Multivariate analysis of factors influencing quality of life and utility in patients with hemophilia (original) (raw)
Health Related Quality of Life (HRQoL) in patients with Hemophilia
2019
Background:Besides striving to achieve greater strides in treating Hemophilia, the importance of assessing patient’s perspective with Hemophilia is the need of the hour. Aim:A study to assess the Health Related Quality of Lifeamong patients with Hemophilia in the Hematology Outpatient Department of Christian Medical College, Vellore Methodology: A Non experimental descriptive design was undertaken. A total of 120 subjects with Hemophilia aged between 18-59 years were selected based on total enumeration sampling technique. The HRQoL was assessed using ‘A36 Hemophilia-QoL’questionnaire. Descriptive and inferential non parametric statistics such as frequency distributions, mean, standard deviation, ANOVA, Independent‘t’ test were used in this study Results: 83.3% of the population had Hemophilia ‘A’. 87.5% had severe Hemophilia. 87.5% of the population had joint bleed. 74.2% had knee as a target joint. Range of motion was predominantly impaired in knee(75.8%) and elbow(60.8%) joints. A...
Haemophilia, 2005
Summary. Although there is a worldwide interest in the assessment of health-related quality-of-life (HRQoL) in haemophilia patients, no non-disease specific instruments (for adults) are readily available. In this paper, a haemophilia-specific quality-of-life assessment measure for adults (the Hemofilia-QoL questionnaire) has been developed and tested for psychometric properties in 121 adults with haemophilia living in Spain. The Hemofilia-QoL questionnaire is a self-report modular instrument that assesses nine relevant HRQoL domains for patients with haemophilia (e.g. physical health, daily activities, joint damage, pain, treatment satisfaction, treatment difficulties, emotional functioning, mental health, relationships and social activity). Psychometric examination involved the assessment of data quality, scaling assumptions, reliability (internal consistency and test–retest) and validity (concurrent; external clinical criterion and sensitivity). The Hemofilia-QoL 36-item version questionnaire had acceptable internal consistency and retest reliability values. The questionnaire shows excellent concurrent validity (with the SF-36 Health Survey) and external clinical criterion validity (haemophilia clinical status) and sensitivity (health status changes) as well. The Hemofilia-QoL is now available for adult assessment and is ready for use in clinical research in Spain.
Health status and health‐related quality of life associated with hemophilia
American Journal of Hematology, 2002
The hemophilias are a group of disorders associated with a chronic burden of morbidity and early mortality. Improvements in these adverse features have been achieved by the use of clotting factor concentrates within comprehensive centers of specialized care providing home infusion programs. Offsetting effects from transfusion-transmitted hepatitis and HIV infection are in recent decline. The net impact of these changes merits assessment. To test the a priori hypotheses that increasing severity of factor VIII deficiency would be associated with an increasing burden or morbidity and that hepatitis and HIV positivity would impair health status further, a cross-sectional study of a populationbased cohort was undertaken in a regional hemophilia program in Ontario, Canada. A survey was made of mild, moderate, and severe hemophiliacs over 13 years of age who self-reported their health status using a standard 15-item questionnaire. The responses were converted to levels in the Health Utilities Index Mark 2 (HUI2) and Mark 3 (HUI3) health status classification systems to form multi-element vectors from which singleattribute morbidity and overall health-related quality of life utility scores were determined. The burden of morbidity was greater in hemophiliacs than in the general population and correlated with the category of disease (mild < moderate < severe). Hepatitis and HIV positivity conferred additional burdens of morbidity, which were mainly in the attributes of mobility (HUI2), ambulation (HUI3), and pain (HUI2/3), all of these differences reaching levels of statistical significance. Despite demonstrable improvements in the safety, effectiveness, and utilization of clotting factor concentrates, hemophiliacs continue to experience an important burden of morbidity. Measurement of this burden, as reported here, provides a basis for future economic evaluation of the costs and consequences of health care interventions provided to this population. Am.
Haemophilia, 2004
A haemophilia-specific health-related quality-of-life questionnaire (named ÔHemofilia-QoLÕ) was developed to assess quality-of-life in adults with haemophilia, and was psychometrically tested. Seventy-three interviews with haemophilia patients and health care professionals were used to generate the items included in the questionnaire, and expert ratings on the items formulated were used to screen them for potential omission. This was followed by psychometric testing in a sample of 35 patients. Preliminary psychometric testing of the revised questionnaire version, which contains 10 domains (physical health, physical role, joint damage, pain, treatment satisfaction, emotional role, mental health, social support), showed acceptable reliability (a ¼ 0.94 for the Hemofilia-QoL total score) and validity, and this will be examined in a subsequent study with a larger patient sample.
Pediatric Hematology and Oncology, 2010
The aim of this study is to describe the health status, health care received, and their impact on the quality of life in patients with hemophilia. Patients with severe factor VIII or IX deficiency without inhibitors or other chronic disease were enrolled. Turkish version of the Hemophilia-Specific Quality of Life Index (Haemo-QoL) questionnaire was administered to the pediatric patients aged 4 to 16 years and Haem-A-QoL to the adult patients. Joints were evaluated according to the World Federation of Hemophilia (WFH) orthopedic joint scores.Thirty-nine children/adolescents and 31 adult patients were enrolled. Mean Haemo-QoL scores were 39.6 ± 15.0 for the children and mean Haem-A-QoL 47.4 ± 14.1 for the adult patients, respectively. Internal consistency reliability was generally sufficient. Total Cronbach's alpha coefficient was >.70 (range .77-.96) in all the age groups. Mean total WFH orthopedic joint scores were 1.83 ± 2.7, 4.9 ± 4.96, and 6.94 ± 6.15 in 4-7, 8-12, and 13-16-year-old groups, respectively. They were more impaired in the adult patients (16.23 ±14.12). These results show that the Turkish version of the Haemo-QoL and Haem-A-QoL are reliable instruments to measure the quality of life in the pediatric and adult patients with
Background and Objectives. Health Related Quality of Life (HRQoL) is an important health outcome measure in haemophilia. The aim of this study was to assess the psychometric properties of the Greek version of Haem-A-QoL, a disease-specific questionnaire for haemophiliacs. Methods. Haem-A-QoL and SF-36 were administered to 118 adult haemophilia patients. Hypothesized scale structure, internal consistency (Cronbach's), and test-retest reliability, as well as various types of construct validity were evaluated. Results. Scale structure of Haem-A-QoL was confirmed, with good item convergence (87%) and discrimination (80.6%) rates. Cronbach's was >0.70 for all but one dimension (dealing) and test-retest reliability was significantly high. The strength of Spearman's correlations between Haem-A-QoL and SF-36 scales ranged from 0.25 to 0.75 (< 0.01). Multiple stepwise linear regression analysis revealed that all but one Haem-A-QoL dimensions were important predictors of SF-36 scales. Known-groups comparisons yielded consistent support of the instruments' construct validity and significant relationships were identified for age, educational level, haemophilia type, disease severity, and viral infections. Conclusion. Overall, the psychometric properties of the Greek version of Haem-A-QoL, resulting from this first time administration of the instrument to Greek adult haemophiliacs, confirmed it as a reliable and valid questionnaire for assessing haemophilia-specific HRQoL in Greece.
2021
Background: Hemophilia affects the patients′ life in many aspects. The major concerns are restriction on physical activities, life-threatening bleeding, arthropathy, etc., and worsening mental issues like anxiety. This study aimed to evaluate the health-related quality of life in hemophilia patients.Methods: In this cross-sectional study, 147 patients with hemophilia had been referred to special patients′ clinic at Kermanshah province (IRAN) for comprehensive medical care services. The patients who met the criteria of this study were selected using counting sampling method and were assessed with the quality of life in hemophilia patients′ questionnaire (A36 Hemofilia-QoL® questionnaire).Results: Out of 147 patients with hemophilia, 139 were male and 8 were female. The mean age of the subjects was 25.85±15.54 years. The results demonstrate that the total score of 65 patients (44.2%) had poor quality, 60 patients (40.8%) had moderate quality and 22 patients (15.0%) had good quality of...
Quality of life assessment in clinical practice in haemophilia treatment
Haemophilia, 2006
The modern management of haemophilia has greatly influenced not only survival of patients, their clinical symptoms and orthopaedic outcome but also their perceived quality of life (QoL). QoL measures recently became an essential part of clinical trials being one of the most important patient-rated outcomes (PROs). Moreover, QoL assessment is essential in pharmacoeconomics. In clinical trials, not only clinical outcomes but also the so-called PROs and health-economic outcomes are included. Different types of economic evaluations may be conducted in order to describe the economic burden of a disease condition, to determine the cost of care and to assess and evaluate alternative treatments in terms of both costs and effects as well as benefits and outcomes with the aim of optimizing the use of resources. For these evaluations, the following analyses are performed: descriptive cost of illness study, incremental cost-effectiveness analysis, incremental cost-utility analysis and incremental cost-benefit analysis. By contrast, PROs are derived from direct patient reports and they allow to evaluate the impact of a disease and its treatment on patientsÕ well-being and functioning. PROs include health-related quality of life, patient preferences/utilities, treatment satisfaction and other PROs such as functional assessment, etc. Choosing a QoL measure, study-and instrument-related aspects have to be taken into account. Finally, QoL assessment will become a part of the regular clinical assessment of persons with haemophilia, in order to provide trustworthy data of perceived well-being to be compared over time and in order to assess treatment efficacy and quality of care.
Measurement properties of the Haem-A-QoL in haemophilia clinical trials
Haemophilia, 2016
Introduction: Patients with haemophilia on long-acting prophylactic treatment may experience an improvement in health-related quality of life (HRQoL) through reductions in breakthrough bleeds and associated complications, including long-term joint damage, compared with episodic treatment. Aim: This analysis examined clinical trial data to understand the psychometric characteristics (reliability, validity and sensitivity to change over time) of the Haem-A-QoL Questionnaire in adult males with haemophilia. Methods: Two recent, multinational, Phase 3 clinical trials of new, long-acting factor concentrates (A-LONG: rFVIIIFc; B-LONG: rFIXFc) assessed HRQoL in adolescent and adult males with severe haemophilia A or B respectively. The adults' baseline assessments, via the 46-item Haem-A-QoL Questionnaire, and change over time at the 6-month assessment were used in the psychometric analyses. Results: Internal consistency reliability was adequate (Cronbach's alpha > 0.70) for nine of the 10 Haem-A-QoL domains and for 'Total Score' in both trials at baseline (A-LONG, n = 133; B-LONG, n = 73). At baseline, several Haem-A-QoL domains and 'Total Score' demonstrated known-groups and convergent validity when compared with other trial measures, including the EQ-5D (items and total scores) and joint impairment. Change score correlations (baseline to 28 weeks) between the EQ-5D and the Haem-A-QoL 'Total Score', and 'Physical Health' and 'Feelings' domains were moderate in magnitude (│r│ ≥ 0.33; P < 0.03), demonstrating sensitivity to change for these outcome measures in ALONG. Conclusion: These psychometric analyses provide evidence of the reliability, validity and ability to detect change of the Haem-A-QoL to assess the HRQoL of adult males with severe haemophilia A and B in longitudinal clinical trials.
Health Status and Quality of Life in Patients with Severe Hemophilia A: A Cross-Sectional Survey
Hematology Reviews, 2019
Among different groups of hemophiliacs, those suffering from Severe Hemophilia A (SHA) are most vulnerable to the complications of the disease. This study investigated the Health-Related Quality of Life (HR-QoL) among adult patients with SHA. A cross-sectional study was designed to gather demographic and clinical information from adult patients with SHA. Patients with inhibitors were excluded. The remaining were asked to complete the HR-QoL questionnaire after being examined for joint health using the Hemophilia Joint Health Score (HJHS). The HR-QoL and joint conditions were measured in 38 patients. The mean EQ-5D value scores were 0.46 (SD=0.23) while the mean Visual Analogous Scale score was 50 (SD=18.7). The clinical examination of patients indicated that the HJHS were as follows: eight patients had a score of 55-75, 12 patients had a score of 40-55, 7 of them (25-40) and 11 patients had a score of 10-25. The results obtained from this study showed that HR-QoL in hemophilia patients was considerably low. Pain, anxiety/depression, and motion limitations were the main causes of the disutility for these patients respectively.
Haemophilia, 2013
In Belgium, where haemophilia affects approximately 1:7000 people (2011), data on patients' quality of life (QoL) is scarce. This project aims to assess physical and mental QoL (P-QoL and M-QoL) simultaneously, and to analyse the influence of different variables on these two aspects of QoL. After Ethics Committee approval, we contacted 84 adult haemophilia A (HA) and haemophilia B (HB) patients, without current inhibitors, on replacement therapy (on-demand or secondary prophylaxis), regularly followed up at our comprehensive treatment centre. Seventy-one (n = 59 HA, n = 12 HB) replied to our questionnaire, which included the SF36v2 QoL assessment forms. We analysed two groups of variables: one including variables previously associated with decreased QoL, and another including variables with unclear impact on QoL (e.g. patients' understanding of haemophilia-related issues, economical concerns). In our population (mean AE SD age: 45.2 AE 14.7 years old), P-QoL appeared more reduced than M-QoL. P-QoL was strongly influenced by the number of arthropathies while M-QoL was primarily affected by patients' concern of personal costs due to haemophilia. Among this latter group, having knowledge of insurance coverage had a positive impact on M-QoL. Scores did not depend on haemophilia type. QoL was impaired in our haemophilia patients. A simultaneous assessment of P-QoL and M-QoL confirmed the benefit of primary prophylaxis in P-QoL, while originally pointing out the major burden of patients' concerns and poor understanding of haemophilia-related economical issues on their M-QoL. This might become a particularly challenging issue in times of financial crisis.
Health and Quality of Life Outcomes
Background: This study examined the psychometric properties of version 2 of the SF-12 Health Survey (SF-12v2) among adults with hemophilia in the United States. Methods: This study employed a cross-sectional design using web-based and paper-based self-administered surveys. Hemophilia patients were recruited using an online panel and at a hemophilia treatment clinic. The psychometric properties of the SF-12v2 were assessed in terms of construct validity, internal consistency reliability, and presence of floor and ceiling effects. Results: A total of 218 adults with hemophilia completed the survey, with most recruited via the online panel (78%). Confirmatory factor analysis using the WLSMV estimator in Mplus supported a two-factor model for the SF-12v2 where the physical functioning, role physical, bodily pain, and general health items loaded onto a latent physical factor (LPF) and the role emotional, mental health, social functioning, and vitality items loaded onto a latent mental factor (LMF). Model fit statistics for the two-factor model were: Chi-square [df] = 172.778 [48]; CFI = 0.972; TLI = 0.962; RMSEA [90% CI] = 0.109 [0.092-0.127]; WRMR = 0.947. Correlated residuals for items belonging to similar domains were estimated and there was a significant correlation between LPF and LMF. All standardized factor loadings were strong and statistically significant, indicating adequate convergent validity. Item-to-other scale correlations were lower than item-to-hypothesized scale correlations suggesting good item discriminant validity. Model testing revealed that LPF and LMF were not perfectly correlated, suggesting adequate construct discriminant validity. Increasing levels of symptom severity were associated with significant decreases in physical component summary (PCS) and mental component summary (MCS) scores, supporting known-groups validity. Internal consistency reliability was satisfactory, with Cronbach's alpha of 0.848 for the LPF and 0.785 for the LMF items. Finally, none of the participants received the least or maximum possible PCS or MCS score, indicating the absence of floor and ceiling effects. Conclusions: Overall, the SF-12v2 was found to have adequate psychometric validity in our sample of adults with hemophilia. These results add to the growing evidence of psychometric validity of the SF-12v2 in different patient populations including hemophilia.
Assessing health-related quality of life in patients with severe hemophilia A and B
Psychology Health and Medicine
Background: Health Related Quality of Life (HRQoL) is an important outcome measure in Inflammatory Bowel Disease (IBD). The aim of our study was to assess HRQoL in a population of 135 Greek patients with IBD. Methods: A cohort of 135 patients with IBD, 81 with ulcerative colitis (UC) and 54 with Crohn's disease (CD) were enrolled in our study. Demographic and disease-related data were recorded. HRQoL was assessed by a disease-specific and a generic questionnaire, IBDQ and SF-36, respectively. Disease activity was assessed by Harvey-Bradshaw Index and the Colitis Activity Index for CD and UC patients, respectively. Results: Among all variables recorded in our study, only disease activity had a significant effect on HRQoL. Patients with active disease scored significantly lower on both IBDQ and SF-36 when compared to those in remission. Only two among the four IBDQ dimensions, bowel and systemic, had significant ability in distinguishing best patients in remission from those with active disease. Conclusions: IBD has a negative impact on HRQoL. Patients with active disease are more impaired than patients in remission. In our population of patients bowel and systemic dimensions had a predominant value in patients' perception of quality of life. Patients in our study using the same instrument scored higher than previously reported.
Haemophilia, 2006
Summary. Disease-specific measures of quality of life (QoL) for children with haemophilia are now available for use in clinical studies [Haemophilia, 10, 2004, 9–16]. One of these measures, the Canadian Haemophilia Outcomes – Kids’ Life Assessment Tool (CHO-KLAT), was developed in Canada with emphasis on the perspectives of children [Pediatr Blood Cancer, 47, 2006, 305–11; Haemophilia, 10, 2004, 34–43]. Another, the Haemo-QoL, was developed in Europe, with emphasis on the perspectives of clinicians [Haemophilia, 8, 2002, 47–54; Haemophilia, 10, 2004, 17–25]. While these two measures are unique and independent, researchers from both studies were collaboratively linked throughout development and testing. This study presents the results of a joint assessment of the two measures with respect to their strengths, limitations and unique contributions.The primary questions addressed were:1 What is the relationship between the CHO-KLAT and the Haemo-QoL in terms of summary scores and item content?2 What are the methodological strengths, limitations and unique contributions of each measure?We conducted a retrospective analysis of data from field testing of both measures. The analysis included a comparative assessment of the basic validity, reliability and items used in each measure. Overall, the CHO-KLAT and the Haemo-QoL are promising and valuable measures of QoL for children with haemophilia. Our analyses confirmed the basic psychometric properties of both tools, but identified some discrepancies between them. Additional data will allow for greater understanding of these discrepancies and lend clarity to how the tools should be used in clinical studies (separately or merged). The present recommendation is that the measures be run independently, but preferably concurrently in studies of children with haemophilia.
Quality of life in haemophilia carriers - preliminary results of the ESCHQoL study
Haemophilia, 2008
The signature of the GAP agreement between the WFH and the Ministry of Health in Jordan represented a significant step forward for the Hemophilia community (patients, families, doctors and health care professionals) in the country. The GAP has provided Jordan with the experiences and the means to achieve remarkable improvements in hemophilia care. The main result was raising the number of identified patients. More than 230 patients with hemophilia have been identified and more than 300 patients and their families have received training and education. Other results include the creation of a national committee for supervising the national Hemophilia program as part of the health system, the establishment of a new national registry and the increasing of the government purchases of factor concentrates. Since the start of the GAP project in Jordan, the modality of treatment changed from mainly cryoprecipitate (for hemophilia A) and fresh frozen plasma (for hemophilia B), to factor concentrates. The factor concentrates usage per capita reached 0.4 compared to 0.12 at the start of GAP. The Jordan Hemophilia Society has been actively involved in all the action conducted under the GAP. In addition to organizing awareness activities and training workshops for the board members, the volunteers, patients, families, and youth and mothers groups, the JHS was involved in the organization of symposiums and workshops for educating and training clinicians, nurses, laboratory scientists, physiotherapists (physiotherapy clinic), and other healthcare professionals to ensure adequate expertise in diagnosis and treatment.
Kocaeli Medical Journal, 2021
Hemofiliye özgü hayat kalitesi ölçeği hemofili hastaları için düzenlenmiş bir ankettir. Hemofili tedavi standartlarını, hasta ve ebeveynlerinin duygularını ve yaşadıkları güçlükleri değerlendirmek için değerli bir araçtır. Bu çalışmanın amacı hemofili hastası çocuk ve genç erişkinlerin hayat kalitesini değerlendirmektir. YÖNTEM ve GEREÇLER: Faktör düzeyi <1 olan 4-25 yaştaki hemofili hastaları poliklinik başvuruları sırasında çalışmaya alındı. Hemofiliye özgü yaşam kalitesi anketi çocuklar ve vasilerine (Haemo-QoL) ve erişkin hastalara (Haemo-A-QoL) uygulandı. (www.haemoqol.de). Hepatit ve insan immün yetersizlik virusu serolojisi için taramaları da yapıldı.