RESUMO
INTRODUCTION: The physical pain and disability affecting many people with haemophilia A (PwHA) are known detractors from psychological wellbeing. While psychosocial support is considered a core tenet of the haemophilia comprehensive care structure, the extent to which mental health challenges are detected and monitored by the individuals treating haematologist remains relatively unexplored. AIM: To describe prevalence of anxiety and depression in a real-world cohort of adult PwHA and evaluate the congruence in reporting of anxiety or depression (A/D) between PwHA and their treating physicians. METHODS: Data for PwHA without inhibitors was drawn from the European 'Cost of Haemophilia: A Socioeconomic Survey II' (CHESS II) study. Haematologist-indicated comorbidities of anxiety and depression were unified into a single A/D indicator. The EQ-5D-5L health status measure was used to characterise self-reported A/D, with individuals stratified into two non-mutually exclusive subgroups based on level of A/D reported (Subgroup A: 'some' or above; Subgroup B: 'moderate' or above). RESULT: Of 381 PwHA with evaluable EQ-5D-5L responses, 54% (n = 206) self-reported at least some A/D (Subgroup A) and 17% (n = 66) reported at least moderate A/D (Subgroup B). Patient-physician congruence in A/D reporting was 53% and 76% for Subgroups A and B, respectively. Descriptive analysis suggested that individuals with physician- and/or self-reported A/D experienced worse clinical outcomes (bleeding events, joint disease, chronic pain). CONCLUSION: While adverse clinical outcomes appear to correlate with A/D, self-reports of moderate-severe symptoms occasionally lacked formal recognition from treating physicians. Cross-disciplinary surveillance of mental health issues could improve both psychological and clinical outcomes among PwHA.
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Ansiedade , Depressão , Hemofilia A , Humanos , Hemofilia A/complicações , Hemofilia A/psicologia , Depressão/epidemiologia , Depressão/etiologia , Depressão/psicologia , Ansiedade/psicologia , Ansiedade/epidemiologia , Adulto , Masculino , Europa (Continente) , Pessoa de Meia-Idade , Feminino , Médicos/psicologia , Adulto Jovem , Qualidade de VidaRESUMO
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.
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Hemofilia A , Qualidade de Vida , Índice de Gravidade de Doença , Humanos , Hemofilia A/complicações , Hemofilia A/psicologia , Qualidade de Vida/psicologia , Europa (Continente) , Masculino , Adulto , Estudos Transversais , Pessoa de Meia-Idade , Feminino , Inquéritos e Questionários , Estudos Retrospectivos , Análise Multivariada , Adulto Jovem , Adolescente , IdosoRESUMO
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.
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Hemofilia A , Humanos , Adulto , Criança , Hemofilia A/tratamento farmacológico , Qualidade de Vida , Estudos Retrospectivos , Estudos Transversais , Estresse FinanceiroRESUMO
INTRODUCTION: Gene therapy clinical trials measure steady-state clotting factor expression levels (FELs) to evaluate the modulation of the bleeding phenotype, aiming to offer consistent protection against breakthrough bleeding events. The link between FELs and bleeding risk in people with haemophilia B (PwHB) is not well understood. AIM: We evaluated the association between FEL and ABR in PwHB. METHODS: This cross-sectional study extended the CHESS burden of illness studies in Europe and the United States. Recruitment of additional adult males with haemophilia B supplemented the existing CHESS sample size of PwHB and FELs. PwHB receiving prophylaxis were excluded, as fluctuating FELs may have confounded the analysis. Demographic and clinical characteristics were reported descriptively. Any recorded baseline FEL was reported by the haemophilia-treating physicians according to the medical records. Generalised linear models with log link explored the association between changes in FEL and ABR. RESULTS: The study included 407 PwHB and no inhibitors receiving on-demand treatment. Mean age was 36.7 years; 56% from the EU, 44% from the United States. Mean baseline FEL was 9.95 IU/dl (SD, 10.47); mean ABR was 2.4 bleeds/year (SD, 2.64). After adjusting for covariates, the model showed that for every 1% increase in FEL the average ABR decreased by .08 (p < .001). Predicted number of bleeding events according to FEL showed a significant non-linear relationship between FEL and ABR (p < .05). CONCLUSION: This analysis showed a significant relationship between FEL and ABR, where increases in FEL were associated with decreases in ABR among men with HB in Europe and the US.
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Hemofilia A , Hemofilia B , Masculino , Humanos , Hemofilia B/complicações , Hemofilia B/tratamento farmacológico , Hemofilia A/tratamento farmacológico , Estudos Transversais , Hemorragia/complicações , Fatores de Coagulação Sanguínea/uso terapêutico , Fator VIII/uso terapêuticoRESUMO
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.
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Hemofilia A , Adolescente , Adulto , Humanos , Criança , Hemofilia A/epidemiologia , Qualidade de Vida , Escolaridade , Comorbidade , Inquéritos e QuestionáriosRESUMO
BACKGROUND: Skill-based practice (e.g., communication skills) is important for individuals to incorporate into students' learning and can be challenging in large classes. Simulation-based education (SBE) is a method where students can learn and practice skills in a safe environment to use in real world settings with assistance of peer coaching. The COVID-19 pandemic presented challenges to providing students with sufficient SBE. The purpose of this paper is to: a.) describe a SBE approach for health coaching referred to as "Demo, Debrief, and Do" (DDD), b.) discuss how this approach became important in COVID-19 classroom experiences, c.) describe the impact of DDD activity on students in a health sciences curriculum. DDD is a collaborative activity where graduate health coaching students demonstrate coaching skills, debrief their demonstration, and support undergraduate students to demonstrate (or do) their own coaching skills in a small virtual online setting. METHODS: Qualitative feedback from 121 undergraduate students enrolled in 3 sections of a behavior change strategies course and quantitative surveys to examine their confidence in applying the skills and overall satisfaction with DDD were gathered. RESULTS: The overall average confidence level following the lab was 31.7 (0-35). The average satisfaction level following the lab was 23.3 (0-25 range). The most common highlight of this DDD experience described was observing the coaching demonstration (i.e., demo), followed by the feedback (i.e., debrief), and the practice (i.e., do). CONCLUSION: The (DDD) simulation approach fulfilled an educational need during the COVID 19 pandemic and filled a gap in offering SBE opportunities for both graduate and undergraduate students while learning effective client-communication skills health coaching delivery.
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Aprendizagem , Pandemias , Humanos , Currículo , Estudantes , RetroalimentaçãoRESUMO
INTRODUCTION: Gene therapies are poised to become a new therapeutic option for persons with haemophilia (PwH), having begun to show durable efficacy and safety in clinical trials to date. However, value assessment of gene therapies faces challenges from small populations that preclude randomized clinical trials (RCT), ethical considerations when treating a serious genetic disorder with lifelong consequences, and appropriate endpoint selection that captures the full scope of the disorder and its lifelong complications. SUMMARY: Health technology assessment (HTA) for new haemophilia therapies may require greater flexibility in evidence requirements and recognition of factors that vary across patient populations and health systems, including current standard-of-care, gains in survival and health-related quality-of-life (HRQoL), and reduced replacement factor utilization. It will be important for HTAs to consider limitations of RCTs for gene therapy and to consider intra-patient data as evidence of clinical effectiveness. Despite long-term clinical trials with up to 8 years of follow-up, ongoing uncertainties about durability of effect may be informed by extrapolating clinical data out â¼10 years, using different projections of durability. Beyond objective endpoints, such as Petersson scores or annualized bleed rates, health utilities that accompany gene therapy (e.g., mental health, freedom of choice, peace of mind) should be considered in HTA evaluations, particularly for comparisons with low dose or no prophylaxis. CONCLUSION: HTAs of gene therapies in haemophilia are encouraged to rise to the challenge of filling evidence gaps and conducting assessments. KEY POINTS OF CONSIDERATION: It is important for HTA bodies to consider the limitations to conduct randomized controlled trials for gene therapy and to consider intrapatient data as evidence of comparative effectiveness. Given the uncertainties around the long-term gene therapy use, clinical trial data should be extrapolated â¼10 years, using scenarios that consider different durations of effect. The major value drivers in a model, in addition to drug pricing itself, will be based on assumptions about duration of effect and savings/cost offsets from reduced use of replacement therapy. Assessment methodologies and modelling configurations need to evolve to fully capture the value of gene therapy, including patient meaningful outcomes, in a validated and quantitative fashion. Regardless of payment system archetype, the intersection between NGOs, the clinical community's voice, HTA willingness to collaborate, and alignment with regulatory acceptance of benefit is critical. [Correction added on 21 February 2022, after first online publication: the 'Key Points of Consideration' have been added in this version.].
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Hemofilia A , Avaliação da Tecnologia Biomédica , Análise Custo-Benefício , Terapia Genética , Hemofilia A/genética , Hemofilia A/terapia , Humanos , Resultado do TratamentoRESUMO
INTRODUCTION: With the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions. OBJECTIVE: To examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs). METHODS: Semi-structured interviews were conducted with PIH (n = 14) and clinical experts (n = 6) who ranked pre-defined treatment attributes by importance. Framework analysis was conducted to identify key themes and treatment attributes; points were allocated based on the rankings. Synthesis of results by a multidisciplinary group informed development of a profile of gene therapy characteristics for use in future research. RESULTS: Key themes identified by PIH and clinical experts included patient relevant features and the importance of 'informed decision making'. The six top-ranked treatment attributes were 'effect on factor level' (79 points), 'uncertainty regarding long-term risks' (57 points), 'impact on daily life' (41 points), 'frequency of monitoring' (33 points), 'impact on ability to participate in physical activity' (29 points), and 'uncertainty regarding long-term benefits' (28 points). The final treatment characteristics were categorised as therapeutic option, treatment effectiveness, safety concerns, impact on self-management and quality of life (role limitations). CONCLUSION: We identified several gene therapy characteristics important to PIH and clinicians in the UK. These characteristics will be used in a future DCE to further investigate patient preferences for gene therapy.
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Comportamento de Escolha , Hemofilia A , Terapia Genética , Hemofilia A/genética , Hemofilia A/terapia , Humanos , Preferência do Paciente , Qualidade de Vida , Reino UnidoRESUMO
BACKGROUND: Long-term prophylactic therapy is considered the standard of care for hemophilia A patients. This study models the long-term clinical and cost outcomes of two factor VIII (FVIII) products using a pharmacokinetic (PK) simulation model in a Chinese population. METHODS: Head-to-head PK profile data of BAY 81-8973 (KOVALTRY®) and antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM, ADVATE®) were applied to a two-state (alive and dead) Markov model to simulate blood FVIII concentrations at a steady state in prophylactically-treated patients with hemophilia A. Worsening of the Pettersson score was simulated and decline was associated with the probability of having orthopaedic surgery. The only difference between the compounds was FVIII concentration at a given time; each subject was treated with 25 IU/kg every 3 days. The model used a lifetime horizon, with cycle lengths of 1 year. RESULTS: Cumulative bleeding events, joint bleeding events, and major bleeding events were reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. Hospitalizations and hospitalization days were also reduced by 19.3% for BAY 81-8973 compared to rAHF-PFM. BAY 81-8973 resulted in both cost savings and a gain in quality adjusted life years (QALYs) compared to rAHF-PFM. CONCLUSION: Based on modeled head-to-head comparisons, differences in PK-properties between BAY 81-8973 and rAHF-PFM result in a reduced number of bleeding events, leading to reduced costs and increased quality of life for BAY 81-8973. These results should be used to inform clinical practice in China when caring for patients with severe hemophilia A.
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Fator VIII , Hemofilia A , Atenção à Saúde , Fator VIII/farmacocinética , Fator VIII/uso terapêutico , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemorragia/tratamento farmacológico , Hemorragia/prevenção & controle , Humanos , Qualidade de Vida , Proteínas Recombinantes/efeitos adversos , Proteínas Recombinantes/farmacocinética , Albumina Sérica/uso terapêuticoRESUMO
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.
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Hemofilia A , Absenteísmo , Eficiência , Hemofilia A/complicações , Humanos , Presenteísmo , Qualidade de Vida , Inquéritos e QuestionáriosRESUMO
AIMS: There is evidence that people with haemophilia A still experience morbidity and functional limitation due to joint damage despite prophylaxis. This study aimed to compare their quality of life and work-related function with that of the general population and patients with osteoarthritis. METHODS: Data from the Cost of Haemophilia in Europe: a Socioeconomic Survey (CHESS) database were compared with published data from normative populations and patients with osteoarthritis in Europe and the United States. RESULTS: In the predominantly young (age 18-35 years) adult CHESS population treated with primary prophylaxis, about 30% reported a target joint; the average frequency of bleeds was one per year; half reported chronic pain. Levels of anxiety and depression were similar to those reported by people using on-demand treatment. Employment and productivity were lower than in the general population. The level of presenteeism (attending work with impairment) was comparable with that reported for a much older population with osteoarthritis who had more extensive joint damage and greater prevalence of pain. CONCLUSION: Compared with the general population, clinical outcomes and quality of life are indicated to be impaired for young adults whose haemophilia is managed by primary prophylaxis. Primary prophylaxis is not associated with lower levels of anxiety and depression than on-demand treatment, and pain is common. The level of presenteeism is comparable to that reported in people with osteoarthritis, an older population with more joint disease. Further studies are needed to fully assess the implications of compromised work performance among young adults with haemophilia as they seek to build a career.
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Hemofilia A , Adolescente , Adulto , Efeitos Psicossociais da Doença , Fator VIII , Hemofilia A/complicações , Hemofilia A/tratamento farmacológico , Hemorragia , Humanos , Qualidade de Vida , Inquéritos e Questionários , Adulto JovemRESUMO
INTRODUCTION: People with inherited and long-term conditions such as haemophilia have been shown to adapt to their levels of disability, often reporting better quality of life (QoL) than expected from the general population (the disability paradox). AIM: To investigate the disability paradox in people with haemophilia in the United States by examining preference differences in health state valuations versus the general population. METHODS: We conducted a discrete choice experiment including duration to capture valuations of health states based on patient-reported preferences. Participants indicated their preferences for hypothetical health states using the EQ-5D-5L, where each participant completed 15 of the 120 choice tasks. Response inconsistencies were evaluated with dominated and repeated scenarios. Conditional-logit regressions with random sampling of the general population responses were used to match the sample of patients with haemophilia. We compared model estimates and derived preferences associated with EQ-5D-5L health states. RESULTS: After removing respondents with response inconsistencies, 1327/2138 (62%) participants remained (177/283 haemophilia; 1150/1900 general population). Patients with haemophilia indicated higher preference value for 99% of EQ-5D-5L health states compared to the general population (when matched on age and gender). The mean health state valuation difference of 0.17 indicated a meaningful difference compared to a minimal clinically important difference threshold of 0.07. Results were consistent by haemophilia type and severity. CONCLUSION: Our findings indicated the presence of a disability paradox among patients with haemophilia, who reported higher health states than the general population, suggesting the impact of haemophilia may be underestimated if general population value sets are used.
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Hemofilia A , Qualidade de Vida , Nível de Saúde , Humanos , Medidas de Resultados Relatados pelo Paciente , Inquéritos e QuestionáriosRESUMO
INTRODUCTION: Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments. AIM: We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes. METHODS: We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on mental health, and post-treatment requirements. The relative importance of each attribute was analysed overall and for subgroups based on haemophilia type and severity. RESULTS: A total of 183 males with haemophilia A (n = 120) or B (n = 63) were included. Half (47%) had severe haemophilia; most (75%) were White. Overall, participants gave effect on bleeding rate the greatest relative importance (31%), followed by dose frequency/durability (26%), uncertainty regarding safety issues (17%), and impact on daily activities (11%). Dose frequency/durability had the greatest importance for those with haemophilia B (35%). CONCLUSION: People with haemophilia prioritised reduced bleeding and treatment burden; the former was more important in haemophilia A and the latter in haemophilia B, followed by safety and impact on daily life in this DCE of gene therapy attributes. These findings and differences can inform clinical and health policy decisions to improve health equity for people with haemophilia.
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Hemofilia A , Adulto , Comportamento de Escolha , Terapia Genética , Hemofilia A/genética , Hemofilia A/terapia , Hemorragia/terapia , Humanos , Masculino , Preferência do Paciente , Inquéritos e QuestionáriosRESUMO
INTRODUCTION: The benefits of physical activity (PA) for people with haemophilia (PWH) may include improvements in joint, bone and muscle health. However, the factor VIII activity level required to avoid a bleeding episode associated with PA is unknown. AIM: To elicit the opinion of clinical experts on the minimum level and ideal factor VIII activity ('level') required to avoid a bleeding episode during participation in different types of PA for PWH. METHODS: Based on the 2017 National Hemophilia Foundation PA descriptions, clinical experts estimated a minimally acceptable and an ideal factor level at which a bleed could be avoided. The uncertainty around estimates was quantified using an approach to construct a probability distribution to represent expert opinion. RESULTS: Minimum and ideal factor level increased with higher risk PA, whether or not joint morbidity was present, as did the experts' uncertainty in their estimates (ie the range between lowest and highest estimates for minimum and ideal levels). Mean minimum levels ranged from 4% to 48% for low to high risk for people without joint morbidity, and from 7% to 47% for those with joint morbidity. For ideal factor levels, corresponding figures were 9%-52% and 12%-64%, respectively. CONCLUSION: To support a patient-centric outcome, expert opinion indicates that the clinical norm of 0.01 IU/mL (1%) trough level is insufficient. It is anticipated that introducing a more targeted approach to meet the needs of patients who are increasingly physically active will benefit patients further in addition to recent treatment advances.
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Exercício Físico/fisiologia , Hemartrose/prevenção & controle , Hemofilia A/terapia , Hemorragia/prevenção & controle , Adolescente , Adulto , Idoso , Criança , Pré-Escolar , Conferências de Consenso como Assunto , Fator VIII/análise , Hemartrose/diagnóstico , Hemartrose/etiologia , Hemofilia A/sangue , Hemofilia A/complicações , Hemorragia/etiologia , Humanos , Lactente , Artropatias/sangue , Artropatias/diagnóstico , Artropatias/patologia , Pessoa de Meia-Idade , Medição de Risco , Adulto JovemRESUMO
Historically, treatment based on the availability of clotting factor replacement has resulted in an arcane guideline for the correction of factor deficiencies in people with haemophilia (PwH). While all other disease entities seek to restore function to a normal level, PwH are restricted to factor nadirs still equivalent to mild or moderate disease, resulting in continued risk of bleeding. A new treatment paradigm is needed based on the defined needs of PwH. A treatment model was developed by a panel of haemophilia providers, patient advocates and health economists to establish specific treatment milestones and targeted outcomes. The panel defined a series of treatment milestones to characterize the activity and outcomes linked to level of factor deficiency correction. All agreed that the ultimate goal should be 'functional cure' and 'health equity'. Seven levels to achieving a functional cure were identified, (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds; (d) Attainment of 'normal' mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis. A parallel set of patient-reported outcomes to achieve health equity was identified. These guidelines are now comparable with other disorders where the goal is to replace missing proteins to attain normal activity levels. As we are no longer limited by plasma supply due to the manufacture of recombinant factors, mimetics, and the early success of gene therapy, health equity is now achievable.
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Equidade em Saúde , Hemofilia A/epidemiologia , Atividades Cotidianas , Hemostasia , Humanos , Estilo de Vida , Qualidade de VidaRESUMO
INTRODUCTION: Clinical severity and impact of haemophilia on quality of life have been generally considered to be lower for haemophilia B (HB) compared with haemophilia A (HA) patients. AIMS: To compare annual bleeding rate (ABR), target joint development and health-related quality of life (HRQoL) between adult (≥18 years) severe HA and HB patients using recent data from the Cost of Haemophilia in Europe: a Socioeconomic Survey (CHESS) study. METHODS: Multivariate generalized linear models (GLM) were constructed to assess the relationship between haemophilia type, ABR, HRQoL (derived from EQ-5D index scores) and the presence of target joints while controlling for covariates. RESULTS: Of the 1225 patients included, 77% (n = 949) had HA and 23% (n = 278) had HB. Of the 514 patients who completed the EQ-5D, 78% (n = 405) had HA, and 22% (n = 110) had HB. Unadjusted mean ABR was 3.79 in HA and 4.60 in HB. The presence of ≥1 target joint was reported in 59% and 54% of patients with HA and HB, respectively. Unadjusted mean EQ-5D index score was 0.78 in HA and 0.76 in HB. Haemophilia type was not a significant predictor of ABR, target joints or HRQoL when adjusted for confounding factors such as BMI, age and replacement therapy regimen. CONCLUSION: Data suggest comparable ABR, incidence of target joints and HRQoL between patients with HB and HA indicating comparable clinical severity and disease impact on patient quality of life.
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Hemofilia A/complicações , Hemofilia B/complicações , Hemorragia/etiologia , Qualidade de Vida/psicologia , Adulto , Estudos Transversais , Feminino , Hemofilia A/patologia , Hemofilia B/patologia , Hemorragia/patologia , Humanos , Masculino , Estudos RetrospectivosRESUMO
BACKGROUND: Joint damage remains a major complication associated with haemophilia and is widely accepted as one of the most debilitating symptoms for persons with severe haemophilia. The aim of this study is to describe how complications of haemophilia such as target joints influence health-related quality of life (HRQOL). METHODS: Data on hemophilia patients without inhibitors were drawn from the 'Cost of Haemophilia across Europe - a Socioeconomic Survey' (CHESS) study, a cost-of-illness assessment in severe haemophilia A and B across five European countries (France, Germany, Italy, Spain, and the UK). Physicians provided clinical and sociodemographic information for 1285 adult patients, 551 of whom completed corresponding questionnaires, including EQ-5D. A generalised linear model was developed to investigate the relationship between EQ-5D index score and target joint status (defined in the CHESS study as areas of chronic synovitis), adjusted for patient covariates including socio-demographic characteristics and comorbidities. RESULTS: Five hundred and fifteen patients (42% of the sample) provided an EQ-5D response; a total of 692 target joints were recorded across the sample. Mean EQ-5D index score for patients with no target joints was 0.875 (standard deviation [SD] 0.179); for patients with one or more target joints, mean index score was 0.731 (SD 0.285). Compared to having no target joints, having one or more target joints was associated with lower index scores (average marginal effect (AME) -0.120; SD 0.0262; p < 0.000). CONCLUSIONS: This study found that the presence of chronic synovitis has a significant negative impact on HRQOL for adults with severe haemophilia. Prevention, early diagnosis and treatment of target joints should be an important consideration for clinicians and patients when managing haemophilia.
Assuntos
Hemofilia A/complicações , Hemofilia B/complicações , Qualidade de Vida , Sinovite/etiologia , Adulto , Doença Crônica , Efeitos Psicossociais da Doença , Europa (Continente) , Hemofilia A/psicologia , Hemofilia B/psicologia , Humanos , Pessoa de Meia-Idade , Estudos Retrospectivos , Índice de Gravidade de Doença , Inquéritos e Questionários , Sinovite/psicologiaRESUMO
OBJECTIVES: To evaluate postoperative health resource utilisation and secondary care costs for radical prostatectomy and partial nephrectomy in National Health Service (NHS) hospitals in England, via a comparison of robot-assisted, conventional laparoscopic and open surgical approaches. PATIENTS AND METHODS: We retrospectively analysed the secondary care records of 23 735 patients who underwent robot-assisted (RARP, n = 8 016), laparoscopic (LRP, n = 6 776) or open radical prostatectomy (ORP, n = 8 943). We further analysed 2 173 patients who underwent robot-assisted (RAPN, n = 365), laparoscopic (LPN, n = 792) or open partial nephrectomy (OPN, n = 1 016). Postoperative inpatient admissions, hospital bed-days, excess bed-days and outpatient appointments at 360 and 1 080 days after surgery were reviewed. RESULTS: Patients in the RARP group required significantly fewer inpatient admissions, hospital bed-days and excess bed-days at 360 and 1 080 days than patients undergoing ORP. Patients undergoing ORP had a significantly higher number of outpatient appointments at 1 080 days. The corresponding total costs were significantly lower for patients in the RARP group at 360 days (£1679 vs £2031 for ORP; P < 0.001) and at 1 080 days (£3461 vs £4208 for ORP; P < 0.001). In partial nephrectomy, Patients in the RAPN group required significantly fewer inpatient admissions and hospital bed-days at 360 days compared with those in the OPN group; no significant differences were observed in outcomes at 1 080 days. The corresponding total costs were lower for patients in the RAPN group at 360 days (£779 vs £1242 for OPN, P = 0.843) and at 1 080 days (£2122 vs £2889 for ORP; P = 0.570). For both procedure types, resource utilisation and costs for laparoscopic surgeries lay at the approximate midpoint of those for robot-assisted and open surgeries. CONCLUSION: Our analysis provides compelling evidence to suggest that RARP leads to reduced long-term health resource utilisation and downstream cost savings compared with traditional open and laparoscopic approaches. Furthermore, despite the limitations that arise from the inclusion of a small sample, these results also suggest that robot-assisted surgery may represent a cost-saving alternative to existing surgical options in partial nephrectomy. Further exploration of clinical cost drivers, as well as an extension of the analysis into subsequent years, could lend support to the wider commissioning of robot-assisted surgery within the NHS.