PB1237 - Functional Independence in People with Hemophilia A on Emicizumab Prophylaxis

Tuesday, June 25, 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)

LH

Luila Clicia Moura Henriques

Student
Faculty of Pharmacy, Universidade Federal de Minas Gerais (UFMG)
Belo Horizonte, Minas Gerais, Brazil
MS

Melissa Palis Santana

Hematologist
Centro de Hematologia e Hemoterapia do Piauí (HEMOPI)
Teresina, Piaui, Brazil
SF

Sandra Sibele Figueiredo

Hematologist
Centro de Hematologia e Hemoterapia da Paraíba (HEMOÍBA)
João Pessoa, Paraiba, Brazil
EC

Edilma Silva Casaretto

Hematology Nurse
Centro de Hematologia e Hemoterapia da Paraíba (HEMOÍBA)
João Pessoa, Paraiba, Brazil
AV

Ana Maria Vanderlei

Hematologist
Fundação de Hematologia e Hemoterapia de Pernambuco (HEMOPE)
Recife, Pernambuco, Brazil
NC

Neuza Cavalcanti Moraes Costa

Hematology Nurse
Fundação de Hematologia e Hemoterapia de Pernambuco (HEMOPE)
Recife, Pernambuco, Brazil
IC

Iris Maciel Costa

Hematology Nurse
Fundação de Hematologia e Hemoterapia de Pernambuco (HEMOPE)
Recife, Pernambuco, Brazil
TG

Tania Maria Rocha Guimaraes

Hematology Nurse
Fundação de Hematologia e Hemoterapia de Pernambuco (HEMOPE)
Recife, Pernambuco, Brazil
AG

Andrea Aparecida Garcia

Hematologist
Hemocentro de São José do Rio Preto
São José do Rio Preto, Sao Paulo, Brazil
BC

Barbara Cabrera

Hematology Nurse
Hemocentro de São José do Rio Preto
São José do Rio Preto, Sao Paulo, Brazil
JA

Juliana Alvares-Teodoro

Professor of Pharmacy
Faculty of Pharmacy, Universidade Federal de Minas Gerais (UFMG)
Belo Horizonte, Minas Gerais, Brazil

Background: Myosteoarticular bleeds lead to musculoskeletal dysfunction in people with hemophilia A (PwHA). Emicizumab prophylaxis reduces the frequency of these bleeds, but little is known about its impact on functional independence.

Aims: We aimed to evaluate functional independence in PwHA on emicizumab prophylaxis.

Methods: PwHA on emicizumab prophylaxis for at least 1 year were invited at several Brazilian Hemophilia Treatment Centers. Clinical and treatment data before (12 months) and during (12 months) emicizumab prophylaxis were collected. Bleeds were classified according to their cause as spontaneous, post-traumatic, and unknown, and only counted if they received hemostatic treatment. Functional Independence Score in Haemophilia (FISH) was applied. Higher scores correspond to higher functional independence.

Results: Of the total 10 PwHA included, 4/10 (40%) had severe hemophilia A and 7/9 (78%) were on prophylaxis. Immune tolerance induction was performed in 7/10 (70%) PcHA and all failed, then receiving bypassing agents. The median age at emicizumab prophylaxis start was 20 years (range 14-27). The total number of treated bleeding events reduced from 35 (22 spontaneous), in inhibitor-negative PwHA on factor VIII prophylaxis, from 12 (8 spontaneous and 4 post-traumatic), in inhibitor-positive PwHA and prophylaxis with bypassing agents, and from 10 (2 spontaneous and 8 post-traumatic), in inhibitor-positive PwHA on exclusive episodic treatment with bypass agents, to 0 in all groups. The medians (ranges) of the total FISH score were 29 (21-32) before and 30 (19-32) in the first year of emicizumab prophylaxis. There was an improvement in the total FISH score in 3/10 (30%) PcHA and a worsening in 3/10 (30%) PcHA, all of them inhibitor-positive.

Conclusion(s): In this small PwHA population, functional independence did not improve with emicizumab prophylaxis.