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Introduction: Haemophilia A (HA) is a congenital bleeding disorder caused by a deficiency/absence of factor VIII (FVIII) and characterised by frequent, acute and prolonged spontaneous or traumatic bleeding events, often leading to haemophilic arthropathy and progressive joint deterioration. HA severity is characterized by endogenous FVIII activity: mild (> 5-40%), moderate (1-5%), or severe (< 1%). HA poses a substantial clinical and socioeconomic burden on people with HA (PWHA), their caregivers, and society. This analysis evaluates clinical and patient-centric outcomes of a cohort of individuals with non-inhibitor HA sampled from France, Germany, Italy, Spain, and the UK in the 'Cost of Haemophilia in Europe: A Socioeconomic Survey II' (CHESS II) study., Methods: CHESS II was a cross-sectional burden-of-illness study collecting clinical and socioeconomic data on adult (≥ 18 years) individuals with haemophilia A or B of any severity with or without inhibitors from eight European countries. Descriptive analyses were conducted examining physician-reported demographics, clinical and health resource utilisation information. PWHA-reported health-related quality of life (HRQoL) using the EQ-5D-5L and Work Productivity and Activity Impairment (WPAI) were also examined. Outcomes were stratified by HA severity and reported at country level., Results: Demographics and clinical characteristics of the cohort (N = 880) were generally consistent across countries. Individuals with severe HA experienced more frequent bleeding events and joint disease despite broad use of factor replacement therapy long-term prophylaxis. A minority of those with mild or moderate HA also experienced such challenges. HRQoL and workforce participation diminished, and chronic pain increased, with increasing HA severity., Conclusion: This analysis provides up-to-date insights on the impact of HA across five European countries. Increasing HA severity was generally associated with worse clinical outcomes, HRQoL and workforce participation. These findings suggest a place for continued evidence-based tailored treatment and clinical management approaches in addressing the residual burden of HA., (© 2024. The Author(s), under exclusive licence to Springer Healthcare Ltd., part of Springer Nature.)

Background: Haemophilia A (HA; Factor VIII deficiency) is a congenital X-linked bleeding disorder characterized by trauma-related or spontaneous bleeding events, most notably arising within the intraarticular space and resulting in chronic inflammation and degeneration of affected joints. Endogenous clotting factor activity relative to normal levels determines the severity of HA symptoms, as mild (> 5-40%), moderate (1-5%), or severe (< 1%). Within the current environment of rapid evolution in HA management, we seek to understand the interplay of condition severity and health-related quality of life (HRQoL) to characterise and differentiate unmet needs among people with HA (PwHA)., Methods: A generalised linear regression model (GLM) was developed to explore the relationship between HA severity and EQ-5D-5 L index score from adult HA patients sampled in the "Cost of Haemophilia across Europe - a Socioeconomic Survey II" (CHESS II) cross-sectional, retrospective burden of illness study among adults with hereditary haemophilia A or B from eight European countries. HA patients of any severity with no active inhibitors during the 12 months prior to data capture and a completeEQ-5D-5 L response were included. A base GLM model was specified with covariates for demographic and clinical characteristics (age, body mass index, country, employment, HA severity, annual bleeding rate, problem joints, and chronic pain)., Results: Of 381 evaluable patients, 221 (58.0%) had severe HA, 96 (25.2%) had moderate HA, and 64 (16.8%) had mild HA. Among the covariates included in the GLM model and after controlling for haemophilia-related outcomes, a significant association was observed between mild HA and higher EQ-5D-5 L index score (average marginal effects, 0.084; p = 0.016) relative to severe HA. Patient country of residence and magnitude of HA-related chronic pain were also associated with significant differences in index scores, with the latter showing a negative relationship with HRQoL outcomes., Conclusions: Condition severity and chronic pain are significant predictors of HRQoL in PwHA. Durable bleeding protection and effective management of chronic pain have the potential to address unmet treatment needs in this population., (© 2024. The Author(s).)

Background: Haemophilia A carries a substantial healthcare burden, affecting health-related quality of life (HRQoL). The Cost of Haemophilia in Men: a Socioeconomic Survey II (CHESS II), a retrospective real-world study, characterised the burden of haemophilia and its impact on HRQoL and work productivity. The current analysis explored the impact of bleeding events on HRQoL and work productivity in Europe. This analysis focused on data collected from males aged 18 to 64 years with haemophilia A without inhibitors who were receiving replacement factor products or a monoclonal antibody and were not participating in a clinical trial at the time of study recruitment. Descriptive statistics were analysed using scores from EuroQoL's EQ-5D-5L index and EQ-VAS analogue scale and the Work Productivity and Activity Index Specific Health Problem (WPAI:SHP) percentage scores stratified by the number of annual bleeding events (ABs) 0, 1, 2, 3-4, or ≥ 5., Results: Of 918 males with haemophilia A in CHESS II, 318 met inclusion criteria and had data available for HRQoL measures; mean age (SD) was 33.8 (12.1) years and 96% were White. Mean (SD) ABs of 2.7 (2.9) occurred over the preceding 12 months: 20% had 3 or 4 ABs; 17% had ≥ 5 ABs. Mean EQ-5D-5L index scores for patients with 0, 1, 2, 3-4, or ≥ 5 ABs were 0.92, 0.76, 0.76, 0.71, and 0.56, respectively. Mean (SD) EQ-VAS scores were 86.9 (13.6), with 0 ABs versus 69.5 (19.1) for 3 or 4 ABs and 61.2 (17.2) for ≥ 5 ABs. Mean percentage of overall work productivity loss on the WPAI:SHP questionnaire ranged from 9.70 to 0 ABs to 47.65 for ≥ 5 ABs., Conclusions: In this European sample of adult men with haemophilia A, HRQoL and work productivity scores were lower among those reporting more AB events. Bleeding burden appears to affect HRQoL and productivity; however, this cross-sectional analysis limits the ability to draw firm conclusions on causality., (© 2023. Institut National de la Santé et de la Recherche Médicale (INSERM).)

Background: Haemophilia bears substantial humanistic and economic burden on children and their caregivers. Characterising the differential impact of severe versus moderate paediatric haemophilia is important for clinical and health policy decisions. We analysed health-related quality of life (HRQoL), annual direct medical (excluding factor treatment costs), non-medical and societal costs among children and adolescents with moderate and severe haemophilia A or B without inhibitors from the European CHESS-PAEDs study. Information was reported by physicians and caregivers; patients aged ≥ 8 years self-reported their HRQoL. Descriptive statistics summarised demographic and clinical characteristics, costs, and HRQoL scores (EQ-5D-Y). Regression models estimated differences in HRQoL and costs for moderate versus severe haemophilia adjusting for age, body mass index z-score, country, number of comorbidities, and weight-adjusted annual clotting factor consumption., Results: The analytic sample comprised 794 patients with a mean age of 10.5 years; most had haemophilia A (79%) and 58% had severe haemophilia. Mean predicted direct medical costs in moderate patients were two-thirds of the predicted costs for severe disease (€3065 vs. €2047; p < 0.001; N = 794), while societal costs were more than half of the predicted costs for children with severe haemophilia (€6950 vs. €3666; p < 0.001; N = 220). Mean predicted HRQoL scores were 0.74 and 0.69 for moderate and severe disease, respectively (p < 0.05; N = 185)., Conclusion: Children with haemophilia and their caregivers displayed a significant economic and humanistic burden. While severe patients showed the highest direct medical and societal costs, and worse HRQoL, the burden of moderate haemophilia on its own was substantial and far from negligible., (© 2022. The Author(s).)

Few studies in amyotrophic lateral sclerosis (ALS) have profiled disease-specific features of the condition in conjunction with assessment of caregivers' burden, distress, quality of life, and investigated patient survival. Eighty-four ALS patients and their primary caregivers were enrolled. Patients completed ALS-specific measures of physical and cognitive function, while caregivers completed measures of anxiety, depression, caregiver burden, and quality of life. Patient-caregiver dyads were interviewed about their health-service utilisation. Survival data were obtained through the Irish register for ALS. Participants were dichotomised into low/high groups according to the severity of self-reported caregiver burden, based on statistically derived cut-off scores. High-burdened caregivers (n = 43) did not significantly differ from low-burdened caregivers (n = 41) with respect to disease-specific characteristics, i.e., ALSFRS-R, bulbar- or spinal-onset ALS, disease duration, or survival data. However, significant differences were reported on subjective measures of anxiety (p < 0.000), depression (p < 0.001), distress (p < 0.000), and quality of life (p < 0.000). These data demonstrate the limited impact of ALS patient-related variables, i.e., ALSFRS-R and onset, on caregiver burden in ALS, and identify the importance of the psychological composition of caregivers. This study suggests that the subjective experience of individual caregivers is an important factor influencing the severity of experienced caregiver burden.

This study provides a quantitative synthesis of meta-analytic evidence for the effectiveness of very broadly defined positive psychological interventions (PPIs), i.e. interventions that enhance well-being through pathways consistent with positive psychology theory. The definition covers a wide range of PPIs including single and multi-element PPI programs as well as mindfulness, mind-body, and physical exercise-based interventions. Five databases were searched. One hundred and ninety eight meta-analyses involving 4,065 primary studies and 501,335 participants were selected for review. Using the AMSTAR-2 criteria, the selected meta-analyses were found to be of moderate or high quality. At post-intervention, PPIs had a significantly small to medium effect on well-being, QoL, strengths, depression, anxiety, and stress. Gains were partially maintained at 7.5-month follow-up. Mind-body PPIs such as yoga were particularly effective. Individuals who engaged in longer, face-to-face programs benefited most. This mega-analysis shows that PPIs have an extensive evidence base supporting their effectiveness. [ABSTRACT FROM AUTHOR]

Introduction: Adequate prophylactic treatment and physical activity improve joint health and clinical outcomes for people with haemophilia A (HA). However, non‐clinical joint‐related burden of moderate (MHA) and severe (SHA) HA has not been well characterised. Aim: To quantify the joint health‐related humanistic and economic burden of MHA and SHA in Europe. Methods: A retrospective analysis of the cross‐sectional CHESS population studies using a patient‐centric measure of joint health (problem joints, PJs: chronic joint pain and/or limited range of movement due to compromised joint integrity with or without persistent bleeding) was conducted. Descriptive statistics summarised health‐related quality of life (HRQoL), work productivity/activity impairment and costs by number of PJs (0, 1 or ≥2) and HA severity. Results: A total of 1171 patients were included from CHESS‐II (n = 468) and CHESS‐PAEDs (n = 703). In both studies, 41 and 59% of patients had MHA and SHA, respectively. Prevalence of ≥2 PJs was similar with MHA and SHA (CHESS‐II: 23 and 26%; CHESS‐PAEDs: 4 and 3%, respectively). HRQoL was worse with an increasing number of PJs (CHESS‐II:.81 vs..66 with 0 and ≥2 PJs, respectively, for MHA;.79 vs..51 for SHA; CHESS‐PAEDs:.64 vs..26 and.72 vs..14). Total costs increased with increasing PJs regardless of severity in CHESS‐II (€2923 vs. €22,536 with 0 and ≥2 PJs, respectively, for MHA; €11,022 vs. €27,098 for SHA) and CHESS‐PAEDs (€6222 vs. €11,043 for MHA; €4457 vs. €14,039 for SHA). Conclusion: Presence of PJs was associated with a substantial humanistic and economic burden on patients with MHA or SHA across the lifespan. [ABSTRACT FROM AUTHOR]

Introduction: The "problem joint" (PJ) concept was developed to address patient‐centric needs for a more holistic assessment of joint morbidity for people with haemophilia (PwH). Aim: To quantify the humanistic burden of PJs in PwH to further support validation of the PJ outcome measure. Methods: Multivariable regression models evaluated the relationship between PJs and health‐related quality of life (HRQoL, EQ‐5D‐5L) and overall work productivity loss (WPL) using data from the 'Cost of HaEmophilia: a Socioeconomic Survey' population studies (adults: CHESS II, CHESS US+; children/adolescents: CHESS‐Paeds). Covariates included were haemophilia severity, age, comorbidities and education. Results: The CHESS II sample included 292 and 134 PwH for HRQoL and WPL analyses, mean age 38.6 years (39% ≥1 PJ, 61% none). CHESS US+ included 345 and 239 PwH for HRQoL and WPL, mean age 35 years (43% ≥1 PJ, 57% none). CHESS‐Paeds included 198 PwH aged 4–17 (HRQoL only), mean age 11.5 years (19% ≥1 PJ, 81% none). In CHESS II and CHESS US+, presence of PJs was associated with worse HRQoL (Both p <.001). Few CHESS‐Paeds participants had PJs, with no significant correlation with HRQoL. In CHESS II, upper body PJs were significantly correlated to WPL (p <.05). In CHESS US+, having ≥1 PJ or upper and lower body PJs were significantly correlated to WPL (vs. none; both p <.05). Conclusion: This study has shown a meaningful burden of PJs on PwH, which should be considered in clinical and health policy assessments of joint health. [ABSTRACT FROM AUTHOR]

Introduction: Few studies have examined the real‐world impact of haemophilia on daily activities and work productivity in people with severe haemophilia A (PWSHA). Aim: To determine clinical attributes and treatment characteristics associated with impairment in daily activities and work among PWSHA using the patient‐reported Work Productivity and Activity Impairment‐General Health Questionnaire (WPAI‐GH). Methods: PWSHA were asked to complete the WPAI‐GH as part of the Cost of Haemophilia in Europe: A Socioeconomic Survey (CHESS) study. Outcomes were determined for activity impairment (AI), absenteeism, presenteeism and overall work productivity loss (WPL). Descriptive statistics and regression analyses were used to evaluate the association between these outcomes and clinical and treatment attributes. Results: Overall, 376 participants completed the AI element of WPAI‐GH; 175 were employed and thus also reported on work impact. Mean ± standard deviation scores were as follows: AI = 34.2% ± 25.8%; absenteeism = 0.06% ±0.2%; presenteeism = 26.8% ± 22.4%; WPL = 28.6% ± 24.0%. Increased AI and WPL were associated with high haemophilia‐related morbidity, measured both as chronic pain (p <.001 for both) and joint synovitis (AI: p <0.001; WPL: p =.017). In descriptive and multivariate analyses, lifelong prophylaxis was associated with reduced AI (p <.001 and p =.031, respectively); high therapy adherence was associated with reduced AI (p =.001 and p =.012, respectively) and with reduced WPL (p <.001 and p =.012, respectively). Conclusion: The WPAI‐GH identified haemophilia‐related morbidity and treatment characteristics, including therapy regimen and adherence, as key attributes impacting functional impairment and work contributions of PWSHA. Early prophylactic intervention and greater adherence to therapy may lead to lower AI and WPL in PWSHA. [ABSTRACT FROM AUTHOR]

Background: Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients' functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The 'Cost of Severe Hemophilia across the US: a Socioeconomic Survey' (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US.Results: We conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931.Conclusions: This analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies. [ABSTRACT FROM AUTHOR]

Objective RA is a progressive, chronic autoimmune disease. We summarize the impact of disease activity as measured by the DAS in 28 joints (DAS28-CRP scores) and pain on productivity and ability to work using the Work Productivity and Activity Impairment questionnaire (WPAI) scores, in addition to the impact of disease duration on the ability to work. Methods Data were drawn from the Burden of RA across Europe: a Socioeconomic Survey (BRASS), a European cross-sectional study in RA. Analyses explored associations between DAS28-CRP score and disease duration with stopping work because of RA, and regression analyses assessed impacts of pain and DAS28-CRP on early retirement and WPAI. Results Four hundred and seventy-six RA specialist clinicians provided information on 4079 adults with RA, of whom 2087 completed the patient survey. Severe disease activity was associated with higher rates of stopping work or early retirement attributable to RA (21%) vs moderate/mild disease (7%) or remission (8%). Work impairment was higher in severe (67%) or moderate RA (45%) compared with low disease activity [LDA (37%)] or remission (28%). Moreover, patients with severe (60%) or moderate pain (48%) experienced increased work impairment [mild (34%) or no pain (19%)]. Moderate to severe pain is significant in patients with LDA (35%) or remission (22%). A statistically significant association was found between severity, duration and pain vs work impairment, and between disease duration vs early retirement. Conclusion Results demonstrate the high burden of RA. Furthermore, subjective domains, such as pain, could be as important as objective measures of RA activity in affecting the ability to work. [ABSTRACT FROM AUTHOR]

Background: Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European countries in recent years. However, few studies have taken a standardised methodology across multiple countries simultaneously, and sought to amalgamate all three levels of burden for severe disease. The overall aim of the 'Cost of Haemophilia in Europe: a Socioeconomic Survey' (CHESS) study was to capture the annualised economic and psychosocial burden of severe haemophilia in five European countries. A cross-section of haemophilia specialists (surveyed between January and April 2015) provided demographic and clinical information and 12-month ambulatory and secondary care activity for patients via an online survey. In turn, patients provided corresponding direct and indirect non-medical cost information, including work loss and out-of-pocket expenses, as well as information on quality of life and adherence. The direct and indirect costs for the patient sample were calculated and extrapolated to population level.Results: Clinical reports for a total of 1,285 patients were received. Five hundred and fifty-two patients (43% of the sample) provided information on indirect costs and health-related quality of life via the PSC. The total annual cost of severe haemophilia across the five countries for 2014 was estimated at EUR 1.4 billion, or just under EUR 200,000 per patient. The highest per-patient costs were in Germany (mean EUR 319,024) and the lowest were in the United Kingdom (mean EUR 129,365), with a study average of EUR 199,541. As expected, consumption of clotting factor replacement therapy represented the vast majority of costs (up to 99%). Indirect costs are driven by patient and caregiver work loss.Conclusions: The results of the CHESS study reflect previous research findings suggesting that costs of factor replacement therapy account for the vast majority of the cost burden in severe haemophilia. However, the importance of the indirect impact of haemophilia on the patient and family should not be overlooked. The CHESS study highlights the benefits of observational study methodologies in capturing a 'snapshot' of information for patients with rare diseases. [ABSTRACT FROM AUTHOR]