Dataset Information

Association Between Myasthenia Gravis-Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities.

ABSTRACT:

Introduction

For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown.

Methods

Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a measure of health-related quality of life (HRQoL), were collected biweekly up to 26 weeks. Utility values were derived from the EQ-5D-5L data with the United Kingdom value set. Descriptive statistics were reported for MG-ADL and EQ-5D-5L at baseline and follow-up. A normal identity-link regression model estimated the association between utility and the eight MG-ADL items. A generalized estimating equations (GEE) model was estimated to predict utility based on the patient's MG-ADL score and treatment received.

Results

A total of 167 patients (84 EFG + CT, 83 PBO + CT) contributed 167 baseline and 2867 follow-up measurements of MG-ADL and EQ-5D-5L. EFG + CT-treated patients experienced more improvements than PBO + CT-treated patients in most MG-ADL items and EQ-5D-5L dimensions, with the largest improvements observed in chewing, brushing teeth/combing hair, eyelid droop (MG-ADL); self-care, usual activities, mobility (EQ-5D-5L). The regression model indicated that individual MG-ADL items contributed differently to utility values, with the largest impact from brushing teeth/combing hair, rising from a chair, chewing, and breathing. The GEE model showed that each unit improvement in MG-ADL led to a statistically significant utility increase of 0.0233 (p < 0.001). In addition, a statistically significant improvement of 0.0598 (p = 0.0079) in utility was found for patients in the EFG + CT group compared to the PBO + CT group.

Conclusion

Among patients with gMG, improvements in MG-ADL were significantly associated with higher utility values. MG-ADL scores alone were not sufficient to capture the utility gained from efgartigimod therapy.

SUBMITTER: Dewilde S

PROVIDER: S-EPMC10070298 | biostudies-literature | 2023 Apr

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Association Between Myasthenia Gravis-Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities.

Dewilde Sarah S Qi Cynthia Z CZ Phillips Glenn G Iannazzo Sergio S Janssen Mathieu F MF

Advances in therapy 20230303 4

<h4>Introduction</h4>For patients with generalized myasthenia gravis (gMG), the association between symptom severity, often measured with the Myasthenia Gravis Activities of Daily Living (MG-ADL) instrument, and utility values is unknown.<h4>Methods</h4>Data was analyzed from the phase 3 ADAPT trial, which included adult patients with gMG randomly assigned to treatment with efgartigimod + conventional therapy (EFG + CT) or placebo + CT (PBO + CT). MG-ADL total symptom scores and the EQ-5D-5L, a ...[more]

PMID: 36867327

Similar Datasets

Project description:BackgroundThe progression of Duchenne muscular dystrophy (DMD) is characterized by loss of ambulation, respiratory insufficiency, cardiomyopathy, and early mortality. DMD profoundly impacts health-related quality-of-life (HRQoL). However, few health state utility data exist; published utilities tend to be derived from small samples for a limited number of health states and are often based on caregiver-reported patient health status. This study estimated utility values for varied clinical and functional health states in DMD, based on patient-reported health status.MethodsIndividuals with DMD in the US aged 12-40 years completed the EQ-5D (5-level) and Health Utilities Index (HUI) preference-based instruments. Based on responses to a clinical questionnaire, participants self-classified into functional health states according to level of lower and upper limb function, use of respiratory support, and presence of cardiomyopathy. Mean [standard deviation (SD)] utility and EQ-5D visual analogue scale (VAS) scores were estimated according to health state; and median (interquartile range) attribute levels calculated to understand which domains of health are most severely affected in DMD.ResultsOf 63 males with DMD, mean (SD) age was 19.8 (6.1) years and 11 (17.5%) were ambulatory. Mean (SD) utility values were 0.92 (0.08; HUI2), 0.84 (0.20; HUI3), and 0.84 (0.13; EQ-5D) for ambulatory patients without cardiomyopathy (n = 10). For non-ambulatory patients with moderately impaired upper limb function, night and daytime ventilation without cardiomyopathy, mean (SD) utilities were 0.49 (0.07) for the HUI2, 0.16 (0.15) for the HUI3 and 025 (0.14) for the EQ-5D. Mean (SD) VAS scores for the same health states were 91 (9) and 83 (21), respectively. In addition to impairments in mobility/ambulation, and self-care, attributes like usual activities and pain also showed notable effects of DMD.ConclusionsIn DMD, although a relationship between disease progression and HRQoL is observed, there is large variability in utility within functional health states, and across instruments. Utility values for less severe non-ambulatory health states described by level of upper limb function are novel. These utility values, derived based on direct patient feedback rather than from caregiver report, are relevant to individuals of varying functional statuses and augment scarce DMD-specific utility data.

Project description:BACKGROUND:The objective of this study was to obtain utilities by means of EQ-5D-5L for different health states in patients with knee osteoarthritis (KOA) or hip osteoarthritis (HOA) in Spain, and to compare these values with those used in foreign studies with the aim of discussing their transferability for their use in economic evaluations conducted in Spain. METHODS:Primary study: Observational prospective study of KOA or HOA patients in Spain. Sociodemographic and clinical characteristics were collected to characterize the sample. Utilities were elicited using the EQ-5D-5L questionnaire. ANOVA and bivariable analyses were conducted to identify differences between health states. LITERATURE REVIEW:Using the bibliographic databases NSH EED and CEA Registry, we conducted searches of model-based cost utilities analyses of technologies in KOA or HOA patients. Health states and utilities were extracted and compared with values obtained from the Spanish sample. RESULTS:Three hundred ninety-seven subjects with KOA and 361 subjects with HOA were included, with average utilities of 0.544 and 0.520, respectively. In both samples, differences were found in utilities according to level of pain, stiffness and physical function (WOMAC) and severity of symptoms (Oxford scales), so that the worst the symptoms, the lower the utilities. The utilities after surgery were higher than before surgery. Due to limitations from our study related to sample size and observational design, it was not possible to estimate utilities for approximately half the health states included in the published models because they were directly related to specific technologies. For almost 100% of health states of the selected studies we obtained very different utilities from those reported in the literature. CONCLUSIONS:To our knowledge this is the first article with detailed utilities estimated using the EQ-5D-5L in Spain for KOA and HOA patients. In both populations, utilities are lower for worse health states in terms of level of pain, stiffness and physical function according to WOMAC, and according to the Oxford scales. Most utilities obtained from the Spanish sample are lower than those reported in the international literature. Further studies estimating utilities from local populations are required to avoid the use of foreign sources in economic evaluations.

Project description:BackgroundThe majority of patient reported outcome measures (PROMs) don't have population norms in Romania. This is the case with the EQ-5D as well. Therefore, we aimed to estimate population norms for the Romanian versions of the EQ-5D-5L, EQ-5D-3L, their indexes, and the EQ-VAS.MethodsA cross-sectional survey was conducted in all regions of Romania from November 2018 to November 2019. A three-stage probability sampling procedure stratified by region and settlement size was used to select a representative sample. Interviews were computer-assisted and conducted in respondents' homes by trained interviewers. Health status was assessed with the EQ-5D-5L, the EQ-5D-3L and the EQ VAS. Descriptive statistics were used to estimate population norms by age groups and sex for the EQ-5D-5L, the EQ-5D-3L, their indexes and the EQ VAS. Population norms were weighted using survey weights. Indexes for the EQ-5D questionnaires were estimated using the recently developed Romanian value sets.ResultsData from 1,649 interviews was analysed in the present study. Survey weights were used so that sex and place of residence ratios for the weighted sample matched the Romanian general population distribution. Participants' mean age was 47.4 years (SE = 1.157) and 50.3% of them reported being in good health. The dimension for which people reported the highest number of problems for both questionnaires was the pain/discomfort dimension. Men aged 35 plus reported fewer problems with pain/discomfort than women for both the EQ-5D-5L and EQ-5D-3L. Health decreased with age as shown by the decrease from age group 18-24 to age group 75 plus in the indexes of both questionnaires: from 0.977 (SE = 0.005) to 0.765 (SE = 0.017) for EQ-5D-5L and from 0.981 (SE = 0.005) to 0.784 (SE = 0.019) for EQ-5D-3L. There was 29.9 points drop in the EQ VAS score between the youngest and oldest group.ConclusionsPopulation norms for the Romanian versions of the EQ-5D-5L, EQ-5D-3L, their indexes, and the EQ VAS are now available. These can now be used as reference values by healthcare professionals, researchers and decision-makers leading to a further development of health-related quality of life research in Romania.

Project description:ObjectivesThis study aimed to provide normative data obtained in response to the EQ-5D-5L questionnaire in Italy and compare this with data from other countries.MethodsA sample of the Italian adult population (aged ≥ 18 years) was recruited and interviewed online using videoconferencing software (Zoom) between November 2020 and February 2021. The distribution of answers was estimated as per the descriptive system of the EQ-5D-5L, and descriptive statistics were calculated for the EQ VAS score and EQ-5D-5L index value in the whole sample and relevant subgroups. An ordinary least square (OLS) regression was performed to evaluate the impact of sociodemographic variables on EQ-5D-5L results. Lastly, a comparison was made with EQ-5D-5L population norms of other countries. Data analysis was performed using Microsoft Excel and Stata 13.ResultsOverall, 1182 people representative of the Italian population (2020) in terms of sex and geographical area responded to the survey. Of the 3125 potential EQ-5D-5L health states, only 106 (3.4%) were selected, and the '11111' and '11112' states were chosen by half of the participants. In terms of EQ-5D-5L dimensions, the frequency of any problems (from slight to extreme) associated with anxiety and depression was high among the very young (18-24 years, 56.0%) and in women of all ages (49.7%). The mean index value (± standard deviation [SD]) was 0.93 (± 0.11) for the entire sample and gradually decreased with age, moving from 0.95 (± 0.06) in the youngest group (18-24 years) to 0.91 (± 0.13) in the oldest age group (≥ 75 years). Similarly, the mean EQ VAS score (± SD) was 81.8 (± 13.5), and decreased from 87.0 (± 8.9) in the 18-24 years age group to 75.1 (± 16.4) among participants > 75 years of age. The existence of self-reported chronic conditions (e.g., cardiovascular disease), female sex, and social assistance recipiency were negatively associated with the EQ-5D index value, while the EQ VAS score was significantly lower in people with chronic conditions and aged > 55 years. Conversely, higher income levels had a positive impact on both the EQ-5D index value and the EQ VAS score. Lastly, both the EQ-5D index value and EQ VAS score in Italy were, on average, higher than in most European countries.ConclusionsEQ-5D-5L population norms provide useful insights into the health status of the Italian population and can be used as a reference for other surveys using the same instrument.

Project description:BackgroundThis study evaluates the health-related quality of life (HRQoL) of persons with diffuse large B-cell lymphoma (DLBCL) by using EQ-5D-5L and SF-6Dv2 and compares the measurement properties of the two instruments.MethodDLBCL patients were identified via a patient group and were surveyed using web-based questionnaires. Demographic information, socioeconomic status (SES), clinical characteristics, and EQ-5D-5L and SF-6Dv2 responses were collected and statistically described. The association between the EQ-5D-5L and SF-6Dv2 dimensions were analyzed using the Spearman's correlation coefficient, whereas the correlation of the utility scores was evaluated using Pearson's correlation coefficient. The agreement between the responses of the two instruments were examined using a Bland-Altman (B-A) plot. A one-way analysis of variance (ANOVA) was performed to compare the utility scores across subgroups in different clinical states (a t-test was used if there were two subgroups). In addition, the graded response model (GRM) was used to describe the discrimination ability and difficulty characteristics of the dimensions in the two instruments.ResultsIn total, 582 valid responses were collected, among which 477 respondents were associated with initial-treatment and 105 respondents were relapsed/refractory (RR) patients. The mean (standard deviation [SD]) EQ-5D-5L and SF-6Dv2 utility scores of the DLBCL patients were 0.828 (0.222) and 0.641 (0.220), respectively. The correlation between the EQ-5D-5L and SF-6Dv2 dimensions ranged from 0.299 to 0.680, and the correlation between their utility scores was 0.787. The B-A plot demonstrated an acceptable but not strong agreement between EQ-5D-5L and SF-6Dv2 utility scores. The GRM model results indicated that all dimensions of each instrument were highly discriminating overall, but EQ-5D-5L had suboptimal discriminative power among patients with good health.ConclusionBoth the EQ-5D-5L and SF-6Dv2 showed valid properties to assess the HRQoL of DLBCL patients. However, utility scores derived from the two instruments had substantial difference, thereby prohibiting the interchangeable use of utilities from the two instruments.

Dataset Information

Association Between Myasthenia Gravis-Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities.

Introduction

Methods

Results

Conclusion

Publications

Association Between Myasthenia Gravis-Activities of Daily Living (MG-ADL) and EQ-5D-5L Utility Values: The Additional Effect of Efgartigimod on Utilities.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets