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Two heads are better than one: benefits of joint models for ALS trials
  1. Ruben P A van Eijk1,2,
  2. James Rooney3,
  3. Orla Hardiman3,4,
  4. Leonard H van den Berg1
  1. 1 Neurology, Brain Center Rudolf Magnus, University Medical Center Utrecht, Utrecht, The Netherlands
  2. 2 Biostatistics & Research Support, Julius Center for Health Sciences and Primary Care, University Medical Center Utrecht, Utrecht, The Netherlands
  3. 3 Academic Unit of Neurology, Trinity Biomedical Sciences Institute, Trinity College Dublin, Dublin, Ireland
  4. 4 Neurology, Beaumont Hospital, Dublin, Ireland5
  1. Correspondence to Dr Ruben P A van Eijk, Neurology, Brain Center Rudolf Magnus, University Medical Center Utrecht, Utrecht 3584, The Netherlands; R.P.A.vanEijk-2{at}umcutrecht.nl

https://doi.org/10.1136/jnnp-2019-320553

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    Kaji et al recently conducted a phase II/III clinical trial to evaluate the safety and efficacy of methylcobalamin in 343 patients with amyotrophic lateral sclerosis (ALS).1 Both coprimary endpoints (survival and ALS Functional Rating Scale [ALSFRS-R]) failed to show significant benefit (p=0.19 and p=0.13, respectively). The choice for two coprimary endpoints is interesting and may have circumvented important pitfalls encountered by previous ALS trials.2 As ALS significantly reduces the patient’s life expectancy, evaluating a drug’s therapeutic potential to improve survival is fundamental. Survival time, however, may be influenced by life-extending interventions (eg, gastrostomy or tracheostomy) and provides little information about the patient’s functioning during life. The ALSFRS-R is a welcome alternative that is clinically relevant, easily obtained and highly predictive of survival time. Unfortunately, positive phase II results on the ALSFRS-R have until now …

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    Footnotes

    • Contributors RPAvE: study concept, design, analysis, interpretation of data and drafting the manuscript. JR: study concept, design, analysis, interpretation of data and drafting the manuscript. OH: study concept and critical revision of the manuscript for intellectual content. LHvdB: study concept and critical revision of the manuscript for intellectual content.

    • Funding This study was funded by the Netherlands ALS Foundation (grant number: TRICALS).

    • Competing interests None declared.

    • Patient consent for publication Not required.

    • Provenance and peer review Not commissioned; internally peer reviewed.