Treatment Recommendations Should Take Account of Individual Patient Variation Not Just Group Responses

2009 ◽  
Vol 27 (1) ◽  
pp. 31-32 ◽  
Author(s):  
Thomas Lundeberg ◽  
Iréne Lund

Recommendations for treatment are commonly based on results evaluating variation in systematic effects (group responses) from randomised controlled trials without taking the individual patient's variation into account. In the evaluation of acupuncture-related treatment effects, the trial design and statistical analysis used are a challenge since the assessed variables commonly have subjective properties and are based on the person's own self-report. Thus, the results that are seen are often varied, most likely due to inter-individual variation in rating of the actual variable such that the treatment effects are expressed more (or less) in some individuals than in others. The basis for the individual variation is probably multi-modal and could be related to the individuals’ expectation, gender, genetic polymorphisms and the aetiology of the condition. The assessment methods used should preferably have proven useful in controlled trials, and the methods for statistical analysis should consider the non-metric properties of the variable and the contribution of the individuals’ variation in the results. In order to evaluate the treatment effects more properly and increase the possibility of detecting any effectiveness, it is therefore important to assess the level of perceived dysfunction or symptom, taking into account the individual variation as well as the systematic effects (the effects of the group). In the evaluation of acupuncture effects, both systematic and individual variation should be reported allowing for the detection of subgroup effects and thereby leading to treatment recommendations that are more likely to be based on each individual's specific needs.

2006 ◽  
Vol 24 (3) ◽  
pp. 109-117 ◽  
Author(s):  
Iréne Lund ◽  
Thomas Lundeberg

Pain is a major clinical problem that causes great suffering for the individual and incurs costs for society. Accurate assessment and evaluation of perceived pain is necessary for diagnosis, for choice of treatment, and for the evaluation of treatment efficacy. The assessment of an individual's pain is a challenge since pain is a subjective, multidimensional experience, and assessment is based on the person's own self-report. The results are often varied, possibly due to inter-individual variation, but also in relation to gender and aetiology. A gold standard for pain assessment is still lacking, but rating scales, questionnaires, and methods derived from psychophysical concepts, such as threshold assessments and perceptual matching, are used. In the evaluation of pain and associated variables, both systematic and individual variation should be taken into account, as should pain-associated symptoms. Recommendations for pain treatment should be based on the patient's specific needs. Therefore, it is important to assess the level of perceived pain taking individual variation into account. The methods used should preferably have proved to be useful in randomised controlled trials, and analysis of pain assessment should consider its non-metric properties. In the future, the use of studies with a naturalistic protocol together with individual assessment of individual pain responses could increase the internal and external validity.


BMJ ◽  
2012 ◽  
Vol 344 (may18 1) ◽  
pp. e2809-e2809 ◽  
Author(s):  
E. A. Akl ◽  
M. Briel ◽  
J. J. You ◽  
X. Sun ◽  
B. C. Johnston ◽  
...  

2020 ◽  
Vol 3 ◽  
pp. 82
Author(s):  
Robert Murphy ◽  
Emer McGrath ◽  
Aoife Nolan ◽  
Andrew Smyth ◽  
Michelle Canavan ◽  
...  

Background: A run-in period is often employed in randomised controlled trials to increase adherence to the intervention and reduce participant loss to follow-up in the trial population. However, it is uncertain whether use of a run-in period affects the magnitude of treatment effect. Methods: We will conduct a sensitive search for systematic reviews of cardiovascular preventative trials and a complete meta-analysis of treatment effects comparing cardiovascular prevention trials using a run-in period (“run-in trials”) with matched cardiovascular prevention trials that did not use a run-in period (“non-run-in trials”). We describe a comprehensive matching process which will match run-in trials with non-run-in trials by patient populations, interventions, and outcomes. For each pair of run-in trial and matched non-run-in trial(s), we will estimate the ratio of relative risks and 95% confidence interval. We will evaluate differences in treatment effect between run-in and non-run-in trials and our and our priamry outcome will be the ratio of relative risks for matched run-in and non-run-in trials for their reported cardiovascular composite outcome. Our secondary outcomes are comparisons of mortality, loss to follow up, frequency of adverse events and methodological quality of trials. Conclusions: This study will answer a key question about what influence a run-in period has on the magnitude of treatment effects in randomised controlled trials for cardiovascular prevention therapies.


2020 ◽  
Vol 6 (4) ◽  
pp. 00246-2020
Author(s):  
Alexandra Jauhiainen ◽  
Lieke E.J.M. Scheepers ◽  
Anne L. Fuhlbrigge ◽  
Tim Harrison ◽  
James Zangrilli ◽  
...  

BackgroundCompEx Asthma, a novel composite end-point combining severe exacerbations (SevEx) with asthma-worsening events, was recently developed. Further characterisation of CompEx Asthma is needed to illustrate the applicability of this end-point. The objective was to evaluate CompEx Asthma as a rate end-point to determine how seasonal and geographical factors impact this novel outcome.MethodsSeven 24–56-week randomised controlled trials of budesonide/formoterol (BUD/FORM) and benralizumab were analysed. Annualised event rates (AERs) and treatment effects (hazard ratio (HR)) were analysed with Poisson and Andersen–Gill models, respectively. Seasonality was analysed by month and five geographical regions were evaluated.ResultsThe studies included 10 815 patients (63% female, mean age 42–49 years). CompEx Asthma AER mirrored seasonal variations in SevEx AER. CompEx Asthma AERs were higher versus SevEx in BUD/FORM and benralizumab trials (range 2.7–4.5-fold and 1.3–2.0-fold increase, respectively) and were less variable versus SevEx between regions (ratios of greatest:smallest AERs: 1.36 for CompEx versus 2.28 for SevEx (BUD/FORM); 1.81 for CompEx versus 2.22 for SevEx (benralizumab)). Treatment effects for CompEx Asthma and SevEx were generally similar across regions and months. However, in Eastern Europe, where SevEx rates were lowest, treatment effect was greater with CompEx Asthma versus SevEx, reaching statistical significance in the benralizumab studies (HR (95% CI): 0.67 (0.53–0.85) versus 0.87 (0.65–1.15)).ConclusionThis study confirmed the reliability of CompEx Asthma as a rate end-point and allowed detection of variations in seasonal SevEx rates, reduction of variation in rates across regions and potential greater sensitivity to treatment effects.


BMJ ◽  
2014 ◽  
Vol 348 (mar07 4) ◽  
pp. g1905-g1905
Author(s):  
P. Sedgwick ◽  
K. Joekes

Sign in / Sign up

Export Citation Format

Share Document