PB1269 - Can long-term lower standard dose of Hemlibera prevent microbleeds in pediatric patients with hemophilia A: A single center experience

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Mehran karimi, MD.

prof. of pediatric hematology oncology
Pediatric Hematology-Oncology department, American Hospital Dubai
Dubai, Dubai, United Arab Emirates

Co-Author(s)

ME

Maroun El khoury

Hematology –Oncology clinic
Hematology –Oncology clinic, American Hospital Dubai, Dubai
Dubai, Dubai, United Arab Emirates
NZ

Naji Zaher

Pediatric Hematology-Oncology
Pediatric Hematology-Oncology department, American Hospital Dubai
Dubai, Dubai, United Arab Emirates
KF

Khan Faraz

Hematology –Oncology clinic
Hematology –Oncology clinic, American Hospital Dubai, Dubai
Dubai, Dubai, United Arab Emirates
YE

Yasmin Elsayed

Pediatric Hematology-Oncology
Internal Medicine Department, American Hospital Dubai, Dubai, UAE
Dubai, Dubai, United Arab Emirates

Background: Hemlibera as a bispecific antibody, mimics the action of FVIIIa by binding with both FIXa and FX.The long-term efficacy of Hemlibera to prevent microbleeds is a matter of debate.

Aims: Our aim is to evaluate long-term efficacy of Hemlibera in two pediatric patients with hemophilia A

Methods: A cross section study was conducted at American Hospital Dubai, UAE in 2023 on two patients with hemophilia A
A consent form was taken, and the study complied with the Declaration of Helsinki.

Results: Case 1: 11-year-old boy was diagnosed with severe hemophilia A with no inhibitor.
He was on prophylaxis with recombinant FVIIIa (Three times weekly with dose of 25 unit/kg/dose). He had target joint in left elbow and knee treated with extra dose of rFVIIIa. He has been on Hemlibera,150 mg (5 mg/kg/SC) every 4 weeks since 2021. All target joints resolved. He plays football every day without having any breakthrough bleeding in the last 3 years. Regular images including US joints done every 6 months and did not show any microbleeds.
Case 2: A 4-year-old boy was diagnosed with severe hemophilia A. He was on on-demand regimen with recombinant FVIII and had frequent right knee joint bleeds and developed inhibitor at the age of one year (15 BU).He was switched to Hemlibera prophylaxis with dose of 60 mg /SC every 3 weeks (3.4 mg/kg) in May 2020. The inhibitor trend was going down to 0.8 BUand he had no microbleeds as per serial US of all joints.

Conclusion(s): Long-term lower standard dose of Hemlibera prophylaxis demonstrated a promising efficacy profile in preventing microbleeds in patients with severe