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Oral Disease-Modifying Treatments for Relapsing Multiple Sclerosis: A Likelihood to Achieve No Evidence of Disease Activity or Harm Analysis

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A Correction to this article was published on 20 December 2018

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Abstract

Background

The likelihood to help or harm (LHH) is an absolute measure of the benefit versus risk profile of a medication, which can be used to assess the potential for benefit versus harm of different disease-modifying treatments (DMTs) for relapsing multiple sclerosis (R-MS) and facilitate clinical decision-making.

Objective

The objective of this study was to assess absolute differences in benefit:risk ratios of oral DMTs for R-MS, using LHH analysis with no evidence of disease activity (NEDA) as beneficial outcome.

Design/Methods

The number needed to treat for a paient to achieve NEDA (NNTBNEDA) was used as an effect size metric of efficacy and the number needed to treat for a patient  to experience  an adverse event (NNTHAE), a serious adverse event (NNTHSAE), or treatment discontinuation due to an adverse event (NNTHAE-D) were used as measures of risk. The LHH—which is the ratio of NNTH:NNTB—values were calculated from published phase III trial data for oral DMTs.

Results

The values for likelihood to achieve NEDA than experience any AE ratio (LHH(AE/NEDA)) were 3.9, 6.8, 12.5 and 3.7, the likelihood to achieve NEDA than experience a SAE ratio (LHH(SAE/NEDA)) values were 3.5, 15, 23.5 and 2.8, and the likelihood to achieve NEDA versus discontinue treatment (LHH(AE-D/NEDA)) values were 20.3, 4.3, 3.9 and 3.1 for cladribine, dimethyl-fumarate, fingolimod, and teriflunomide, respectively.

Conclusions

With all of the oral DMTs examined, R-MS patients are more likely to achieve NEDA than experience any adverse event.

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Change history

  • 20 December 2018

    NEDA was evaluated as the proportion of patients free of relapses, 3-month confirmed disability progression, and free of new or newly enlarging T2 lesion and Gadolinium enhancing lesions.

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Acknowledgements

The authors thank Dr S.J. Kedikoglou, MD, MPH, PhD and Mrs. E. Delicha, MSc for their expert statistical advice.

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Authors and Affiliations

  1. Multiple Sclerosis Centre and Neurology Section, Athens Medical Centre - Paleo Phaliro Clinic, 175-62, Athens, Greece

    Dimitrios Papadopoulos

  2. 1st Neurology Department, Aeginition Hospital, Medical School, National and Kapodistrian University of Athens, 72-74 V. Sofia’s Avenue, 11528, Athens, Greece

    Dimos-Dimitrios D. Mitsikostas

Authors
  1. Dimitrios Papadopoulos
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Corresponding author

Correspondence to Dimos-Dimitrios D. Mitsikostas.

Ethics declarations

Ethical Approval and Consent

No ethics approval or patient consent was obtained because all data used in this study were collected from previously published peer-reviewed articles.

Conflict of interest

DP has received consulting, speaking fees, and travel grants from Bayer, Genesis Pharma, Merck, Novartis, Roche, Sanofi-Aventis, Specifar, and Teva. DDM has received consulting, speaking fees, and travel grants from Allergan, Amgen, Cefaly, ElectroCore, Genesis Pharma, Eli Lily, Merck-Serono, Novartis, Roche, Sanofi-Genzyme, Speficar, and Teva.

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No funding source had a role in the conception and preparation of this article.

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Papadopoulos, D., Mitsikostas, DD.D. Oral Disease-Modifying Treatments for Relapsing Multiple Sclerosis: A Likelihood to Achieve No Evidence of Disease Activity or Harm Analysis. CNS Drugs 32, 1069–1078 (2018). https://doi.org/10.1007/s40263-018-0547-z

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