Investigating generalizability of results from a randomized controlled trial of the management of chronic widespread pain

the MUSICIAN study

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Abstract

The generalisability of randomised controlled trials will be compromised if markers of treatment outcome also affect trial recruitment. In a large trial of chronic widespread pain (CWP), we aimed to determine the extent to which randomised participants represented eligible patients, and whether factors predicting randomisation also influenced trial outcome. Adults from eight UK general practices were surveyed to determine eligibility for a trial of two interventions (exercise, and cognitive behavioural therapy (CBT)). Amongst those eligible, logistic regression identified factors associated with reaching the randomisation step in the recruitment process . The main trial analysis was recomputed, weighting for the inverse of the likelihood of reaching the randomisation stage , and the numbers needed to treat (NNT) were calculated for each treatment. 884 persons were identified as eligible for the trial, of whom 442 (50%) were randomised. Several factors were associated with the likelihood of reaching the randomisation stage: higher Body Mass Index (BMI) (odds ratio: 1.99; 0.85-4.61); more severe/disabling pain (1.90; 1.21-2.97); having a treatment preference (2.11; 1.48-3.00); and expressing positivity about interventions offered (exercise: 2.66; 1.95-3.62; CBT: 3.20; 2.15-4.76). Adjusting for this selection bias decreased the treatment effect associated with exercise and CBT but increased that observed for combined therapy. All were associated with changes in NNT. This has important implications for the design and interpretation of pain trials generally.
Original languageEnglish
Pages (from-to)96-102
Number of pages7
JournalPain
Volume158
Issue number1
Early online date29 Sep 2016
DOIs
Publication statusPublished - Jan 2017

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Random Allocation
Chronic Pain
Cognitive Therapy
Randomized Controlled Trials
Numbers Needed To Treat
Exercise
Pain
Selection Bias
Therapeutics
General Practice
Body Mass Index
Logistic Models
Odds Ratio

Keywords

  • chronic widespread pain
  • fibromyalgia
  • RCTs
  • methodology
  • external validity

Cite this

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title = "Investigating generalizability of results from a randomized controlled trial of the management of chronic widespread pain: the MUSICIAN study",
abstract = "The generalisability of randomised controlled trials will be compromised if markers of treatment outcome also affect trial recruitment. In a large trial of chronic widespread pain (CWP), we aimed to determine the extent to which randomised participants represented eligible patients, and whether factors predicting randomisation also influenced trial outcome. Adults from eight UK general practices were surveyed to determine eligibility for a trial of two interventions (exercise, and cognitive behavioural therapy (CBT)). Amongst those eligible, logistic regression identified factors associated with reaching the randomisation step in the recruitment process . The main trial analysis was recomputed, weighting for the inverse of the likelihood of reaching the randomisation stage , and the numbers needed to treat (NNT) were calculated for each treatment. 884 persons were identified as eligible for the trial, of whom 442 (50{\%}) were randomised. Several factors were associated with the likelihood of reaching the randomisation stage: higher Body Mass Index (BMI) (odds ratio: 1.99; 0.85-4.61); more severe/disabling pain (1.90; 1.21-2.97); having a treatment preference (2.11; 1.48-3.00); and expressing positivity about interventions offered (exercise: 2.66; 1.95-3.62; CBT: 3.20; 2.15-4.76). Adjusting for this selection bias decreased the treatment effect associated with exercise and CBT but increased that observed for combined therapy. All were associated with changes in NNT. This has important implications for the design and interpretation of pain trials generally.",
keywords = "chronic widespread pain, fibromyalgia, RCTs, methodology, external validity",
author = "Jones, {Gareth T.} and Jones, {Elizabeth A.} and Beasley, {Marcus J.} and Macfarlane, {Gary J.}",
note = "The MUSICIAN trial was supported by an award from Arthritis Research UK, Chesterfield, UK. Grant number: 17292. Ethical approval for the study was granted by Cheshire NHS Research Ethics Committee; reference number: 07/Q1506/61. All participants provided written consent.",
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AU - Macfarlane, Gary J.

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N2 - The generalisability of randomised controlled trials will be compromised if markers of treatment outcome also affect trial recruitment. In a large trial of chronic widespread pain (CWP), we aimed to determine the extent to which randomised participants represented eligible patients, and whether factors predicting randomisation also influenced trial outcome. Adults from eight UK general practices were surveyed to determine eligibility for a trial of two interventions (exercise, and cognitive behavioural therapy (CBT)). Amongst those eligible, logistic regression identified factors associated with reaching the randomisation step in the recruitment process . The main trial analysis was recomputed, weighting for the inverse of the likelihood of reaching the randomisation stage , and the numbers needed to treat (NNT) were calculated for each treatment. 884 persons were identified as eligible for the trial, of whom 442 (50%) were randomised. Several factors were associated with the likelihood of reaching the randomisation stage: higher Body Mass Index (BMI) (odds ratio: 1.99; 0.85-4.61); more severe/disabling pain (1.90; 1.21-2.97); having a treatment preference (2.11; 1.48-3.00); and expressing positivity about interventions offered (exercise: 2.66; 1.95-3.62; CBT: 3.20; 2.15-4.76). Adjusting for this selection bias decreased the treatment effect associated with exercise and CBT but increased that observed for combined therapy. All were associated with changes in NNT. This has important implications for the design and interpretation of pain trials generally.

AB - The generalisability of randomised controlled trials will be compromised if markers of treatment outcome also affect trial recruitment. In a large trial of chronic widespread pain (CWP), we aimed to determine the extent to which randomised participants represented eligible patients, and whether factors predicting randomisation also influenced trial outcome. Adults from eight UK general practices were surveyed to determine eligibility for a trial of two interventions (exercise, and cognitive behavioural therapy (CBT)). Amongst those eligible, logistic regression identified factors associated with reaching the randomisation step in the recruitment process . The main trial analysis was recomputed, weighting for the inverse of the likelihood of reaching the randomisation stage , and the numbers needed to treat (NNT) were calculated for each treatment. 884 persons were identified as eligible for the trial, of whom 442 (50%) were randomised. Several factors were associated with the likelihood of reaching the randomisation stage: higher Body Mass Index (BMI) (odds ratio: 1.99; 0.85-4.61); more severe/disabling pain (1.90; 1.21-2.97); having a treatment preference (2.11; 1.48-3.00); and expressing positivity about interventions offered (exercise: 2.66; 1.95-3.62; CBT: 3.20; 2.15-4.76). Adjusting for this selection bias decreased the treatment effect associated with exercise and CBT but increased that observed for combined therapy. All were associated with changes in NNT. This has important implications for the design and interpretation of pain trials generally.

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