Cross‐cultural development and psychometric evaluation of a patient‐reported health‐related quality of life questionnaire for adults with haemophilia

Summary. Co‐morbidities of haemophilia, such as arthropathy and blood‐borne infections, can adversely affect the quality of life of adult patients with haemophilia. The purpose of this study was to develop and validate a haemophilia‐specific health‐related quality of life questionnaire for adults (HAEMO‐QoL‐A). Subjects with varying severities of haemophilia completed the HAEMO‐QoL‐A at baseline and 4 weeks. Other assessments included the SF‐36 and Health Assessment Questionnaire – Functional Disability Index (HAQ‐FDI). Two‐hundred and twenty‐one participants completed the 41‐item HAEMO‐QoL‐A covering six domains (Physical Functioning, Role Functioning, Worry, Consequences of Bleeding, Emotional Impact and Treatment Concerns) and four independent items. Internal consistency was good‐to‐excellent (Cronbach’s α‐range: 0.75–0.95). Test–retest reproducibility was good, with intraclass correlation coefficients >0.80 except for the Emotional Impact domain (0.79). Concurrent validity between the HAEMO‐QoL‐A total and subscale scores and all SF‐36 subscale scores were generally good (correlations range: 0.13–0.87). Significant correlations between the HAEMO‐QoL‐A and the HAQ‐FDI ranged from ?0.14 to ?0.69. There were non‐significant correlations with the Treatment Concerns subscale and with the Worry subscale. The HAEMO‐QoL‐A discriminated significantly between adults with haemophilia by severity and HIV status. The Physical Functioning subscale discriminated between patients receiving prophylactic or on‐demand therapy. The HAEMO‐QoL‐A is a valid and reliable instrument for assessing quality of life in haemophilia patients.     

Validation of the Sarcoidosis Health Questionnaire in a non-US population

Background and objective: Sarcoidosis is a multi‐system disease with an unpredictable course and variable clinical manifestations that are often associated with impaired quality of life. The Sarcoidosis Health Questionnaire (SHQ), which was developed for an 80% African American population, assesses health‐related quality of life in sarcoidosis patients. The aim of this study was to validate the SHQ in a predominantly European population of sarcoidosis patients. Methods: Consecutive outpatients (n = 92) with sarcoidosis, who were attending a teaching hospital clinic, completed three questionnaires (SHQ, Short Form (SF) 36, Fatigue Assessment Scale (FAS)) and pulmonary function tests were performed. Results: The mean age of the patients was 51 years, 52% were males and 74% were of European ethnicity. The mean number of organs involved was 1.3, with pulmonary involvement in 95% of patients (mean forced expiratory volume in 1 s 74.4%, forced vital capacity 84.6%). Seventy percent of patients had current symptoms and 26.5% were receiving immunosuppressant therapy. The SHQ total score (mean 5.13) was significantly correlated with the SF 36 physical component score (46.7, r = 0.78) and the FAS (20.8, r = ?0.7) but only weakly correlated with pulmonary function. There were significant differences in SHQ scores when patients were stratified according to symptoms, oral therapy, health status (P < 0.0001 for all), forced expiratory volume in 1 s ≥70% (P = 0.008) and forced vital capacity ≥70% (P = 0.01). Conclusions: The SHQ correlated well with health‐related quality of life and fatigue measures in a predominantly European population of sarcoidosis patients, despite differences in organ involvement and disease burden, when compared with the development study.     

Validation of the Finnish ISAAC questionnaire on asthma against anti-asthmatic medication reimbursement database in 5-year-old children

Background: Valid identification of childhood asthma at the population level for epidemiological purposes remains a challenge. We aimed at validating the Finnish version of the International Study of Asthma and Allergies in Childhood (ISAAC) questionnaire based on parental‐reported childhood asthma. Materials and Methods: The ISAAC questionnaire has been validated against anti‐asthmatic medication reimbursement data of the Finnish Social Insurance Institution, being the gold standard, among 2236 5‐year‐old consecutively born children (1996–2004) carrying human leukocyte antigen (HLA)‐conferred susceptibility to type 1 diabetes. Two combined questionnaire questions (any wheezing symptom or use of asthma medication during the preceding 12 months plus ever asthma; any wheezing symptom or use of asthma medication during the preceding 12 months plus ever doctor‐diagnosed asthma) were validated against valid reimbursement with purchase of at least one anti‐asthmatic medication during a 12‐month period. The validity of the questionnaire was estimated as the sensitivity, specificity, positive predictive value, negative predictive value, and Youden's index. Results: The sensitivity 0.98 [95% confidence interval (CI) = 0.92–0.99]; specificity 0.98 (95% CI = 0.97–0.98); negative predictive value 1.00 (95% CI = 1.00–1.00); and Youden's index 0.96 (95% CI = 0.96–0.96) were the same for each of the two sets of combined questions. The positive predictive value for the first combined question was 0.63 (95% CI = 0.55–0.71), while it was 0.64 (95% CI = 0.57–0.72) for the second combined question. Conclusion: The Finnish ISAAC questionnaire was highly valid and is an acceptable instrument for the survey of the prevalence of parental‐reported childhood asthma for epidemiological purposes. Please cite this paper as: Nwaru BI, Lumia M, Kaila M, Luukkainen P, Tapanainen H, Erkkola M, Ahonen S, Pekkanen J, Klaukka T, Veijola R, Simell O, Knip M and Virtanen SM. Validation of the Finnish ISAAC questionnaire on asthma against anti‐asthmatic medication reimbursement database in 5‐year‐old children. Clin Respir J 2011; 5: 211–218.     

无创呼吸机治疗COPD伴重度CO2潴留的疗效观察

目的探讨无创机械通气在慢性阻塞性肺病伴重度CO2潴留患者中的治疗效果。方法回顾性分析2005年12月到2007年10月我科收治的30例COPD病人资料,均为PaCO2〉80mmHg的COPD伴Ⅱ型呼衰患者,在常规药物治疗及氧疗基础上应用无创呼吸机辅助通气（NIPPV）治疗,通过自身对照观察患者NIPPV治疗前、后血气指标变化和病情改善程度。结果30例患者中2例不能耐受无创通气,放弃治疗,24例使用无创后好转,病情稳定出院,4例无创通气失败,改行气管插管,死亡2例。NIPPV治疗有效患者2h后血气指标明显改善,PaCO2显著下降（P〈0．001）,48h后病情明显好转。结论无创正压机械辅助通气可显著改善COPD伴重度高碳酸血症患者,疗效肯定。     

Validation of the Japanese version of the Rome II modular questionnaire and irritable bowel syndrome severity index

Background Instruments for measuring the presence and severity of specific irritable bowel syndrome (IBS) symptoms, comparable to those used in Western countries, have been lacking in Japan. The aim of this study was to develop, validate, and confirm the reliability of the Japanese version of the Rome II modular questionnaire for IBS (RIIMQ-J) and the IBS severity index (IBSSI-J). Methods Forty-nine patients in the university hospital with chronic or recurrent abdominal pain and discomfort and/or altered bowel habits were enrolled. With Rome II criteria, 27 patients were diagnosed as having IBS, and the other 22 patients were evaluated as having other functional bowel disorders (FBDs). The English versions of RIIMQ and IBSSI were translated into Japanese. After back-translation and approval of the questionnaire, subjects completed both questionnaires twice within 14 days. Results Cronbach's alpha of the RIIMQ-J was high (0.72). The sensitivity of RIIMQ-J for the diagnosis of IBS was also high (89%). The specificity of RIIMQ-J for denial of IBS among patients with other FBD was satisfactory (73%). The IBSSI-J showed high internal consistency (0.69) and reproducibility (intraclass correlation coefficient, 0.86, P < 0.001). Conclusions The RIIMQ-J and IBSSI-J are valid, reliable, and appropriate instruments for detecting and assessing the severity of IBS status in Japanese patients.     

Adherence to prophylaxis is associated with better outcomes in moderate and severe haemophilia: results of a patient survey

Severe haemophilia is associated with bleeding into joints and development of arthropathy. Prophylactic treatment with infusion of replacement clotting factor is known to prevent bleeding, preserve joint functioning and result in higher health‐related quality of life (HRQoL) than episodic treatment; however, adhering to standard prophylaxis schedules can be difficult, and little is known about the relationship between adherence to prophylactic treatment and outcomes. The aim of this study was to assess the relationship between self‐reported adherence to prophylaxis and health outcomes, including HRQoL and bleeding episodes. Adults with haemophilia (n = 55) and caregivers of children with haemophilia (n = 55) in Australia, Canada, and the United States completed an online questionnaire which included measures of HRQoL (SF‐12v2 for adults and SF‐10 for caregivers of children), self‐reported bleeding episodes, and the VERITAS‐Pro measure of adherence to prophylaxis in haemophilia. Regression analysis was used to test the association between VERITAS‐Pro total score and outcomes. Poorer adherence (higher VERITAS‐Pro scores) was associated with a greater number of self‐reported bleeding episodes in the past year among adults (p < 0.01), more days of work/school missed among paediatric patients (p < 0.01), and lower physical health status scores among paediatric patients (p < 0.05). This study highlights the benefits of adherence to prophylaxis among those with severe haemophilia and provides evidence for the utility of the VERITAS‐Pro by demonstrating a relationship between adherence and outcomes.     

Psychometric evaluation of a patient-reported outcome measure in pancreatic exocrine insufficiency (PEI)

Background/objectives

Pancreatic exocrine insufficiency (PEI) is commonly caused by chronic pancreatitis (CP) or cystic fibrosis (CF). There are no PEI-specific patient-reported assessments of symptoms and impacts. The PEI Questionnaire (PEI-Q) was developed through qualitative research with PEI patients and expert clinical input. This study evaluated the psychometric properties of the PEI-Q.

Validation of the English version of the quality of life questionnaire of the European Foundation for Osteoporosis (QUALEFFO) in Malaysia

Background: Osteoporotic fractures will soon become a common problem in Asian countries including Malaysia, as the growth in the elderly population will be more marked in this region. This leads to loss of independence and reduced quality of life (QOL). QOL is used as an outcome measure in clinical trials to focus the management of diseases on the patient rather than the disease. To date, no such instrument for measuring QOL of osteoporosis patients has been validated in Malaysia. Aim: To investigate the reliability and validity of the English version of the Quality of Life Questionnaire of the European Foundation for Osteoporosis (QUALEFFO) in Malaysia. Methods: QUALEFFO was administered twice to postmenopausal osteoporotic women on alendronate. The patient group consisted of 46 women with back pain while the control group consisted of 42 women without back pain. Results: High internal consistency was seen in all domains in the QUALEFFO with Cronbach α of 0.74–0.95 and 0.53–0.89 in the patient and control group, respectively. Factor analysis also showed that each domain consisted of one component except for the social domain. The test‐retest reliability showed high correlation coefficient in all domains (0.50–0.90, P < 0.001). Patients with back pain showed significantly worse QOL in the overall total QUALEFFO score compared to the control group (P < 0.0001). Conclusions: The English version of QUALEFFO was found to be reliable and valid for the evaluation of patients with osteoporosis and who understand English in Malaysia. This study also indicates that patients with back pain have poorer QOL.     

哮喘控制问卷与哮喘生命质量问卷在哮喘患者中的应用价值研究

目的探讨哮喘控制问卷（ACQ）与哮喘生命质量问卷（AQLQ）在哮喘患者中的应用价值。方法选取中国医科大学附属第一医院呼吸内科就诊的哮喘患者132例,其中男性60例,女性72例,平均年龄（47．64-12．3）岁。所有患者填写ACQ、AQLQ问卷并完成肺功能测试。按FEV-％pred将患萏分为三组：1组FEVi％pred≥80％;2组60％≤FEV1％pred〈80％;3组FEV1％pred〈60％。采用Pearson相关分析ACQ分值、AQLQ分值与肺功能指标之间的相关性。结果3组患者的ACQ、AQLQ评分差异有统计学意义（F：32．27、4．65,P〈0．01）,且ACQ、AQI。Q评分能很好的反映肺功能的差异。ACQ评分与肺功能指标呈负相关,AQI。Q评分与肺功能指标呈正相关。结论哮喘患者的肺功能指标与ACQ评分、AQLQ评分有很好的相关性,能更准确的评价患者病情,ACQ与AQLQ在哮喘患者中有很好的应用价值。     

Bone density and health‐related quality of life in adult patients with severe haemophilia

Summary. Severe haemophilia and reduced bone density can negatively influence perception of patient’s health‐related quality of life (HRQoL), especially considering future aspects, the risk of losing independence or pain suffering. The aim of this study was to assess levels of HRQoL in severe haemophilia patients and to compare HRQoL to those of the general population as well as to determine whether reduced bone density is correlated to the perceived HRQoL. Patients were divided into two groups based on timing of being treated with prophylaxis: Group A (started prophylaxis at age of ≤3 years; n = 22); Group B (at age of >3 years; n = 15). The bone mineral density (BMD g cm^?2) of different measured sites was measured by dual energy X‐ray absorptiometry (DXA). HRQoL was assessed using SF‐36 questionnaire. Group A have mean BMD T‐score >?1.0 (i.e. normal score) at all measured sites, and have almost similar scores in the SF‐36 domains compared with the reference population. Group B have mean BMD T‐score ?1.0 at lumbar spine and total body, and their scores in the SF‐36 domains were lower compared with the reference population. Moreover, significant correlations were found between BMD at femoral neck and total body with physical domains. With adequate long‐term prophylaxis since early childhood, adult patients with haemophilia report a comparable BMD and HRQoL to the Swedish reference population. Reduced BMD in group B correlated with impaired physical health, which underscores the importance of early onset of adequate prophylactic treatment.     

Impact of obesity on quality of life and depression in patients with heart failure

BACKGROUND: The effect of obesity on health related quality of life (HRQOL) and depression in a number of disease states is well documented, but its impact in heart failure (HF) patients remains speculative. We therefore examined the relationship between obesity, HRQOL, and depression in 358 patients with HF. METHODS AND RESULTS: Comparative analyses were conducted to determine if body mass index (BMI) was associated with HRQOL and depression in three groups of patients with HF-normal weight (BMI 18.5-24.9 kg/m2, n = 100), overweight (BMI 25-29.9 kg/m2, n = 141), and obese (BMI > or = 30 kg/m2, n = 117). Obese patients were younger than normal and overweight participants; all other demographic and clinical characteristics were similar. HRQOL and depression scores were significantly higher (worse) for obese patients. Body mass index was significantly correlated with all 3 scales of HRQOL (overall, r2 = .160; physical, r2 = .162; and mental, r2 = .217) as well as with depression (r2 = .166). CONCLUSION: Obese patients with HF are more likely to have poorer HRQOL, physical health, emotional well-being and depressive symptoms. Poorer HRQOL is predictive of worse outcomes in patients with HF; however, given the apparent obesity paradox in HF, further investigation of the impact of obesity in HF is urgently required.