Preventing Bleeds in Pediatric Patients With Hemophilia A: Which Factor Replacement Therapy Offers the Best Protection and at What Cost?
Author(s)
Izabella Lunk, Msc, Daphne Arzoumanidou, Msc, Sharon Eastwood, Dr.
Bayer Consumer Care AG, Basel, Switzerland.
Bayer Consumer Care AG, Basel, Switzerland.
OBJECTIVES: Bleed prevention is crucial in the treatment of pediatric hemophilia A population, to protect developing joints of children from irreversible damage and chronic pain. Despite regular prophylactic treatment, breakthrough bleeds still happen, needing additional factor VIII (FVIII) replacement. There are several FVIII treatment options available, including few recently approved agents, damoctocog alfa pegol (Jivi) and efanesoctocog alfa (Altuviiio/Altuvoct). The aim is to estimate the number of bleeds during 5 years of prophylaxis treatment, between age 7 and 11, with the currently available treatment options for hemophilia A; and present its economic impact.
METHODS: Annualized bleeding rates (ABR) and factor consumption were derived from pivotal clinical trials of all FVIII treatments. Recent long-term extension studies were used for the two treatments receiving regulatory approvals most recently. Number of total bleeds, total factor consumption and costs were calculated per patient, for the five years of treatment between ages 7 and 11.
RESULTS: The total number of bleeds over 5 years was lowest for damoctocog alfa pegol (Jivi), with 3.70. Second lowest number of bleeds was achieved with efanesoctocog alfa (Altuviiio/Altuvoct) with 3.90 bleeds, following Efmoroctocog alfa (Elocta), turoctocog alfa pegol (Esperoct), turoctocog alfa (NovoEight), simoctocog alfa (Nuwiq), octocog alfa (Kovaltry), Afstyla (lonoctocog alfa) and octocog alfa (Advate) with 9.80, 9.85, 9.85, 11.85, 16.85, 18.45 and 18.75 bleeds, respectively. Cost of treating bleeds was estimated to be ranging from €4,479 (damoctocog alfa pegol, Jivi) to €23,243 (octocog alfa, Advate) in Germany and $6,400 (Jivi) to $29,953 (turoctocog alfa pegol, Esperoct) in USA.
CONCLUSIONS: Prevention of bleeds is of utmost importance when treating pediatric patients with hemophilia A. Choosing a treatment with the lowest possible bleeding rates can support the physical development of the patient. This analysis showed that the lowest number of bleeds and lowest costs are estimated to be reached by damoctocog alfa pegol (Jivi).
METHODS: Annualized bleeding rates (ABR) and factor consumption were derived from pivotal clinical trials of all FVIII treatments. Recent long-term extension studies were used for the two treatments receiving regulatory approvals most recently. Number of total bleeds, total factor consumption and costs were calculated per patient, for the five years of treatment between ages 7 and 11.
RESULTS: The total number of bleeds over 5 years was lowest for damoctocog alfa pegol (Jivi), with 3.70. Second lowest number of bleeds was achieved with efanesoctocog alfa (Altuviiio/Altuvoct) with 3.90 bleeds, following Efmoroctocog alfa (Elocta), turoctocog alfa pegol (Esperoct), turoctocog alfa (NovoEight), simoctocog alfa (Nuwiq), octocog alfa (Kovaltry), Afstyla (lonoctocog alfa) and octocog alfa (Advate) with 9.80, 9.85, 9.85, 11.85, 16.85, 18.45 and 18.75 bleeds, respectively. Cost of treating bleeds was estimated to be ranging from €4,479 (damoctocog alfa pegol, Jivi) to €23,243 (octocog alfa, Advate) in Germany and $6,400 (Jivi) to $29,953 (turoctocog alfa pegol, Esperoct) in USA.
CONCLUSIONS: Prevention of bleeds is of utmost importance when treating pediatric patients with hemophilia A. Choosing a treatment with the lowest possible bleeding rates can support the physical development of the patient. This analysis showed that the lowest number of bleeds and lowest costs are estimated to be reached by damoctocog alfa pegol (Jivi).
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
CO187
Topic
Clinical Outcomes, Economic Evaluation
Disease
Pediatrics, Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)