PB0227 - Cost evaluation of clotting factors concentrates before, during and after immune tolerance Induction according to outcome in Congenital Haemophilia A and inhibitor

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Ricardo Mesquita Camelo, MD, MBA, MSc, PhD, PostD

Professor
Department of Internal Medicine, Faculty of Medicine, Universidade Federal de Minas Gerais (UFMG), Belo Horizonte, Brazil
Belo Horizonte, Minas Gerais, Brazil

Co-Author(s)

VF

Vivian Karla Brognoli Franco

Hematologist
Faculty of Medicine, Universidade Federal de Santa Catarina, Florianópolis, Brazil
Florianópolis, Santa Catarina, Brazil
Maíse Moreira Dias

Hematologist
Faculty of Medicine, Universidade Federal de Minas Gerais (UFMG)
Belo Horizonte, Minas Gerais, Brazil
AO

Andréa Oliveira

Hematologist
Fundação Hemominas, Belo Horizonte, Brazil
Belo Horizonte, Minas Gerais, Brazil
NB

Nathália Martins Beserra

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

Claudia Santos Lorenzato

Hematologist
Centro de Hematologia e Hemoterapia do Paraná (Hemepar), Curitiba, PR, Brasil
Rio de Janeiro, Parana, Brazil
RR

Rosângela de Albuquerque Ribeiro

Hematologist
Centro de Hematologia e Hemoterapia do Ceará (HEMOCE)
Fortaleza, Ceara, Brazil
JA

Juliana Alvares-Teodoro

Professor of Pharmacy
Faculty of Pharmacy, Universidade Federal de Minas Gerais (UFMG)
Belo Horizonte, Minas Gerais, Brazil
Suely M. Rezende, MD, PhD

Professor of Internal Medicine
Universidade Federal de Minas Gerais
Belo Horizonte, Minas Gerais, Brazil

Background: Immune Tolerance Induction (ITI) is the standard treatment for eradicating neutralizing antibodies to exogenous factor VIII (FVIII) inhibitors, but it is costly.

Aims: To assess the costs of clotting factor concentrates (CFC) used before, during and after ITI in people with haemophilia A (PwHA) according to ITI outcomes.

Methods: This is a retrospective study with PwHA who underwent ITI in 4 Haemophilia Treatment Centers in Brazil. We evaluated medical records in three periods: i) pre-ITI (12 months before ITI), ii) during ITI (annualized period), and iii) post-ITI (12 months after ITI). Outcomes were defined according to international criteria as failure, complete and partial success. Costs were adjusted for the Brazilian Extended National Consumer Price Index (June 2022) and purchasing power parity (PPP dollar 2022).

Results: We enrolled 91 PwHA (86 [94.5%] with FVIII < 0.01 IU/mL). Complete success, partial success and failure were achieved in 29 (31.9%), 35 (38.4%) and 27 (29.7%) PwHA, respectively. Annualized Bleeding Rate (ABR) was increased in PwHA who had ITI failure in all three periods, when compared to partial/complete success: pre-ITI (p=0.020), during ITI (p=0.003) and post-ITI (p < 0.001). Before ITI, the mean cost/kg (± standard deviation [SD]) of CFC was 5,468.57 (± 6,150.06), 5,441.41 (± 4,341.18) and 9,344.79 (± 8,285.61) PPP dollars in complete success, partial success and failure, respectively (p < 0.05). During ITI, the mean cost/kg was 5,060.57 (± 3,766.81), 8,124.30 (± 7,379.13) and 25,271.66 (± 12,132.84) in complete success, partial success and failure, respectively (p < 0.001). After ITI, the mean cost/kg was 2,767.89 (± 1,242.61), 4,246.28 (± 8,616.35) and 20,835.99 (± 12,972.86) in complete success, partial success and failure, respectively (p < 0.05).

Conclusion(s): Failure group had the highest costs related to ITI, possibly due to the highest ABR, which required a high consumption of CFC, especially bypassing agents.