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Clinical Trials

ALS/SURV: a modification of the CAFS statistic

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Pages 576-583 | Received 30 Oct 2018, Accepted 07 Jul 2019, Published online: 23 Jul 2019

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.
  • Ensor H, Lee RJ, Sudlow C, Weir CJ. Statistical approaches for evaluating surrogate outcomes in clinical trials: a systematic review. J Biopharm Stat. 2016;26:859–79.
  • Huque MF, Alosh M, Bhore R. Addressing multiplicity issues of a composite endpoint and its components in clinical trials. J Biopharm Stat. 2011;21:610–34.
  • Berry JD, Miller R, Moore DH, et al. The Combined Assessment of Function and Survival (CAFS): a new endpoint for ALS clinical trials. Amyotroph Lateral Scler Frontotemporal Degener. 2013;14:162–8.
  • Cudkowicz ME, Titus S, Kearney M, Yu H, Sherman A, Schoenfeld D, et al. Safety and efficacy of ceftriaxone for amyotrophic lateral sclerosis: a multi-stage, randomised, double-blind, placebo-controlled trial. Lancet Neurol. 2014;13:1083–91.
  • Cudkowicz ME, van den Berg LH, Shefner JM, Mitsumoto H, Mora JS, Ludolph A, et al. Dexpramipexole versus placebo for patients with amyotrophic lateral sclerosis (EMPOWER): a randomised, double-blind, phase 3 trial. Lancet Neurol. 2013;12:1059–67.
  • Finkelstein DM, Schoenfeld DA. Combining mortality and longitudinal measures in clinical trials. Stat Med. 1999;18:1341–54.
  • Goutman SA, Brown MB, Glass JD, Boulis NM, Johe K, Hazel T, et al. Long-term Phase 1/2 intraspinal stem cell transplantation outcomes in ALS. Ann Clin Transl Neurol. 2018;5:730–40.
  • Spearman C. The proof and measurement of association between two things. Am J Psychol. 1904;15:72–101.
  • Plana-Ripoll O, Gomez G. Selecting the primary endpoint in a randomized clinical trial: the ARE method. J Biopharm Stat. 2016;26:880–98.
  • Rauch G, Beyersmann J. Planning and evaluating clinical trials with composite time-to-first-event endpoints in a competing risk framework. Stat Med. 2013;32:3595–608.
  • Tseng CH, Wong WK. Analysis of a composite endpoint with longitudinal and time-to-event data. Stat Med. 2011;30:1018–27.
  • Huang Z, Zhang H, Boss J, Goutman SA, Mukherjee B, Dinov ID, et al. Complete hazard ranking to analyze right-censored data: an ALS survival study. PLoS Comput Biol. 2017;13:e1005887.
  • Tang M, Gao C, Goutman SA, et al. Model-based and model-free techniques for amyotrophic lateral sclerosis diagnostic prediction and patient clustering. Neuroinformatics. 2019;17:407–21.
  • Elamin M, Bede P, Montuschi A, Pender N, Chio A, Hardiman O. Predicting prognosis in amyotrophic lateral sclerosis: a simple algorithm. J Neurol. 2015;262:1447–54.
  • Pfohl SR, Kim RB, Coan GS, Mitchell CS. Unraveling the complexity of amyotrophic lateral sclerosis survival prediction. Front Neuroinform. 2018;12:36.
  • van Eijk RP, Eijkemans MJ, Rizopoulos D, van den Berg LH, Nikolakopoulos S. Comparing methods to combine functional loss and mortality in clinical trials for amyotrophic lateral sclerosis. Clin Epidemiol. 2018;10:333–41.

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