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Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration Volume 19, 2018 - Issue 3-4
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Research Article

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

Iyas Daghlas1 Harvard Medical School, Boston, MA, USA, Correspondence[email protected]
ORCID Iconhttps://orcid.org/0000-0001-8735-284X
ORCID Icon,
Teresa E. Lever2 Department of Otolaryngology-Head and Neck Surgery, University of Missouri School of Medicine, Columbia, MO, USA, and
&
Emily Leary3 Department of Orthopaedic Surgery, University of Missouri School of Medicine, Columbia, MO, USAORCID Iconhttps://orcid.org/0000-0003-0836-7440
ORCID Icon
Pages 206-211 | Received 02 May 2017, Accepted 02 Dec 2017, Published online: 20 Dec 2017

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. BDNF ALS study group (Phase III). J Neurol Sci. 1999;169:13–21.
  • Gordon PH, Miller RG, Moore DH. ALSFRS-R. Amyotroph Lateral Scler Other Motor Neuron Disord. 2004;5(Suppl 1):90–3.
  • Kimura F, Fujimura C, Ishida S, Nakajima H, Furutama D, Uehara H, et al. Progression rate of ALSFRS-R at time of diagnosis predicts survival time in ALS. Neurology. 2006;66:265–7.
  • Atassi N, Berry J, Shui A, Zach N, Sherman A, Sinani E, et al. The PRO-ACT database: design, initial analyses, and predictive features. Neurology. 2014;83:1719–25.
  • Kahan BC, Jairath V, Doré CJ, Morris TP. The risks and rewards of covariate adjustment in randomized trials: an assessment of 12 outcomes from 8 studies. Trials. 2014;15:139.
  • Yoo W, Mayberry R, Bae S, Singh K, Peter He Q, Lillard JW. Jr. A study of effects of multicollinearity in the multivariable analysis. Int J Appl Sci Technol. 2014;4:9–19.
  • 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.
  • 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.
  • Meininger V, Drory VE, Leigh PN, Ludolph A, Robberecht W, Silani V. Glatiramer acetate has no impact on disease progression in ALS at 40 mg/day: a double- blind, randomized, multicentre, placebo-controlled trial. Amyotroph Lateral Scler. 2009;10:378–83.
  • 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. Ann Neurol. 2009;66:227–34.
  • Gordon PH, Cheng B, Salachas F, Pradat PF, Bruneteau G, Corcia P, et al. Progression in ALS is not linear but is curvilinear. J Neurol. 2010;257:1713–17.
  • Mandrioli J, Biguzzi S, Guidi C, Sette E, Terlizzi E, Ravasio A, et al. Heterogeneity in ALSFRS-R decline and survival: a population-based study in Italy. Neurol Sci. 2015;36:2243–52.
  • Watanabe H, Atsuta N, Nakamura R, Hirakawa A, Watanabe H, Ito M, et al. Factors affecting longitudinal functional decline and survival in amyotrophic lateral sclerosis patients. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16:230–6.
  • Curran PJ, Obeidat K, Losardo D. Twelve frequently asked questions about growth curve modeling. J Cogn Dev. 2010;11:121–36.
  • Bates D, Maechler M, Bolker B, Walker S. Fitting linear mixed-effects models using lme4. J Stat Soft. 2015;67:1–48.
  • Alexandra K, Per B, Rune Haubo Bojesen C. (2016). lmerTest: Tests in linear mixed effects models. R package version 2.0–32. https://CRAN.R-project.org/package=lmerTest
  • Proudfoot M, Jones A, Talbot K, Al-Chalabi A, Turner MR. The ALSFRS as an outcome measure in therapeutic trials and its relationship to symptom onset. Amyotroph Lateral Scler Frontotemporal Degener. 2016;17:414–25.
  • Chiò A, Canosa A, Gallo S, Cammarosano S, Moglia C, Fuda G, et al. ALS clinical trials: do enrolled patients accurately represent the ALS population? Neurology. 2011;77:1432–7.
  • Heeren T, D'Agostino R. Robustness of the two independent samples t-test when applied to ordinal scaled data. Statist Med. 1987;6:79–90.
  • Vickers AJ. Comparison of an ordinal and a continuous outcome measure of muscle soreness. Int J Technol Assess Health Care. 1999;15:709–16.
  • Carifio J, Perla R. Resolving the 50-year debate around using and misusing likert scales. Med Educ. 2008;42:1150–2.
  • Norman G. Likert scales, levels of measurement and the “laws” of statistics. Adv Health Sci Educ Theory Pract. 2010;15:625–32.
  • Wulfsohn MS, Tsiatis AA. A joint model for survival and longitudinal data measured with error. Biometrics. 1997;53:330–9.
  • Gonzalez-Bermejo J, Morélot-Panzini C, Tanguy ML, Meininger V, Pradat PF, Lenglet T. Early diaphragm pacing in patients with amyotrophic lateral sclerosis (RespiStimALS): a randomised controlled triple-blind trial. Lancet Neurol. 2016;15:1217–27.

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