Patient-Perceived Outcomes and Quality of Life in ALS.
ABSTRACT: A variety of outcome measures are used in clinical practice and in research to assess patients with amyotrophic lateral sclerosis (ALS). However, there may be discordance between traditional outcome measures such as strength and physical function, and patient-perceived measures of well-being. One such self-perceived measure, reflecting the patient's view, is quality of life (QOL). QOL in patients with severe medical disorder is often underestimated by others. Patients with ALS often have high QOL, and this may persist throughout the disease due to shifting expectations and to reprioritization of factors contributing to QOL. QOL instruments can measure health-related QOL (HRQOL) or global QOL, and can be generic or disease-specific. HRQOL refers primarily to physical and mental health. Global QOL is much broader, and is also determined by non-health-related factors. The choice of a QOL instrument depends on whether the setting is routine patient care or clinical research, whether or not the outcome of a specific intervention is being assessed, and upon the expected efficacy or toxicity of the intervention. Global QOL instruments are best for individual clinical patient care or for comparing groups. HRQOL or a combination of HRQOL and global QOL instruments are most appropriate for assessing specific interventions.
Project description:<label>Background</label>Health-related quality of life (HRQoL) impairment is common in pulmonary hypertension (PH), but its clinical predictors are not well established. This study aims to characterize the HRQoL of patients with pulmonary arterial hypertension (PAH) and other precapillary forms of PH (pcPH) and to explore its clinical correlates.<label>Materials and Methods</label>A cross-sectional, observational study of patients with documented PAH and other forms of pcPH. Patients completed two patient-reported outcome measures (PROM): Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) and Nottingham Health Profile (NHP). Clinical characteristics were retrieved from electronic medical records.<label>Results</label>Mean CAMPHOR and NHP scores for the study population were indicative of a moderate HRQoL impairment. Patients in World Health Organisation Functional Classes (WHO FC) III/IV showed significantly worse HRQoL. The main clinical correlates of HRQoL were WHO FC, 6-minute walking distance (6MWD), and Borg dyspnoea index. Overall quality of life (QoL), assessed through CAMPHOR's QoL domain, showed patterns comparable to HRQoL measured by both instruments.<label>Conclusions</label>HRQoL, measured by two different PROMs, is impaired in Portuguese patients with PAH and other forms of pcPH, particularly in patients with increased disease severity. WHO FC, 6MWD, and Borg dyspnoea index are highly correlated with HRQoL and QoL.
Project description:<b>Background:</b> A key component to monitoring and investigating patient QOL is through patient reported health related quality of life (HRQOL) outcome measures. Many instruments have been used to assess HRQOL in bladder cancer and each instrument varies in its development, validation, the context of its usage in the literature and its applicability to certain disease states. <b>Objective:</b> In this review, we sought to summarize how clinicians and researchers should most appropriately utilize the available HRQOL instruments for bladder cancer. <b>Methods:</b> We performed a comprehensive literature search of each instrument used in bladder cancer, paying particular attention to the outcomes assessed. We used these outcomes to group the available instruments into categories best reflecting their optimal usage by stage of disease. <b>Results:</b> We found 5 instruments specific to bladder cancer, of which 3 are validated. Only one of the instruments (the EORTC-QLQ-NMIBC24) was involved in a randomized, prospective validation study. The most heavily used instruments are the EORTC-QLQ-BLM30 for muscle-invasive disease and the FACT-Bl which is used across all disease states. Of the 5 available instruments, 4 are automatically administered with general instruments, while the BCI lacks modularity, and requires co-administration with a generalized instrument. <b>Conclusion:</b> There are multiple strong instruments for use in gauging HRQOL in bladder cancer patients. We have divided these instruments into three categories which optimize their usage: instruments for use following NMIBC treatments (EORTC-QLQ-NMIBC24), instruments for use following radical cystectomy (FACT-Bl-Cys and EORTC-QLQ-BLM30) and more inclusive instruments not limited by treatment modality (BCI and FACT-Bl).
Project description:Over the past 10 years, several instruments developed specifically for the assessment of Quality of Life (QOL) in dementia have been introduced. The goal of the current review is to present, compare, and critique existing QOL measures for dementia populations to assist investigators and clinicians in selecting the optimal inventory for their specific needs. Nine measures are reviewed with a focus on conceptualizations of QOL, psychometric data, targeted patient population, and administration and scoring procedures. Critical discussion and comparison of the instruments is presented after the scales are described individually. Differences in definitions of QOL, assessment procedures, and methods that were used to establish the validity of instruments are highlighted. An important direction for future research on QOL scales for dementia is to establish their responsiveness to change over time. It will also be important to identify factors that affect reports of QOL, determine the how perceived QOL affects decisions regarding the care of dementia patients, and evaluate interventions to increase patient QOL.
Project description:PURPOSE:Quality of life (QOL) is an important concept in the field of health and medicine. QOL is a complex concept that is interpreted and defined differently within and between disciplines, including the fields of health and medicine. The aims of this study were to systematically review the literature on QOL in medicine and health research and to describe the country of origin, target groups, instruments, design, and conceptual issues. METHODS:A systematic review was conducted to identify research studies on QOL and health-related quality of life (HRQOL). The databases Scopus, which includes Embase and MEDLINE, CINAHL, and PsycINFO were searched for articles published during one random week in November 2016. The ten predefined criteria of Gill and Feinstein were used to evaluate the conceptual and methodological rigor. RESULTS:QOL research is international and involves a variety of target groups, research designs, and QOL measures. According to the criteria of Gill and Feinstein, the results show that only 13% provided a definition of QOL, 6% distinguished QOL from HRQOL. The most frequently fulfilled criteria were: (i) stating the domains of QOL to be measured; (ii) giving a reason for choosing the instruments used; and (iii) aggregating the results from multiple items. CONCLUSION:QOL is an important endpoint in medical and health research, and QOL research involves a variety of patient groups and different research designs. Based on the current evaluation of the methodological and conceptual clarity of QOL research, we conclude that the majority QOL studies in health and medicine have conceptual and methodological challenges.
Project description:<h4>Background</h4>Symptomatic prolapse impairs quality of life. Health-related quality of life (HRQoL) is considered an important outcome of pelvic organ prolapse (POP) surgery. However, it is rarely reported, and measures are inadequately used. Thus, studies reporting patient-reported surgical outcomes in low-income contexts are needed. This study aims to evaluate the effect of prolapse surgery on patient HRQoL and determine the predictive factors for change in HRQoL.<h4>Methods</h4>A total of 215 patients who had prolapse stage III or IV were enrolled. Patients underwent vaginal native tissue repair, and their HRQoL was evaluated at baseline, 3 and 6 months postoperatively. Effect of surgery on subjective outcomes were measured using validated Prolapse Quality of Life (P-QoL-20), Prolapse Symptom Score (POP-SS), Body Image in Prolapse (BIPOP), Patient Health Questionnaire (PHQ-9), and Patient Global Index of Improvement (PGI-I) tools. A linear mixed-effect model was used to compare pre- and postoperative P-QoL scores and investigate potential predictors of the changes in P-QoL scores.<h4>Results</h4>In total, 193 (89.7%) patients were eligible for analysis at 3 months, and 185 (86.0%) at 6 months. Participant's mean age was 49.3?±?9.4 years. The majority of patients had prolapse stage III (81.9%) and underwent vaginal hysterectomy (55.3%). All domains of P-QoL improved significantly after surgery. Altogether more than 72% of patients reported clinically meaningful improvement in condition-specific quality of life measured with P-QoL-20 at 6 months. An improvement in POP-SS, BIPOP, and the PHQ-9 scores were also observed during both follow-up assessments. At 6 months after surgery, only 2.7% of patients reported the presence of bulge symptoms. A total of 97.8% of patients had reported improvement in comparison to the preoperative state, according to PGI-I. The change in P-QoL score after surgery was associated with the change in POP-SS, PHQ, BIPOP scores and marital status (p?<?0.001). However, age, type of surgery, and prolapse stage were not associated with the improvement of P-QoL scores.<h4>Conclusions</h4>Surgical repair for prolapse effectively improves patient's HRQoL, and patient satisfaction is high. The result could be useful for patient counselling on the expected HRQoL outcomes of surgical treatment. Surgical service should be accessible for patients suffering from POP to improve HRQoL.
Project description:Hemolytic uraemic syndrome (HUS) is progressive renal failure disease and determination of their quality of life (QoL) on the basis of patient-reported outcomes (PROs) are becoming increasingly important in the economic evaluations for its treatment with eculizumab (ECU).To perform the systematic evaluation of QoL in HUS patients treated with ECU on the basis of Evaluating Measures of Patient Reported Outcomes (EMPRO) tool.A systematic review was conducted in PubMed, EMBASE, the Cochrane Library, CINAHL and Google Scholar till September 2016 by two independent researchers. Each identified instrument was evaluated for its quality of performance by using the EMPRO tool for its overall score and seven attribute specific scores (range 0-100, worst to best).Five different PROs instruments were identified from 10 articles (n?=?112) which showed eculizumab significantly improves health-related quality of life (HRQOL) in atypical HUS (aHUS) patients. Amongst five instruments viz. EuroQol five dimensions questionnaire (EQ-5?D), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F), Headache Impact Test-6 (HIT-6), 36-Item Short Form Health Survey (SF-36) and Visual Analogue Scale (VAS), the overall EMPRO score was higher for VAS (73.83) and EQ-5?D (73.81). Whereas, FACIT-F and HIT- 6 were just able to meet the minimal threshold of EMPRO scoring (50.24 and 59.09, respectively).Evidence from present investigation support that eculizumab significantly improves HRQoL in patients with aHUS furthermore, EQ-5?D and VAS instrument should be recommended for assessing HRQoL in them. However, selection of PRO instrument for determination of QoL in HUS entirely depend upon the study requirements.
Project description:BACKGROUND:"Asthma-related quality of life" (QOL) refers to the perceived impact that asthma has on the patient's QOL. OBJECTIVE:National Institutes of Health institutes and other federal agencies convened an expert group to recommend standardized measures of the impact of asthma on QOL for use in future asthma clinical research. METHODS:We reviewed published documentation regarding the development and psychometric evaluation; clinical research use since 2000; and extent to which the content of each existing QOL instrument provides a unique, reliable, and valid assessment of the intended construct. We classified instruments as core (required in future studies), supplemental (used according to the study's aims and standardized), or emerging (requiring validation and standardization). This work was discussed at an National Institutes of Health-organized workshop convened in March 2010 and finalized in September 2011. RESULTS:Eleven instruments for adults and 6 for children were identified for review. None qualified as core instruments because they predominantly measured indicators of asthma control (symptoms and/or functional status); failed to provide a distinct, reliable score measuring all key dimensions of the intended construct; and/or lacked adequate psychometric data. CONCLUSIONS:In the absence of existing instruments that meet the stated criteria, currently available instruments are classified as either supplemental or emerging. Research is strongly recommended to develop and evaluate instruments that provide a distinct, reliable measure of the patient's perception of the impact of asthma on all of the key dimensions of QOL, an important outcome that is not captured in other outcome measures.
Project description:To evaluate the effects of onabotulinumtoxinA on patient-reported outcomes including health-related quality of life (HRQOL), treatment satisfaction, and treatment goal attainment in patients with urinary incontinence (UI) due to neurogenic detrusor overactivity (NDO).In this multicenter, double-blind, randomized, placebo-controlled, phase III, 52-week study (ClinicalTrials.gov NCT00311376), patients with UI due to NDO who were not adequately managed with anticholinergic therapy were treated with intradetrusor injections of onabotulinumtoxinA (200 or 300 U) or placebo (0.9% saline). HRQOL measures included the Incontinence Quality of Life (I-QOL) Questionnaire total score, and the 3 domain scores (avoidance and limiting behavior, psychosocial, and social embarrassment), the modified Overactive Bladder Patient Satisfaction with Treatment Questionnaire (OAB-PSTQ), and Patient Global Assessment. Assessments were made at baseline, posttreatment week 6 (primary time point), week 12, and at 12-week intervals.Patients (mean age of 46 years with 30.5 weekly UI episodes at baseline) were randomized to receive placebo (n = 149) or onabotulinumtoxinA (200 U [n = 135] or 300 U [n = 132]). At week 6, improvements from baseline in I-QOL Questionnaire total score were greater (p < 0.001) in both onabotulinumtoxinA-treated groups vs placebo. Responses to the OAB-PSTQ also demonstrated greater mean improvements from baseline (p < 0.001) in both onabotulinumtoxinA-treated groups vs placebo at week 6. Patients who received onabotulinumtoxinA also reported greater improvement in the Patient Global Assessment than those in the placebo group (p ? 0.001 vs placebo).Patients with UI due to NDO reported greater improvement in HRQOL and treatment satisfaction with onabotulinumtoxinA than with placebo consistently across several patient-reported outcome instruments.This study provides Class I evidence that onabotulinumtoxinA intradetrusor injections (200 or 300 U) can improve quality of life measures in patients with NDO not adequately managed with anticholinergic therapy.
Project description:Clinical interpretation of health related quality of life (HRQOL) scores is challenging. The purpose of this analysis was to interpret score changes and identify minimal clinically important differences (MCID) on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (QLQ-C30) before (T1) and during (T2) cancer treatment.Patients (N = 627) in stem cell transplant (SCT) and medical (MED) or radiation (RAD) oncology at two comprehensive cancer centers, enrolled in the Electronic Self-Report Assessment-Cancer study and completed the QLQ-C30 at T1 and T2. Perceived changes in five QOL domains, physical (PF), emotional (EF), social (SF), cognitive functioning (CF) and global quality of life (QOL), were reported using the Subject Significance Questionnaire (SSQ) at T2. Anchored on SSQ ratings indicating "improvement", "the same", or "deterioration", means and effect sizes were calculated for QLQ-C30 score changes. MCID was calculated as the mean difference in QLQ-C30 score changes reflecting one category change on SSQ rating, using a two-piece linear regression model.A majority of SCT patients (54%) perceived deteriorating global HRQOL versus improvement (17%), while approximately equal proportions of MED/RAD patients perceived improvement (25%) and deterioration (26%). Global QOL decreased 14.2 (SCT) and 2.0 (MED/RAD) units, respectively, among patients reporting "the same" in the SSQ. The MCID ranged 5.7-11.4 (SCT) and 7.2-11.8 (MED/RAD) units among patients reporting deteriorated HRQOL; ranged 2.7-3.4 units among MED/RAD patients reporting improvement. Excepting for the global QOL (MCID =6.9), no meaningful MCID was identified among SCT patients reporting improvement.Cancer treatment has greater impact on HRQOL among SCT patients than MED/RAD patients. The MCID for QLQ-C30 score change differed across domains, and differed for perceived improvement and deterioration, suggesting different standards for self-evaluating changes in HRQOL during cancer treatment. Specifically, clinical attention can be focused on patients who report at least a 6 point decrease, and for patients who report at least a 3 point increase on QLQ-C30 domains.The trial was registered with ClinicalTrials.gov: NCT00852852.
Project description:OBJECTIVE:Studies that have investigated quality of life (QoL) in eating disorders (EDs) have been focussed on the impact of the ED on QoL and little is known regarding the possible reciprocal impact of QoL on EDs. The aim of this study was to provide a first-time investigation of possible bidirectional relationships between EDs and both health-related QoL (HRQoL) and psychological distress (PD). METHOD:Structural equation modeling was applied to longitudinal data collected from a community sample of Australian women (N = 828) surveyed at baseline, five annual follow-ups, and again after nine years. Participants reported height and weight (from which body mass index, BMI, was calculated) and completed measures of ED symptoms (Eating Disorder Examination Questionnaire), HRQoL (12-item Medical Outcomes Study Short Form), and PD (Kessler Psychological Distress Scale). RESULTS:Overall, evidence was found for a bidirectional relationship, whereby ED symptoms predicted reduced HRQoL and greater PD over time, while lower levels of HRQoL and greater PD in turn predicted increased levels of ED symptoms. These relationships were stable, observable within 12 months, and remained observable over a time period of at least four years. However, also observed were some inconsistent findings where ED symptoms predicted a short term (one year) improvement in mental HRQoL. This short term boost was not sustained at longer follow-ups. CONCLUSIONS:Not only do ED symptoms impact on HRQoL and PD, but perceived poor HRQoL and PD also contribute to ED symptom development or exacerbation. This supports a movement away from symptom-centric approaches whereby HRQoL is conceptualized as a passive outcome expected to be rectified by addressing ED symptoms. Improvement in QoL and PD might rather be viewed as targets to be pursued in their own right under broader approaches in the treatment of EDs.