Background: Hereditary Transthyretin Amyloidosis Polyneuropathy is a rare life-threatening neurologic disease that imposes considerable mortality and it is associated with progressive related disabilities. In this study, we aimed to assess the effect of the disease across health-related quality of life dimensions, in both carriers of the mutation and patients, to compare health-related quality of life with gene...
Background: Hereditary Transthyretin Amyloidosis Polyneuropathy is a rare life-threatening neurologic disease that imposes considerable mortality and it is associated with progressive related disabilities. In this study, we aimed to assess the effect of the disease across health-related quality of life dimensions, in both carriers of the mutation and patients, to compare health-related quality of life with gene...
Background Hereditary Transthyretin Amyloidosis Polyneuropathy is a rare life-threatening neurologic disease that imposes considerable mortality and it is associated with progressive related disabilities. In this study, we aimed to assess the effect of the disease across health-related quality of life dimensions, in both carriers of the mutation and patients, to compare health-related quality of life with gener...
Background: Hereditary Transthyretin Amyloidosis Polyneuropathy is a rare life-threatening neurologic disease that imposes considerable mortality and it is associated with progressive related disabilities. In this study, we aimed to assess the effect of the disease across health-related quality of life dimensions, in both carriers of the mutation and patients, to compare health-related quality of life with gene...
The burden of pneumococcal disease in adults is substantial from a social and economic point of view. This study assessed the cost-effectiveness of the 13-valent pneumococcal conjugate vaccine (PCV13) for the prevention of invasive pneumococcal disease and pneumococcal pneumonia in adults versus "no vaccination" and versus vaccination with the 23-valent pneumococcal polysaccharide vaccine (PPSV23). A Markov mod...
Transthyretin-associated familial amyloid polyneuropathy (TTR-FAP) is a neurological disease that affects severely patients and their families and caregivers over generations. It is a rare, progressive, and if untreated fatal autosomal dominant hereditary disorder. The disease may affect multiple organ systems and if untreated progress rapidly to death. TTR-FAP affects nearly 10,000 people worldwide, with known...
OBJECTIVE: To assess the natural history and treatment effect on survival among patients with transthyretin-associated familial amyloid polyneuropathy (TTR-FAP) stage 1 Val30Met. METHODS: Multi-institutional, hospital-based study of patients with TTR-FAP Val30Met prospectively followed up until December 2016, grouped into untreated (n = 1,771), liver transplant (LTx)-treated (n = 957), or tafamidis-treated (n =...
Introduction: The recently created Health Technology Assessment System (SiNATS) in Portugal will allow the reassessment of health technologies in a clinical practice context. This position paper intends to list and briefly describe the numerous challenges and limitations that may influence the premises and the Outcomes of the health technology reassessments and therefore its own purpose. This paper reflects the...
Introduction: The recently created Health Technology Assessment System (SiNATS) in Portugal will allow the reassessment of health technologies in a clinical practice context. This position paper intends to list and briefly describe the numerous challenges and limitations that may influence the premises and the outcomes of the health technology reassessments and therefore its own purpose. This paper reflects the...
Introdução e objetivos: Os non-vitamin K antagonist oral anticoagulants (NOAC) foram recentemente comparticipados para a fibrilhação auricular não-valvular (FA), sendo relevante determinar o seu custo-efetividade para a realidade portuguesa. Métodos: Foi especificado um modelo Markov para simular a progressão dos doentes com FA no decurso da sua vida. Os dados de efetividade relativa para os eventos acidente va...