Approaches and Recommendations for Estimating Minimally Important Differences for Health-Related Quality of Life Measures

Hays, Ron D.; Farivar, Sepideh S.; Liu, Honghu H.

doi:10.1081/copd-200050663

Cited by 206 publications

(208 citation statements)

References 11 publications

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“…The impact of a change by .1 point is considered significant on a 5-category scale. 33,34 HRQL data were analyzed for changes in mean HRQL score from pretransplant measurement and performed as an exploratory analysis, given the small sample size. A paired Student t test was used to assess changes from baseline to each posttransplant time point (day 100, 6 months, and 1 year).…”

Section: Discussionmentioning

confidence: 99%

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

Shenoy

Eapen

Panepinto

et al. 2016

Blood

174

158

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• Children with sickle cell disease engrafted unrelated donor marrow after reduced intensity conditioning.• A high incidence of GVHD and associated mortality compromised safety of the trial.Children with sickle cell disease experience organ damage, impaired quality of life, and premature mortality. Allogeneic bone marrow transplant from an HLA-matched sibling can halt disease progression but is limited by donor availability. A Blood and Marrow Transplant Clinical Trials Network (BMT CTN) phase 2 trial conducted from 2008 to 2014 enrolled 30 children aged 4 to 19 years; 29 were eligible for evaluation. The primary objective was 1-year event-free survival (EFS) after HLA allele-matched (at HLA-A, -B, -C, and -DRB1 loci) unrelated donor transplant. The conditioning regimen included alemtuzumab, fludarabine, and melphalan. Graft-versus-host disease (GVHD) prophylaxis included calcineurin inhibitor, short-course methotrexate, and methylprednisolone. Transplant indications included stroke (n 5 12), transcranial Doppler velocity >200 cm/s (n 5 2), ‡3 vaso-occlusive pain crises per year (n 5 12), or ‡2 acute chest syndrome episodes (n 5 4) in the 2 years preceding enrollment. Median follow-up was 26 months (range, 12-62 months); graft rejection was 10%. The 1-and 2-year EFS rates were 76% and 69%, respectively. The corresponding rates for overall survival were 86% and 79%. The day 100 incidence rate of grade II-IV acute GVHD was 28%, and the 1-year incidence rate of chronic GVHD was 62%; 38% classified as extensive. There were 7 GVHD-related deaths. A 34% incidence of posterior reversible encephalopathy syndrome was noted in the first 6 months. Although the 1-year EFS met the prespecified target of ‡75%, this regimen cannot be considered sufficiently safe for widespread adoption without modifications to achieve more effective GVHD prophylaxis.

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Section: Discussionmentioning

confidence: 99%

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

Shenoy

Eapen

Panepinto

et al. 2016

Blood

174

158

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“…The data source was the combined data analysis of two randomized, double-blind, placebo-controlled trials that assessed the efficacy and safety of an 16 Efficacy assessments used in this analysis included the QEQ (total score, transformed onto a 0 (worst) to 100 (best) scale) and the Erectile Function domain of the IIEF (score range, 1 (worst) to 30 (best)), which can be used to categorize ED by severity category as no ED (score X26), mild ED (22)(23)(24)(25), mild-tomoderate ED (17)(18)(19)(20)(21), moderate ED (11-16) and severe ED (1-10). 5,6 Analyses were based on observed mean (s.e., 95% confidence interval (CI)) QEQ total scores within the entire intent-to-treat population (patients within the active treatment group plus the placebo group who took at least one dose of study medication and who provided sufficient efficacy data for at least one efficacy analysis during that period) at baseline, end of treatment (week 12) and their difference (change scores).…”

Section: Methodsmentioning

confidence: 99%

“…Although anchor-based approaches to estimating MCMI are useful, 11,14,22 the validity of such an estimate depends on the strength of the correlation between the disease-related anchor and the PRO measure. 23 As reported previously for these data, improvement in IIEF Erectile Function domain scores showed moderate-to-high correlations with improvement in QEQ item scores (range, 0.52-0.73; Po0.0001) and with the QEQ total score (0.67; Po0.0001).…”

Section: Quality Of Erection Questionnaire K Hvidsten Et Almentioning

confidence: 99%

Clinically meaningful improvement on the quality of erection questionnaire in men with erectile dysfunction

et al. 2009

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Defining the minimal clinically meaningful improvement (MCMI) is crucial to understanding the treatment effects on health-status measures. We estimated the MCMI on the quality of erection questionnaire (QEQ), a validated measure specific to assess erectile quality during sexual intercourse. Data were from two controlled trials of an investigational phosphodiesterase type 5 inhibitor. Improvement on the Erectile Function domain of the International Index of Erectile Function was used as the anchor. For men who improved by exactly 1 erectile dysfunction severity category (anchor group (n ¼ 95)), clinically meaningful improvement (CMI, estimated with mean QEQ total change score from baseline to end of treatment) and MCMI (estimated with the lower limit of the 95% confidence interval of the mean) were 22.4 (s.d., 2.2) and 18.0 points, respectively. For the difference between the anchor group and men with no change in severity category (n ¼ 116), CMI and MCMI were 17.7 (s.d., 2.9) and 12 points, respectively. Distribution-based analyses (baseline s.e. of measurement (s.e.m.) ¼ 7.99, end-of-treatment s.e.m. ¼ 8.22 and s.e. of difference ¼ 11.46) supported a proposed MCMI of 12 points. Convergence of anchor-based and distribution-based criteria supports at least a 12-point difference in QEQ scores between treatments as clinically important.

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“…To further assess divergent validity, domain scores between the teenagers with SB and controls were compared using a t-test. To quantify effect size, we used a previously established method of dividing the mean difference between teenagers with SB and controls by the SD of the control population [28,29]. Several distribution-based approaches were used to determine what minimally important difference could be considered clinically significant.…”

Section: Phase 5 Internal Validationmentioning

confidence: 99%

Validation of QUALAS-T, a health-related quality of life instrument for teenagers with spina bifida

Szymański

Misseri

Whittam

et al. 2017

CEJU

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Introduction We aimed to develop and validate a self-reported QUAlity of Life Assessment in Spina bifida for Teenagers (QUALAS-T). Material and methodsWe drafted a 46-question pilot instrument using a patient-centered comprehensive item generation/refinement process. A group of 13-17 years olds with spina bifida (SB) was recruited online via social media and in person at SB clinics (2013)(2014)(2015). Healthy controls were recruited during routine pediatrician visits. Final questions were identified based on clinical relevance, factor analysis and domain psychometrics. Teenagers with SB completed the validated generic Kidscreen-27 instrument. Results Median age of 159 participants was 15.2 years (42.0% male, 77.4% Caucasian), similar to 58 controls (p ≥ 0.06). There were 102 online and 57 clinic participants (82.8% of eligible). Patients, parents and an expert panel established face and content validity of the 2-domain, 10-question QUALAS-T. Internal consistency and test-retest reliability were high for the Family and Independence and Bladder and Bowel domains (Cronbach's alpha: 0.76-0.78, ICC: 0.72-0.75). The Bladder and Bowel domain is the same for QUALAS-T , QUALAS-A for adults and QUALAS-C for children. Correlations between QUALAS-T domains were low (r = 0.34), indicating QUALAS-T can differentiate between distinct HRQOL components. Correlations between QUALAS-T and Kidscreen-27 were also low (r ≤0.41). QUALAS-T scores were lower in teenagers with SB than without (p <0.0001). Conclusions QUALAS-T is a short, valid HRQOL tool for adolescents with SB, applicable in clinical and research settings. Since the Bladder & Bowel domains for all QUALAS versions are the same, Bladder and Bowel HRQOL can be measured on the same scale from age 8 through adulthood.

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Approaches and Recommendations for Estimating Minimally Important Differences for Health-Related Quality of Life Measures

Cited by 206 publications

References 11 publications

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease

Clinically meaningful improvement on the quality of erection questionnaire in men with erectile dysfunction

Validation of QUALAS-T, a health-related quality of life instrument for teenagers with spina bifida

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