13 documents found, page 1 of 2

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Health-related quality of life in hereditary transthyretin amyloidosis polyneur...

Inês, Mónica; Coelho, Teresa; Conceição, Isabel; Ferreira, Lara; Carvalho, Mamede de; Costa, João

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...


Health-related quality of life in hereditary transthyretin amyloidosis polyneur...

Inês, Mónica; Coelho, Teresa; Conceição, Isabel; Ferreira, Lara; de Carvalho, Mamede; Costa, João

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...


Health-related quality of life in hereditary transthyretin amyloidosis polyneur...

Inês, Mónica; Coelho, Teresa; Conceição, Isabel; Ferreira, Lara; de Carvalho, Mamede; Costa, João

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...


Health-related quality of life in hereditary transthyretin amyloidosis polyneur...

Inês, Mónica; Coelho, Teresa; Conceição, isabel; Ferreira, Lara; Carvalho, Mamede; Costa, João

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...


Cost-effectiveness of the 13-valent pneumococcal conjugate vaccine in adults in...

Gouveia, Miguel; Jesus, Gonçalo; Inês, Mónica; Costa, João; Borges, Margarida

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...


Survival in transthyretin familial amyloid polyneuropathy : a review

Inês, Mónica; Costa, João

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...


Natural history and survival in stage 1 Val30Met transthyretin familial amyloid...

Coelho, Teresa; Inês, Mónica; Conceição, isabel; Soares, Marta; Carvalho, Mamede de; Costa, João

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 =...


The Challenges of Health Technology Assessment in Portugal

Laires, Pedro; Inês, Mónica; Gouveia, Miguel; Mateus, Céu; Miguel, Luis Silva; Gouveia Pinto, Carlos

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...


The Challenges of Health Technology Assessment in Portugal

Laires, Pedro; Inês, Mónica; Gouveia, Miguel; Mateus, Céu; Silva Miguel, Luis; Gouveia Pinto, Carlos

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...


Custo-efetividade dos novos anticoagulantes orais na fibrilhação auricular em P...

Costa, João; Fiorentino, Francesca; Caldeira, Daniel; Inês, Mónica; Pereira, Catarina Lopes; Pinheiro, Luís; Vaz-Carneiro, António; Borges, Margarida

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...


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