Disease-specific Health-related Quality Of Life Instrument Research Articles

Health-related quality of life assessment instruments for extracorporeal membrane oxygenation survivors: A scoping review.

Extracorporeal membrane oxygenation (ECMO) has been successfully and widely used in adult patients for the past 5years. About 50% of these patients can survive and are discharged from hospitals. Health-related quality of life (HRQOL) is crucial for evaluating survived ECMO patients. This scoping review aims to identify instruments commonly used to measure HRQOL of ECMO survivors and give pertinent instrument characteristics. A systematic search was conducted in PubMed, Web of Science, EMBASE (OVID), MEDLINE (OVID), CINAHL (EBSCO), Cochrane Library, and three Chinese databases from January 2012 to December 2021. Two reviewers independently reviewed publication selection and data extraction. Twenty-nine studies met the inclusion criteria. Most studies (93%) were cross-sectional, and the median (or average) follow-up time ranged from 3months to 9years. Two prospective studies (7%) followed patients longitudinally until 1year after discharge. ECMO survivors had poorer long-term HRQOL than the general population. However, it is comparable to or better than patients with other critical or chronic illnesses. Identified HRQOL assessment instruments show four generic HRQOL instruments, one disease-specific HRQOL instrument, and nineteen single-dimensional instruments. Seven instruments were used in more than three articles. SF-36 (86.2%), IES/IES-R (41.4%), and HADS (37.9%) were the most frequently used instruments. The timing, frequency, and tools for HRQOL assessment of ECMO survivors are variable. No ECMO-specific HRQOL instrument was developed and validated. Further studies on assessment instruments are warranted. Research is also needed to identify interventions that may enhance HRQOL in ECMO survivors.

Abstract 15211: Age and Improvement in Health- Related Quality of Life After Percutaneous Coronary Intervention

Background: The purpose of percutaneous coronary intervention (PCI) is to relieve chest pain, improve health-related quality of life (HRQoL), or reduce the risk of cardiac adverse events and death among patients with coronary artery disease. The aim of this study was to investigate whether age is related to HRQoL improvement in patients undergoing PCI and which clinical characteristics explain the changes in HRQoL during 12 months. Methods: Altogether 301 patients undergoing PCI were divided into three age groups: ≤65 (n=94), 66-74 (n=117), and ≥75 (n=90). Patients responded to the generic 15D and disease-specific SAQ-7 HRQoL instruments at baseline and 1 and 12 months after PCI. Results: The median ages in groups were 60, 69, and 79 years, and the proportions of women were 19.1%, 32.5%, and 35.6%, respectively. Baseline instrument scores were lower in the oldest group compared to the youngest groups (15D p <0.001, SAQ-7 p =0.006). 15D and SAQ-7 improved statistically ( p <0.001 for all) and clinically significantly in all groups at 1 month. At 12 months, the 15D score decreased in all groups but remained statistically and clinically higher compared to baseline in the two youngest groups, and in the oldest group, it was statistically ( p =0.005) and nearly clinically higher (threshold 0.015, Δ score 0.013, 95% CI -0.003-0.030). In the oldest group, deterioration in the 15D between 1 and 12 months was due to dimensions “mobility”, “vision”, and “excretion” but not in “cardiac-related” dimensions. Significant improvement in SAQ-7 score at one-year follow-up was observed in all groups ( p <0.001 for all). The improvements in 15D and SAQ-7 scores at 12 months showed no difference between the age groups ( p =0.381). Conclusions: There was no difference in the improvement of generic or disease-specific HRQoL after PCI between the age groups. Importantly, both 15D and SAQ-7 improved equally in the oldest groups as in the younger patients.

Translation and validation of the Italian version of the Wisconsin Stone Quality of Life Questionnaire (I-WISQOL) for assessing quality of life in patients with urolithiasis

Urolithiasis is a chronic condition, and it has been associated with a significant negative impact on patients' health-related quality of life (HRQOL). Several tools to assess patients' HRQOL have been validated in Italian, however disease-specific HRQOL instruments are still lacking. We aimed to develop and validate the Italian version of the WISQOL (I-WISQOL) in patients with urolithiasis. The Italian version of the WISQOL was developed in a multistep process involving primary translation, back-translation, and pilot testing among a group of patients (N.=10). Patients presenting with urolithiasis were prospectively recruited from the outpatient stone clinics and completed both questionnaire WISQOL and SF-36. Demographic information, as well as medical and surgical data, were obtained through an interview. Internal consistency of the I-WISQOL was obtained with Cronbach's α. Correlation of total scores of the I-WISQOL and SF36 was assessed to determine convergent validity using Spearman Rho. Correlations between clinical variables and results from the I-WISQOL were analyzed to descriptively assess the association of interest. A total of 93 participants were evaluated and completed the Italian version of the I-WISQOL. The I-WISQOL demonstrated excellent internal consistency (Cronbach's α 0.95) and good convergent validity with the validated SF-36 (Spearman Rho r=0.70, P<0.001). Using ANOVA analysis, a significant decline in WISQOL Score was noted with the increasing number of renal colics (P=0.0543), ER visits (P=0.037), number of inpatient hospitalization (P=0.025). At multivariate analysis, worse WISQOL total score was predicted by a greater number of renal colic events (ß=-4.92 [-8.81-1.04], P=0.014) and by a greater number inpatient hospitalization (ß=-7.31 [-14.35 -0.26], P=0.042). The I-WISQOL is an internally consistent and valid instrument to assess HRQOL in Italian-speaking patients with kidney stones. Its use in clinical practice should be implemented in order to tailor the management of each patient.

Sarcopenia and health-related quality of life: A systematic review and meta-analysis.

The decrease of physical abilities and functional decline that can be caused by musculoskeletal conditions such as sarcopenia, can lead to higher levels of dependency and disability. Therefore, it may influence patient reported outcome measures (PROM), such as the health-related quality of life (HRQoL). The purpose of this systematic review and meta-analysis is to provide a comprehensive overview of the relationship between sarcopenia and HRQoL. Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) were followed throughout the whole process of this work. A protocol was previously published on PROSPERO. The electronic databases MEDLINE, Scopus, Allied and Complementary Medicine (AMED), EMB Review - ACP Journal Club, EBM Review - Cochrane Central of Register of Controlled Trials and APA PsychInfo were searched until October 2022 for observational studies reporting a HRQoL assessment in both sarcopenic and non-sarcopenic individuals. Study selection and data extraction were carried out by two independent researchers. Meta-analysis was performed using a random effect model, reporting an overall standardized mean difference (SMD) and its 95% confidence interval (CI) between sarcopenic and non-sarcopenic individuals. Study quality was measured using the Newcastle-Ottawa Scale and the strength of evidence was assessed using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) tool. The search strategy identified 3725 references from which 43 observational studies were eligible and included in this meta-synthesis study. A significantly lower HRQoL was observed for sarcopenic individuals compared with non-sarcopenic ones (SMD -0.76; 95% CI -0.95; -0.57). Significant heterogeneity was associated with the model (I2 =93%, Q test P-value <0.01). Subgroup analysis showed a higher effect size when using the specific questionnaire SarQoL compared with generic questionnaires (SMD -1.09; 95% CI -1.44; -0.74 with the SarQoL versus -0.49; 95% CI -0.63; -0.36 with generic tools; P-value for interaction <0.01). A greater difference of HRQoL between sarcopenic and non-sarcopenic was found for individuals residing in care homes compared with community-dwelling individuals (P-value for interaction <0.001). No differences were found between age groups, diagnostic techniques, and continents/regions. The level of evidence was rated as moderate using the GRADE assessment. This systematic review and meta-analysis combining 43 observational studies shows that HRQoL is significantly reduced in sarcopenic patients. The use of disease-specific HRQoL instruments may better discriminate sarcopenic patients with respect to their quality of life.

Assessment of health-related quality of life in Australian patients with idiopathic pulmonary fibrosis: a comparison of the EQ-5D-5L and the AQoL-8D

PurposeIdiopathic pulmonary fibrosis (IPF) is a progressive and debilitating chronic lung disease with a high symptom burden, which has a substantial impact on health-related quality of life (HRQoL). Our study aimed to assess the suitability of the EuroQol five-dimension (EQ-5D-5L) and the Assessment of Quality of Life- eight-dimension (AQoL-8D) questionnaires in measuring HRQoL as health state utility values (HSUVs) in an Australian IPF cohort.MethodsData for estimation of health state utility values (HSUVs) were collected from participants of the Australian IPF Registry (AIPFR) using self-administered surveys which included the EQ-5D-5L and the AQoL-8D. Data on lung function and disease specific HRQoL instruments were collected from the AIPFR. Performance of the two instruments was evaluated based on questionnaire practicality, agreement between the two instruments and test performance (internal and construct validity).ResultsOverall completion rates for the EQ-5D-5L and AQoL-8D were 96% and 85%, respectively. Mean (median) HSUVs were 0.65 (0.70) and 0.69 (0.72) for the EQ-5D-5L and AQoL-8D, respectively. There was reasonable agreement between the two instruments based on the Bland–Altman plot mean difference (−0.04) and intraclass correlation coefficient (0.84), however there were some fundamental differences. A larger range of values was observed with the EQ-5D-5L (−0.57–1.00 vs 0.16–1.00). The EQ-5D-5L had a greater divergent sensitivity and efficacy in relation to assessing HSUVs between clinical groupings. The AQoL-8D ,however, had a higher sensitivity to measure psychosocial aspects of HRQoL in IPF.ConclusionThe EQ-5D-5L demonstrated superior performance when compared to AQoL-8D in persons with IPF. This may be attributable to the high symptom burden which is physically debilitating to which the EQ-5D-5L may be more sensitive.

The Gilles de la Tourette Syndrome-Quality of Life Scale (GTS-QOL): A Validation in Japanese Patients.

Background: Though Gilles de la Tourette's syndrome (GTS) has significant impact on the quality of life of its patients, measures of health-related quality of life (HR-QOL) specific to adolescents and adults with GTS were not developed until recently. The present study provides evidence on the validity of the Gilles de la Tourette Syndrome-Quality of Life Scale (GTS-QOL), the first disease-specific HR-QOL instrument for GTS patients, for the first time in an East Asian sample.Methods: One hundred and two Japanese individuals aged 13 and above with GTS were included in our study. Internal consistency was evaluated using Cronbach's alpha. The 4-factor structure of the GTS-QOL was assessed using confirmatory factor analysis, using goodness of fit indices, factor loadings of each questionnaire item, and covariances between factors. Validity was assessed using interscale correlations. Convergent and discriminate construct validity was evaluated using correlations with other scales such as the 28-item General Health Questionnaire, the Yale Global Tic Severity Scale, and the short version of the Padua Inventory.Results: Scaling assumptions were met. Internal consistency reliability was high, with a Cronbach's alpha of 0.96. Confirmatory factor analysis revealed sufficient factor loadings and goodness of fit. All measures of goodness of fit corroborated the fit of the 4-factor model. Standardized covariances between factors in the confirmatory factor analysis were >0.8. There were significant correlations with other well-validated scales, and thus convergent and discriminate construct validity was sufficient.Conclusion: The GTS-QOL is a valid and reliable instrument to measure disease-specific HR-QOL of GTS patients in Japan.

Psychometric properties of disease-specific health-related quality of life (HRQoL) instruments for food allergy and food intolerance: protocol for a COSMIN-based systematic review

IntroductionFood allergies and food intolerances can bring burdens on patients and their caregivers and reduce health-related quality of life (HRQoL). An increasing number of disease-specific HRQoL instruments for food allergies...

Health-Related Quality of Life Issues Experienced by Thoracic and Breast Sarcoma Patients: A Rare and Understudied Group.

Thoracic and breast sarcomas constitute a rare subgroup within the sarcoma population. There is limited knowledge about their health-related quality of life (HRQoL) and a valid disease-specific HRQoL instrument is lacking. This qualitative study aimed to investigate the HRQoL issues experienced by a small group of thoracic and breast sarcoma patients. Semi-structured interviews with 19 thoracic and four breast sarcoma patients were conducted and thematically analysed. Physical issues mentioned by both groups were fatigue, sleep disturbances, pain, wound infections, and symptoms related to chemotherapy and radiotherapy. Tightness in the back and restrictions in performing tasks above arm height were specific physical issues for breast sarcoma patients, whereas respiratory problems were only mentioned by thoracic sarcoma patients. Body image issues, changes in mood, fear of recurrence, and living with uncertainty were important mental health issues for both subgroups. Social issues in both groups included challenges in work and relationships, financial difficulties, loss of independence, and limitations in social activities. The identified physical, mental, and social health challenges can significantly impact thoracic and breast sarcoma patients’ HRQoL. Results of this qualitative study will guide personalised supportive care for breast and thoracic sarcoma patients and help in determining the best possible HRQoL measurement strategy for sarcoma patients with different primary sarcoma locations.

The relationship between chronic kidney disease-associated pruritus and health-related quality of life: a systematic review.

ABSTRACTBackgroundChronic kidney disease–associated pruritus (CKD-aP) is a common and burdensome condition for end-stage kidney disease (ESKD) patients, especially those receiving haemodialysis. High-quality evidence of the relationship between CKD-aP and health-related quality of life (HRQoL) can therefore inform clinicians and policymakers about treatment choice and reimbursement decisions.MethodsA systematic literature review and narrative synthesis stratified by study design and HRQoL instrument was conducted to evaluate in adult ESKD patients receiving in-centre haemodialysis the relationship between CKD-aP and HRQoL assessed using multi dimensional generic or condition-specific preference- or non-preference-based measures. MEDLINE, Embase, Web of Science, BIOSIS Citation Index, Cochrane Library and PsycINFO from inception to March 2020 were searched, with two reviewers extracting data independently.ResultsSearches identified 2684 unique records, of which 20 papers relating to 18 unique studies [5 randomised controlled trials (RCTs) and 13 observational studies] were included. HRQoL was assessed using four generic and eight disease-specific measures. The impact of CKD-aP was assessed by comparison of means, linear regression and correlation. Observational studies employing comprehensively adjusted multivariable linear regression largely found associations between CKD-aP severities and HRQoL. Analyses suggest this relationship is partially mediated by the sleep disturbance caused by CKD-aP. RCTs showing improvements in CKD-aP severity were associated with clinically meaningful improvements in HRQoL. Compared with generic measures, disease-specific HRQoL instruments reported greater changes with reduced CKD-aP. Heterogeneity in study design and reporting precluded meta-analysis.ConclusionsCKD-aP severity was found to be associated with a worsening of HRQoL in the majority of observational and RCT studies. Parallel improvements in CKD-aP and HRQoL with interventions may support their use (PROSPERO registration 175035).

Health-related quality of life of multiple sclerosis patients: a European multi-country study

BackgroundInconsistent use of generic and disease-specific health-related quality of life (HRQOL) instruments in multiple sclerosis (MS) studies limits cross-country comparability. The objectives: 1) investigate real-world HRQOL of MS patients using both generic and disease-specific HRQOL instruments in the Netherlands, France, the United Kingdom, Spain, Germany and Italy; 2) compare HRQOL among these countries; 3) determine factors associated with HRQOL.MethodsA cross-sectional, observational online web-based survey amongst MS patients was conducted in June–October 2019. Patient demographics, clinical characteristics, and two HRQOL instruments: the generic EuroQOL (EQ-5D-5L) and disease-related Multiple Sclerosis Quality of Life (MSQOL)-54, an extension of the generic Short Form-36 (SF-36) was collected. Health utility scores were calculated using country-specific value sets. Mean differences in HRQOL were analysed and predictors of HRQOL were explored in regression analyses.ResultsIn total 182 patients were included (the Netherlands: n = 88; France: n = 58; the United Kingdom: n = 15; Spain: n = 10; living elsewhere: n = 11). Mean MSQOL-54 physical and mental composite scores (42.5, SD:17.2; 58.3, SD:21.5) were lower, whereas the SF-36 physical and mental composite scores (46.8, SD:22.6; 53.1, SD:22.5) were higher than reported in previous clinical trials. The mean EQ-5D utility was 0.65 (SD:0.26). Cross-country differences in HRQOL were found. A common predictor of HRQOL was disability status and primary progressive MS.ConclusionsThe effects of MS on HRQOL in real-world patients may be underestimated. Combined use of generic and disease-specific HRQOL instruments enhance the understanding of the health needs of MS patients. Consequent use of the same instruments in clinical trials and observational studies improves cross-country comparability of HRQOL.

Clinical and health-related quality of life outcomes of transfusion-dependent thalassaemia patients in Singapore

IntroductionTransfusion-dependent thalassaemia is associated with complications related to iron overload from frequent red cell transfusions which affect quality of life. We collected data on the clinical outcomes, complications, socioeconomic status and health-related quality of life (HRQoL) of transfusion-dependent thalassaemia patients in Singapore, and analysed the associations between clinical and socioeconomic factors with development of transfusion-related complications and HRQoL scores. Materials and methodsThis was a cross-sectional study of transfusion-dependent thalassaemia patients treated at four major public hospitals in Singapore. Clinical information was obtained from retrospective reviews of medical records. Socioeconomic data and patient-reported compliance to iron chelators were obtained from prospective interviews of patients or caregivers using a questionnaire. A validated, disease-specific HRQoL instrument, the TranQOL, was administered to patients and caregivers during a routine clinic or transfusion visit. ResultsLiver iron loading was the most common transfusion-related complication and occurred in 79% of patients. Cardiac iron loading was noted in 28.3% and endocrine complications were present in 34.2%. Liver iron loading was significantly associated with higher mean ferritin level. Cardiac iron loading was significantly associated with increasing age, higher mean ferritin level and type of iron chelator. Endocrine complications were associated with increasing age, higher mean ferritin level, type of iron chelator and poorer patient-reported compliance to iron chelators. The lowest TranQOL scores were reported by caregiver parents of patients aged less than 18 years. Lower TranQOL scores were significantly associated with increasing age, especially in the 31–50 age cohort, and with reception of social assistance. ConclusionThe main morbidities noted in transfusion-dependent thalassaemia patients in Singapore are from complications associated with iron loading. The cohort of older thalassaemia patients aged 31–50 experienced significantly higher rates of cardiac iron loading, endocrine complications and lower TranQOL scores compared to younger age cohorts.

Quality of Life and Health Care Utilization in the CIRCA-DOSE Study.

This study evaluated the impact of contact force-guided radiofrequency ablation versus cryoballoon ablation on quality of life and health care utilization. Traditional outcome parameters, such as arrhythmia-free survival, are insufficient to evaluate the clinical impact of atrial fibrillation (AF), as it fails to the capture patient- and health system-level differences in treatment approaches. The CIRCA-DOSE (Cryoballoon Vs. Contact-Force Atrial Fibrillation Ablation) study randomly assigned 346 patients with drug-refractory paroxysmal AF to contact force-guided radiofrequency or cryoballoon ablation. Health-related quality-of-life (HRQOL) was assessed at baseline, and at 6 and 12months post-ablation using a disease-specific and generic HRQOL instruments. Health care utilization (hospitalization, emergency department visits, and cardioversion) and antiarrhythmic drug use for the 12months preceding ablation was compared with the 12months following ablation. Disease-specific and generic HRQOL was moderately to severely impaired at baseline and improved significantly at 6 and 12months of follow-up (median improvement in AFEQT [Atrial Fibrillation Effect on QualiTy of Life] score 32.4 [interquartile range: 17.7 to 48.9]). When compared with the 12months pre-ablation, the proportion and absolute number of cardioversions decreased significantly (41.1% vs. 10.1% of patients, 137 vs. 35 events; p<0.0001). Similar significant reductions in emergency department visits (66.7% vs. 25.1% of patients, 224 vs. 87 events; p<0.0001), and hospitalizations (25.5% vs. 14.5% of patients, 86 vs. 50 events; p<0.001) were observed. There were no significant differences between randomized groups. In this multicenter randomized trial, catheter ablation with advanced-generation technologies resulted in a significant improvement in HRQOL and a significant reduction in health care utilization in the year following AF ablation. (Cryoballoon vs. Irrigated Radiofrequency Catheter Ablation [CIRCA-DOSE]; NCT01913522).

Impact of cyclosporin treatment on health-related quality of life of patients with alopecia areata

Introduction Alopecia areata (AA) is a disfiguring disease with substantial psychological burden. No studies explore the efficacy of pharmacotherapy through health-related quality of life (HRQoL) using both disease-specific and generic quality of life (QoL) instruments. We present the first study to elicit health utility from patients with AA and to evaluate the efficacy of cyclosporin in relation to HRQoL using both measures. Methods Participants with moderate to severe AA from a placebo-controlled randomized trial investigating cyclosporin were administered the generic preference-based HRQoL instrument, Assessment of Quality of Life-8D (AQoL-8D) and the disease-specific HRQoL instrument, Alopecia Areata Symptom Impact Scale (AASIS). HRQoL was measured at each study visit and compared to baseline. Results A number of 32 participants were analyzed. The mean health utility was 0.748. At 3 months, the cyclosporin group had trends for greater improvement in HRQoL across 6 of 8 AQoL-8D dimensions and 5 of 7 AASIS symptom domains compared to placebo. HRQoL was lower than Australian population norms across 6 of 8 AQoL-8D dimensions. Conclusions Patients with AA had a mean health utility of 0.748. Treatment with cyclosporin 4 mg/kg/d for 3 months resulted in trends for improvement of HRQoL across multiple dimensions in both disease-specific and generic measures. Capsule summary The mean health utility for patients with AA was 0.748. Patients with alopecia areata have impaired health-related quality of life across 6 of 8 AQoL-8D dimensions compared to population norms. Treatment with oral cyclosporin for moderate to severe AA resulted in trends for improvement in QoL across multiple dimensions.

The content comparison of health-related quality of life measures in heart failure based on the international classification of functioning, disability, and health: a systematic review.

Introduction: Due to the necessity of assessing the health-related quality of life (HRQOL) in heart failure (HF) and the increased use of the International Classification of Functioning, Disability, and Health (ICF) for making a content comparison of measurement instruments, the present study aimed to evaluate the relationship between the instruments and ICF. To this aim, the disease-specific HRQOL instruments in HF were identified, and then psychometric properties and content comparison of included instruments were conducted by linking to ICF. Methods: Disease-specific HRQOL instruments in HF were identified through a comprehensive and systematic search strategy. Then, the psychometric properties of included instruments were determined, and their contents were analyzed and compared based on the ICF coding system. In addition, each instrument was independently linked to ICF by two researchers based on standardized linking rules, and finally their degree of agreement was assessed by the Cohen’s kappa coefficient. Results: Ten instruments including a total of 247 items and 417 concepts were linked to 124 different ICF categories. Further, 39 (31.5%), 65 (52.5%), 13 (10.4%), and 7 (5.6%) categories were linked to body function, activity and participation, environmental factors, and body structure, respectively. According to the content analysis approach and psychometric properties, the appropriate measurement instruments were Kansas City Cardiomyopathy and Minnesota living with HF questionnaires, respectively. Conclusion: Content comparison provides researchers with valuable information on the instrument heterogeneity and overlapping, which results in selecting the most appropriate measurement instrument based on a specific clinical context.

Studio di validazione dell’emPHasis-10 per la popolazione italiana

Introduction: Patients with Pulmonary Hypertension (PH) have a severely impaired health-related quality of life (HRQoL). The emPHasis-10, a disease-specific HRQoL instrument for PH, has recently been developed and validated. The aim of this study was to assess the reliability and validity of the translated version of emPHasis-10 in the Italian language. Materials and Methods: The Italian version of the emPHasis-10 scale was sent by post to a group of 100 PH Italian patients recruited from the Associazione Malati di Ipertensione Polmonare (AMIP). Together with the emPHasis-10 two HRQoL validated scales were also sent: the Medical Outcomes Study 36-item Short Form (SF-36) and the Nottingham Health Profile (NHP). A written Informed Consent was sent with the questionnaires for explicit approval. 70 patients completed the three questionnaires in two occasions seven days apart, the first at T0 and the second at T1, and sent back by post with the signed informed consent. Statistical analysis was performed with the statistical software SPSS version 24. Results: Data showed that the scale has an excellent internal consistency (Cronbach’s Alpha&gt; 0.9) comparable with the values of the original version, and a significant reproducibility (p &lt;0.01) evaluated by comparing the T0 and T1 compilations. The scale correlated with the SF-36 and NHP scales demonstrating a good convergent validity. Discussion: The Italian version of the emPHasis-10 has been shown to be valid and reliable in the Italian population and can be recommended for use in clinical practice.

Health-related quality of life in pre-adolescent liver transplant recipients with biliary atresia: A cross-sectional study

Pediatric recipients of liver transplantation (LT) often report lower Health-Related Quality of Life (HRQOL) than healthy controls when assessed on generic HRQOL measurement tools. The recent addition of the Pediatric Liver Transplant Quality of Life (PeLTQL), a novel disease-specific HRQOL instrument for pediatric LT recipients, into the clinical armamentarium of tools now routinely available to clinical care teams, provides the unique opportunity to identify disease-related challenges in children who have undergone this life-saving intervention. This study assesses HRQOL in pre-adolescent aged patients with a primary diagnosis of biliary atresia (BA) who underwent LT as an infant, using both generic and disease-specific HRQOL instruments validated for children. We also examined modifiable factors associated with HRQOL after pediatric LT. HRQOL was the primary outcome of this study assessed using the disease-specific PeLTQL and the generic Pediatric Quality of Life Inventory 4.0 (PedsQL). Exposure variables of interest included medication status (e.g., monotherapy, dual therapy) and participation in sports. A total of 70 (56% female, mean age 9.89 ± 1.25 years) pediatric LT recipients (mean interval since LT was 9.0 ± 1.26 years) comprised the study cohort. LT recipients reported significantly lower PedsQL Scores relative to the general population. Immunosuppression monotherapy was associated with higher patient-reported PeLTQL Scores, and sports participation was associated with higher parent-reported PedsQL Scores. Pre-adolescents who underwent LT as an infant with BA, self-report low HRQOL on both disease-specific and generic HRQOL tools. Further research targeting sports participation and simplifying immunosuppression may further optimize quality of life years restored by life-saving LT.

Life after per-oral endoscopic myotomy: long-term outcomes of quality of life and their association with Eckardt scores

The clinical efficacy of per-oral endoscopic myotomy (POEM) has been commonly established by reduction in the Eckardt score (<3) after the procedure. However, achalasia can lead to significant impairment in the patient's quality of life that may go beyond the 4 classic achalasia symptoms as measured by the Eckardt score. The aims of our study were to evaluate the effect of POEM on short-term and long-term health-related quality of life (HRQOL) and to assess the association between HRQOL and Eckardt scores. Single-center, prospective, cohort study of consecutive POEMs during a 3-year period. Eckardt and HRQOL scores as measured by the short-form survey questionnaire (SF-36) were obtained at baseline and at various intervals after POEM. Comparison of the mean scores was described by using univariate linear regression. The association between Eckardt scores and HRQOL were calculated by using a linear, mixed-model analysis. POEM was performed in 143 consecutive patients (54% male; mean ± standard deviation [SD] age, 56.9 ± 17.9 years). At long-term follow-up (mean 16.4 months, range 12-40), both the HRQOL baseline mental and physical component scores improved significantly from 61.5 ± 2.2 to 71.2 ± 3.6; (P< .021) and from 55.8 ± 2.2 to 63.6 ± 3.3; (P=< .034), respectively. Mixed-model analysis showed a significant association between Eckardt and all HRQOL scores (P< .001). POEM improved all dimensions of HRQOL as measured by the SF-36 survey at both short-term and long-term follow-up. A strong association was seen between Eckardt scores and all HRQOL domains. Further studies with the use of disease-specific HRQOL instruments are warranted. (Clinical trial registration number: NCT01832779.).

Late consequences of venous thromboembolism: Measuring quality of life after deep vein thrombosis and pulmonary embolism

The identification of specific post-thrombotic complications as well as an increased appreciation of the importance of patient-reported outcome measures have triggered an interest in studying health-related quality of life (HRQoL) in patients with a history of venous thromboembolism (VTE).In order to enable a comprehensive assessment of HRQoL in clinical trials it is recommended that both generic and disease-specific questionnaires be used. SF-36 and EQ-5D are the most widely used generic questionnaires in VTE studies, whereas several disease-specific questionnaires have been developed to evaluate HRQoL following deep vein thrombosis (DVT) and in chronic venous disease (CVD). The most widely used of these is the VEINES-QOL/Sym, which was developed for CVD. VT-QOL and DVTQOL are both questionnaires developed to evaluate HRQoL after DVT, while PEmb-QoL is currently the only available disease-specific questionnaire developed specifically for pulmonary embolism (PE).Although studies show inconsistent results, patients who sustain DVT seem in general to report similar HRQoL compared to the general population. However, patients who develop PTS after DVT report poorer HRQoL using both generic and disease-specific questionnaires. HRQoL appears to be impaired in patients who sustain PE compared to the general population. In these patients, persistent dyspnea after PE has been shown to be a predictor of reduced HRQoL.Further work is needed to develop practical, patient-derived, valid, reliable and responsive disease-specific HRQoL instruments covering the specific aspects of these diseases.

The development of a health-related quality-of-life instrument for young people with narcolepsy: NARQoL-21

BackgroundNarcolepsy is a lifelong sleep disorder with a prevalence of between 0.03% and 0.06% and onset at around puberty. It is associated with psychiatric comorbidities and cognitive difficulties. No valid and reliable condition-specific health-related quality-of-life (HrQoL) instrument has been developed for this population.MethodsA questionnaire based on four mixed-gender age-defined focus group discussions and a patient panel analysis was administered to young people with narcolepsy and a control group. External reliability was measured by a test-retest procedure and internal reliability was measured using Cronbach’s alpha. Convergent validity with the KIDSCREEN-10 index was assessed using with intraclass correlation coefficients (ICC) and receiver operating characteristic (ROC) curves. Factor analysis techniques were used to identify suitable items and confirm the factor structure. Baseline values were assessed for convergent validity, ceiling effects, agreement and sensitivity. Comparison with KIDSCREEN-10 was made on the basis of area under the curve (AUC).ResultsOne hundred young people with narcolepsy and 95 control subjects returned questionnaires. The factor structure revealed two main factors with five domains and 21 questions, which was confirmed with confirmatory factor analysis. The domains of the NARQoL-21 showed good independence while the floor and ceiling effects were acceptable. The external reliability (0.928), convergent validity (rs = 0.769) and internal consistency (Cronbach’s alpha = 0.886) were excellent. A Bland–Altman plot revealed some proportional bias. Good discriminant validity was detected for control/patient (Cohen’s d = 2.114). ROC analysis showed significantly better AUC for NARQoL-21 (0.939) than KIDSCREEN (0.877). A cut-off score equivalent to KIDSCREEN-10 for suboptimal HrQoL which maximized sensitivity (84%) and specificity (92%) was found at NARQoL-21 score below 42.ConclusionsEstablishing the validity of a disease-specific HrQoL instrument in a population of people with a rare condition poses significant challenges. The mixed-methods approach adopted here has resulted in a questionnaire of 21 items with good discrimination and convergent validity, and excellent internal and external reliability, allowing precise and stable measurements. The cut-off score can be useful to identify patients with very poor HrQoL and thus improve the design of treatment options. Further testing in a longitudinal cohort is recommended in order to establish responsiveness.

Determinants of generic and specific health-related quality of life in patients with Parkinson's disease.

ObjectivesGeneric and disease-specific health-related quality of life (HRQoL) instruments may reflect different aspects of lives in patients with Parkinson’s disease (PD) and thus be associated with different determinants. We used the same cluster of predictors for the generic and disease-specific HRQoL instruments to examine and compare the determinants of HRQoL.MethodHRQoL was measured in 92 patients with PD by the 36-item Short-Form Health Survey (SF-36) and the 39-item Parkinson’s Disease Questionnaire (PDQ-39). The predictors included demographic and disease characteristics, and motor and non-motor symptoms. Multiple regression analyses were used to identify HRQoL determinants.ResultsDepressive symptoms and motor difficulties of daily living were the first two significant determinants for both instruments. The other significant determinant for the SF-36 was fatigue and non-motor difficulties of daily living, and for the PDQ-39 was motor signs of PD.ConclusionsThe results suggest the importance of the evaluation and intervention focused on depressive symptoms and motor difficulties of daily living in patients with PD. In addition, the SF-36 seems more related to non-motor symptoms, while the PDQ-39 appears more associated with motor symptoms. This information is important for understanding results from these two instruments and for choosing which to use.