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)
Autor(a) principal: | |
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Data de Publicação: | 2024 |
Outros Autores: | , , , , |
Tipo de documento: | Artigo |
Idioma: | por |
Título da fonte: | HU Revista (Online) |
Texto Completo: | https://periodicos.ufjf.br/index.php/hurevista/article/view/43113 |
Resumo: | 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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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)Aspectos clínicos e diagnósticos, qualidade de vida, utilidade, adesão terapêutica e custos para angioedema hereditário associado à deficiência de C1-esterase: Série temática: Avaliação de Tecnologias em Saúde Hospitalar (ATS-H)Hereditary angioedema; Rare diseases; Health Economic EvaluationAngioedemas HereditáriosDoenças RarasAvaliação em SaúdeIntroduction: 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.Introdução: O angioedema hereditário associado à deficiência de C1 esterase (AEH-C1-INH) é uma doença rara (DR) que se manifesta com a ocorrência de episódios recorrentes de angioedema não pruriginoso subcutâneo ou submucoso, o que gera impactos em todos os aspectos da vida dos indivíduos. Objetivos: Avaliar a qualidade de vida e a jornada clínica e assistencial dos pacientes com AEH-C1-INH. Material e Métodos: Trata-se de um estudo observacional ambispectivo em que foram aplicados questionários relacionados à qualidade de vida e jornada assistencial, além da coleta de dados clínicos dos prontuários nos tempos 0, 6 e 12 meses. Resultados: Foram recrutados 15 indivíduos com AEH-C1-INH e a mediana (I.I.Q) de idade da amostra foi de 38 anos (30-43). O tempo médio entre os primeiros sintomas e o diagnóstico foi de 8 anos. Os dados clínicos demonstraram história familiar positiva expressiva, ocorrência importante de edema de laringe em algum momento da vida e altos índices de recorrência das crises de angioedema durante os 12 meses de estudo. A qualidade de vida apresentou prejuízo importante principalmente em aspectos físicos, emocionais e vitalidade, sem variações significativas no tempo de estudo. Além disso, vale destacar a perda de produtividade expressiva associada a gastos médios de R$3.017,00 para medicamentos e R$598,00 para exames complementares em 12 meses. Conclusão: Observa-se um panorama de perda significativa de qualidade de vida relacionada ao AEH-C1-INH, principalmente por impactos da saúde física e emocional no exercício das atividades rotineiras. Ressaltam-se os impactos econômicos da jornada terapêutica, tanto pela perda de produtividade quanto pela necessidade de financiar medicamentos e exames que deveriam ser responsabilidade do Estado por meio do Sistema Único de Saúde (SUS). Portanto, fica clara a importância de medidas públicas que busquem amenizar os impactos causados pela doença nos indivíduos acometidos.Editora UFJF2024-04-04info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionArtOrapplication/pdfhttps://periodicos.ufjf.br/index.php/hurevista/article/view/4311310.34019/1982-8047.2023.v49.43113HU Revista; v. 49 (2023); 1-81982-80470103-3123reponame:HU Revista (Online)instname:Universidade Federal de Juiz de Fora (UFJF)instacron:UFJFporhttps://periodicos.ufjf.br/index.php/hurevista/article/view/43113/27322Copyright (c) 2024 Alex de Cerqueira Silveira Figueiredo, Angelina Xavier Acosta, Regis de Albuquerque Campos, Camila Ferreira Ramos, Temis Felix, Ney Boa-Sortehttps://creativecommons.org/licenses/by/4.0info:eu-repo/semantics/openAccessde Cerqueira Silveira Figueiredo, AlexXavier Acosta, Angelinade Albuquerque Campos, RegisFerreira Ramos, Camila Felix, TemisBoa-Sorte, Ney2024-01-10T12:00:06Zoai:periodicos.ufjf.br:article/43113Revistahttps://periodicos.ufjf.br/index.php/hurevistaPUBhttps://periodicos.ufjf.br/index.php/hurevista/oairevista.hurevista@ufjf.edu.br1982-80470103-3123opendoar:2024-01-10T12:00:06HU Revista (Online) - Universidade Federal de Juiz de Fora (UFJF)false |
dc.title.none.fl_str_mv |
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) Aspectos clínicos e diagnósticos, qualidade de vida, utilidade, adesão terapêutica e custos para angioedema hereditário associado à deficiência de C1-esterase: Série temática: Avaliação de Tecnologias em Saúde Hospitalar (ATS-H) |
title |
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) |
spellingShingle |
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) de Cerqueira Silveira Figueiredo, Alex Hereditary angioedema; Rare diseases; Health Economic Evaluation Angioedemas Hereditários Doenças Raras Avaliação em Saúde |
title_short |
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) |
title_full |
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) |
title_fullStr |
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) |
title_full_unstemmed |
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) |
title_sort |
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) |
author |
de Cerqueira Silveira Figueiredo, Alex |
author_facet |
de Cerqueira Silveira Figueiredo, Alex Xavier Acosta, Angelina de Albuquerque Campos, Regis Ferreira Ramos, Camila Felix, Temis Boa-Sorte, Ney |
author_role |
author |
author2 |
Xavier Acosta, Angelina de Albuquerque Campos, Regis Ferreira Ramos, Camila Felix, Temis Boa-Sorte, Ney |
author2_role |
author author author author author |
dc.contributor.author.fl_str_mv |
de Cerqueira Silveira Figueiredo, Alex Xavier Acosta, Angelina de Albuquerque Campos, Regis Ferreira Ramos, Camila Felix, Temis Boa-Sorte, Ney |
dc.subject.por.fl_str_mv |
Hereditary angioedema; Rare diseases; Health Economic Evaluation Angioedemas Hereditários Doenças Raras Avaliação em Saúde |
topic |
Hereditary angioedema; Rare diseases; Health Economic Evaluation Angioedemas Hereditários Doenças Raras Avaliação em Saúde |
description |
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. |
publishDate |
2024 |
dc.date.none.fl_str_mv |
2024-04-04 |
dc.type.driver.fl_str_mv |
info:eu-repo/semantics/article info:eu-repo/semantics/publishedVersion ArtOr |
format |
article |
status_str |
publishedVersion |
dc.identifier.uri.fl_str_mv |
https://periodicos.ufjf.br/index.php/hurevista/article/view/43113 10.34019/1982-8047.2023.v49.43113 |
url |
https://periodicos.ufjf.br/index.php/hurevista/article/view/43113 |
identifier_str_mv |
10.34019/1982-8047.2023.v49.43113 |
dc.language.iso.fl_str_mv |
por |
language |
por |
dc.relation.none.fl_str_mv |
https://periodicos.ufjf.br/index.php/hurevista/article/view/43113/27322 |
dc.rights.driver.fl_str_mv |
https://creativecommons.org/licenses/by/4.0 info:eu-repo/semantics/openAccess |
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https://creativecommons.org/licenses/by/4.0 |
eu_rights_str_mv |
openAccess |
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application/pdf |
dc.publisher.none.fl_str_mv |
Editora UFJF |
publisher.none.fl_str_mv |
Editora UFJF |
dc.source.none.fl_str_mv |
HU Revista; v. 49 (2023); 1-8 1982-8047 0103-3123 reponame:HU Revista (Online) instname:Universidade Federal de Juiz de Fora (UFJF) instacron:UFJF |
instname_str |
Universidade Federal de Juiz de Fora (UFJF) |
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UFJF |
institution |
UFJF |
reponame_str |
HU Revista (Online) |
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HU Revista (Online) |
repository.name.fl_str_mv |
HU Revista (Online) - Universidade Federal de Juiz de Fora (UFJF) |
repository.mail.fl_str_mv |
revista.hurevista@ufjf.edu.br |
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1796798239258705920 |