Assessing clinically meaningful treatment effects in controlled trials: Chronic migraine as an example

David W. Dodick, Catherine C. Turkel, Ronald E. Degryse, Hans Christoph Diener, Richard B. Lipton, Sheena K. Aurora, Marissa E. Nolan, Stephen D. Silberstein

Research output: Contribution to journalArticle

19 Scopus citations

Abstract

In addition to headache, persons with chronic migraine (CM) experience multiple symptoms, both ictal and interictal, that may contribute to their suffering. Translating clinical trial results into practice requires assessment of the results' clinical meaningfulness. When examining treatment benefit in this disabled patient population, multiple headache-symptom measures should be considered to fully reflect clinical relevance. Currently, only onabotulinumtoxinA is approved specifically for headache prophylaxis in adults with CM. Topiramate is the only other therapeutic agent with double-blind, placebo-controlled evidence in this population. Herein we evaluate the clinical meaningfulness of onabotulinumtoxinA and topiramate as headache prophylaxis in CM by comparing primary endpoints from the placebo-controlled, double-blind phase of the Phase 3 REsearch Evaluating Migraine Prophylaxis Therapy (PREEMPT) clinical program and the topiramate clinical trial (frequency of headache days [primary endpoint in PREEMPT; secondary in topiramate trial] and migraine/migrainous days [primary in topiramate trial, or "migraine/probable-migraine days"; secondary in PREEMPT]). Additionally, outcome measures such as responder rates, health-related quality of life, discontinuation rates, safety, and tolerability profiles are important clinical considerations. The clinical data indicate that statistically significant, clinically relevant treatment benefits exist for both onabotulinumtoxinA and topiramate. These data support these treatments as meaningful headache prophylaxis in adults with CM. Perspective CM is a chronic pain condition. We sought to determine the clinical relevance of recent trials in this disabled population. Clinical data indicate that statistically significant, clinically relevant treatment benefits exist for both onabotulinumtoxinA and topiramate, and support use of these treatments as meaningful headache prophylaxis in CM.

Original languageEnglish (US)
Pages (from-to)164-175
Number of pages12
JournalJournal of Pain
Volume16
Issue number2
DOIs
StatePublished - Feb 1 2015

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Keywords

  • Chronic migraine
  • clinical meaningfulness
  • onabotulinumtoxinA
  • prophylactic treatment
  • topiramate

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology
  • Anesthesiology and Pain Medicine

Cite this

Dodick, D. W., Turkel, C. C., Degryse, R. E., Diener, H. C., Lipton, R. B., Aurora, S. K., Nolan, M. E., & Silberstein, S. D. (2015). Assessing clinically meaningful treatment effects in controlled trials: Chronic migraine as an example. Journal of Pain, 16(2), 164-175. https://doi.org/10.1016/j.jpain.2014.11.004