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

References

• Cedarbaum JM, Stambler N, Malta E, Fuller C, Hilt D, Thurmond B, et al. The ALSFRS-R: a revised ALS functional rating scale that incorporates assessments of respiratory function. J Neurol Sci. 1999;169:13–21.
   PubMed Web of Science ®Google Scholar
• van Eijk RPA, de Jongh AD, Nikolakopoulos S, McDermott CJ, Eijkemans MJC, Roes KCB, et al. An old friend who has overstayed their welcome: the ALSFRS-R total score as primary endpoint for ALS clinical trials. Amyotroph Lateral Scler Frontotemporal Degener. 2021;22:300–7.
   PubMed Web of Science ®Google Scholar
• European Medicines Agency. Guideline on clinical investigation of medicinal products for the treatment of amyotrophic lateral sclerosis. London (UK): European Medicines Agency; 2016.
   Google Scholar
• US Food and Drug Administration. Guidance for industry: amyotrophic lateral sclerosis: developing drugs for treatment. Silver Spring (MD): US Food and Drug Administration; 2019.
   Google Scholar
• Kaufmann P, Levy G, Thompson JL, Delbene ML, Battista V, Gordon PH, et al. The ALSFRSr predicts survival time in an ALS clinic population. Neurology 2005;64:38–43.
   PubMed Web of Science ®Google Scholar
• Kollewe K, Mauss U, Krampfl K, Petri S, Dengler R, Mohammadi B. ALSFRS-R score and its ratio: a useful predictor for ALS-progression. J Neurol Sci. 2008;275:69–73.
   PubMed Web of Science ®Google Scholar
• @kelsieswrites. I think ALSFRS fails everyone. And I have no reason to believe the science won’t bear out here, in the end. Just need a bit more time to study how trial participants do longer term. Thankfully this trial has a very robust OLE and so the data is there for @biogen to do that. ❤️ [Twitter]. 2021 Available at: https://twitter.com/kelsieswrites/status/1449919426917126145.
   Google Scholar
• @alli_naquin. I am a pALS that has been excluded from drug trials due to “slow progression”. How do we transition away from ALSFRS as the sole primary endpoint? Is this trial design hurting or helping the search for treatments? [Twitter]. 2021 Available at: https://twitter.com/alli_naquin/status/1450484302932856834.
   Google Scholar
• Bacci ED, Staniewska D, Coyne KS, Boyer S, White LA, Zach N, et al. Item response theory analysis of the amyotrophic lateral sclerosis functional rating scale-revised in the pooled resource open-access ALS clinical trials database. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17:157–67.
   PubMed Web of Science ®Google Scholar
• Franchignoni F, Mandrioli J, Giordano A, Ferro S, ERRALS Group. A further Rasch study confirms that ALSFRS-R does not conform to fundamental measurement requirements. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16:331–7.
   PubMed Web of Science ®Google Scholar
• Rooney J, Burke T, Vajda A, Heverin M, Hardiman O. What does the ALSFRS-R really measure? A longitudinal and survival analysis of functional dimension subscores in amyotrophic lateral sclerosis. J Neurol Neurosurg Psychiatry. 2017;88:381–5.
   PubMed Web of Science ®Google Scholar
• Castrillo-Viguera C, Grasso DL, Simpson E, Shefner J, Cudkowicz ME. Clinical significance in the change of decline in ALSFRS-R. Amyotroph Lateral Scler. 2010;11:178–80.
   PubMed Web of Science ®Google Scholar
• Newton J, Jayaprakash K, Glasmacher SA, McEleney A, Bethell A, Fraser E, et al. Excellent reliability of the ALSFRS-R administered via videoconferencing: a study of people with motor neuron disease in Scotland. J Neurol Sci. 2020;416:116991.
   PubMed Web of Science ®Google Scholar
• Berry JD, Paganoni S, Carlson K, Burke K, Weber H, Staples P, et al. Design and results of a smartphone-based digital phenotyping study to quantify ALS progression. Ann Clin Transl Neurol. 2019;6:873–81.
   PubMed Web of Science ®Google Scholar
• Kasarskis EJ, Dempsey-Hall L, Thompson MM, Luu LC, Mendiondo M, Kryscio R. Rating the severity of ALS by caregivers over the telephone using the ALSFRS-R. Amyotroph Lateral Scler Other Motor Neuron Disord. 2005;6:50–4.
   PubMed Web of Science ®Google Scholar
• Bakker LA, Schroder CD, Tan HHG, Vugts S, van Eijk RPA, van Es MA, et al. Development and assessment of the inter-rater and intra-rater reproducibility of a self-administration version of the ALSFRS-R. J Neurol Neurosurg Psychiatry. 2020;91:75–81.
   PubMed Web of Science ®Google Scholar
• Montes J, Levy G, Albert S, Kaufmann P, Buchsbaum R, Gordon PH, et al. Development and evaluation of a self-administered version of the ALSFRS-R. Neurology 2006;67:1294–6.
   PubMed Web of Science ®Google Scholar
• Charmaz K. Constructing grounded theory: a practical guide through qualitative analysis. Thousand Oaks (CA): Sage Publications; 2006.
   Google Scholar
• Manera U, Cabras S, Daviddi M, Vasta R, Torrieri MC, Palumbo F, et al. Validation of the Italian version of self-administered ALSFRS-R scale. Amyotroph Lateral Scler Frontotemporal Degener. 2021;22:151–3.
   PubMed Web of Science ®Google Scholar
• Wicks P, Massagli MP, Wolf C, Heywood J. Measuring function in advanced ALS: validation of ALSFRS-EX extension items. Eur J Neurol. 2009;16:353–9.
   PubMed Web of Science ®Google Scholar
• Guedes K, Pereira C, Pavan K, Valerio BC. Cross-cultural adaptation and validation of als Functional Rating Scale-Revised in Portuguese language. Arq Neuropsiquiatr. 2010;68:44–7.
   PubMed Web of Science ®Google Scholar
• Maksymowicz S, Kukołowicz P, Siwek T, Rakowska A. Validation of the revised Amyotrophic Lateral Sclerosis Functional Rating Scale in Poland and its reliability in conditions of the medical experiment. Neurol Sci. 2021;42:943–9.
   PubMed Web of Science ®Google Scholar
• Rashed HR, Tork MA, Soliman R, Serag R, Fahmy N. Arabic adaptation and validation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R): Egyptian study. Amyotroph Lateral Scler Frontotemporal Degener. 2021;22:220–2.
   PubMed Web of Science ®Google Scholar
• Fournier CN, Bedlack R, Quinn C, Russell J, Beckwith D, Kaminski KH, et al. Development and validation of the Rasch-built overall amyotrophic lateral sclerosis disability scale (ROADS). JAMA Neurol. 2020;77:480–8.
   PubMed Web of Science ®Google Scholar
• Helleman J, Kruitwagen-van Reenen ET, Bakers J, Kruithof WJ, van Groenestijn AC, Jaspers Focks RJH, et al. Using patient-reported symptoms of dyspnea for screening reduced respiratory function in patients with motor neuron diseases. J Neurol. 2020;267:3310–8.
   PubMed Web of Science ®Google Scholar
• Smith RA, Macklin EA, Myers KJ, Pattee GL, Goslin KL, Meekins GD, et al. Assessment of bulbar function in amyotrophic lateral sclerosis: validation of a self-report scale (Center for Neurologic Study Bulbar Function Scale). Eur J Neurol. 2018;25:907–e66. Epub 2018 Apr 16. Erratum in: Eur J Neurol. 2018 Oct;25(10):1303.
   PubMed Web of Science ®Google Scholar