PB0246 - Exploring the Efficacy and Economic Impact of Emicizumab Prophylaxis in children with Severe Hemophilia A and Inhibitors : A Cohort Cost-Effectiveness Study in Iran

Sunday, June 23, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Peyman Eshghi, MD

MD, Professor of Pediatric Hematology & Oncology
Pediatric Congenital Hematologic Disorders Research Center, Research Institute for Children’s Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran

Co-Author(s)

SM

Soha Mohammadimoghaddam

MD, Research assistant
Pediatric Congenital Hematologic Disorders Research Center, Research Institute for Children’s Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran
ZG

Zahra Gharib-Naseri

Pharm.D, PhD
Department of Pharmacoeconomics & Pharmaceutical Management ,School of Pharmacy, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran
MN

Majid Naderi

MD, Associate Professor of Hematology and Oncology
Genetics of Non-Communicable Diseases Research Center, School of Medicine, Zahedan University of Medical Sciences,Zahedan,Iran.
Zahedan, Sistan va Baluchestan, Iran
BK

Bijhan Keikhaei

MD, Professor of Hematology and Oncology
Thalassemia & Hemoglobinopathy Research Center, Health Research Institute, Ahvaz Jundishapur University of Medical Sciences, Ahvaz, Iran.
Ahvaz, Khuzestan, Iran
AB

Asghar Bazrafshan

MD, Subspecialist in Pediatrics Hematology and Oncology
Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran
Shiraz, Fars, Iran
BH

Behnaz Habibpanah

MsC, Nurse
Pediatric Congenital Hematologic Disorders Research Center, Research Institute for Children’s Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran
LK

Leila Khanali Mojen

Nurse PhD, Assistant Professor
Pediatric Congenital Hematologic Disorders Research Center, Research Institute for Children’s Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran
MA

Mehdi Aghapour

MD
Pediatric Congenital Hematologic Disorders Research Center, Research Institute for Children’s Health, Shahid Beheshti University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran
RH

Razieh Hantooshzadeh

MD
Management Center for special Diseases, Deputy of Health, Ministry of Health and medical Education, Tehran, Iran.
Tehran, Tehran, Iran
AS

Ahmadreza Shamshiri

PHD, Assistant Professor Of Epidemiology
Department Community Oral Health , School of Dentistry,Research Center for Caries Prevention, Dental Research Institute, Tehran University of Medical Sciences, Tehran, Iran
Tehran, Tehran, Iran

Background: The high effectiveness of Emicizumab in preventing bleeding in Hemophilia A(HA) patients is overshadowed by its high cost, especially in low or middle-income countries (LMIC).

Aims: This study aims to assess the cost and effectiveness of Emicizumab prophylaxis (EmP) in severe HA patients with FVIII inhibitors in Iran as a model of LMIC.

Methods: A multicenter, open-label cohort study-approved by Institutional Ethical Committee- was conducted on 12 severe HA children with FVIII inhibitors in 2023 in 5 Comprehensive Care Centers in Iran on the cost and effectiveness of EmP, compared with their previous On-Demand treatment with Bypassing agents (BPAs). Endpoints included all treated bleeding rates, number of target joints, Heamophilia Joint Health Score (HJHS), Heamophilia‐specific Quality of Life assessment for children and adolescents (Heamo-QOL), and pharmaceutical expenditures including Both BPAs and Emicizumab (As the main component of direct medical costs).

Results: 12 enrolled participants aged 2-14 (mean: 6.54) years received EmP for 16-20 (mean: 17.6) weeks till the time of report. They had a mean Annualized Bleeding Rate (ABR) of 25 (18-36) last year despite of On-Demand treatment with BPAs. During the period of EmP, all participants (100%) reported zero spontaneous bleeding. However, 2 patients (16.66%) experienced traumatized bleeding (one femur fracture, and one lip laceration) leading to surgery. Not only there was not any new target joints, but also HJHS score improved from 26.17 to 7.33 during 12 weeks. Additionally, Haemo-Qol score improved in age group:4-7y, 8-12y, 13-16y from 71.42%,61.71%,39.7% to 23.74%, 24.52%,14.68%respectively. The mean direct cost of treatment decreased significantly from 483.29 to 262.08 US$/kg/month/patient (saving of 2654$/month for all cases). No adverse event was reported except one mild local and transient Emicizumab injection-site reaction.

Conclusion(s): EmP effectively controlled bleeding with a significant decrease in treatment costs, and improved heamophilia-related quality of life in this study population in Iran.