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The minimally important difference and patient acceptable symptom state for the Raynaud's condition score in patients with Raynaud's phenomenon in a large randomised controlled clinical trial.

ABSTRACT: The Raynaud's condition score (RCS) is a validated outcome measure for Raynaud's phenomenon (RP).To assess the minimally important difference (MID) and patient acceptable symptom state (PASS) for RCS in patients with RP.and methods Patients with active RP (n=162) (mean RCS >25 (0-100 visual analogue scale) participated in a placebo-controlled, crossover randomised clinical trial (RCT). Data from the two treatment groups were combined for this analysis. Retrospective and prospective anchors were administered during the RCT. MID groups were defined as the group who reported being somewhat better (anchor #1) and a one-step change from "unbearable" to "very severe", etc (anchor #2). Patients were considered to have achieved PASS if they rated their Raynaud's condition as "very mild" or "mild" at the last study visit.The mean age of participants was 48.9 years and the mean baseline RCS was 46.4 points. The RCS change score for the MID improvement group ranged from -13.9 to -14.3 points and PASS estimate was 34.0 points.The MID and PASS estimates for RCS are 14-15 points for improvement and 34 points, respectively, on a 0-100 scale in a large RCT of patients with active RP. This information can aid in interpreting RCS in future RP trials.

SUBMITTER: Khanna PP

PROVIDER: S-EPMC2837770 | biostudies-literature | 2010 Mar

REPOSITORIES: biostudies-literature

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The minimally important difference and patient acceptable symptom state for the Raynaud's condition score in patients with Raynaud's phenomenon in a large randomised controlled clinical trial.

Khanna Puja P PP Maranian Paul P Gregory Jeff J Khanna Dinesh D

Annals of the rheumatic diseases 20090412 3

<h4>Background</h4>The Raynaud's condition score (RCS) is a validated outcome measure for Raynaud's phenomenon (RP).<h4>Objective</h4>To assess the minimally important difference (MID) and patient acceptable symptom state (PASS) for RCS in patients with RP.<h4>Subjects</h4>and methods Patients with active RP (n=162) (mean RCS >25 (0-100 visual analogue scale) participated in a placebo-controlled, crossover randomised clinical trial (RCT). Data from the two treatment groups were combined for this ...[more]

PMID: 19364728

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Project description:BackgroundThe results of clinical trials should be assessed for both statistical significance and importance of observed effects to patients. Minimal important difference (MID) is a threshold denoting a difference that is important to patients. Patient acceptable symptom state (PASS) is a threshold above which patients feel well.ObjectiveTo determine MID and PASS for common outcome instruments in patients with subacromial pain syndrome (SAPS).MethodsWe used data from the FIMPACT trial, a randomised controlled trial of treatment for SAPS that included 193 patients. The outcomes were shoulder pain at rest and on arm activity, both measured with the 0-100?mm visual analogue scale (VAS), the Constant-Murley score (CS), and the Simple Shoulder Test (SST). The transition question was a five-point global rating of change. We used three anchor-based methods to determine the MID for improvement: the receiver operating characteristic (ROC) curve, the mean difference of change and the mean change methods. For the PASS, we used the ROC and 75th percentile methods and calculated estimates using two different anchor question thresholds.ResultsDifferent MID methods yielded different estimates. The ROC method yielded the smallest estimates for MID: 20?mm for shoulder pain on arm activity, 10 points for CS and 1.5 points for SST, with good to excellent discrimination (areas under curve (AUCs) from 0.86 to 0.94). We could not establish a reliable MID for pain at rest. The PASS estimates were consistent between methods. The ROC method PASS thresholds using a conservative anchor question threshold were 2?mm for pain at rest, 9?mm for pain on activity, 80 points for CS and 11 points for SST, with AUCs from 0.74 to 0.83.ConclusionWe recommend the smallest estimate from different methods as the MID, because it is very unlikely that changes smaller than the smallest MID estimate are important to patients: 20?mm for pain VAS on arm activity, 10 points for CS and 1.5 points for SST. We recommend PASS estimates of 9?mm for pain on arm activity, 80 points for CS, and 11 points for SST.Trial registrationClinicalTrials.gov NCT00428870 (first registered January 29, 2007).

Project description:BackgroundMany different drugs have been suggested for the symptomatic treatment of primary Raynaud's phenomenon. Apart from calcium channel blockers, which are considered the drugs of choice, the evidence of the effects of alternative pharmacological treatments is limited. This is an update of a review first published in 2008.ObjectivesTo assess the effects of various drugs with vasodilator actions on primary Raynaud's phenomenon.Search methodsFor this update the Cochrane Peripheral Vascular Diseases Group Trials Search Co-ordinator searched the Specialised Register (last searched 14 May 2012), CENTRAL (Issue 4, 2012) and clinical trials databases. We contacted one pharmaceutical company and one trial author for additional information. In addition, the reference lists of relevant studies were searched for additional citations. There were no language restrictions.Selection criteriaRandomised controlled trials evaluating the effects of oral formulations of any drug with vasodilator effects on subjective symptoms in primary Raynaud's phenomenon. Treatment with, or comparison with, calcium channel blockers was not assessed in this review.Data collection and analysisTwo members of the review team independently assessed the trials for inclusion and their quality and extracted the data. Data extraction included adverse events. We contacted trial authors for missing data.Main resultsEight studies involving 290 participants were included. Two trials examined the effects of captopril, the rest were single trials on single drugs. All comparisons were with placebo. The methodological quality of most trials was poor.Enalapril was associated with a small increase in the frequency of attacks per week (difference in means 0.8; 95% CI 0.43 to 1.17). The difference between the intervention groups on a subjective improvement score was non-significant. There was a significant effect of buflomedil on the frequency of attacks per week (weighted mean difference (WMD) -8.8; 95% CI -17.55 to -0.09), but there was no evidence of effect on the severity score. The proportion with fewer attacks was significantly higher on moxisylyte than on placebo (relative risk (RR) 4.33; 95% CI 1.36 to 13.81). For captopril, beraprost, dazoxiben and ketanserin there was no evidence of an effect on the frequency, severity or duration of attacks. Beraprost and moxisylyte gave significantly more adverse effects than placebo.Authors' conclusionsPoor methodological quality, small sample sizes and the limited data available resulted in low precision of the statistical results and limited value of the overall results .The overall results show that there is no evidence for an effect of vasodilator drugs on primary Raynaud's phenomenon.

Project description:BackgroundSymptom assessment requires psychometrically validated questionnaires that are easy to use, relevant to the disease, and quick to administer. The MD Anderson Symptom Inventory for malignant pleural mesothelioma (MDASI-MPM) was adapted from the general (core) MDASI to assess the severity of cancer-related and treatment-related symptoms specific to patients with this condition. The MDASI-MPM includes the 13 core MDASI symptoms, which are experienced by most cancer patients, and 6 MPM-specific items developed via qualitative interviewing, a method favored by the US Food and Drug Administration for instrument item generation and development. Qualitative interviewing that summarizes the item generation and development for the MDASI-MPM is detailed in a separate report. The psychometric study reported here was the next step in developing the validation dossier for the MDASI-MPM.ResultsIn this secondary analysis of data from a Phase II trial, 248 patients provided MDASI-MPM data at multiple timepoints during therapy. Over time, fatigue, pain, shortness of breath, feeling of malaise, and muscle weakness were consistently the worst symptoms reported; symptoms interfered most with work and general activity and least with relations with others. Cronbach coefficient alpha values for all MDASI-MPM subscales were at least 0.88 at baseline and 0.91 during treatment, indicating good internal consistency reliability. Intraclass correlations of at least 0.86 for all MDASI-MPM subscales administered a cycle apart (n = 82) were indicative of good test-retest reliability. Correlations between MDASI-MPM subscales and LCSS-Meso scores were at least 0.70 (P < 0.001 for all comparisons). Patients with good performance status had significantly lower scores than did patients with poor performance status (all P < 0.05), supporting evidence for known-group validity and sensitivity. Effect-size differences were 0.69 and higher, indicating medium-to-large effects. The minimally important difference in the MDASI-MPM subscales ranged from 1.0 to 1.5 points on a 0-10 scale.ConclusionsSymptoms specific to a particular cancer, treatment method, or treatment site can be added to the core MDASI to create a tailored, "fit for purpose" instrument. We found the MDASI-MPM to be a valid, reliable, and responsive (sensitive) instrument for assessing the severity of symptoms of patients with MPM and their interference in patients' daily functioning.

Dataset Information

The minimally important difference and patient acceptable symptom state for the Raynaud's condition score in patients with Raynaud's phenomenon in a large randomised controlled clinical trial.

Publications

The minimally important difference and patient acceptable symptom state for the Raynaud's condition score in patients with Raynaud's phenomenon in a large randomised controlled clinical trial.

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