The Huntington's Disease health-related Quality of Life questionnaire (HDQoL): a disease-specific measure of health-related quality of life

Hocaoglu MB, Gaffan EA, Ho AK. The Huntington's disease health-related quality of life questionnaire: a disease-specific measure of health-related quality of life. Huntington's disease (HD) is a genetic neurodegenerative disorder characterized by motor, cognitive and psychiatric disturbances, and yet there is no disease-specific patient-reported health-related quality of life outcome measure for patients. Our aim was to develop and validate such an instrument, i.e. the Huntington's Disease health-related Quality of Life questionnaire (HDQoL), to capture the true impact of living with this disease. Semi-structured interviews were conducted with the full spectrum of people living with HD, to form a pool of items, which were then examined in a larger sample prior to data-driven item reduction. We provide the statistical basis for the extraction of three different sets of scales from the HDQoL, and present validation and psychometric data on these scales using a sample of 152 participants living with HD. These new patient-derived scales provide promising patient-reported outcome measures for HD.

Health-related quality of life in Huntington’s Disease patients: a comparison of proxy assessment and patient self-rating using the disease-specific Huntington’s Disease health-related quality of life questionnaire (HDQoL)

Huntington’s disease (HD) is a fatal, neurodegenerative disease for which there is no known cure. Proxy evaluation is relevant for HD as its manifestation might limit the ability of persons to report their health-related quality of life (HrQoL). This study explored patient–proxy ratings of HrQoL of persons at different stages of HD, and examined factors that may affect proxy ratings. A total of 105 patient–proxy pairs completed the Huntington’s disease health-related quality of life questionnaire (HDQoL) and other established HrQoL measures (EQ-5D and SF-12v2). Proxy–patient agreement was assessed in terms of absolute level (mean ratings) and intraclass correlation. Proxies’ ratings were at a similar level to patients’ self-ratings on an overall Summary Score and on most of the six Specific Scales of the HDQoL. On the Specific Hopes and Worries Scale, proxies on average rated HrQoL as better than patients’ self-ratings, while on both the Specific Cognitive Scale and Specific Physical and Functional Scale proxies tended to rate HrQoL more poorly than patients themselves. The patient’s disease stage and mental wellbeing (SF-12 Mental Component scale) were the two factors that primarily affected proxy assessment. Proxy scores were strongly correlated with patients’ self-ratings of HrQoL, on the Summary Scale and all Specific Scales. The patient–proxy correlation was lower for patients at moderate stages of HD compared to patients at early and advanced stages. The proxy report version of the HDQoL is a useful complementary tool to self-assessment, and a promising alternative when individual patients with advanced HD are unable to self-report.

Impact of socioeconomic and clinical factors on child oral health-related quality of life (COHRQoL)

Child oral health-related quality of life (COHRQoL) has been increasingly assessed; however, few studies appraised the influence of socioeconomic status on COHRQoL in developing countries. This study assessed the relationship of COHRQoL with socioeconomic backgrounds and clinical factors. This study followed a cross-sectional design, with a multistage random sample of 792 schoolchildren aged 12 years, representative of Santa Maria, a southern city in Brazil. Participants completed the Brazilian version of the Child Perceptions Questionnaire (CPQ(11-14)), their parents or guardians answered questions on socioeconomic status, and a dental examination provided information on the prevalence of caries, dental trauma and occlusion. The assessment of association used hierarchically adjusted Poisson regression models. Higher impacts on COHRQoL were observed for children presenting with untreated dental caries (RR 1.20; 95% CI 1.07-1.35) and maxillary overjet (RR 1.19; 95% CI 1.02-1.40). Socioeconomic factors also associated with COHRQoL; poorer scores were reported by children whose mothers have not completed primary education (RR 1.30; 95% CI 1.17-1.44) and those with lower household income (RR 1.13; 95% CI 1.02-1.26). Poor socioeconomic standings and poor dental status have a negative impact on COHRQoL; reducing health inequalities may demand dental programmes and policies targeting deprived population.

Factors associated with health-related quality of life after successful kidney transplantation: a population-based study

Kidney transplantation improves the quality of life of end-stage renal disease patients. The quality of life benefits, however, pertain to patients on average, not to all transplant recipients. The aim of this study was to identify factors associated with health-related quality of life after kidney transplantation. Population-based study with a cross-sectional design was carried out and quality of life was assessed by SF-36 Health Survey Version 1. A multivariate linear regression model was constructed with sociodemographic, clinical and laboratory data as independent variables. Two hundred and seventy-two kidney recipients with a functioning graft were analyzed. Hypertension, diabetes, higher serum creatinine and lower hematocrit were independently and significantly associated with lower scores for the SF-36 oblique physical component summary (PCSc). The final regression model explained 11% of the PCSc variance. The scores of oblique mental component summary (MCSc) were worse for females, patients with a lower income, unemployed and patients with a higher serum creatinine. The regression model explained 9% of the MCSc variance. Among the studied variables, comorbidity and graft function were the main factors associated with the PCSc, and sociodemographic variables and graft function were the main determinants of MCSc. Despite comprehensive, the final regression models explained only a little part of the heath-related quality of life variance. Additional factors, such as personal, environmental and clinical ones might influence quality of life perceived by the patients after kidney transplantation.

QUALITY OF LIFE IN SYSTEMIC LUPUS ERYTHEMATOSUS PATIENTS IN NORTHEASTERN BRAZIL: IS HEALTH-RELATED QUALITY OF LIFE A PREDICTOR OF SURVIVAL FOR THESE PATIENTS?

Objective: To identify social, demographic and clinical characteristics that influence survival of patients with systemic lupus erythematosus (SLE). Methods: Sixty-three patients with a diagnosis of SLE were studied at our medical services in 1999 and then reviewed in 2005. We utilized a protocol to obtain demographic and clinical traits, activity and damage indices, and health-related quality of life via the SF-36. All statistical tests were performed using a significance level of 5%. Results: Out of the 63 patients examined in 1999, six died, four were lost for the follow-up and the previous protocol was applied to the remaining 53 patients. The six patients who died presented the worst recorded health-related quality of fife, in all aspects. The most important observed predictor of death was a mean lower score in the Role-Emotional Domain of the mental health component of the SF-36 (p<0.01). Conclusion: Health-related quality of life may be used as possible predictive factor of mortality among patients with SLE.

A new short and simple health-related quality of life measurement for paediatric rheumatic diseases: initial validation in juvenile idiopathic arthritis

Objective. To develop and validate a new short and simple measure of health-related quality of life (HRQL) in children with juvenile idiopathic arthritis (JIA).Methods. The Paediatric Rheumatology Quality of Life Scale (PRQL) is a 10-item questionnaire that explores HRQL in two domains: physical health (PhH) and psychosocial health (PsH). Validation of the parent proxy report and child self-report versions of the instrument was accomplished by evaluating 472 JIA patients and similar to 800 healthy children. Validation analyses included assessment of feasibility, face and content validity; construct and discriminative ability; internal structure and consistency; test-retest reliability; responsiveness to clinical change; and minimal clinically important difference.Results. The PRQL was found to be feasible and to possess both face and content validity. The PRQL score correlated in the predicted range with most of the other JIA outcome measures, thereby demonstrating good construct validity, and discriminated well between different levels of disease severity. Assessment of internal structure (factor analysis) revealed that the PhH and PsH subscales identify two unambiguously separated domains. The internal consistency (Cronbach's alpha) was 0.86. The intraclass correlation coefficient for test-retest reliability was 0.91. The PRQL revealed fair responsiveness, with a standardized response mean of 0.67 in improved patients. Overall, the PRQL appeared to be more able to capture physical HRQL than psychosocial HRQL.Conclusion. The PRQL was found to possess good measurement properties and is, therefore, a valid instrument for the assessment of HRQL in children with JIA. This tool is primarily proposed for use in standard clinical care.

Health related quality of life among the elderly: a population-based study using SF-36 survey

Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP)

Validation of the Brazilian versions of two inventories for measuring oral health-related quality of life of edentulous subjects

Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP)

Proxy-reported health-related quality of life of patients with juvenile idiopathic arthritis: Pediatric rheumatology international trials organization multinational quality of life cohort study

Objective. To investigate the proxy-reported health-related quality of life (HRQOL) and its determinants in patients with juvenile idiopathic arthritis (JIA).Methods. In this multinational, multicenter, cross-sectional study, HRQOL of patients with JIA was assessed through the Child Health Questionnaire (CHQ) and was compared with that of healthy children of similar age from the same geographic area. of joint inflammation, Childhood Health Assessment Questionnaire (CHAQ), and erythrocyte sedimentation rate.Results. A total of 6,639 participants (3,324 with JIA and 3,315 healthy) were enrolled from 32 countries. The mean SD physical and psychosocial summary scores of the CHQ were significantly lower in patients with JIA than in healthy children (physical: 44.5 +/- 10.6 versus 54.6 +/- 4.0, P < 0.0001; psychosocial: 47.6 +/- 8.7 versus 51.9 +/- 7.59 P < 0.0001), with the physical well-being domain being most impaired. Patients with persistent oligoarthritis had better HRQOL compared with other subtypes, whereas HRQOL was similar across patients with systemic arthritis, polyarthritis, and extended oligoarthritis. A CHAQ score > 1 and a pain intensity rating > 3.4 cm on a 10-cm visual analog scale were the strongest determinants of poorer HRQOL in the physical and psychosocial domains, respectively.Conclusion. We found that patients with JIA have a significant impairment of their HRQOL compared with healthy peers, particularly in the physical domain. Physical well-being was mostly affected by the level of functional impairment, whereas the intensity of pain had the greatest influence on psychosocial health.

Assessment of health-related quality of life in children with functional defecation disorders

Objective: To evaluate the health-related quality of life in children with functional defecation disorders. Methods: One hundred children seen consecutively were enrolled and subdivided into three subsets according to the Roma II classification criteria: functional constipation (n = 57), functional fecal retention (n = 29) and nonretentive functional soiling (n = 14). The generic instrument Child Health Questionnaire - Parent Form 50 (CHQ-PF50®), was used to measure quality of life and to assess the impact of these disorders from the point of view of parents. The instrument measures physical and psychosocial wellbeing in 15 health domains, each of which is graded on a scale from 0 to 100, with higher values indicating better health and greater wellbeing. Ten of these are then used to obtain two aggregated and summary scores: the physical and psychosocial scores. Results: No statistically significant differences were detected between subsets in terms of demographic or anthropometric characteristics. In 14 domains, children with defecation disorders scored lower than healthy children. When subsets were compared, statistically significant differences were detected between children with nonretentive functional soiling (lower scores) and those with functional constipation. Physical and psychosocial scores for the entire sample were lower than those for the group of healthy children used as controls. Conclusions: The CHQ-PF50® was considered adequate for demonstrating compromised quality of life in children with functional defecation disorders, as has been reported for other diseases, being a useful tool for making treatment decisions and for patient follow-up. Copyright © 2006 by Sociedade Brasileira de Pediatria.

Methotrexate improves the health-related quality of life of children with juvenile idiopathic arthritis

Objectives: To examine the change in health-related quality of life (HRQOL) and its determinants in children with juvenile idiopathic arthritis (JIA) treated with methotrexate (MTX). Methods: Patients were extracted from the PRINTO clinical trial which aimed to evaluate the efficacy and safety profile of MTX administered in standard, intermediate or higher doses (10, 15 and 30 mg/m2/week respectively). Children with polyarticular-course JIA, who were less than 18 years and had a complete HRQOL assessment were included. Results: A total of 521 children were included. At baseline, patients with JIA showed poorer HRQOL (p<0.01) than healthy children. In 207/412 (50%) and 63 (15%) children, HRQOL values were 2 standard deviations below the mean of healthy controls in the physical and psychosocial summary scale, respectively. After 6 months of treatment with standard dose MTX, there was a statistically significant improvement in all HRQOL health concepts, particularly the physical ones. Similar improvements were observed in those who did not respond to a standard dose of MTX and were subsequently randomised to a higher dose. The presence of marked disability at baseline was associated with a fivefold increased risk of retaining poor physical health after 6 months of active treatment with standard dose MTX. Other less important determinants of retaining poor physical well-being were the baseline level of systemic inflammation, pain intensity and an antinuclear-antibody-negative status. Conclusions: MTX treatment produces a significant improvement across a wide range of HRQOL components, particularly in the physical domains, in patients with JIA.

Dornase alfa improves the health-related quality of life among Brazilian patients with cystic fibrosis - A one-year prospective study

Background: Health-related quality of life (HRQOL) measurements provide valuable information about the psychological and social impact of treatment on patients with cystic fibrosis (CF). This study evaluated the HRQOL of Brazilian patients with CF and assessed the changes in HRQOL domains over 1 year after dornase alfa (Pulmozyme) introduction. Patients and Methods: One hundred fifty-six stable patients with CF and 89 caregivers answered the Portuguese-validated version of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at baseline (T 0), and at 3 (T 1), 6 (T 2), 9 (T 3), and 12 (T 4) months of follow-up. Eighteen patientswere excluded because they did not fulfill the inclusion criteria. The patients were analyzed in two groups: those aged 6-11 years and those aged 14 years and older. ANOVA for observed repeated results and the last observation carried forward (LOCF) method for missing data were used for the statistical analysis. Results: After 1 year of follow-up, there was significant improvement in respiratory symptoms (T 4-T 0=8.1; 95% confidence interval (95% CI)=[2.1;14.0]; effect size (ES)=0.35; P<0.001), Emotional Functioning (T 4-T 0=5.6; 95% CI=[1.1;10.1]; ES=0.31; P<0.05), Social Functioning (T 4-T 0=6.0; 95% CI=[1.3;11.7]; ES=0.31; P<0.05), Body Image (T 4-T 0=11.9; 95% CI=[4.1;19.7]; ES=0.42; P<0.05), and Treatment Burden (T 4-T 0=5.3; 95% CI=[0.3;10.3]; ES=0.24; P<0.05) domains in the younger group. A significant improvement in Role Functioning (T 4-T 0=6.1; 95% CI=[1.1;11.1]; ES=0.40; P<0.05), Body Image (T 4-T 0=12.6; 95% CI=[3.5;21.7]; ES=0.46; P<0.05), and Weight (T 4-T 0=11.7; 95% CI=[1.8;21.6]; ES=0.40; P<0.05) was obtained in the older group. The caregivers' CFQ-R showed improvements in the Digestive Symptoms (T 4-T 0=5.5; 95% CI=[1.5;9.4]; ES=0.30; P<0.05), Respiratory Symptoms (T 4-T 0=7.6; 95% CI=[3.9;11.4]; ES=0.48; P<0.05), and Weight (T 4-T 0=10.1; 95% CI=[1.6;18.6]; ES=0.26; P<0.05) domains. Conclusion: The introduction of dornase alfa improved the HRQL of the patients with CF during the first year of treatment. © 2010 Wiley-Liss, Inc.

Oral health-related quality of life and satisfaction before and after treatment with complete dentures in a Dental School in Brazil

Purpose: The aim of this study was to evaluate the impact of treatment with new complete dentures on oral health-related quality of life and satisfaction of edentulous patients attending the Prosthodontic Department in a Dental School in Brazil. Methods: A total of 70 edentulous subjects (37-86 years) treated by undergradute students at the Araçatuba Dental School, University of State of São Paulo, were accessed. A specific questionnaire for edentulous patients (EDENT) based on the Oral Health Impact Profile (OHIP-EDENT) was applied to collect information on patient oral health-related quality of life. Questions related to the personal satisfaction of the edentulous patients with their complete dentures were also included. The patients were accessed before the treatment, and 3 months after receiving the new dentures. Results: After rehabilitation with new complete dentures, all domains of OHIP-EDENT showed significant improvements. There was also a significant improvement in patient satisfaction after placement of new complete dentures. Furthermore, it was possible to observe association between upper denture satisfaction and age. Conclusion: Conventional complete dentures may have a positive impact on oral health-related quality of life and satisfaction of edentulous patients. © 2012 Japan Prosthodontic Society.