PB0638 - Early initiation of prophylaxis does not necessarily prevent Intracranial hemorrhage in infants with severe haemophilia A – a single center experience

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

SL

Sarina Levy-Mendelovich

Pediatric hematologist in the National hemophilia and thrombosis center, Israel
National Hemophilia Center and Thrombosis & Hemostasis Institute, Sheba Medical Center, Ramat Gan 52621, Israel
Kiryat ono, HaMerkaz, Israel

Co-Author(s)

NG

Noa Greenberg-kushnir

resident in pediatric hemato-oncology
National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Israel., Israel
RAMAT GAN, HaMerkaz, Israel
IB

Ivan Budnik

Senior researcher
Department of Internal Medicine, Division of Hematology/Oncology, University of Iowa, Iowa City, IA, USA
Iowa City, Illinois, United States

Presenting Author(s)

Assaf A. Barg, MD

Head of pediatric thrombosis clinic
Sheba Medical Center
Ramat Gan, HaMerkaz, Israel

Co-Author(s)

ea

einat avishai

Researcher
National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Israel., Israel
Tel aviv, Tel Aviv, Israel
OC

Omri Cohen

Senior hematologist
National Hemophilia Center, Sheba Medical Center
Ramat Gan, HaMerkaz, Israel
TB

Tami Brutman-Barazani

Researcher
Sheba Medical Center
Tel Hashomer, HaMerkaz, Israel
tL

tami Livnat

Senior researcher- Head of Amalia Biron institute for thrombosis research
National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Israel; Amalia Biron Research Institute of Thrombosis and Hemostasis, Tel Aviv University, Israel., Israel
Ramat Gan, HaMerkaz, Israel
GK

Gili Kenet

CONSULTANT PAEDIATRIC HAEMATOLOGIST
National Hemophilia Center Sheba Medical center, Tel Hashomer & Amalia Biron Research Institute of Thrombosis & Hemostasis, Tel Aviv University, Israel,
Tel Aviv, Tel Aviv, Israel

Background: Children with haemophilia A (HA) have a significantly higher risk of intracranial hemorrhage (ICH) than the general population. Notably, the hallmark of HA therapy is prophylaxis, aimed at spontaneous bleeding prevention, and recently emicizumab provides a viable alternative to intravenous factor replacement therapy. Non the less, whether earlier prophylaxis initiation would actually prevent ICH remains to be elucidated.

Aims: To evaluate the incidence and timing of ICH in infants and toddlers with severe HA before and after initiation of prophylaxis.

Methods: In a single center open arm prospective study we included severe HA patients under one year who started prophylaxis by either FVIII (n=3) or emicizumab (n=27) since 2019, with longitudinal follow-up. The study collected data on demographics and on ICH occurrence before and after prophylaxis initiation.

Results: A total of 30 infants were enrolled, whose median age at prophylaxis initiation was 7 months, 24 (80%) primarily choose to start emicizumab prophylaxis. Six a children started Factor replacement prophylaxis. All children were followed for a median of 24.3 months (IQR 8.1-39.4 months). The median age for prophylaxis initiation decreased from about 1 year (2019) to 3 months in 2023. Four (13.3%) children were diagnosed with ICH, all cases occurred during the first week of life and were associated with birth trauma. Following prophylaxis initiation, no further ICH were observed despite repeated incidences (n=7) of head trauma, that required further therapy with additional doses of FVIII or bypass agents and tranexamic acid.

Conclusion(s): Our study highlights a shift towards early prophylaxis in the era of non-replacement therapies. The early incidence of ICH in neonates and infants should be considered when discussing the optimal timing for initiating prophylaxis in patients with severe HA.