Browsing by Issue Date, starting with "2020-03-06"
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- Uso off-label de medicamentos em pediatriaPublication . Domingues, Ana Margarida Alcario Lavaredas; Ramalhinho, Isabel Maria Pires SebastiãoDefine-se como uso off-label de medicamentos quando a sua utilização não é coincidente com as indicações terapêuticas, posologia, via de administração ou faixa etária aprovadas oficialmente pelas entidades reguladoras. Este tipo de utilização de medicamentos tem sido uma prática comum e frequente em situações para as quais não se encontram ainda descritas alternativas terapêuticas. O uso off-label levanta várias questões, nomeadamente, de natureza científica, ética e jurídica, por todos os riscos que acarreta. Contudo, pode ser, por vezes, a única alternativa em grupos especiais, tal como a Pediatria. A escassa investigação sobre a população pediátrica, por parte da indústria farmacêutica, justifica-se pelo expectável baixo retorno económico e consequentemente reduzido investimento, para além de eventuais questões éticas associadas à investigação neste grupo. O objetivo desta dissertação foi quantificar e caracterizar o uso off-label na população pediátrica, em Portugal, no distrito de Faro. Para tal efetuou-se um estudo descritivo, transversal deste tipo de prescrição em farmácias comunitárias do Sotavento e Barlavento Algarvio. A amostra foi constituída por doentes até aos 18 anos de idade, selecionados aleatoriamente, tendo sido aplicado um questionário após a dispensa dos medicamentos. Do estudo efetuado foi possível concluir que a taxa de prescrição off-label foi de 15,5%. As especialidades clínicas de Pediatria e Medicina Geral e Familiar foram as mais solicitadas para a amostra estudada. Os tipos de uso em off-label foram principalmente a dose e a faixa etária, incidindo apenas nos subgrupos dos lactentes e das crianças. Os medicamentos anti-infeciosos para uso sistémico e sistema respiratório foram os que apresentaram maior taxa de prescrição off-label. Este estudo é um contributo para melhorar o conhecimento da realidade do uso off-label de medicamentos, também em contexto de farmácia comunitária, identificando padrões de prescrição, tendências, prioridades e oportunidades de melhoria da utilização segura e eficaz de medicamentos nesta população especial.
- Resiliência regional no Algarve: uma pesquisa qualitativa baseada na perceção de atores estratégicos da regiãoPublication . Santos, Fernanda Rosenberg; Pinto, Hugo Emanuel dos Reis Sales da CruzThis dissertation is based in a qualitative and exploratory research to understand the socio-economic resilience on the Portuguese region of the Algarve since it was impacted by the 2008 global economic crisis. The study is the result of a qualitative analysis exercise by applying a bottom-up method and includes specific aspects of the literature review based on evolutionary resilience. The study investigated the current path of the Algarve after the crisis, through objective questions based on the perception of the key actors in this region. Respondents sought to answer the main research questions: (a) What are the negative aspects of the 2008 crisis? and (b) What did the actors of the region draw as lessons from this crisis? In order to collect the main data for this research, the interviews were recorded and transcribed directly into a specific data storage system for qualitative analysis – MAXQDA. The results obtained show that the Algarve, as a tourist destination, is highly specialized in this sector. But sometimes this excessive specialisation causes constraints on the development of the other sectors and in the whole economy. In this research, the actors gain voice and share their views about the development of the region. Thus, for the Algarve region to guarantee the opportunity to growth, it has to share a governance system among the region's municipalities, improve public accessibility through inter municipal public transportation in the region, as well as to establish tools to strengthen the unique and expressive image of the Algarve.
- Health-related quality of life in hereditary transthyretin amyloidosis polyneuropathy: a prospective, observational studyPublication . Inês, Mónica; Coelho, Teresa; Conceição, Isabel; Ferreira, Lara; de Carvalho, Mamede; Costa, JoãoBackground 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 general population, as well as to explore health-related quality of life prognostic factors among patients, including disease progression and treatment. Methods This study was a multi-institutional, longitudinal, prospective, observational study of hereditary Transthyretin Amyloidosis Polyneuropathy Portuguese adult subjects (621 asymptomatic carriers and 733 symptomatic patients) enrolled in the Transthyretin Amyloidosis Outcomes Survey. Health-related quality of life was captured with the preference-based instrument EQ-5D-3 L. For general population the dataset included all subjects enrolled in a representative national study (n = 1500). Different econometric models were specified; multivariate probit, generalized linear model and generalized estimating equations model; including demographic and clinical covariates. Results Hereditary Transthyretin Amyloidosis Polyneuropathy patients have their health status severely impaired in all quality of life dimensions and more anxiety/depression problems were found among asymptomatic carriers. No differences on utility were found between carriers and general population (p = 0.209). Among patients, the utility value is estimated to be 0.51 (0.021), a decrement of 0.27 as compared with general population utility. Higher disease duration, advanced disease stage and not receiving treatment are associated with impaired health-related quality of life. No differences were found between genders (p = 0.910) or between late (≥50 years) and early-onset patients (p = 0.254). The utility estimate ranged from 0.63 (0.009) in stage I to 0.01 (0.005) in stage IV. Conclusions Hereditary Transthyretin Amyloidosis Polyneuropathy symptoms and progressive associated disabilities substantially decrease patient’s health-related quality of life. Clinical strategies focused on health-related quality of life preservation such as close follow-up of asymptomatic carriers, prompt diagnosis and adequate, early treatment would benefit patient’s long-term outcomes, slowing the progressive decline in health-related quality of life.