Item response theory analysis of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised in the Pooled Resource Open-Access ALS Clinical Trials Database

References

• Council MR. Aid to the investigation of peripheral nerve injuries. In: Medical Research Council. War memorandum. In: Office HMsS, editor. 2nd edn. London:1943. pp 11–46.
   Google Scholar
• Talakad NS, Pradhan C, Nalini A, Thennarasu K, Raju TR. Assessment of pulmonary function in amyotrophic lateral sclerosis. Indian J Chest Dis Allied Sci. 2009.
   Google Scholar
• Gordon PH, Corcia P, Lacomblez L, Pochigaeva K, Abitbol J-L, Cudkowicz M, et al. Defining survival as an outcome measure in amyotrophic lateral sclerosis. Archives of Neurology. 2009;66:758–61.
   PubMed Web of Science ®Google Scholar
• The Amyotrophic Lateral Sclerosis Functional Rating Scale. Assessment of activities of daily living in patients with amyotrophic lateral sclerosis. The ALS CNTF treatment study (ACTS) phase I-II Study Group. Arch Neurol. 1996;53:141–7.
   PubMed Web of Science ®Google Scholar
• 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. BDNF ALS Study Group (Phase III). Journal of the Neurological Sciences. 1999;169:13–21.
   PubMed Web of Science ®Google Scholar
• Aggarwal SP, Zinman L, Simpson E, McKinley J, Jackson KE, Pinto H, et al. Safety and efficacy of lithium in combination with riluzole for treatment of amyotrophic lateral sclerosis: a randomized, double-blind, placebo-controlled trial. Lancet Neurology. 2010;9:481–8.
   PubMed Web of Science ®Google Scholar
• Anagnostou E, Rentzos M, Alexakis T, Zouvelou V, Zambelis T, Evdokimidis I. Volume matters: the influence of different botulinum toxin-A dilutions for sialorrhea in amyotrophic lateral sclerosis. Muscle & Nerve. 2013;47:276–8.
   PubMed Web of Science ®Google Scholar
• Beghi E, Chio A, Inghilleri M, Mazzini L, Micheli A, Mora G, et al. A randomized controlled trial of recombinant interferon beta-1a in ALS. Italian Amyotrophic Lateral Sclerosis Study Group. Neurology. 2000;54:469–74.
   Google Scholar
• de Carvalho M, Pinto S, Costa J, Evangelista T, Ohana B, Pinto A. A randomized, placebo-controlled trial of memantine for functional disability in amyotrophic lateral sclerosis. Amyotrophic lateral sclerosis: official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases. 2010;11:456–60.
   PubMed Web of Science ®Google Scholar
• Piepers S, Veldink JH, de Jong SW, van der Tweel I, van der Pol WL, Uijtendaal EV, et al. Randomized sequential trial of valproic acid in amyotrophic lateral sclerosis. Annals of Neurology. 2009;66:227–34.
   PubMed Web of Science ®Google Scholar
• Rudnicki SA, Berry JD, Ingersoll E, Archibald D, Cudkowicz ME, Kerr DA, et al. 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. Amyotroph Lateral Scler Frontotemporal Degener. 2013;14:44–51.
   PubMed Web of Science ®Google Scholar
• Cedarbaum JM, Stambler N. Performance of the Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS) in multicenter clinical trials. Journal of the Neurological Sciences. 1997;152 (Suppl 1):S1–9.
   PubMed Web of Science ®Google Scholar
• Jenkinson C, Fitzpatrick R, Swash M, Peto V, Group A-HS. The ALS Health Profile Study: quality of life of amyotrophic lateral sclerosis patients and carers in Europe. Journal of Neurology. 2000;247:835–40.
   PubMed Web of Science ®Google Scholar
• Kaufmann P, Levy G, Montes J, Buchsbaum R, Barsdorf AI, Battista V, et al. Excellent inter-rater, intra-rater, and telephone-administered reliability of the ALSFRS-R in a multicenter clinical trial. Amyotrophic lateral sclerosis: official publication of the World Federation of Neurology Research Group on Motor Neuron Diseases. 2007;8:42–6.
   PubMed Web of Science ®Google Scholar
• Andrich D. Rasch models for measurement. Sage Publications; 1988.
   Google Scholar
• Samejima F. Estimation of a latent ability using a response pattern of graded scores. Psychometrika Monographs. Richmond, VA: Psychometric Society; 1969. Available from: http://psychometricsociety.org/sites/default/files/pdf/MN17.pdf.
   Google Scholar
• Lord FM. A theory of test score (Psychometric Monograph No. 7). Iowa City, IA: Psychometric Society. 1952.
   Google Scholar
• Franchignoni F, Mora G, Giordano A, Volanti P, Chio A. Evidence of multidimensionality in the ALSFRS-R Scale: a critical appraisal on its measurement properties using Rasch analysis. Journal of Neurology, Neurosurgery, and Psychiatry. 2013;84:1340–5.
   PubMed Web of Science ®Google Scholar
• Franchignoni F, Mandrioli J, Giordano A, Ferro S. A further Rasch study confirms that ALSFRS-R does not conform to fundamental measurement requirements. Amyotroph Lateral Scler Frontotemporal Degener. 2015(ahead of print):1–7.
   Web of Science ®Google Scholar
• Pooled Resource Open-Access ALS Clinical Trials Database. Data Sets. 2013 (December 18, 2013); Available from: https://nctu.partners.org/ProACT/Data.
   Google Scholar
• Brooks B. Amyotrophic lateral sclerosis clinimetric scales: guidelines for administration and scoring. Handbook of Clinical Neurological Scales New York: Demos Vermande. 1997:27–9.
   Google Scholar
• Muthén L, Muthén B. Mplus User’s Guide. Los Angeles, CA:1998–2004.
   Google Scholar
• Steiger JH. Understanding the limitations of global fit assessment in structural equation modeling. Personality and Individual Differences. 2007;42:893–8.
   Web of Science ®Google Scholar
• Yu CY. Evaluating cut-off criteria of model fit indices for latent variable models with binary and continuous outcomes. Doctoral dissertation; University of California, Los Angeles 2002.
   Google Scholar
• RUMM2030. Interpreting RUMM2030 - Part I: dichotomous items. Perth, Australia: RUMM Laboratory Pt Ltd; 2009.
   Google Scholar
• Cai L, Thissen D, S dT*. IRTPRO 2.1 for Windows: Item response theory for patient-reported outcomes. Skokie, IL: Scientific Software International, Inc; 2011.
   Google Scholar
• Embretson SE, Reise SP. Item response theory for psychologists: Psychology Press; 2000.
   Google Scholar
• Reise SP. The Emergence of Item Response Theory Models and the Patient Reported Outcomes Measurement Information Systems. Austrian J Statistics. 2009;38:211–20.
   Google Scholar
• Orlando M, Thissen D. Likelihood-based item-fit indices for dichotomous item response theory models. Applied Psychological Measurement. 2000;24:50–64.
   Web of Science ®Google Scholar
• Campos TS, Rodríguez-Santos F, Esteban J, Vázquez PC, Mora Pardina JS, Carmona AC. Spanish adaptation of the revised amyotrophic lateral sclerosis functional rating scale (ALSFRS-R). Amyotroph Lateral Scler. 2010;11:475–7.
   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. 2005;6:50–4.
   PubMed Web of Science ®Google Scholar
• Kyngdon A. Comparing factor analysis and the Rasch model for ordered response categories: an investigation of the scale of gambling choices. J Appl Meas. 2004;5:398–418.
   PubMedGoogle Scholar
• Food and Drug Administration (FDA). Guidance for industry on patient-reported outcome measures: use in medical product development to support labeling claims. Federal Register. 2009;74:65132–3.
   Google Scholar