ABSTRACT Background: Cirrhosis is one of the final stages of chronic liver disease. Common causes of cirrhosis include alcoholism and viral hepatitis infections. Cirrhosis can progress from an asymptomatic, compensated phase to decompensation and the appearance of overt symptoms. There is no specific treatment for decompensated cirrhosis. The ANSWER trial positioned long-term albumin infusions as a potential treatment for patients with cirrhosis and uncomplicated ascites. Objective: This study assesses the economic impact of albumin infusions following the ANSWER trial regimen in Brazilian patients with decompensated cirrhosis from the public and private healthcare systems perspectives. Methods: The incremental cost per patient per year was calculated for standard medical treatment (SMT) plus long-term albumin infusions versus SMT alone. Costs of diuretics and albumin were obtained from Banco de Preços em Saúde and the Drug Market Regulation Chamber. Costs for complication and procedures were gathered from the published literature. Costs were transformed to 2021 Brazilian reals (BRL). Incidences of clinical complications and treatments were gathered from the ANSWER trial. Univariate sensitivity analysis was performed by increasing and decreasing all inputs by 20%. Results: The cost per patient per year was 118,759 BRL and 189,675 BRL lower for patients treated with SMT and albumin (compared to SMT only) for the public and private healthcare systems, respectively. The additional cost of albumin was offset by reduced complications and treatments (149,526 BRL and 249,572 BRL, respectively). The univariate sensitivity analysis showed cost savings for both healthcare systems in all the scenarios assessed. Conclusion: This economic analysis suggests that, if the ANSWER trial clinical outcomes translate into real-world effectiveness, addition of albumin infusions to SMT in patients with decompensated cirrhosis may lead to cost savings for the public and private healthcare systems in Brazil.

RESUMO Contexto: A cirrose representa o estágio final da doença hepática crônica. Causas comuns de cirrose incluem alcoolismo e infecções por hepatite viral. A cirrose pode progredir de uma fase compensada assintomática para descompensação e aparecimento de sintomas evidentes. Não há tratamento específico para cirrose descompensada. O estudo ANSWER demonstrou que a administração de albumina a longo prazo pode representar um potencial tratamento para pacientes com cirrose e ascite não complicada. Objetivo: Nosso estudo avalia o impacto econômico da administração de albumina a longo prazo seguindo o protocolo do estudo ANSWER em pacientes brasileiros com cirrose descompensada, sob a perspectiva dos sistemas de saúde público e privado. Métodos: O custo incremental por paciente por ano foi calculado para o tratamento médico padrão (SMT) associado a administração de albumina a longo prazo comparado a SMT apenas. Os custos de diuréticos e albumina foram obtidos no Banco de Preços em Saúde e na Câmara de Regulação do Mercado de Medicamentos. Os custos de complicações e procedimentos foram coletados da literatura publicada. Os custos foram transformados em Reais de 2021 (BRL). As incidências de complicações clínicas e tratamentos foram coletadas do estudo ANSWER. Uma análise de sensibilidade univariada foi realizada aumentando e diminuindo todas as variáveis em 20%. Resultados: O custo por paciente por ano foi de R$ 118.759 e R$ 189.675 menor para pacientes tratados com SMT e albumina (comparado apenas com SMT) para os sistemas de saúde público e privado, respectivamente. O custo adicional da albumina foi compensado pela redução de complicações e tratamentos (149.526 BRL e 249.572 BRL, respectivamente). A análise de sensibilidade univariada mostrou redução de custos para ambos os sistemas de saúde em todos os cenários avaliados. Conclusão: Esta análise econômica sugere que, se os resultados clínicos do estudo ANSWER se confirmarem no mundo real, a administração de albumina associada ao SMT em pacientes com cirrose descompensada pode levar a redução de custos para os sistemas de saúde público e privado no Brasil.

Resumen Objetivo: Estimar el uso de recursos y costes asociados al seguimiento de pacientes con infección por el virus de la inmunodeficiencia humana tras discontinuación del tratamiento antirretroviral actual debido a falta de efectividad o toxicidad inaceptable y cambio a un nuevo tratamiento antirretroviral, comparado con el seguimiento habitual de los pacientes con tratamiento antirretroviral, desde la perspectiva del Sistema Nacional de salud español. Método: Se identificó el uso de recursos (pruebas clínicas, visitas médicas, visitas a la farmacia hospitalaria) asociado al seguimiento de pacientes con infección por el virus de la inmunodeficiencia humana en tres perfiles de pacientes (estable, discontinuación y cambio por falta de efectividad, discontinuación y cambio por toxicidad inaceptable), a partir de las guías de práctica clínica y un panel de expertos multidisciplinar (. = 5). Los expertos consensuaron los principales eventos adversos que conducían a la discontinuación, agrupándolos en: alteraciones gastrointestinales, renales, óseas, musculoesqueléticas, dermatológicas, hepáticas y del perfil lipídico, trastornos neuropsiquiátricos y sexuales. Los costes unitarios se identificaron a partir de bases de datos oficiales costes sanitarios y de la literatura. Se estimó el coste (€, 2020) del seguimiento en cada perfil de paciente, sin incluir el coste derivado del tratamiento antirretroviral, en un horizonte temporal de dos años. Resultados: El coste por paciente a dos años se estimó en 4.148 € (pruebas: 2.293 €; visitas: 1.855 €) para el seguimiento del paciente estable. El seguimiento del paciente tras discontinuación por falta de efectividad y cambio de tratamiento antirretroviral se estimó en 5.434 € (pruebas: 2.777 €; visitas: 2.657 €). El coste del seguimiento tras la discontinuación por toxicidad inaceptable y cambio de tratamiento antirretroviral varió en función del evento adverso que motivó el cambio, oscilando entre 4.690 € para las alteraciones del perfil lipídico, y 5.304 € para las alteraciones musculoesqueléticas. En este perfil de pacientes, las pruebas variaron entre 2.403 € y 3.017 € y las visitas entre 2.287 € y 2.842 €. Conclusiones: El coste asociado al seguimiento del paciente con infección por el virus de la inmunodeficiencia humana tras discontinuación y cambio a un nuevo tratamiento antirretroviral es mayor comparado con el seguimiento habitual, sin tener en cuenta el coste farmacológico. La tasa de discontinuación del tratamiento antirretroviral es un factor relevante a la hora de seleccionar la terapia más adecuada para cada paciente.

Abstract Objective: To assess the use of resources and the costs associated with following up patients infected with the human immunodeficiency virus after discontinuation of an antiretroviral treatment and initiation of a new one due to a lack of effectiveness or unacceptable toxicity, as compared to the costs involved in the routine follow-up of patients on antiretroviral treatment, from the Spanish National Health System perspective. Method: The use of resources (clinical tests, medical visits, and hospital pharmacy visits) associated with following three profiles of patients infected with the human immunodeficiency virus (stable ones, those discontinuing an existing antiretroviral treatment and being switched to a new one due to a lack of effectiveness, and those discontinuing an existing antiretroviral treatment and being switched to a new one due to unacceptable toxicity) was identified, based on clinical practice guidelines and the findings of a multidisciplinary expert panel (n = 5). The experts agreed on the main adverse events leading to discontinuation, classifying them into gastrointestinal, renal, osseous, musculoskeletal, dermatological, hepatic, lipid profile-related, neuropsychiatric and sexual alterations. Unit costs were identified from official healthcare costs databases. The cost (€, 2020) of following up each patient profile was estimated, excluding the cost of the antiretroviral treatment itself, with a time horizon of two years. Results: The per-patient cost of following up stable patients over two years was estimated at €4,148 (tests: €2,293; visits: €1,855). Patient follow-up after discontinuation of an existing antiretroviral treatment and initiation of a different one due to a lack of effectiveness was estimated at €5,434 (tests: €2,777; visits: €2,657). The cost of follow-up after discontinuation of an existing regimen and initiation of a new one due to unacceptable toxicity varied according to the adverse event prompting the switch, ranging from €4,690 for lipid profile dysregulation, to €5,304, for musculoskeletal alterations. In this patient profile, the cost of tests ranged from €2,403 to €3,017, and that of visits from €2,287 to €2,842. Conclusions: The cost associated with following up of patients infected with the human immunodeficiency virus after discontinuation of an existing antiretroviral regimen and initiation of a new one is higher tan that of routine follow-up, without taking the cost of drugs into account. The treatment discontinuation rate is a relevant factor when selecting the most appropriate therapy for each patient.

Introduction: Establishing a home enteral nutrition (HEN) that is adapted to the patient's needs and preferences can improve their quality of life and adherence, contributing to a higher treatment efficiency, better prognosis and cost reduction. As in most cases, patients need total or partial help from their caregivers, and there is a need for evaluating how well do caregivers know their patients' priorities. Objectives: Exploring patients' preferences for HEN characteristics in Spain, and the concordance between patients and caregivers. Methods: A cross-sectional observational study based on the discrete choice experiment methodology. A set of eight choice scenarios generated from six attributes with two levels each was presented along with an ad hoc questionnaire on the importance and satisfaction with HEN. The relative importance (RI) of each attribute and the patient-caregiver concordance was estimated in every question. Results: One hundred and forty-eight patients participated, and in 77 cases both the patient and the caregiver took the survey. The most important attributes (RI) for HEN were adaptation to comorbidities (33%), tolerability (33%), nutrients and calories (26%) and package characteristics (8%). Patients showed a high degree of satisfaction with the assistance received. Concordance between patient and caregiver was found in every question, being it moderate to good. Conclusions: According to patients, an ideal HEN product would be adaptable to comorbidities, easy to tolerate, providing the necessary nutrients and calories, with an easy to handle package. Caregivers know well their patients' preferences.

Introducción: establecer una nutrición enteral domiciliaria (NED) adaptada a las necesidades y preferencias del paciente puede mejorar la calidad de vida y la adherencia, contribuyendo a una mayor eficiencia del tratamiento, mejor pronóstico y reducción de costes. En muchos casos los pacientes requieren ayuda total o parcial de cuidadores, por lo que es importante evaluar hasta qué punto estos conocen sus preferencias. Objetivos: explorar las preferencias de los pacientes por las características de la NED en España, y la concordancia entre paciente y cuidador. Métodos: estudio observacional, transversal, basado en la metodología del experimento de elección discreta. Se presentaron ocho escenarios de elección generados a partir de seis atributos con dos niveles, y un cuestionario ad hoc de importancia y satisfacción con la NED. Se estimó la importancia relativa (IR) de cada atributo y la concordancia paciente-cuidador en todas las cuestiones. Resultados: participaron 148 pacientes; en 77 casos cumplimentaron las cuestiones los pacientes y sus cuidadores. Los atributos más importantes (IR) en la NED fueron adaptabilidad a comorbilidades (33%), tolerabilidad (33%), aporte de nutrientes y calorías (26%) y características del envase (8%). Los pacientes mostraron un elevado grado de satisfacción con la asistencia recibida. Se encontró concordancia paciente-cuidador en todas las cuestiones, siendo esta de moderada a buena. Conclusiones: según los pacientes, el producto para NED ideal se adapta a las comorbilidades, es fácilmente tolerable, y aporta los nutrientes y calorías necesarios, con un envase de fácil manejo. Los cuidadores conocen bien las preferencias del paciente.