PB0492 - Fourth interim analysis of the HEM-POWR Study: A post hoc analysis of real-world effectiveness and safety of damoctocog alfa pegol in previously treated patients with haemophilia A in Taiwan

Monday, June 24, 2024

13:45 – 14:45 ICT

Room: Exhibition Hall

Co-Author(s)

JW

Jiaan-Der Wang, PhD

Director
Center for Rare Disease and Hemophilia, Department of Pediatrics, Taichung Veterans General Hospital
Taichung, Taichung, Taiwan (Republic of China)
CP

Ching-Tien Peng

Advisor
Division of Pediatric Hematology and Oncology, China Medical University Children's Hospital
Taichung City, Taichung, 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)
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)
CL

Chia-Jen Liu

Attending Physician
Division of Hematology, Department of Medicine, Taipei Veterans General Hospital
Taipei, Taipei, Taiwan (Republic of China)

Presenting Author(s)

Shyh-Shin Chiou, MD, PhD

President/Professor/Director/Doctor
Division of Pediatric Hematology and Oncology, Kaohsiung Medical University Hospital
Kaohsiung, Kaohsiung, Taiwan (Republic of China)

Background: The HEM-POWR study (NCT03932201) assesses real-world effectiveness and safety of damoctocog alfa pegol an extended half-life factor VIII (FVIII) product for treatment of previously treated patients (PTPs) aged ≥12 years with haemophilia A (HA).

Aims: To present the fourth interim analysis of a subgroup of HEM-POWR patients from Taiwan.

Methods: HEM-POWR is a Phase 4, prospective cohort study of PTPs with HA receiving on-demand or prophylactic damoctocog alfa pegol. The primary endpoint was annualised bleeding rate (ABR); safety was a secondary endpoint. The full analysis set (FAS) included PTPs fulfilling all inclusion criteria with a documented dose of damoctocog alfa pegol in the study and during the observation period. The safety analysis set (SAF) included PTPs with ≥1 dose in the observation period. Data were collected from patient diaries and physician records.

Results: At data cut-off (1 August, 2023), 18 PTPs were included in the SAF and 14 in the FAS. In the FAS, the median (Q1, Q3) observation period was 645.0 (170.0, 848.0) days (Table 1). Every 2 days (4/14, 28.6%) and twice weekly (4/14, 28.6%) were the most common FVIII treatment regimens before damoctocog alfa pegol initiation (missing: 6/14, 42.9%). The most common prophylaxis regimens of damoctocog alfa pegol at baseline were every 5 days (5/12, 41.7%), twice weekly (4/12, 33.3%) and every 7 days (3/12, 25.0%). Patients were aged ≥18 to < 60 years (12/14; 85.7%) and had severe disease (13/14; 92.9%). Total mean (SD) ABR during the observation period compared with prior to damoctocog alfa pegol initiation was –8.3 (8.0) (Table 2). In this subgroup there were no study drug-related treatment-emergent adverse events, deaths or inhibitors.

Conclusion(s): Results from this subgroup of patients from Taiwan study sites demonstrate the real-world effectiveness and safety of damoctocog alfa pegol in PTPs with HA.

Funded by Bayer.