Session: 321. Blood Coagulation and Fibrinolytic Factors: Poster III
Hematology Disease Topics & Pathways:
Bleeding Disorders, Hemophilia, Diseases, Bleeding and Clotting, Hemostasis, Pediatric, Clinically relevant
In the NuProtect study, PUPs with severe haemophilia A who were treated with simoctocog alfa for 100 exposure days (EDs) showed a 17.6% cumulative incidence of high-titre inhibitors [Liesner RJ, Neufeld EJ. Blood 2019; 134 (Suppl. 1): 903], while the hamster cell line-derived rFVIII arm of the SIPPET study showed a cumulative incidence of 28.4% (Peyvandi F, et al. New Engl J Med 2016; 374:2054-34). In the NuProtect study, children who received continuous prophylaxis had a median spontaneous annualised bleeding rate (ABR) of 0 [mean (SD): 0.54 (1.07)]. In the extension study, the dosing regimen and frequency of infusions were determined by the Investigator based on the patient’s clinical situation.
Forty-eight patients continued into the NuProtect extension study at 15 centres in 9 countries. The median (range) age at enrolment into the extension study was 2.8 (1.3–11.9) years. All 48 patients were included in the safety analysis. One patient was lost to follow-up after 1 ED. The remaining 47 patients received prophylaxis for a mean (SD) of 20.6 (6.5) months and 179 (72) EDs and were included in the efficacy analysis. By the end of the study, 85% of patients were on a twice-weekly infusion schedule. No patient developed FVIII inhibitors during the NuProtect extension study. During prophylaxis, the median (IQR) spontaneous ABR was 0 (0–0.50) [mean (SD): 0.29 (0.66), estimated (negative binomial counting regression model) mean (95% CI): 0.28 (0.15, 0.53)]. The median (IQR) total ABR was 1.0 (0–1.95) [mean (SD): 1.63 (2.25), estimated mean (95% CI): 1.62 (1.09, 2.42)]. Of the 47 patients, 34% experienced no bleeding episodes (BEs) and 72% experienced no spontaneous BEs. Treatment efficacy was rated either “excellent” or “good” in 86 of 110 (78.2%) rated BEs treated with simoctocog alfa. Three patients had 4 surgeries that were treated with simoctocog alfa; an overall efficacy assessment was performed jointly by the haematologist and surgeon for 2 of the surgeries, and both ratings were "excellent". There were no treatment-related adverse events as assessed by the Investigator.
In conclusion, no children with severe haemophilia A developed FVIII inhibitors during the NuProtect extension study. Most patients received twice-weekly prophylaxis. Simoctocog alfa was effective for both the prevention and treatment of BEs and was well-tolerated, with no new safety signals in a mean follow-up of 20.6 months.
Disclosures: Liesner: Novo Nordisk, Octapharma, Swedish Orphan Biovitrum: Speakers Bureau; Bayer, Takeda: Consultancy; Octapharma, Bayer, Takeda, Novo Nordisk, Roche: Research Funding. Neufeld: Takeda: Consultancy; Bayer: Other: DSMB; ApoPharma/Chiezi: Other: DSMB service; Pfizer: Membership on an entity's Board of Directors or advisory committees; Novo Nordsik: Consultancy; Octapharma: Consultancy; Imara Pharma: Other: DSMB service; Acceleron Pharma: Consultancy, Other: DSMB; genetech: Consultancy; BMS: Consultancy.
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