Abstract Number: VPB0697
Meeting: ISTH 2022 Congress
Theme: Hemophilia and Rare Bleeding Disorders » Hemophilia - Clinical
Background: SCT800 is the first domestic recombinant factor VIII (rFVIII) approved in China for treatment of hemophilia A in adult patients, and is currently under investigation for pediatric indications.
Aims: The phase III study was designed to investigate the efficacy, safety and pharmacokinetics (PK) of SCT800 for prophylaxis and treatment of bleeding episodes in severe pediatric hemophilia A patients who have been previously treated with factor VIII (FVIII)
Methods: Study patients include boys < 12yr with severe hemophilia A (FVIII:C < 1%) and no history of FVIII inhibitors. For patients 0-5yr, the previous FVIII treatment should be >50 exposure days (ED); for those aged 6-11yr, ED should be >150. Eligible patients received SCT800 25-50 IU/kg every other day or three times a week for approximately 24 weeks. PK was evaluated after single injections of 50 IU/kg. The primary efficacy endpoint was annualized bleeding rate (ABR).
Results: Sixty-eight (68) patients were enrolled (34 each for age group of 0-5yr and 6-11) and the mean exposure to SCT800 was 78.9 days. The median ABRs of the two groups were similar, 2.21 vs 2.17(0-5yr vs 6-11yr. The median ABRs for spontaneous bleeds were 0.00 for both groups. Hemostatic efficacy was rated as “excellent” or “good” for 92.1% of bleeds. 83% of 127 reported bleeds were treated with ≤2 injections. Detailed ABR data for each group were included in the table below. Drug-related adverse events (AEs) occurred in 2.9% of the patients. Only one 2-year-old patient developed inhibitors and was successfully treated with high dose SCT800 using an immune tolerance induction (ITI) scheme. The higher clearance and shorter t1/2 were observed in patients 0-5yr compared to those of 6-11yr.
Conclusion(s): The data indicate that SCT800 is effective and safe for the prophylaxis and treatment of bleeds in previously FVIII-treated severe pediatric hemophilia A patients.
Table. Annualized Bleeding Rates
To cite this abstract in AMA style:
Wu R, Wang X, Zhao X, Chen Y, Zhou Z, Sun J, Xu M, Li W, Xiao J, Yang F, Chen X, Xu W, Huang J, Ma C, Gai W, Xie L, Yang R. Efficacy, Safety and Pharmacokinetics of Recombinant Human Coagulation Factor VIII (SCT800) in Previously Treated Children with Severe Hemophilia A. [abstract]. https://abstracts.isth.org/abstract/efficacy-safety-and-pharmacokinetics-of-recombinant-human-coagulation-factor-viii-sct800-in-previously-treated-children-with-severe-hemophilia-a/. Accessed March 22, 2024.« Back to ISTH 2022 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-safety-and-pharmacokinetics-of-recombinant-human-coagulation-factor-viii-sct800-in-previously-treated-children-with-severe-hemophilia-a/