RESUMO
BACKGROUND: Treatment paradigms in autoimmune hepatitis (AIH) have remained largely unchanged for decades. Studies report ≤20% of patients have sub-optimal treatment response with most requiring long-term therapy. AIM: The United Kingdom Autoimmune Hepatitis (UK-AIH) study was established to evaluate current treatment practice and outcomes, determine the unmet needs of patients, and develop and implement improved treatment approaches. METHODS: The United Kingdom Autoimmune Hepatitis study is a cross-sectional cohort study examining secondary care management of prevalent adult patients with a clinical diagnosis of autoimmune hepatitis. Enrolment began in March 2014. Prevalent cases were defined as having been diagnosed and treated for >1 year. Demographic data, biochemistry, treatment history and response, and care location were collected. RESULTS: In total, 1249 patients were recruited; 635 were cared for in transplant units and 614 in non-transplant centres (81% female with median age at diagnosis 50 years). Overall, 29 treatment regimens were reported and biochemical remission rate was 59%. Remission rates were significantly higher in transplant compared to non-transplant centres (62 vs 55%, P = 0.028). 55% have ongoing corticosteroid exposure; 9% are receiving prednisolone monotherapy. Those aged ≤20 years at diagnosis were more likely to develop cirrhosis and place of care was associated with an aggressive disease phenotype. CONCLUSIONS: There are significant discrepancies in the care received by patients with autoimmune hepatitis in the UK. A high proportion remains on corticosteroids and there is significant treatment variability. Patients receiving care in transplant centres were more likely to achieve and maintain remission. Overall poor remission rates suggest that there are significant unmet therapeutic needs for patients with autoimmune hepatitis.
Assuntos
Disparidades em Assistência à Saúde/tendências , Hepatite Autoimune/epidemiologia , Hepatite Autoimune/terapia , Adolescente , Corticosteroides/economia , Corticosteroides/uso terapêutico , Adulto , Idoso , Idoso de 80 Anos ou mais , Estudos de Coortes , Estudos Transversais , Feminino , Disparidades em Assistência à Saúde/economia , Hepatite Autoimune/economia , Humanos , Cirrose Hepática/economia , Cirrose Hepática/epidemiologia , Cirrose Hepática/terapia , Masculino , Pessoa de Meia-Idade , Prednisolona/economia , Prednisolona/uso terapêutico , Resultado do Tratamento , Reino Unido/epidemiologia , Adulto JovemRESUMO
BACKGROUND AND AIM: Little is known about population-based epidemiology and disease burden of autoimmune hepatitis (AIH). The aim of this study was to investigate the prevalence, incidence, comorbidity and direct medical cost of AIH in South Korea. METHODS: The data was from the nationwide, population-based National Health Insurance Service claims database and the Rare Intractable Disease registration program. Age and gender-specific prevalence rates were calculated, and data on comorbidity, diagnostic tests, prescribed drugs, and medical costs were retrieved for patients registered under the disease code K75.4 (AIH) from 2009 to 2013. RESULTS: A total of 4,085 patients with AIH were identified between 2009 and 2013 with a female-to-male ratio of 6.4. The age-adjusted prevalence rate was 4.82/100,000 persons and gender adjusted prevalence rates were 8.35 in females and 1.30 in males. The age-adjusted calculated incidence rate was 1.07/100,000 persons (gender-adjusted 1.83 in females and 0.31 in males). Ascites, variceal bleeding, and hepatocellular carcinoma were found in 1.4%, 1.3%, and 2.2% of the patients, respectively. Forty-six patients (1.1%) underwent liver transplantation during the study period. Case-fatality was 2.18%. Corticosteroid and azathioprine were prescribed in 44.1% and 38.0% of prevalent patients with AIH in 2013, respectively. The nationwide total direct medical cost was less than 4.0 million USD, and the average cost for each patient was 1,174 USD in 2013. CONCLUSION: This is the first report on the nationwide epidemiology of AIH in Korea, and it showed a lower prevalence than that of Western countries with considerable disease burden.
Assuntos
Efeitos Psicossociais da Doença , Hepatite Autoimune/economia , Adolescente , Corticosteroides/uso terapêutico , Adulto , Idoso , Idoso de 80 Anos ou mais , Azatioprina/uso terapêutico , Carcinoma Hepatocelular/complicações , Carcinoma Hepatocelular/diagnóstico , Carcinoma Hepatocelular/epidemiologia , Criança , Pré-Escolar , Comorbidade , Bases de Dados Factuais , Feminino , Hepatite Autoimune/diagnóstico , Hepatite Autoimune/epidemiologia , Hepatite Autoimune/terapia , Humanos , Lactente , Recém-Nascido , Neoplasias Hepáticas/complicações , Neoplasias Hepáticas/diagnóstico , Neoplasias Hepáticas/epidemiologia , Transplante de Fígado , Masculino , Pessoa de Meia-Idade , Prevalência , República da Coreia/epidemiologia , Taxa de Sobrevida , Adulto JovemRESUMO
Autoimmune hepatitis has a variable occurrence, clinical phenotype, and outcome, and the factors contributing to this variability are uncertain. The goals of this review are to examine the global disparities in the occurrence and outcome of autoimmune hepatitis, suggest bases for these disparities, and encourage investigations that extend beyond single-center experiences. Disparities in the incidence and prevalence of autoimmune hepatitis in different age groups, genders, ethnicities, and geographical regions suggest that factors other than genetic predisposition are involved. Age- and gender-related antigen exposures from the external (infections, toxins, and medications) and internal (intestinal microbiome) environment may affect the incidence of the disease, and the timeliness and nature of treatment may influence its prevalence. The increasing incidence of autoimmune hepatitis in Spain, Denmark, and the Netherlands suggests that a new etiological trigger has been introduced or that the susceptible population has changed. Variations in mortality between Western and Asian-Pacific countries may result from differences in disease detection or management, and variations in gender predilection, peak age of onset, frequency of concurrent immune diseases, and serological profile may reflect gender-biased and age-related antigen exposures and genetic predispositions. Global collaborations, population-based epidemiological studies that identify case clustering, and controlled interview-based surveys are mechanisms by which to understand these disparities and improve management. In conclusion, autoimmune hepatitis has a rising incidence in some countries and variable occurrence, phenotype, and outcome between countries and subgroups within countries. These disparities suggest that unrecognized population-based environmental, infectious, or socioeconomic factors are affecting its character.
Assuntos
Saúde Global/tendências , Disparidades em Assistência à Saúde/tendências , Hepatite Autoimune/epidemiologia , Hepatite Autoimune/terapia , Animais , Saúde Global/economia , Disparidades em Assistência à Saúde/economia , Hepatite Autoimune/economia , Humanos , Incidência , Resultado do TratamentoRESUMO
OBJECTIVE: Therapy for autoimmune hepatitis has been prednisone based for decades; however, budesonide may be equally effective with fewer side effects. Our aim was to evaluate quality-adjusted life years and health care costs of three different treatment regimens. MATERIALS AND METHODS: Treatment using prednisone, budesonide or a combination of both over a three-year period in newly diagnosed children with type I autoimmune hepatitis were simulated with a Markov model. Transition probabilities were calculated over consecutive three-month period. Costs were determined from a hospital database and health utilities were estimated from the literature. A Monte Carlo probabilistic sensitivity analysis was used to simulate the outcomes of 5000 patients in each treatment arm. RESULTS: Compared to standard therapy, budesonide leads to a gain of 0.09 quality-adjusted life years, costing $17,722 per QALY over a three-year period. Standard therapy led to significantly lower QALY's compared to other strategies (p < 0.001). Health utilities of patients in remission in each treatment group had the greatest impact on the model. Budesonide remained the treatment of choice if the probability of inducing remission was 55% or greater. CONCLUSIONS: Budesonide therapy in non-cirrhotic, treatment naïve patients with type I autoimmune hepatitis yielded greater QALY's compared to the current standard therapy with an acceptable increase in costs.
Assuntos
Anti-Inflamatórios/uso terapêutico , Budesonida/uso terapêutico , Custos de Cuidados de Saúde/estatística & dados numéricos , Hepatite Autoimune/tratamento farmacológico , Anos de Vida Ajustados por Qualidade de Vida , Adolescente , Anti-Inflamatórios/economia , Budesonida/economia , Criança , Pré-Escolar , Técnicas de Apoio para a Decisão , Esquema de Medicação , Quimioterapia Combinada , Seguimentos , Hepatite Autoimune/economia , Humanos , Illinois , Cadeias de Markov , Mercaptopurina/análogos & derivados , Mercaptopurina/economia , Mercaptopurina/uso terapêutico , Método de Monte Carlo , Prednisona/economia , Prednisona/uso terapêutico , Resultado do TratamentoRESUMO
In > 80% of patients with autoimmune hepatitis, steroid therapy alone or in combination with azathioprine results in disease remission. Treatment response results in reversal of fibrosis and excellent long-term survival in many patients, whereas untreated patients may expect a 10-year survival of < 30%. The use of azathioprine monotherapy (2 mg/kg/day) has gained widespread acceptance in maintaining remission in clinical practice. Although all patients with autoimmune hepatitis may not need treatment, particularly those with mild disease, alternative strategies are required in patients who have failed to achieve remission on standard therapy of steroids with or without azathioprine, or patients with azathioprine-induced drug toxicity. In such circumstances, the use of salvage therapy in the form of ciclosporin, tacrolimus or mycophenolate mofetil may be warranted. Liver transplantation is the treatment of choice for patients who present with subacute liver failure or decompensated cirrhosis. Salvage therapy results in an exponential rise in cost with each increment in therapeutic escalation. As an alternative to standard therapy, it has also been suggested that novel therapies such as ciclosporin, tacrolimus or mycophenolate mofetil be initiated to achieve remission. However, a > 10-fold cost differential exists between the charges associated with more potent immunosuppression and standard therapy. Therefore, in evaluating novel immunosuppression in autoimmune hepatitis, it behoves clinicians not only to consider end points pertaining to efficacy, but also end points pertaining to cost-effectiveness. Moreover, the exact role of pharmacogenomics and genotyping of thiopurine methyltransferase in patients with autoimmune hepatitis needs to be fully defined.
Assuntos
Hepatite Autoimune/tratamento farmacológico , Hepatite Autoimune/economia , Preparações Farmacêuticas/economia , Corticosteroides/economia , Corticosteroides/uso terapêutico , Azatioprina/economia , Azatioprina/uso terapêutico , Análise Custo-Benefício/economia , Análise Custo-Benefício/tendências , Humanos , Fatores Imunológicos/economia , Fatores Imunológicos/uso terapêutico , Preparações Farmacêuticas/administração & dosagemRESUMO
AIH is a chronic liver disease that has been associated with hepatic failure and death in the absence of liver transplantation. As a result, AIH imparts significant medical and economic burdens on affected patients and health care delivery systems, respectively. The use of accepted methodologies for outcomes and health services research has identified emerging information on the epidemiology and natural history, HRQoL, and resource utilization for similar autoimmune chronic liver diseases such as PBC and PSC. Similar efforts are needed in AIH, and they are supported on the basis of existing data which suggest similar levels of disease burden compared to PBC and PSC. As a result, the ability to plan for disease management strategies in AIH that require the allocation of scarce resources will be feasible.