Abstract Number: PB0886
Meeting: ISTH 2020 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: N8-GP is a glycoPEGylated extended half-life recombinant factor VIII developed for prophylaxis/treatment of HA. In the main phase (MP) of pathfinder 2, a pivotal trial that evaluated N8-GP in adults/adolescents with severe HA, HRQoL was improved/maintained from baseline (BL) and a high level of TS reported.
Aims: To examine HRQoL and TS in adults (≥17 years) receiving N8-GP prophylaxis in pathfinder 2 from BL to end of trial (EOT).
Methods: pathfinder 2 was a phase 3, multinational, open-label, partially randomised trial. Prophylaxis cohorts: N8-GP (50 IU/kg) every 4 days (Q4D) or biweekly (MP); N8-GP (50 IU/kg) Q4D or N8-GP (75 IU/kg) every 7 days (randomised extension phase). Informed consent was obtained; the study was approved by relevant ethics committees.
Patient-reported outcome questionnaires administered throughout the trial included: Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL, scored 0-100; lower=better HRQoL), EuroQol 5 Dimensions-3 Levels (EQ-5D-3L, scored 0-1; visual analogue scale [VAS] scored 0-100; higher=better HRQoL); Hemophilia Satisfaction questionnaire (Hemo-SAT, scored 0-100; lower=higher TS).
Results: Adults (n=154; median age: 30 years; range: 17-66) had a median treatment duration of 54.7 months (range: 2.6-77.7). Mean (SD) Haem-A-QoL total score was improved/maintained from BL-EOT (-2.06 [11.53]) and from MP-EOT (+0.05 [9.43]) (Figure 1). Overall HRQoL was improved/maintained (EQ-5D-3L mean [SD] change from BL-EOT: index +0.01 [0.16], VAS +3.36 [12.04]; from MP-EOT: index -0.01 [0.19], VAS +0.18 [12.08]). Improvement in TS was maintained to EOT (Hemo-SAT general satisfaction mean [SD] change: -2.59 [13.28] from BL-EOT; +0.81 [12.00] from MP-EOT). Haem-A-QoL total score response rate (threshold: -7 points) increased from 20.8% (MP) to 28.8% (EOT) (Figure 2).
Conclusions: Prophylactic N8-GP for HA had a positive impact on maintaining and improving HRQoL for 4.5 years or longer in the pathfinder 2 trial.
[Figure 1. Change in HRQoL from BL to EOT and from MP until EOT, according to Haem-A-QoL scores.]
[Figure 2. Percentage of responders for Haem-A-QoL total score improved over time*]
To cite this abstract in AMA style:
Giangrande P, Lee XY, Curry N, Benchikh El Fegoun S. Prophylaxis with Turoctocog Alfa Pegol (N8-GP) Maintains Improvements in Health-Related Quality of Life (HRQoL) and Treatment Satisfaction (TS) over ≥4.5 Years in Patients with Haemophilia A (HA) [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/prophylaxis-with-turoctocog-alfa-pegol-n8-gp-maintains-improvements-in-health-related-quality-of-life-hrqol-and-treatment-satisfaction-ts-over-%e2%89%a54-5-years-in-patients-with-haemophilia-a/. Accessed September 22, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/prophylaxis-with-turoctocog-alfa-pegol-n8-gp-maintains-improvements-in-health-related-quality-of-life-hrqol-and-treatment-satisfaction-ts-over-%e2%89%a54-5-years-in-patients-with-haemophilia-a/