PB1155 - Cost of factor concentrates in the Brazilian Protocol of Immune Tolerance Induction: preliminary results of the Co$tIT Study

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

SM

Suely M. Meireles Rezende, MD, PhD

Full Professor
Universidade Federal de Minas Gerais
Belo Horizonte, Minas Gerais, Brazil

Co-Author(s)

VF

Vivian K B Franco

Researcher
Centro de Hematologia e Hemoterapia de Santa Catarina (Hemosc), Santa Catarina, Brazil
Florianópolis, Santa Catarina, Brazil
JA

Jamila Angela da Silva

Pharmacist
Centro de Hematologia e Hemoterapia de Santa Catarina - HEMOSC
Florianópolis, Santa Catarina, Brazil
Maíse M. Dias

Researcher
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto, Universidade de São Paulo, São Paulo, Brazil
Ribeirão Preto, Sao Paulo, Brazil
Ad

Andrea G. de Oliveira

Researcher
Fundação Centro de Hematologia e Hemoterapia de Minas Gerais (Hemominas), Minas Gerais, Brazil
Belo Horizonte, Minas Gerais, Brazil
AV

Andréa Vilela De Oliveira Santos

Pharmacist
Fundação HEMOMINAS, Belo Horizonte, Brazil
Belo Horizonte, Minas Gerais, Brazil
RR

Rosângela A. Ribeiro

Researcher
Centro de Hematologia e Hemoterapia do Ceará (Hemoce), Ceará, Brazil
Fortaleza, Ceara, Brazil
NM

Nathália Martins Beserra

Pharmacist
Centro de Hematologia e Hemoterapia do Ceará (HEMOCE), Fortaleza, Brazil
Fortaleza, Ceara, Brazil
CS

Claudia Santos Lorenzato

Haematologist
Centro de Hematologia e Hemoterapia do Paraná (HEMEPAR),
Curitiba, Parana, Brazil
JA

Juliana Alvares-Teodoro

Pharmacist
Faculty of Pharmacy, Universidade Federal de Minas Gerais
Belo Horizonte, Minas Gerais, Brazil
Ricardo M. Camelo, MD, MSc, MBA

Physician
Faculty of Pharmacy, Universidade Federal de Minas Gerais
Belo Horizonte, Minas Gerais, Brazil

Background: Immune tolerance induction (ITI) is the standard treatment for eradicating inhibitors in haemophilia A (HA), but is costly. The Brazilian Ministry of Health purchases factor concentrates (FC) in a centralized manner. Therefore, the prices tend to be one of the lowest in the world market of hemophilia. However, assessment of cost on expenditure of FC in ITI is still missing in Brazil.

Aims: To access the costs of FC used during ITI in patients with HA (PwHA) using a national ITI protocol.

Methods: Co$tIT is a subset of the BrazIT, a Brazilian Cohort Study aimed at evaluating the predictors of ITI response. The protocol was based on a low-dose ITI regimen. For this study, we collected number of bleeding episodes, duration/outcome of ITI, and cost of FC use during ITI of PwHA from four hemophilia treatment centers in Brazil. We analysed the costs according to the outcome of ITI, which was defined as complete/partial success, and failure. All patients/guardians signed informed consent. The study was approved by ethical committees.

Results: We evaluated 62 severe (FVIII < 1%) PwHA on ITI. Total/partial success was achieved in 42 (68%), and 20 (32%) failed. Duration of ITI was longer (37 months vs 24 months) and there were more bleeding episodes (16.5 vs 4.5) in patients who failed compared with total/partial success, respectively. During ITI, the median total cost with FVIII (US$379,448.25 vs US$138,180.20), activated prothrombin complex concentrate (US$191,420.25 vs US$28,280.00) and recombinant activated factor VII (US$580,720. 25 vs US$0.00) was higher in patients who failed compared with total/partial success, respectively.

Conclusion(s): ITI represents a substantial economic impact for the Brazilian Public Health System, especially when failure occurs. Predictors of response to ITI are awaited once could be used to focus on non-responders PwHA and drive management to other therapeutic strategies.