How Do We Define a Meaningful Change in Quality of Life for Patients with Sarcoidosis?
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SUBMITTER: Aronson KI
PROVIDER: S-EPMC7919151 | biostudies-literature |
REPOSITORIES: biostudies-literature
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Project description:Background Among older patients with atrial fibrillation, there are limited data examining clinically meaningful changes in quality of life (QoL). We examined the extent of, and factors associated with, clinically meaningful change in QoL over 1-year among older adults with atrial fibrillation. Methods and Results Patients from cardiology, electrophysiology, and primary care clinics in Massachusetts and Georgia were enrolled in a cohort study (2015-2018). The Atrial Fibrillation Effect on Quality-of-Life questionnaire was used to assess overall QoL and across 3 subscales: symptoms, daily activities, and treatment concern. Clinically meaningful change in QoL (ie, difference between 1-year and baseline QoL score) was categorized as either a decline (≤-5.0 points), no clinically meaningful change (-5.0 to +5.0 points), or an increase (≥+5.0 points). Ordinal logistic models were used to examine factors associated with QoL changes. Participants (n=1097) were on average 75 years old, 48% were women, and 87% White. Approximately 40% experienced a clinically meaningful increase in QoL and 1 in every 5 patients experienced a decline in QoL. After multivariable adjustment, women, non-Whites, those who reported depressive and anxiety symptoms, fair/poor self-rated health, low social support, heart failure, or diabetes mellitus experienced clinically meaningful declines in QoL. Conclusions These findings provide insights to the magnitude of, and factors associated with, clinically meaningful change in QoL among older patients with atrial fibrillation. Assessment of comorbidities and psychosocial factors may help identify patients at high risk for declining QoL and those who require additional surveillance to maximize important clinical and patient-centered outcomes.
| S-EPMC7726984 | biostudies-literature
Project description:ObjectiveTo evaluate the measurement properties of all three domains of the Migraine-Specific Quality of Life questionnaire version 2.1 (MSQ v2.1) electronic patient-reported outcome (ePRO) to assess the functional impact of migraine in patients with episodic or chronic migraine (CM); and identify meaningful within-patient change thresholds for the Role Function-Restrictive (RFR), Role Function-Preventive (RFP), and Emotional Function (EF) domains.MethodsData were drawn from three double-blind, placebo-controlled, and randomized Phase 3 clinical studies (episodic migraine [EM]: EVOLVE-1 and EVOLVE-2; CM: REGAIN). The psychometric properties of the MSQ v2.1 ePRO domains were demonstrated by evaluating reliability (internal consistency and test-retest), construct validity (convergent and known groups), and responsiveness. Meaningful within-patient change thresholds for domains were estimated using anchor-based approaches, supplemented by empirical cumulative distribution function curves and probability density function plots to enable interpretation of meaningful change over 3 months. The Patient Global Impression of Severity (PGI-S) and Patient Global Impression of Improvement served as anchors.ResultsA total of 2,850 patients with either EM (EVOLVE-1: 851; EVOLVE-2: 909) or CM (REGAIN: 1,090) were included. The Cronbach's alpha estimates of internal consistency exceeded the recommended threshold of ≥0.70 for all domains from the three studies, indicating adequate internal consistency. Test-retest reliability intraclass correlation coefficients were ≥0.80 for all domains across all three studies, demonstrating almost perfect agreement. Convergent validity was supported by moderate-to-strong correlation (r ≥ 0.30) between all domains of MSQ v2.1 ePRO and studied anchors (Migraine Disability Assessment Score and PGI-S scores) across all three studies. Known group validity was established between all domains and subgroups of patients stratified by baseline PGI-S scores and baseline number of monthly migraine headache days for all three studies. The 3-month meaningful within-patient change thresholds were the same for EM and CM for RFP: 20.00 and EF: 26.67; and for RFR: 25.71.ConclusionsThese findings demonstrate that all three domains of the MSQ v2.1 ePRO have sufficient reliability, validity, responsiveness, and appropriate interpretation standards. Our results suggest that MSQ v2.1 ePRO is a well-defined and reliable patient-reported outcome instrument that is suitable for use in clinical studies for evaluating the impact of migraine on patient functioning in episodic and CM.
| S-EPMC8048965 | biostudies-literature
Project description:BackgroundMajor depressive disorder (MDD) directly impacts patients' lives including symptoms, functioning and health-related quality-of-life (HRQoL). Patient-reported outcomes can capture these impacts, however interpretation of clinical meaningfulness of these measurements are often not readily available. Meaningful change thresholds (MCTs) can be derived for clinical outcome assessments to quantify the change in symptoms that is meaningful to the patient following pharmacologic treatment or other interventions. The objective of this analysis was to determine the within-patient MCT of the self-reported Quality-of-Life in Depression Scale (QLDS) among patients with MDD and active suicidal ideation with intent (MDSI) using an anchor-based approach.MethodsData from 2 randomized phase-3 trials of esketamine nasal spray (ASPIRE I and ASPIRE II) were analyzed. The Montgomery-Åsberg Depression Rating Scale (MADRS) was the primary anchor with three different severity criteria. Other anchor variables utilized were Clinical Global Impression of Severity of Suicidality-revised version, Clinical Global Impression of Imminent Suicide Risk, and EuroQol Visual Analog Scale [EQ-VAS]. Spearman correlation coefficients between the change in QLDS and anchor variables were calculated. The mean change in QLDS score at Day 25 from baseline was calculated based on the categorical change in the anchor. Coefficient yield from linear regression of the mean changes in EQ-VAS and QLDS, and distribution-based approach with ½ SD of change in QLDS were considered.ResultsIn ASPIRE I, mean (SD) improvement in QLDS score among patients with one category improvement in MADRS from baseline to Day 25 was - 8.22 (8.87), - 8.30 (9.01), and - 8.20 (8.92) using severity criteria #1, #2, and #3, respectively. Patients who achieved a 7-point improvement (MCT) in EQ-VAS yielded a mean - 9.69-point improvement in QLDS at Day 25. The ½ SD of change in QLDS was 5.63. Similar results were obtained for ASPIRE II. The MCTs identified using multiple anchors across both trials ranged from - 11.4 to - 6.7 and had an overall mean of - 7.90 (ASPIRE I) and - 7.92 (ASPIRE II). Thus, an 8-point change was recommended as the MCT for QLDS.ConclusionThe recommended MCT will help quantify within-person changes in HRQoL using patient-reported QLDS and determine meaningful treatment benefit in an MDD patient population with acute suicidal ideation or behavior.Trial registrationName of the registry: ClinicalTrials.gov.Trial registration numberASPIRE I (NCT03039192), ASPIRE II (NCT03097133). Date of registration: February 01, 2017; March 31, 2017. URL of trial registry record: https://clinicaltrials.gov/ct2/show/NCT03039192 ; https://clinicaltrials.gov/ct2/show/NCT03097133 .
| S-EPMC9273809 | biostudies-literature
Project description:BackgroundAlthough psoriatic arthritis is complex and involves multiple domains, recent advances in treatments have made remission or near-remission of most symptoms a potentially achievable goal for many patients. We sought to evaluate whether achieving minimal disease activity (MDA) criteria represented meaningful improvement from the patient perspective.MethodsData were combined from two randomized, multinational, 24 week clinical studies of ixekizumab, a high-affinity monoclonal antibody selectively targeting interleukin-17A, in biological drug-naïve or experienced adults. MDA required 5 of 7 of: tender joint count ≤1; swollen joint count ≤1; Psoriasis Area and Severity Index total score ≤ 1 or body surface area ≤ 3%; patient's assessment of pain visual analogue scale (VAS) ≤15; patient's global assessment of disease activity VAS ≤20; Health Assessment Questionnaire Disability Index ≤0.5; and tender entheseal points ≤ 1. MDA responders and non-responders were compared for mean change from baseline on the 36-Item Short Form Health Survey (SF-36), European Quality of Life 5 Dimension 5 Level Health Questionnaire (EQ-5D-5 L); EQ-5D-5 L VAS; and Work Productivity and Activity Impairment-Specific Health Problem (WPAI-SHP) questionnaire.ResultsMDA responders had significantly greater improvements versus non-responders in each SF-36 domain and in the SF-36 physical summary score; improvements were also greater in the EQ-5D-5 L and EQ-5D-5 L VAS, and in 3 of the 4 WPAI-SHP domains. MDA responders were more likely to achieve minimal clinically important differences than non-responders.ConclusionThese findings support MDA response as being strongly associated with achieving improved disease status based on measures of patient reported health-related quality of life and productivity.Trial registrationSPIRIT-P1, NCT01695239, First Posted: September 27, 2012; and SPIRIT-P2, NCT02349295, First Posted: January 28, 2015.
| S-EPMC6390571 | biostudies-literature
Project description:Patients with advanced myeloma experience a high symptom burden particularly near the end of life, making timely hospice use crucial. Little is known about the quality and determinants of end-of-life care for this population, including whether potential increases in hospice use are also accompanied by "late" enrollment (? 3 days before death). Using the Surveillance, Epidemiology, and End-Results-Medicare database, we identified patients ? 65 years diagnosed with myeloma between 2000 and 2013 who died by December 31, 2013. We assessed prevalence and trends in hospice use, including late enrollment. We also examined six established measures of potentially aggressive medical care at the end of life. Independent predictors of late hospice enrollment and aggressive end-of-life care were assessed using multivariable logistic regression analyses. Of 12,686 myeloma decedents, 48.2% enrolled in hospice. Among the 6111 who enrolled, 17.2% spent ? 3 days there. There was a significant trend in increasing hospice use, from 28.5% in 2000 to 56.5% by 2013 (Ptrend <0.001), no significant rise in late enrollment (12.2% in 2000 to 16.3% in 2013, Ptrend =0.19), and a slight decrease in aggressive end-of-life care (59.2% in 2000 to 56.7% in 2013, Ptrend =0.01). Patients who were transfusion-dependent, on dialysis, or survived for less than one year were more likely to enroll late in hospice and experience aggressive medical care at the end of life. Gains in hospice use for myeloma decedents were not accompanied by increases in late enrollment or aggressive medical care. These findings suggest meaningful improvements in end-of-life care for this population.
| S-EPMC6068022 | biostudies-literature
Project description:OBJECTIVE:To demonstrate challenges in the estimation of change in quality of life (QOL). METHODS:Data were taken from a completed clinical trial with negative results. Responses to 13 QOL items were obtained 12 months apart from 258 persons with Alzheimer's disease (AD) participating in a randomized, placebo-controlled clinical trial with two treatment arms. Two analyses to estimate whether "change" in QOL occurred over 12 months are described. A simple difference (later - earlier) was calculated from total scores (standard approach). A Qualified Change algorithm (novel approach) was applied to each item: differences in ratings were classified as either: improved, worsened, stayed poor, or stayed "positive" (fair, good, excellent). The strengths of evidence supporting a claim that "QOL changed", derived from the two analyses, were compared by considering plausible alternative explanations for, and interpretations of, results obtained under each approach. RESULTS:Total score approach: QOL total scores decreased, on average, in the two treatment (both -1.0, p < 0.05), but not the placebo (=-0.59, p > 0.3) groups. Qualified change approach: Roughly 60% of all change in QOL items was worsening in every arm; 17% - 42% of all subjects experienced change in each item. CONCLUSIONS:Totalling the subjective QOL item ratings collapses over items, and suggests a potentially misleading "overall" level of change (or no change, as in the placebo arm). Leaving the items as individual components of "quality" of life they were intended to capture, and qualifying the direction and amount of change in each, suggests that at least 17% of any group experienced change on every item, with 60% of all observed change being worsening. DISCUSSION:Summarizing QOL item ratings as a total "score" collapses over the face-valid, multi-dimensional components of the construct "quality of life". Qualified Change provides robust evidence of changes to QOL or "enhancements of" life quality.
| S-EPMC4514524 | biostudies-literature
Project description:BACKGROUND:Carcinoid syndrome is associated with a reduced quality of life that can be attributed to symptoms such as diarrhea and fatigue as well as social and financial issues. This study was conducted to psychometrically assess meaningful change in bowel movement frequency among carcinoid syndrome patients using data from the TELESTAR clinical study. METHODS:An anchor-based approach for deriving meaningful change thresholds consisted of mapping change from baseline bowel movement frequency to other patient-reported assessments of change. These included the European Organization for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire - Core Questionnaire (QLQ-C30) Diarrhea Symptom responders, the EORTC Gastrointestinal NET questionnaire (GI.NET21) GI Symptom responders, and reported adequate relief at Week 12 (? 10-point score decrease from Day 1 to Week 12). Parameters included within-group mean change from baseline to Week 12, t-tests of the change (Wilcoxon rank sum for adequate relief), and effect size. RESULTS:There were 135 carcinoid syndrome patients with a mean baseline frequency of 5.7 bowel movements a day. A distribution-based method yielded meaningful change estimates of 0.62 bowel movements a day for overall frequency and 0.83 bowel movements a day at Week 12. Anchor-based analysis indicated a large effect size among patients who reported adequate relief at Week 12 (-?1.58; n?=?18; P?=?0.014), the QLQ-C30 Diarrhea domain responders (-?1.24; n?=?40; P?<?0.001), and the GI.NET21 GI Symptoms Domain responders (-?1.49; n?=?25; P?=?0.005). Exit interview data for meaningful change yielded effect size estimates of -?1.57 for overall change during the Double-blind Treatment Period and?-?1.97 for change between Baseline and Week 12. CONCLUSIONS:Meaningful change derivation is critical to interpret patient outcomes for evaluating treatment efficacy. In this study, carcinoid syndrome patients experienced clinically meaningful reductions in bowel movement frequency of ?30% over 12?weeks with telotristat ethyl treatment. TRIAL REGISTRATION:NCT01677910 .
| S-EPMC6815313 | biostudies-literature
Project description:BACKGROUND:Numerous studies have investigated the association between hormone receptor expression and clinical outcome in ovarian carcinoma (OC); however, these have largely focussed on serous OCs, with few studies reporting specifically on endometrioid OCs (EnOC). Where analyses have been stratified by histotype, expression has been assessed using the percentage of positive tumor cells, without accounting for nuclear expression intensity. METHODS:Here we assess the expression levels of progesterone receptor (PR), estrogen receptor alpha (ER) and androgen receptor (AR) using histoscore - a nuclear scoring method incorporating both proportion of positive cells and the intensity of nuclear staining - across a cohort of 107 WT1 negative EnOCs. RESULTS:Hierarchical clustering by PR, ER and AR histoscores identified four EnOC subgroups (PR+/ER+, PR+/ER-, PR-/ER+ and PR-/ER-). EnOC patients in the PR+/ER+ and PR+/ER- groups displayed favorable outcome (multivariable HR for disease-specific survival 0.05 [0.01-0.35] and 0.05 [0.00-0.51]) compared to the PR-/ER+ group. Ten-year survival for stage II PRhigh and PRlow cases was 94.1% and 42.4%. ERhigh EnOC patients (PR+/ER+, PR-/ER+) had higher body mass index compared to ERlow cases (P?=?0.015) and high grade serous OC patients (P?<?0.001). CONCLUSION:These data demonstrate that endometrioid OC cases with high PR expression display markedly favorable outcome. Stage II EnOCs with high PR expression represent potential candidates for de-escalation of first-line therapy. Future work should seek to characterise the sensitivity of PR and ER positive EnOCs to endocrine therapy.
| S-EPMC6854467 | biostudies-literature
Project description:BackgroundSarcoidosis is a systemic inflammatory granulomatous disease, frequently affecting the lung. If left untreated, it may end in lung fibrosis. Proangiogenic and profibrotic vascular endothelial growth factor (VEGF), transforming growth factor (TGF)-β1, fibroblast growth factor (FGF)-2 and platelet-derived growth factor (PDGF)-AB are a known therapeutical target in pulmonary fibrosing diseases, e.g. IPF, but there is no targeted therapy option for pulmonary fibrosis in sarcoidosis.ObjectivesThe aim of our study was to determine the association of these markers' serum levels on lung function and the patients' quality of life in a long-term follow-up of sarcoidosis patients, to provide further information for finding targeted therapy options for pulmonary sarcoidosis.Methods54 patients with sarcoidosis underwent blood sampling, pulmonary function testing and answered the King's Brief Interstitial Lung Disease (K-BILD) questionnaire at baseline and at three-years follow-up. Serum levels of profibrotic and angiogenic markers were assessed at baseline by enzyme-linked immunosorbent assay.ResultsBetween 2015 and 2018, 54 patients with biopsy proven sarcoidosis were enrolled. Throughout the observation period, there was a significant decrease in the diffusion capacity for carbon monoxide (DLCO) [%] (-6.5504 ± 13,39, p = 0.001) and forced expiratory volume in one second predicted (FEV1) [%] (-6.07 ± 12.09, p = 0.001). Patients with greater impairment of forced vital capacity (FVC) did have significantly higher serum levels of VEGF (p = 0.03) and PDGF-AB (p<0.001). The K-BILD questionnaire did not change significantly during follow-up. However, patients with worsening K-BILD scores did have significantly higher serum-levels of PDGF-AB (2.67 pg/ml ± 0.93 vs. 1.88 pg/ml ± 0.60, p = 0.004) at baseline, compared to those with unchanged or increasing K-BILD scores.ConclusionsAmong patients with pulmonary sarcoidosis, baseline serum levels of VEGF and PDGF-AB were associated with pulmonary function impairment. Furthermore, PDGF-AB was associated with worsening K-BILD scores. No such association was observed for FGF-2 and TGF-ß1. VEGF and PDGF-AB may be possible prognostic and therapeutic targets in sarcoidosis as a fibrosing ILD beyond IPF.
| S-EPMC7899331 | biostudies-literature
Project description:ObjectivesTo examine SLEDAI-2000 cut-off scores for definition of active SLE and to determine the sensitivity to change of SLEDAI-2000 for the assessment of SLE disease activity and minimal clinically meaningful changes in score.MethodsData from two multi-centre studies were used in the analysis: in a cross-sectional and a longitudinal fashion. At every assessment, data were collected on SLEDAI-2000 and treatment. The cross-sectional analysis with receiver operating characteristic (ROC) curves was used to examine the appropriate SLEDAI-2000 score to define active disease and increase in therapy was the reference standard. In the longitudinal analysis, sensitivity to change of SLEDAI-2000 was assessed with multinomial logistic regression. ROC curves analysis was used to examine possible cut-points in score changes associated with change in therapy, and mean changes were estimated.ResultsIn the cross-sectional analysis, the most appropriate cut-off scores for active disease were 3 or 4. In the longitudinal analysis, the best model for predicting treatment increase was with the change in SLEDAI-2000 score and the score from the previous visit as continuous variables. The use of cut-points was less predictive of treatment change than the use of continuous score. The mean difference in the change in SLEDAI-2000 scores, adjusted for prior score, between patients with treatment increase and those without was 2.64 (95% CI 2.16, 3.14).ConclusionsAn appropriate SLEDAI-2000 score to define active disease is 3 or 4. SLEDAI-2000 index is sensitive to change. The use of SLEDAI-2000 as a continuous outcome is recommended for comparative purposes.
| S-EPMC3077910 | biostudies-literature