consistent among subjects regardless of baseline characteristics.

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration

Evaluating how multidimensionality affects the performance of the ALSFRS-R in clinical trials proposes determining treatment benefit on a subscale level, prior to stating whether a treatment is generally effective.

Amyotrophic lateral sclerosis disease progression model

  • R. GomeniM. Fava
  • Psychology
    Amyotrophic lateral sclerosis & frontotemporal degeneration
  • 2014
A longitudinal model to describe amyotrophic lateral sclerosis disease progression using the revised Amyotrophic Lateral Sclerosis Functional Rating Scale and a probabilistic model to estimate the presence of clusters of trajectories in ALS progression over 12 months of treatment showed that the degree of ALS disease progression quantified by the ALSFRS-R symptomatic change on placebo is highly heterogeneous.

Exploring the diagnosis delay and ALS functional impairment at diagnosis as relevant criteria for clinical trial enrolment*

At time of inclusion in therapeutic trial closed to diagnosis, ΔFS or diagnosis delay may discriminate the rate of progression, according to diagnosis delay.
...

References

SHOWING 1-6 OF 6 REFERENCES

Performance of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials

Outcome measures for early phase clinical trials

The ALSFRS‐R most strongly predicted survival and provided the most complete data, but large changes may be necessary before patients perceive treatment effects, but all could be used in short‐duration, early phase trials.

Amyotrophic lateral sclerosis: a consensus viewpoint on designing and implementing a clinical trial

  • P. LeighM. Swash L. H. van den Berg
  • Medicine
    Amyotrophic lateral sclerosis and other motor neuron disorders : official publication of the World Federation of Neurology, Research Group on Motor Neuron Diseases
  • 2004
The panel recommended the use of the ALS Functional Rating Scale (ALSFRS‐R) to measure primary endpoints and a review of other key issues in this area including regional variations in the epidemiology, diagnosis and management of ALS.

Riluzole for amyotrophic lateral sclerosis (ALS)/motor neuron disease (MND).

  • R. MillerJ. MitchellM. LyonD. Moore
  • Medicine
    Amyotrophic lateral sclerosis and other motor neuron disorders : official publication of the World Federation of Neurology, Research Group on Motor Neuron Diseases
  • 2003
The efficacy of riluzole in prolonging survival, and in delaying the use of surrogates (tracheostomy and mechanical ventilation) to sustain survival, is examined.

Phase III randomized trial of gabapentin in patients with amyotrophic lateral sclerosis

Analysis of the combined data from the phase II and III trials revealed a significantly more rapid decline of forced vital capacity in patients treated with gabapentin.

The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function