Building the National Program of Inherited Bleeding Disorders in Brazil

Rezende, Suely Meireles; Jardim, Letícia Lemos; Magalhães, Vera Lúcia; Melo, Helder Teixeira; Araújo, João Paulo Baccara; Genovez, Guilherme

doi:10.1182/bloodadvances.2019gs121559

Cited by 4 publications

(4 citation statements)

References 3 publications

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“…In Brazil, as mentioned, the Ministry of Health is responsible for purchasing and distributing procoagulants for the treatment of HA. 16,17,30 The trading process is responsible for lowering the costs of overall HA treatment. 29,30 However, the cost-effectiveness of different treatment options needs to be evaluated based on real-world data.…”

Section: Discussionmentioning

confidence: 99%

“…2,14,15 In Brazil, the treatment of HA is guaranteed by the Public Health System (in Portuguese, Sistema Único de Saúde [SUS]). 16,17 Since 2011, the Brazilian ITI Protocol was implemented with the aim of providing a treatment option for PsHAhri. In 2019, emicizumab was incorporated into the SUS for the treatment of those who failed ITI.…”

Section: ) Introductionmentioning

confidence: 99%

“…In 2019, emicizumab was incorporated into the SUS for the treatment of those who failed ITI. 16,17 According to the Brazilian Immune Tolerance (BrazIT) Study, which aims to register the factors related to the outcome of the Brazilian ITI Protocol, the success rate is about 65%, 18 which means that the remaining 35% PsHAhri who failed ITI will be offered emicizumab as prophylaxis. However, emicizumab is not currently indicated by the Brazilian Ministry of Health as prophylaxis during ITI.…”

Section: ) Introductionmentioning

confidence: 99%

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Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab

Camelo

Barbosa

Araújo

et al. 2023

Value in Health Regional Issues

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Section: Discussionmentioning

confidence: 99%

Section: ) Introductionmentioning

confidence: 99%

Section: ) Introductionmentioning

confidence: 99%

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Economic Evaluation of Immune Tolerance Induction in Children With Severe Hemophilia A and High-Responding Inhibitors: A Cost-Effectiveness Analysis of Prophylaxis With Emicizumab

Camelo

Barbosa

Araújo

et al. 2023

Value in Health Regional Issues

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“…The Brazilian ITI program was implemented as a National Government public policy in 2011 [ 26 ]. Moreover, in Brazil, factor concentrates for haemophilia care are purchased centrally by the Ministry of Health (MoH) and distributed to HTCs across the country [ 27 , 28 ]. Currently, about 450 PwHA have been included in this program.…”

Section: Introductionmentioning

confidence: 99%

Predictors of the outcome of immune tolerance induction in patients with haemophilia A and inhibitors: The Brazilian Immune Tolerance (BrazIT) Study protocol

et al. 2021

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The development of inhibitors is the main complication of haemophilia A (HA) treatment. Immune tolerance induction (ITI) is the treatment of choice for inhibitor eradication. We describe the methodology of the Brazilian Immune Tolerance Induction (BrazIT) Study, aimed to identify clinical, genetic, and immune biomarkers associated with response to ITI and inhibitor recurrence. This cohort study includes people with HA (PwHA) and inhibitors (a) who require bypassing agents to treat and/or prevent bleeding, and (b) who are at any stage of ITI treatment. Patients are included in each haemophilia treatment centre (HTC). Factor VIII (FVIII) and inhibitor assessments are performed at local laboratories of each HTC. The ITI regimen followed the national protocol of the Brazilian Ministry of Health. All PwHA starts with low-dose ITI (50 IU/kg three times weekly); high-dose regimen (100 IU/kg daily) is used if there is lack of response to the low-dose ITI. Outcomes are classified as total or partial success, and failure. Standardized case report forms with clinical, laboratory, and treatment data are collected from medical files and interviews. Blood samples are collected for genetic and immune biomarkers at the time of inclusion in the study and at the end of ITI. The study is ongoing and, currently, 202/250 (80.8%) PwHA from 15 HTCs have been included. BrazIT Study is the largest cohort of PwHA and inhibitor under treatment with the same ITI regimen reported to date. This study is likely to contribute with novel predictors of ITI response.

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