Abstract Number: PB0929
Meeting: ISTH 2020 Congress
Background: Hemlibra® (Roche), the bispecific humanised antibody bridging FIXa and FX, was recently approved as prophylaxis for haemophilia A (HA) patients with and without inhibitors. Data in young children are limited, and moreover even less evidence exists regarding real world use. The risk for adverse events during long-term use and concomitant use with FVIII /bypass agents in children is to be established.
Aims: To evaluate safety and efficacy on PedNet patients using Hemlibra.
Methods: The PedNet Registry is a prospective, multicentre cohort study that includes all children with haemophilia born since January 1st 2000 and diagnosed and treated in one of the 31 participating haemophilia centres in Europe, Canada and Israel. The present study focuses on patients using Hemlibra at the cut-off date of January 1st 2020.
Results: 70 children (n=67 severe HA) are currently treated with Hemlibra, of whom 63 had a FVIII inhibitor at start of treatment. 17 infants started Hemlibra before the age of 2 years (2 before 6 months). The median treatment period was 6.9 (IQR 2.1-14.6) months. 23 patients (22 with inhibitors) reported a total of 40 bleeding episodes; the remaining 47 patients (41 with inhibitors) had zero bleeds. Details of bleedings can be found in the table. Concomitant treatment with FVIII was reported in 12 children (11 with inhibitors, of whom 6 received ITI). Ten children received rFVIIa therapy. No inhibitors were diagnosed in patients treated with Hemlibra; only a local skin reaction was reported so far. No serious adverse events were reported.
Conclusions: Hemlibra was well tolerated both in inhibitor and non-inhibitor patients. No serious adverse events were reported.
To cite this abstract in AMA style:Kenet G, Oldenburg J, Santagostino E, Motwani J, Liesner R, d'Oiron R, Kurnik K, Glosli H, Male C, Ranta S, Ortiz AS, PedNet Study Group . Hemlibra Treatment in Pediatric Hemophilia A Patients – Real World Data of Safety and Uptake in the PedNet Cohorts [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/hemlibra-treatment-in-pediatric-hemophilia-a-patients-real-world-data-of-safety-and-uptake-in-the-pednet-cohorts/. Accessed September 21, 2023.
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