Abstract Number: PB0536
Meeting: ISTH 2021 Congress
Background: Management options for bleeding episodes (BEs) in pediatric patients with hemophilia A or B with inhibitors (PwHABI) are limited. Suboptimal treatment has negative musculoskeletal consequences.
Aims: We assessed the efficacy and safety of eptacog beta (EB; LFB and HEMA Biologics), a new bypassing agent (recombinant FVIIa) in treating BEs in pediatric PwHABI.
Methods: PERSEPT 2 was a randomized, crossover, international, phase 3 study of EB in pediatric PwHABI. Patients received either an initial dose (ID) of 75 or 225µg/kg EB followed by per protocol dosing of 75µg/kg at prespecified intervals (determined by clinical response). The primary efficacy endpoint using the EMA definition was the proportion of BEs of all severity (mild, moderate and severe) with ‘good’ or ‘excellent’ response at 12h after the initial dose. Secondary/tertiary efficacy endpoints included time to response, number of administrations, pain relief at 12h, bleeding recurrence within 24h, and responses at 24h.
Results: Twenty-five patients (range 1-11 years; median 5.0 years) received EB treatment for 549 BEs (546 mild/moderate, 3 severe). Of these, 239 BEs (67%) treated with 75µg/kg ID regimen (IDR) and 310 BEs (63%) treated with 225µg/kg IDR achieved hemostatic response within 12h; median time to response was 9 and 12h, respectively. Response was achieved with a median of 3 doses with 75µg/kg IDR and 2 doses with the 225µg/kg IDRs. Pain was relieved within 12 hours in the vast majority of cases (>90%). Most BEs (>98%) had no recurrence, and the success proportion at 24h was >97% for both IDRs (Table 1). EB was well tolerated. No thromboembolic, allergic, or treatment-related adverse events were reported. No neutralizing antibodies to EB were observed.
Conclusions: Results from PERSEPT 2 provide clinically meaningful evidence that EB is an effective and safe therapeutic option for the treatment of BEs in pediatric PwHABI.
To cite this abstract in AMA style:Castaman G, Pipe SW, Callaghan M, Ducore J, Escobar M, Hermans C, Mahlangu J, Nowak-Göttl U, Journeycake J, Meeks S, Recht M, Wang M, Young G, Chambron N, AL-Sabbagh A, Bonzo D, Macie C, Mitchell IS, Shapiro A. Results from the Eptacog Beta in the Treatment of Bleeding Episodes of all Severities in Pediatric Hemophilia A or B Patients with Inhibitors [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 1). https://abstracts.isth.org/abstract/results-from-the-eptacog-beta-in-the-treatment-of-bleeding-episodes-of-all-severities-in-pediatric-hemophilia-a-or-b-patients-with-inhibitors/. Accessed September 23, 2021.
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