TQG202, a recombinant B-domain-deleted factor VIII, resulted in effective bleeding control when used on-demand among Chinese patients with moderate-to-severe hemophilia A, according to the results of a single-arm study published in the journal Haemophilia.
Although prophylactic treatment of hemophilia A is preferred, the majority of available prophylactic factor VIII in China is plasma-derived. Due to a severe plasma shortage, few patient are able to receive prophylactic therapy and recombinant factor VIII is currently limited by its expense.
A previous clinical trial demonstrated that TQG202 is effective as a prophylactic treatment. Given that on-demand treatment is currently the most common and feasible strategy in China, the aim of this study was to evaluate the efficacy and safety of TQG202 as on-demand therapy.
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This multicenter, single-arm, open-label study treated 56 patients with moderate to severe hemophilia A with TQG202 as on-demand treatment of bleeding episodes. The coprimary endpoints were infusion efficiency at 15 and 60 minutes after first administration and hemostatic efficacy of first bleeding episode.
The mean age at baseline was 28.1 years and 1% of patients had mild hemophilia A, 33.9% had moderate, and 64.3% had severe. The median number of bleeding episodes in the prior 3 months was 7 and the median number of total joint bleeds in the prior 3 months was 5. There were 76.8% of patients who had received prior on-demand therapy, 3.6% prophylactic treatment, and 19.6% received both in the prior 3 months.
TQG202 resulted in a median infusion efficiency of 155.42 at 15 minutes and 145.17 at 60 minutes. The majority of patients (69.6%) experienced excellent hemostatic efficacy of their first bleeding episode, with 14.3% having good and 1.8% fair efficacy.
For the second bleeding episode, TQG202 efficacy was rated as excellent for 69.6% of patients and good for 28.0%.
Treatment-emergent adverse events (AEs) developed among 19.6% of patients, with the most common including abnormal laboratory tests, gastrointestinal diseases, and nausea. None of the patients withdrew from the study due to treatment-emergent AEs and there were no serious AEs.
The authors concluded that TQG202 “showed effective control of the bleeding symptoms, with a low incidence of AEs and inhibitors development.”
Disclosures: This study was supported by Chia Tai Tianqing Pharmaceutical Group Co., Ltd. Please see the original reference for a full list of disclosures.
Reference
Sun Z, Xi Y, Liu W, et al. 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. Haemophilia. Published online March 13, 2023. doi: 10.1111/hae.14777
