PB1206 - Real-World Assessment of rVIII-SingleChain for Prophylactic Treatment in Severe Hemophilia A Patients in Taiwan

Tuesday, June 25, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Presenting Author(s)

Yeu-Chin Chen, MD

Associate Professor/Doctor, Director of hemophilia care & research center
Division of Hematology/Oncology, Tri-Service General Hospital, Defense Medical Center
Taipei, Taipei, Taiwan (Republic of China)

Co-Author(s)

SC

Shu-Huey Chen

Division head
Department of Pediatrics, Taipei Medical University-Shuang Ho Hospital, Ministry of Health and Welfare
New Taipei City, New Taipei, Taiwan (Republic of China)
DW

De-Fu Weng

Director
Division of Pediatric Hematology/Oncology, Department of Pediatrics, Chung Shan Medical University Hospital
Taichung, Taichung, Taiwan (Republic of China)

Background: rVIII-SingleChain has been reimbursed for the severe HA prophylactic use since 01/09/2020 in Taiwan. However, there is limited real-world evidence across Asian populations.

Aims: To assesses real-world outcomes with rVIII-SingleChain and other long-acting rFVIII products for severe HA prophylaxis treatment in Taiwan.

Methods: We conducted retrospective medical chart review in three hemophilia treatment centers from 01/09/2020 to 31/08/2021. Factor consumption and bleeding events were collected from patient-reported injection record. Only severe people with hemophilia A (PwHA) on regular prophylactic rFVIII treatment for ≥ 12 weeks were included, age and joint severity were balanced between rVIII-SingleChain group and other long-acting products group. Pharmacokinetic parameters was estimated by WAPPS-hemo analysis.

Results: Our cohort encompassed 39 PwHA (38 male, 1 female), with 14 PwHA in rVIII-SingleChain group (mean observation period 198.1, SD 63.4 days) and 25 in the compared group (Efmorocotocog alfa or Rurioctocog alfa pegol, mean 345.3, SD 62.9 days). Demographics and other baseline characteristics are comparable between two groups. Of the rVIII-SingleChain group, median annualized bleed rates (ABRs), annualized spontaneous bleed rates (AsBRs), and annualized joint bleed rates (AjBR) were comparable with other rFVIII group (0.0 vs 2.0, 0.0 vs 1.0, 0.0 vs 0.0, respectively) without statistical differences. The median factor VIII consumption of rVIII-SingleChain group was 97.7 (IQR 92.3-103.2) IU/week/kg, whereas other rFVIII group was 81.4 (IQR 71.0-90.3) IU/week/kg. Pharmacokinetic analysis showed comparable half-life profile among rVIII-SingleChain (mean 16.9, SD 4.2 hours), Efmorocotocog alfa (mean 18.2, SD 4.7 hours), and Rurioctocog alfa pegol (mean 17.0, SD 2.6 hours).

Conclusion(s): Our real-world study demonstrates that rVIII-SingleChain prophylaxis provided robust bleeding prevention in this Asian HA population. The half-life of rVIII-SingleChain prophylaxis in this real-world setting had a similar profile with other long-acting FVIII products. These findings contribute valuable insights for the effectiveness of rVIII-SingleChain use for severe HA prophylaxis therapy.