Efficacy and safety of the B-domain-deleted TQG202 for on-demand treatment in moderate and severe haemophilia A patients: A multicentre, single-arm trial.

INTRODUCTION: On-demand treatment is the most common treatment strategy for haemophilia A in China.

AIM: This study aims to evaluate the efficacy and safety of a human-derived B-domain-deleted recombinant factor VIII (TQG202) in the on-demand treatment of bleeding episodes in moderate/severe haemophilia A patients.

METHODS: This multicentre, single-arm clinical trial enrolled moderate/severe haemophilia patients previously treated with FVIII concentrates for ≥50 exposure days (EDs) from May 2017 to October 2019. TQG202 was injected intravenously on-demand for bleeding episode management. The primary endpoints were the infusion efficiency at 15 and 60 min after the first administration and haemostatic efficacy of the first bleeding episode. Safety was also monitored.

RESULTS: Fifty-six participants were enrolled with a median age of 24.5 (range: 12-64) years old. The median total dose of TQG202 was 29,250 IU per participant (range: 1750-2,02,500), and the median number of administrations was 24.5 (2-116). The median infusion efficiency at 15 and 60 min after the first administration was 155.4% and 145.2%, respectively. Among the 48 first bleeding episodes evaluated, 47 (83.9%, 95% CI: 71.7%-92.4%) had a haemostatic efficacy rating of excellent or good. Eleven (19.6%) participants had treatment-related adverse events (TRAEs), but no ≥grade 3 TRAE was observed. Inhibitor development (0.6BU) was observed in one participant (1.8%) after 22 EDs, but was undetectable after 43 EDs.

CONCLUSION: TQG202 for on-demand treatment in moderate/severe haemophilia A shows effective control of the bleeding symptoms, with a low incidence of adverse events and inhibitors development.