scholarly journals Randomised clinical trial for the cost–utility evaluation of two strategies of perineal reconstruction after abdominoperineal resection in the context of anorectal carcinoma: biological mesh repair versus primary perineal wound closure, study protocol for the GRECCAR 9 Study

BMJ Open ◽  
2021 ◽  
Vol 11 (4) ◽  
pp. e043333
Author(s):  
Etienne Buscail ◽  
Cindy Canivet ◽  
Laurent Ghouti ◽  
Sylvain Kirzin ◽  
Nicolas Carrere ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Abdominoperineal Resection ◽  
Rectal Adenocarcinoma ◽  
Perineal Wound ◽  
Cost Utility ◽  
Biological Mesh ◽  
High Rate ◽  
Sensitivity Analyses ◽  
Ethical Review

IntroductionAbdominoperineal resections performed for anorectal tumours leave a large pelvic and perineal defect causing a high rate of morbidity of the perineal wound (40%–60%). Biological meshes offer possibilities for new standards of perineal wound reconstruction. Perineal fillings with biological mesh are expected to increase quality of life by reducing perineal morbidity.Methods and analysisThis is a multicentre, randomised and single-blinded study with a blinded endpoint evaluation, the experimental arm of which uses a biological mesh and the control arm of which is defined by the primary closure after abdominoperineal resection for cancer. Patients eligible for inclusion are patients with a proven history of rectal adenocarcinoma and anal canal epidermoid carcinoma for whom abdominoperineal resection was indicated after a multidisciplinary team discussion. All patients must have social security insurance or equivalent social protection. The main objective is to assess the incremental cost–utility ratio (ICUR) of two strategies of perineal closure after an abdominoperineal resection performed for anorectal cancer treatment: perineal filling with biological mesh versus primary perineal closure (70 patient in each arm). The secondary objectives focus on quality of life and morbidity data during a 1-year follow-up. Deterministic and probabilistic sensitivity analyses will be performed in order to estimate the uncertainty surrounding the ICUR. CIs will be constructed using the non-parametric bootstrap approach. A cost-effectiveness acceptability curve will be built so as to estimate the probability of efficiency of the biological meshes given a collective willingness-to-pay threshold.Ethics and disseminationThe study was approved by the Regional Ethical Review Board of ‘Nord Ouest 1’ (protocol reference number: 20.05.14.60714; national number: 2020-A01169-30).The results will be disseminated through conventional scientific channels.Trial registration numberClinicalTrials.gov Registry (NCT02841293).

scholarly journals E-health ecosystem with integrated and stepped psychosocial services for breast cancer survivors: study protocol of a multicentre randomised controlled trial

BMJ Open ◽  
2021 ◽  
Vol 11 (3) ◽  
pp. e041548
Author(s):  
Cristian Ochoa-Arnedo ◽  
Joan Carles Medina ◽  
Aida Flix-Valle ◽  
Dimitra Anastasiadou
Keyword(s):  
Breast Cancer ◽  
Quality Of Life ◽  
Randomised Controlled Trial ◽  
Emotional Distress ◽  
Controlled Trial ◽  
Psychosocial Care ◽  
Cost Utility ◽  
Psychosocial Services ◽  
Randomised Controlled

IntroductionPsychosocial interventions for patients with breast cancer (BC) have demonstrated their effectiveness at reducing emotional distress and improving quality of life. The current digitisation of screening, monitoring and psychosocial treatment presents the opportunity for a revolution that could improve the quality of care and reduce its economic burden. The objectives of this study are, first, to assess the effectiveness of an e-health platform with integrated and stepped psychosocial services compared with usual psychosocial care, and second, to examine its cost–utility.Methods and analysisThis study is a multicentre randomised controlled trial with two parallel groups: E-health intervention with integrated and stepped psychosocial services vs usual psychosocial care. An estimated sample of 338 patients with BC in the acute survival phase will be recruited from three university hospitals in Catalonia (Spain) and will be randomly assigned to one of two groups. All participants will be evaluated at the beginning of the study (T1: recruitment), 3 months from T1 (T2), 6 months from T1 (T3) and 12 months from T1 (T4). Primary outcome measures will include number of clinical cases detected, waiting time from detection to psychosocial intervention and proportion of cases successfully treated in the different steps of the intervention, as well as outcomes related to emotional distress, quality of life, post-traumatic stress and growth, treatment adherence and therapeutic alliance. Secondary outcomes will include the acceptability of the platform, patients’ satisfaction and usability. For the cost–utility analysis, we will assess quality-adjusted life years and costs related to healthcare utilisation, medication use and adherence, work absenteeism and infrastructure-related and transport-related costs.Ethics and disseminationThis study was approved by the Ethics committee of the Institut Català d’Oncologia network in Hospitalet, Spain. Findings will be disseminated through peer-reviewed journals, reports to the funding body, conferences among the scientific community, workshops with patients and media press releases.Trial registration numberOnline Psychosocial Cancer Screening, Monitoring and Stepped Treatment in Cancer Survivors (ICOnnectat-B),NCT04372459.

Utilities and Quality-Adjusted Life Years

1989 ◽  
Vol 5 (4) ◽  
pp. 559-575 ◽  
Author(s):  
George W. Torrance ◽  
David Feeny
Keyword(s):  
Quality Of Life ◽  
Cost Utility ◽  
Health Improvement ◽  
Quality Adjusted Life Years ◽  
Utility Analysis ◽  
Quality Adjustment ◽  
Life Years ◽  
Quality Adjusted Life ◽  
Broad Overview

Utilities and quality-adjusted life years (QALYs) are reviewed, with particular focus on their use in technology assessment. This article provides a broad overview and perspective on these two techniques and their interrelationship, with reference to other sources for details of implementation. The historical development, assumptions, strengths/weaknesses, and applications of each are summarized.Utilities are specifically designed for individual decision-making under uncertainty, but, with additional assumptions, utilities can be aggregated across individuals to provide a group utility function. QALYs are designed to aggregate in a single summary measure the total health improvement for a group of individuals, capturing improvements from impacts on both quantity of life and quality of life– with quality of life broadly defined. Utilities can be used as the quality-adjustment weights for QALYs; they are particularly appropriate for that purpose, and this combination provides a powerful and highly useful variation on cost-effectiveness analysis known as cost-utility analysis.

scholarly journals Rituximab in generalized myasthenia gravis: Clinical, quality of life and cost–utility analysis

2017 ◽  
Vol 2 (3) ◽  
pp. 81-85 ◽  
Author(s):  
João Peres ◽  
Rita Martins ◽  
José Delgado Alves ◽  
Ana Valverde
Keyword(s):  
Quality Of Life ◽  
Myasthenia Gravis ◽  
Cost Utility ◽  
Cost Utility Analysis ◽  
Utility Analysis ◽  
Clinical Quality

Final results from ClarIDHy, a global, phase 3, randomized, double-blind study of ivosidenib (IVO) versus placebo (PBO) in patients (pts) with previously treated cholangiocarcinoma (CCA) and an isocitrate dehydrogenase 1 (IDH1) mutation.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 4069-4069
Author(s):  
Ghassan K. Abou-Alfa ◽  
Teresa Macarulla ◽  
Milind M. Javle ◽  
Robin Kate Kelley ◽  
Sam Joseph Lubner ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Primary Endpoint ◽  
Failure Time ◽  
Objective Response ◽  
Idh1 Mutation ◽  
High Rate ◽  
Double Blind Study ◽  
Double Blind ◽  
Grade 3

4069 Background: CCA is a rare cancer for which there are limited effective therapies. IDH1 mutations occur in ̃20% of intrahepatic CCAs, resulting in production of the oncometabolite D-2-hydroxyglutarate, which promotes oncogenesis. IVO (AG-120) is a first-in-class, oral, small-molecule inhibitor of mutant IDH1 (mIDH1). ClarIDHy aimed to demonstrate the efficacy of IVO vs PBO in pts with unresectable or metastatic m IDH1 CCA. The primary endpoint was met with significant improvement in progression-free survival (PFS) by independent radiology center (IRC) with IVO vs PBO (hazard ratio [HR] = 0.37, p < 0.0001). Objective response rate (ORR) and stable disease for IVO were 2.4% (3 partial responses) and 50.8% (n = 63) vs 0% and 27.9% (n = 17) for PBO. IVO pts experienced significantly less decline in physical and emotional functioning domains of quality of life at cycle 2 day 1 vs PBO pts (nominal p < 0.05). Methods: Pts with m IDH1 CCA were randomized 2:1 to IVO (500 mg PO QD) or matched PBO and stratified by prior systemic therapies (1 or 2). Key eligibility: unresectable or metastatic m IDH1 CCA based on central testing; ECOG PS 0–1; measurable disease (RECIST v1.1). Crossover from PBO to IVO was permitted at radiographic progression. Primary endpoint: PFS by IRC. Secondary endpoints included overall survival (OS; by intent-to-treat), ORR, PFS (by investigator), safety, and quality of life. The planned crossover-adjusted OS was derived using the rank-preserving structural failure time (RPSFT) model. Results: As of 31 May 2020, ̃780 pts were prescreened for an IDH1 mutation and 187 were randomized to IVO (n = 126) or PBO (n = 61); 13 remain on IVO. Median age 62 y; M/F 68/119; 91% intrahepatic CCA; 93% metastatic disease; 47% had 2 prior therapies. 70% of PBO pts crossed over to IVO. OS data were mature, with 79% OS events in IVO arm and 82% in PBO. Median OS (mOS) was 10.3 months for IVO and 7.5 months for PBO (HR = 0.79; 95% CI 0.56–1.12; one-sided p = 0.093). The RPSFT-adjusted mOS was 5.1 months for PBO (HR = 0.49; 95% CI 0.34–0.70; p < 0.0001). Common all-grade treatment emergent adverse events (TEAEs, ≥ 15%) in the IVO arm: nausea 41%, diarrhea 35%, fatigue 31%, cough 25%, abdominal pain 24%, decreased appetite 24%, ascites 23%, vomiting 23%, anemia 18%, and constipation 15%. Grade ≥ 3 TEAEs were reported in 50% of IVO pts vs 37% of PBO pts, with grade ≥ 3 treatment-related AEs in 7% of IVO pts vs 0% in PBO. 7% of IVO pts experienced an AE leading to treatment discontinuation vs 9% of PBO pts. There were no treatment-related deaths. Conclusions: IVO was well tolerated and resulted in a favorable OS trend vs PBO despite a high rate of crossover. These data – coupled with statistical improvement in PFS, supportive quality of life data, and favorable safety profile – demonstrate the clinical benefit of IVO in advanced m IDH1 CCA. Clinical trial information: NCT02989857.

Quality of Life Change and Individual Differences in Stimulus Screening in Relation to Incidence of Illness

1977 ◽  
Vol 41 (1) ◽  
pp. 267-278 ◽  
Author(s):  
Albert Mehrabian ◽  
Marion Ross
Keyword(s):  
Quality Of Life ◽  
Individual Differences ◽  
Well Being ◽  
High Rate ◽  
Life Change ◽  
Emotional States ◽  
Life Changes ◽  
Comprehensive Description ◽  
High Arousal

A considerable amount of evidence indicates that a high rate of life changes—a source of continued and unavoidable arousal—is detrimental to health and psychological well-being. The present study hypothesized that sustained high-arousal states are unpreferred and that the persistence of unpreferred emotional states is harmful. Using a conceptual framework for a comprehensive description of emotional states and the differential preferences for these, it is possible to make more precise predictions on the illness consequences of emotionally unpreferred life changes. Particular hypotheses which received support were that more arousing life changes are more conducive to illness; that among the more arousing life changes, unpleasant changes are associated with more illness than pleasant ones; that unpleasant life changes are more detrimental to health when combined with dominance-inducing life changes; and that arousing life changes are particularly harmful to more arousable (non-screening) individuals.

Final results from ClarIDHy, a global, phase III, randomized, double-blind study of ivosidenib (IVO) versus placebo (PBO) in patients (pts) with previously treated cholangiocarcinoma (CCA) and an isocitrate dehydrogenase 1 (IDH1) mutation.

2021 ◽  
Vol 39 (3_suppl) ◽  
pp. 266-266
Author(s):  
Andrew X. Zhu ◽  
Teresa Macarulla ◽  
Milind M. Javle ◽  
Robin Kate Kelley ◽  
Sam Joseph Lubner ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Primary Endpoint ◽  
Failure Time ◽  
Objective Response ◽  
Idh1 Mutation ◽  
High Rate ◽  
Phase Iii ◽  
Double Blind Study ◽  
Grade 3

266 Background: CCA is a rare cancer for which there are limited effective therapies. IDH1 mutations occur in ~20% of intrahepatic CCAs, resulting in production of the oncometabolite D-2-hydroxyglutarate, which promotes oncogenesis. IVO (AG-120) is a first-in-class, oral, small-molecule inhibitor of mutant IDH1 (m IDH1). ClarIDHy aimed to demonstrate the efficacy of IVO vs PBO in pts with unresectable or metastatic m IDH1 CCA. The primary endpoint was met with significant improvement in progression-free survival (PFS) by independent radiology center (IRC) with IVO vs PBO (hazard ratio [HR] = 0.37, p < 0.0001). Objective response rate (ORR) and stable disease for IVO were 2.4% (3 partial responses) and 50.8% (n = 63) vs 0% and 27.9% (n = 17) for PBO. IVO pts experienced significantly less decline in physical and emotional functioning domains of quality of life at cycle 2 day 1 vs PBO pts (nominal p < 0.05). Methods: Pts with m IDH1 CCA were randomized 2:1 to IVO (500 mg PO QD) or matched PBO and stratified by prior systemic therapies (1 or 2). Key eligibility: unresectable or metastatic m IDH1 CCA based on central testing; ECOG PS 0–1; measurable disease (RECIST v1.1). Crossover from PBO to IVO was permitted at radiographic progression. Primary endpoint: PFS by IRC. Secondary endpoints included overall survival (OS; by intent-to-treat), ORR, PFS (by investigator), safety, and quality of life. The planned crossover-adjusted OS was derived using the rank-preserving structural failure time (RPSFT) model. Results: As of 31 May 2020, ~780 pts were prescreened for an IDH1 mutation and 187 were randomized to IVO (n = 126) or PBO (n = 61); 13 remain on IVO. Median age 62 y; M/F 68/119; 91% intrahepatic CCA; 93% metastatic disease; 47% had 2 prior therapies. 70% of PBO pts crossed over to IVO. OS data were mature, with 79% OS events in IVO arm and 82% in PBO. Median OS (mOS) was 10.3 months for IVO and 7.5 months for PBO (HR = 0.79; 95% CI 0.56–1.12; one-sided p = 0.093). The RPSFT-adjusted mOS was 5.1 months for PBO (HR = 0.49; 95% CI 0.34–0.70; p < 0.0001). Common all-grade treatment emergent adverse events (TEAEs, ≥ 15%) in the IVO arm: nausea 41%, diarrhea 35%, fatigue 31%, cough 25%, abdominal pain 24%, decreased appetite 24%, ascites 23%, vomiting 23%, anemia 18%, and constipation 15%. Grade ≥ 3 TEAEs were reported in 50% of IVO pts vs 37% of PBO pts, with grade ≥ 3 treatment-related AEs in 7% of IVO pts vs 0% in PBO. 7% of IVO pts experienced an AE leading to treatment discontinuation vs 9% of PBO pts. There were no treatment-related deaths. Conclusions: IVO was well tolerated and resulted in a favorable OS trend vs PBO despite a high rate of crossover. These data – coupled with statistical improvement in PFS, supportive quality of life data, and favorable safety profile – demonstrate the clinical benefit of IVO in advanced m IDH1 CCA. Clinical trial information: NCT02989857.

Change in Quality of Life and Cost/Utility Analysis in Open Stage-related Surgical Treatment of Elbow Stiffness

Orthopedics ◽  
2013 ◽  
Vol 36 (7) ◽  
pp. e923-e930 ◽  
Author(s):  
Giuseppe Giannicola ◽  
Gianluca Bullitta ◽  
Federico M. Sacchetti ◽  
Marco Scacchi ◽  
David Polimanti ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Surgical Treatment ◽  
Cost Utility ◽  
Cost Utility Analysis ◽  
Utility Analysis ◽  
Elbow Stiffness

Health-Related Quality of Life and Cost–Utility Analysis of Employees With Musculoskeletal Disorders Receiving Vocational Physiotherapy

2020 ◽  
Vol 68 (10) ◽  
pp. 476-479
Author(s):  
Laran Chetty
Keyword(s):  
Quality Of Life ◽  
Musculoskeletal Disorders ◽  
Cost Utility ◽  
Cost Utility Analysis ◽  
Health Related Quality ◽  
Utility Analysis ◽  
Related Quality ◽  
Health Related ◽  
The Cost

Background: The purpose of this project was to evaluate both health-related quality of life (HRQoL) and cost-utility associated with care for employees with musculoskeletal disorders who received vocational physiotherapy at a North London National Health Service (NHS) Foundation Trust in the United Kingdom. Methods: A pre- and post-physiotherapy EuroQol 5 Dimension (EQ-5D) questionnaire was administered to employees presenting to the vocational physiotherapy service (VPS) with musculoskeletal disorders. The cost-utility analysis of the physiotherapy service was calculated using cost data provided by VPS billing information and benefits measured using Quality-Adjusted Life Years (QALYs). Findings: Overall, there was a significant improvement in the EQ-5D index from baseline to discharge in all HRQoL domains. The visual analog scale (VAS) improved from a mean of 31.5 (SD = 18.3) at baseline to 73.2 (SD = 18.5) at discharge. A cost-utility analysis indicated that the VPS would continue to be cost-effective until the cost per employee increased by 82.5%. Conclusion/Application to Practice: The project supports integration of vocational physiotherapy services into an occupational health department.

OP56 Rehabilitation Of Memory In Brain Injury: A Cost-Utility Analysis

2018 ◽  
Vol 34 (S1) ◽  
pp. 20-21
Author(s):  
Shaun Harris ◽  
Deborah Fitzsimmons ◽  
Roshan das Nair ◽  
Lucy Bradshaw
Keyword(s):  
Usual Care ◽  
Cost Effective ◽  
Cost Utility ◽  
Sensitivity Analyses ◽  
Base Case ◽  
Utility Analysis ◽  
Considerable Uncertainty ◽  
Memory Rehabilitation ◽  
The Impact

Introduction:People with traumatic brain injuries (TBIs) commonly report memory impairments which are persistent, debilitating, and reduce quality of life. As part of the Rehabilitation of Memory in Brain Injury trial, a cost-effectiveness analysis was undertaken to examine the comparative costs and effects of a group memory rehabilitation program for people with TBI.Methods:Individual-level cost and outcome data were collected. Patients were randomized to usual care (n=157) or usual care plus memory rehabilitation (n=171). The primary outcome for the economic analysis was the EuroQol-5D quality of life score at 12 months. A UK NHS costing perspective was used. Missing data was addressed by multiple imputation. One-way sensitivity analyses examined the impact of varying different parameters, and the impact of available cases, on base case findings whilst non-parametric bootstrapping examined joint uncertainty.Results:At 12 months, the intervention was GBP 26.89 (USD 35.76) (SE 249.15) cheaper than usual care; but this difference was statistically non-significant (p=0.914). At 12 months, a QALY loss of −0.007 was observed in the intervention group confidence interval (95% CI: −0.025–0.012) and a QALY gain seen in the usual care group 0.004 (95% CI: -0.017–0.025). This difference was not statistically significant (p=0.442). The base case analysis gave an ICER of GBP 2,445 (USD 3,252) reflecting that the intervention was less effective and less costly compared to usual care. Sensitivity analyses illustrated considerable uncertainty. When joint uncertainty was examined, the probability of the intervention being cost-effective at a willingness-to-pay threshold of GBP 20,000 per QALY gain was 29 percent and 24 percent at GBP 30,000.Conclusions:Our cost-utility analysis indicates that memory rehabilitation was cheaper but less effective than usual care but these findings must be interpreted in the light of small statistically non–significant differences and considerable uncertainty was evident. The ReMemBrIn intervention is unlikely to be considered cost-effective for people with TBI.

Sign in / Sign up

Export Citation Format

Share Document