Custo-efetividade do emprego do teste de desencadeamento oral no diagnóstico e tratamento da alergia ao leite de vaca sob a perspectiva do sistema público de saúde
Data
2024-05-29
Autores
Rodrigues, Vanessa Cristina de Castro 
Orientadores
Morais, Mauro Batista de
Tipo
Tese de doutorado
Título da Revista
ISSN da Revista
Título de Volume
Resumo
Objetivo: Estimar a relação de custo-efetividade do teste de desencadeamento oral no diagnóstico e avaliação do desenvolvimento de tolerância de lactentes em aleitamento artificial com manifestações clínicas gastrointestinais de alergia às proteínas do leite de vaca não IgE-mediadas, sob a perspectiva do sistema público de saúde. Métodos: Foi construído um modelo de decisão para comparar duas estratégias: 1. PRÁTICA RECOMENDADA de acordo com as diretrizes, que preconizam realização dos testes de desencadeamento para 100% dos pacientes; 2. PRÁTICA ATUAL, cuja frequência de realização dos testes de desencadeamento baseou-se em dados obtidos em estudo sobre prática médica para manejo da alergia ao leite de vaca gastrointestinal não IgE-mediada, realizado em 2022 com apoio da Sociedade Brasileira de Pediatria. Os dois braços diferenciavam-se apenas pela frequência de indicação médica dos testes de desencadeamento. Déficit antropométrico foi considerado como desfecho para caracterizar a ocorrência de manifestações clínicas graves. Para obtenção desse dado, foi realizada revisão sistemática e meta-análise sobre frequência de déficit antropométrico em lactentes brasileiros de 0 a 6 meses de idade com alergia ao leite de vaca. Considerou-se que 10% dos pais recusariam a realização dos testes de desencadeamento e 3,65 e 24 meses como idades de admissão e horizonte, respectivamente. Foram aplicados outros dados obtidos na pesquisa sobre prática médica, complementados com dados da literatura, custos do sistema público de saúde em moeda local (Real) e taxa de desconto de 5% para custos e desfechos. O desfecho da efetividade foi o tempo em dieta sem exclusão e os valores de AVAQ (anos de vida ajustados pela qualidade): com alergia=0,84; sem alergia=0,94. Foram realizadas análises de sensibilidade univariada e probabilística e de impacto orçamentário. Foram utilizadas as seguintes ferramentas: Formulário Google para coleta e Jamovi para análise estatística da pesquisa sobre prática médica; Próspero para registro da revisão sistemática, Rayyan para seleção dos artigos e ProMeta3 para proceder a metanálise; Microsoft Excel para cálculo da razão de custo-efetividade incremental e análise de sensibilidade. Resultados: O estudo sobre prática médica incluiu 447 participantes, dos quais 56% e 45% informaram indicar o teste de desencadeamento diagnóstico para lactentes com manifestações clínicas leves-moderadas e graves, respectivamente; e 93% o teste de desencadeamento para avaliação do desenvolvimento de tolerância. Essas probabilidades foram aplicadas na PRÁTICA ATUAL. A metanálise incluiu 387 lactentes provenientes de sete estudos. Com os dados agregados de cada estudo, fez-se a síntese utilizando modelo metanalítico de efeitos randômicos. A metanálise resultou em prevalência de déficit antropométrico de 24% (IC95% = 16%; 36%). A PRÁTICA RECOMENDADA gerou, em média, economia de R$1.208, ganho de +0,019 AVAQs e +70 dias em dieta sem exclusão/paciente. Na análise de sensibilidade probabilística, 100% das simulações da custo-efetividade e 95,4% da custo-utilidade mostraram menor custo e maior efetividade. Em cinco anos, a economia seria de R$646 milhões. Conclusões: O uso do teste de desencadeamento oral no diagnóstico e tratamento da alergia ao leite de vaca gastrointestinal não IgE-mediada é uma estratégia dominante, que melhora a qualidade de vida, proporciona mais tempo em dieta sem exclusão e reduz custos.
Objective: To assess the cost-effectiveness of the oral food challenge test in diagnosing and assessing oral tolerance development in non-breastfed infants with gastrointestinal clinical manifestations of non-IgE-mediated cow’s milk protein allergy from a public health system perspective. Methods: A decision model was developed to compare two strategies: 1. RECOMMENDED PRACTICE according to the guidelines, which recommend performing the oral challenge tests for all patients; and 2. CURRENT PRACTICE, which based the frequency of oral challenge tests on data from a 2022 survey on the medical management of non-IgE-mediated gastrointestinal cow’s milk allergy, supported by the Brazilian Society of Pediatrics. The two arms differed only in the frequency of medical indications for the oral challenge tests. Anthropometric deficit was considered an outcome characterizing the occurrence of severe clinical manifestations. To gather these data, a systematic review and meta-analysis were conducted on the frequency of anthropometric deficit in Brazilian infants aged 0 to 6 months with cow’s milk allergy. It was assumed that 10% of parents would refuse the oral challenge tests and that the ages of admission and horizon were 3.65 and 24 months, respectively. Other data obtained from the survey on medical practices supplemented with data from the literature were considered, as well as public health system costs in local currency (Brazilian Real), and a 5% discount rate for costs and outcomes. The effectiveness endpoint was the time without a cow’s milk elimination diet and the quality-adjusted life years (QALY) values: 0.84 with allergy and 0.94 without allergy. Univariate and probabilistic sensitivity analyses and budget impact evaluation were performed. The following tools were used: Google Forms for data collection, and Jamovi for statistical analysis in the survey on medical practice; Prospero for registering the systematic review, Rayyan for article selection, and ProMeta3 for the meta-analysis calculations; Microsoft Excel for calculating the incremental cost-effectiveness ratio and performing sensitivity analyses. Results: The survey on medical practice included 447 participants. Of these, 56% reported using the oral challenge test to diagnose infants with mild to moderate clinical manifestations and 45% used it for those with severe clinical manifestations. A total of 93% of the participants used the oral challenge test to assess the development of tolerance. These probabilities have been applied in ACTUAL PRACTICE. The meta-analysis included 387 infants across seven studies. The aggregated data from each study were synthesized using a random-effects meta-analytical model. The meta-analysis showed a 24% prevalence of anthropometric deficit (95% CI: 16%-36%). The RECOMMENDED PRACTICE resulted in average savings of R$1,208, a gain of +0.019 QALYs, and +70 days without a cow’s milk elimination diet per patient. In the probabilistic sensitivity analyses, all cost-effectiveness simulations and 95.4% of cost-utility simulations demonstrated lower costs and greater effectiveness. Over five years, the savings would amount to R$646 million. Conclusions: The use of the oral food challenge test in diagnosing and treating non-IgE-mediated gastrointestinal cow’s milk allergy is a dominant strategy that improves the quality of life, allows for more time without an elimination diet, and reduces costs.
Objective: To assess the cost-effectiveness of the oral food challenge test in diagnosing and assessing oral tolerance development in non-breastfed infants with gastrointestinal clinical manifestations of non-IgE-mediated cow’s milk protein allergy from a public health system perspective. Methods: A decision model was developed to compare two strategies: 1. RECOMMENDED PRACTICE according to the guidelines, which recommend performing the oral challenge tests for all patients; and 2. CURRENT PRACTICE, which based the frequency of oral challenge tests on data from a 2022 survey on the medical management of non-IgE-mediated gastrointestinal cow’s milk allergy, supported by the Brazilian Society of Pediatrics. The two arms differed only in the frequency of medical indications for the oral challenge tests. Anthropometric deficit was considered an outcome characterizing the occurrence of severe clinical manifestations. To gather these data, a systematic review and meta-analysis were conducted on the frequency of anthropometric deficit in Brazilian infants aged 0 to 6 months with cow’s milk allergy. It was assumed that 10% of parents would refuse the oral challenge tests and that the ages of admission and horizon were 3.65 and 24 months, respectively. Other data obtained from the survey on medical practices supplemented with data from the literature were considered, as well as public health system costs in local currency (Brazilian Real), and a 5% discount rate for costs and outcomes. The effectiveness endpoint was the time without a cow’s milk elimination diet and the quality-adjusted life years (QALY) values: 0.84 with allergy and 0.94 without allergy. Univariate and probabilistic sensitivity analyses and budget impact evaluation were performed. The following tools were used: Google Forms for data collection, and Jamovi for statistical analysis in the survey on medical practice; Prospero for registering the systematic review, Rayyan for article selection, and ProMeta3 for the meta-analysis calculations; Microsoft Excel for calculating the incremental cost-effectiveness ratio and performing sensitivity analyses. Results: The survey on medical practice included 447 participants. Of these, 56% reported using the oral challenge test to diagnose infants with mild to moderate clinical manifestations and 45% used it for those with severe clinical manifestations. A total of 93% of the participants used the oral challenge test to assess the development of tolerance. These probabilities have been applied in ACTUAL PRACTICE. The meta-analysis included 387 infants across seven studies. The aggregated data from each study were synthesized using a random-effects meta-analytical model. The meta-analysis showed a 24% prevalence of anthropometric deficit (95% CI: 16%-36%). The RECOMMENDED PRACTICE resulted in average savings of R$1,208, a gain of +0.019 QALYs, and +70 days without a cow’s milk elimination diet per patient. In the probabilistic sensitivity analyses, all cost-effectiveness simulations and 95.4% of cost-utility simulations demonstrated lower costs and greater effectiveness. Over five years, the savings would amount to R$646 million. Conclusions: The use of the oral food challenge test in diagnosing and treating non-IgE-mediated gastrointestinal cow’s milk allergy is a dominant strategy that improves the quality of life, allows for more time without an elimination diet, and reduces costs.
Descrição
Citação
RODRIGUES, Vanessa Cristina de Castro. Custo-efetividade do emprego do teste de desencadeamento oral no diagnóstico e tratamento da alergia ao leite de vaca sob a perspectiva do sistema público de saúde. 2024. 159 f. Tese (Doutorado em Nutrição) – Escola Paulista de Medicina, Universidade Federal de São Paulo (UNIFESP), São Paulo, 2024.