RESUMO
Assuntos
Angioedemas Hereditários/epidemiologia , Ansiedade/epidemiologia , Depressão/epidemiologia , Absenteísmo , Angioedemas Hereditários/economia , Animais , Efeitos Psicossociais da Doença , Progressão da Doença , Humanos , Prevalência , Qualidade de VidaRESUMO
BACKGROUND: We conducted our first patient survey at the 2013 hereditary angioedema (HAE) patient summit and learned that, despite several novel therapies, the burden of disease was high. OBJECTIVE: To determine, from the patient's perspective, if any improvements in the current state of HAE care occurred over a two-year period between HAE patient summits. METHODS: A patient survey was conducted at the 2015 Hereditary Angioedema Association conference by using paper surveys that aimed at understanding the current state of HAE care. Questions included patient characteristics, burden of disease, and satisfaction with care and treatment options. Comparisons between patients with HAE with C1-inhibitor (HAE-C1INH) and patients with HAE with normal C1-inhibitor (HAE-nlC1INH), as well as between patients with HAE in 2013 and 2015, were performed by using χ2 tests. RESULTS: There were 232 surveys distributed, and 143 surveys were identified as complete for inclusion and analysis from patients with self-reported HAE. Most patients had type I or type II HAE (67.5% [n = 106]), with a smaller number of patients with HAE-nlC1INH (23.6% [n = 37]). In 2015, almost half of the patients with HAE-C1INH (47.1%) and 56.7% of the patients with HAE-nlC1INH experienced a delay of ≥10 years between initial symptoms and diagnosis. Among the patients with HAE-C1INH, 25% reported one or more attacks per week and another 48% reported experiencing one or more attacks per month (fewer than one attack per week). The patients with HAE-nlC1INH reported attacks more frequently than did the patients with HAE-C1INH (p = 0.002), with 59.5% who reported attacks at least once a week. Emergency care was reported one or more times per month in 5% of the patients with HAE-C1INH and in 24.3% of the patients with HAE-nlC1INH. CONCLUSION: Similar to 2013, although significant progress has been made, there is still a high burden of disease that faces patients with HAE.
Assuntos
Angioedemas Hereditários/epidemiologia , Efeitos Psicossociais da Doença , Pacientes , Adolescente , Adulto , Idoso , Idoso de 80 Anos ou mais , Criança , Pré-Escolar , Diagnóstico Tardio , Feminino , Seguimentos , Humanos , Lactente , Recém-Nascido , Masculino , Pessoa de Meia-Idade , Satisfação do Paciente , Qualidade da Assistência à Saúde , Inquéritos e Questionários , Estados Unidos/epidemiologia , Adulto JovemRESUMO
This review will discuss the cost burden of hereditary angioedema on patients, healthcare systems, and society. The impact of availability of and access to novel and specific therapies on morbidity, mortality, and the overall burden of disease will be explored along with potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment. The prevalence of orphan diseases, legislative incentives to encourage development of orphan disease therapies and the impact of orphan disease treatment on healthcare payment systems will be discussed.
RESUMO
This article discusses orphan diseases, their prevalence, legislative incentives to encourage development of therapies, and the impact of treatment on health care payment systems. Specifically, the cost burden of hereditary angioedema on patients, health care systems, and society is reviewed. The impact of availability of and access to novel and specific therapies on morbidity, mortality, and overall burden of disease is explored. Changes in treatment paradigms to improve effect and reduce cost of treatment are presented.
Assuntos
Angioedemas Hereditários/epidemiologia , Angioedemas Hereditários/terapia , Doenças Raras , Efeitos Psicossociais da Doença , Descoberta de Drogas/economia , Descoberta de Drogas/legislação & jurisprudência , Farmacoeconomia , Saúde Global , Custos de Cuidados de Saúde , Humanos , Prevalência , Mecanismo de ReembolsoRESUMO
Hereditary angioedema (HAE) is a chronic disease with a high burden of disease that is poorly understood and often misdiagnosed. Availability of treatments, including C1 esterase inhibitor (C1INH) replacement, ecallantide, and icatibant, marks a significant advance for HAE patients. We aimed to better understand the current state of HAE care, from a patient perspective, after the introduction of several novel therapies. One session of the United States Hereditary Angioedema Association 2013 patient summit was devoted to data collection for this study. Patients attending the summit were self-selected, and HAE diagnosis was self-reported. Survey questions assessed patient characteristics, burden of disease, and treatment. Participant responses were captured using an audience response system. We surveyed 149 (80%) type I and II HAE (HAE-C1INH) and 37 (20%) HAE with normal C1INH (HAE-nlC1INH) patients. HAE-C1INH (72%) and HAE-nlCINH patients (76%) equally reported that HAE had a significant impact on quality of life (QOL). A third of HAE-C1INH patients were diagnosed within one year of their first HAE attack, but another third reported a delay of more than 10 years. Most HAE-C1INH (88%) and HAE-nlC1INH (76%) patients had on-demand treatment available. HAE-C1INH patients frequently had an individual treatment plan (76%) compared with 50% of HAE-nlC1INH patients. Most HAE-C1INH patients went to the emergency department (ED) or were hospitalized less than once every six months (80%). Our findings show that HAE management is improving with good access to on-demand and prophylactic treatment options. However, HAE patients still have a significant burden of disease and continued research and educational efforts are needed.
Assuntos
Angioedemas Hereditários/diagnóstico , Angioedemas Hereditários/terapia , Adolescente , Adulto , Angioedemas Hereditários/epidemiologia , Angioedemas Hereditários/prevenção & controle , Criança , Pré-Escolar , Efeitos Psicossociais da Doença , Gerenciamento Clínico , Progressão da Doença , Humanos , Lactente , Pessoa de Meia-Idade , Recidiva , Inquéritos e Questionários , Adulto JovemRESUMO
Availability of effective treatment for acute attacks is expected to transform the care of hereditary angioedema (HAE) patients. We felt that it would be of interest to test these assumptions by examining the perceptions of HAE patients regarding the impact that these therapies have had on their lives. Patients at a United States HAE Association summit meeting were asked to rate the burden of HAE currently and compare by recall with 2009 when these therapies were not available. Questions covered five domains: psychological/emotional status, ability to carry out daily activities, fear of suffocation, worry about their children inheriting HAE, and medication side effects. Data were analyzed using Wilcoxon signed-rank tests or analysis of variance. Responses were obtained from 134 self-identified HAE subjects: 85 type I, 21 type II, and 28 with normal C1 inhibitor (C1INH). Burden of disease showed significant improvement in all domains except worry about children inheriting HAE. With the introduction of newer therapies, subjects with the most severe burden of illness improved more than those with milder burdens. However, significant burden of illness remained. The availability of the current treatments has substantially improved the quality of life for HAE patients in the United States, similar to a survey of Danish HAE patients regarding the introduction of home treatment. Nevertheless, our study shows that a substantial burden of illness remains for HAE patients.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/prevenção & controle , Percepção , Atividades Cotidianas/psicologia , Angioedemas Hereditários/complicações , Angioedemas Hereditários/psicologia , Criança , Dinamarca , Progressão da Doença , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/etiologia , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/psicologia , Medo/psicologia , Acessibilidade aos Serviços de Saúde , Humanos , Qualidade de Vida , Resultado do Tratamento , Estados UnidosRESUMO
Hereditary angioedema (HAE) is a rare genetic syndrome caused by a deficiency in functional C1 inhibitor that results in recurrent episodes of nonpruritic swelling of the hands, feet, arms, legs, trunk, face, genitalia, bowels, and larynx beginning in childhood or adolescence and continuing throughout the patient's lifetime. Treatment for acute HAE attacks in the United States has been transformed by new therapies that inhibit the underlying mechanisms of angioedema- notably ecallantide, a potent and specific inhibitor of plasma kallikrein, and icatibant, a selective bradykinin receptor antagonist. These treatments, combined with safer formulations of plasma-derived C1 esterase inhibitor concentrate for HAE prophylaxis and acute treatment, have greatly improved the quality of life for people with HAE, many of whom can now lead fairly normal lives. This article reviews the current therapeutic landscape for HAE, including treatment for acute angioedema attacks, short- and long-term HAE prophylaxis, and home-based therapy.
Assuntos
Angioedemas Hereditários/tratamento farmacológico , Angioedemas Hereditários/prevenção & controle , Prevenção Secundária/métodos , Adolescente , Angioedemas Hereditários/fisiopatologia , Bradicinina/análogos & derivados , Bradicinina/uso terapêutico , Criança , Ensaios Clínicos como Assunto , Efeitos Psicossociais da Doença , Feminino , Humanos , Masculino , Peptídeos/uso terapêutico , Gravidez , Resultado do Tratamento , Estados Unidos , Adulto JovemRESUMO
Hereditary angioedema (HAE) is characterized by nonpitting, nonpruritic subcutaneous, or submucosal edema that may be accompanied by a nonpruritic serpentine erythematous rash. The swelling in this autosomal dominantly inherited disorder is mediated by uncontrolled localized bradykinin production. The extremities, face, gastrointestinal tract, genitalia, larynx, and trunk are commonly involved. Nearly one-third of patients who are not treated may suffer asphyxiation from a laryngeal attack. HAE symptoms overlap with other conditions that cause swelling. An accurate diagnosis is therefore crucial when considering therapy. Recently approved treatments for HAE swelling attacks include C1 esterase inhibitor concentrate and ecallantide. This case describes a 41-year-old woman who was treated ineffectively for more than 20 years, was switched to an effective therapy, but suffered near fatal laryngeal edema due to a missed dose of the effective therapy. The case underscores the need for increased awareness of HAE among clinicians and the importance of good compliance to therapy among patients with HAE.
Assuntos
Angioedemas Hereditários/complicações , Angioedemas Hereditários/tratamento farmacológico , Proteínas Inativadoras do Complemento 1/uso terapêutico , Edema Laríngeo/etiologia , Adulto , Proteínas Inativadoras do Complemento 1/administração & dosagem , Feminino , Humanos , Unidades de Terapia Intensiva/economia , Intubação Intratraqueal , Edema Laríngeo/terapia , Adesão à Medicação , Resultado do TratamentoRESUMO
Hereditary angioedema (HAE) is a rare, autosomal dominant disorder characterized by recurrent acute attacks of swelling of the larynx, abdomen, and periphery. This study was designed to assess the humanistic burden of illness associated with HAE. HAE burden was assessed via a web-based survey of patients that solicited information on attack characterization, treatment, side effects, pain, and functional and emotional burden of disease management. In addition to HAE-specific sections, the survey used three standardized instruments to compare HAE patient data to normative (healthy) and chronic disease populations: the 12-Item Short Form (SF-12) Health Survey, the Work Productivity and Activity Impairment-General Health (WPAI-GH) questionnaire, and the Hamilton Depression Inventory-Short Form (HDI-SF). A total of 457 HAE patients responded to the survey (response rate, â¼19%). Patients reported significantly poorer health-related quality of life versus population norms, based on the SF-12 Physical Component Summary (mean, 43.7 versus 49.6; p < 0.001) and Mental Component Summary (mean, 42.6 versus 49.4; p < 0.001). HAE patients also had higher mean HDI-SF scores than population norms (8.1 ± 6.5 versus 3.1 ± 3.0; p < 0.001), with 42.5% of HAE patients scoring >8.5, indicative of depressive symptomatology. Productivity was also markedly impaired in all WPAI-GH categories, including 34% overall work impairment. Because of their most recent HAE attack, workers lost a mean of 3.3 days; students lost a mean of 1.9 days. HAE results in considerable humanistic burden to patients across physical and mental health domains; negatively impacts education, career, and work productivity; and compounds the substantial economic burdens that are reported separately.
Assuntos
Angioedemas Hereditários/psicologia , Efeitos Psicossociais da Doença , Depressão/epidemiologia , Qualidade de Vida , Adulto , Feminino , Nível de Saúde , Inquéritos Epidemiológicos , Humanos , Masculino , Pessoa de Meia-Idade , Escalas de Graduação PsiquiátricaRESUMO
BACKGROUND: Compliance with asthma medications probably results in improved outcomes, but few studies have examined this relationship. OBJECTIVE: To examine the association between medication compliance and exacerbation in asthmatic patients. METHODS: Retrospective analysis of a managed care database. The 97,743 participants (aged 6-99 years; mean age, 32.8 years) had asthma and prescriptions for controller medications. Compliance with the index medication (the first controller medication prescribed) was measured using 2 methods: medication possession ratio (MPR), calculated for 365 days after the index date, and number of prescriptions for each index medication. Exacerbation was defined as 1 or more emergency department visits or hospitalizations within 1 year of the index date. Multivariate models were used to determine the odds of exacerbation based on relative compliance for each definition of compliance. RESULTS: Based on the median MPR, more-compliant patients were less likely to experience exacerbation than less-compliant patients (odds ratio, 0.94; 95% confidence interval, 0.91-0.97; P < .001). Using the 75th percentile MPR, risk of exacerbation was even smaller (odds ratio, 0.89; 95% confidence interval, 0.86-0.92; P < .001). All the cutoff points for compliance (> or = 2 through > or = 6 prescriptions) demonstrated significantly less exacerbations in more-compliant vs less-compliant patients after adjusting for covariates. As the criteria for compliance became more stringent, more-compliant patients became increasingly less likely to have an exacerbation vs less-compliant patients. CONCLUSION: More-compliant asthmatic patients were significantly less likely to experience exacerbation than less-compliant asthmatic patients. These findings demonstrate the importance of improving medication compliance among asthmatic patients to impact outcomes.