Clinical and diagnostic aspects, quality of life, usefulness, therapeutic adherence and costs for hereditary angioedema associated with C1-esterase deficiency
Série temática: Avaliação de Tecnologias em Saúde Hospitalar (ATS-H)
DOI:
https://doi.org/10.34019/1982-8047.2023.v49.43113Keywords:
Hereditary angioedema; Rare diseases; Health Economic EvaluationAbstract
Introduction: Hereditary Angioedema (HAE) associated with C1 esterase deficiency (C1-INH) is a rare disease (RD) that manifests with recurrent episodes of non-pruritic subcutaneous or submucosal angioedema, which impacts on all aspects of the individual's life. Objectives: to evaluate the quality of life and the clinical and care journey of patients with HAE. Material and methods: This is an ambispective observational study in which questionnaires related to quality of life and care journey were applied, in addition to the clinical data collection from medical records at 0, 6 and 12 months. Results: 15 subjects with HAE were recruited and the median (IQR) age of the sample was 38 years (30-43). The average time between the first symptoms and diagnosis was 8 years. The clinical data showed a substantial positive family history, considerable occurrence of laryngeal edema at some point in life and high rates of recurring angioedema crises during the 12 months of the study. Quality of life was significantly impaired, especially in terms of physical and emotional aspects and vitality, with no significant variations over the study period. It is also worth noting the major loss of productivity associated with average costs of R$3,017.00 for medication and R$598.00 for complementary tests over 12 months. Conclusion: There is a noticeable loss of quality of life related to HAE, mainly due to the impact on physical and emotional health when carrying out routine activities. The economic impacts of the therapeutic journey stand out, both due to the loss of productivity and the need to finance medicines and tests that should be the responsibility of the State through the Unified Health System (SUS). Therefore, the importance of public measures that seek to mitigate the impacts caused by the disease on affected individuals is evident.
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Copyright (c) 2024 Alex de Cerqueira Silveira Figueiredo, Angelina Xavier Acosta, Regis de Albuquerque Campos, Camila Ferreira Ramos, Temis Felix, Ney Boa-Sorte
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