Emicizumab in Management of Hemophilia_A Patients with High Titer FVIII Inhibitor - Real-World Evidence from Poland

Windyga, Jerzy; Zdziarska, Joanna; Stefańska-Windyga, Ewa; Chmielewska, Ewa; Chojnowski, Krzysztof; Jamrozik, Michał; Kostyra, Aleksandra; Kozińska, Justyna; Krainiak, Halyna; Oleksiuk, Jolanta; Rupa‐Matysek, Joanna; Zarzycka, Ewa; Buczma, Anna

doi:10.1182/blood-2022-163567

Cited by 1 publication

(1 citation statement)

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“…Although excluded according to the predefined search criteria, several studies reported specifically on concomitant rFVIIa use during surgery (one original research article and five congress abstracts) 26–31 . Across these studies, there were no safety issues or evidence of thrombosis for patients receiving rFVIIa during surgical procedures.…”

Section: Resultsmentioning

confidence: 99%

Safety of recombinant activated factor VII for treatment of breakthrough bleeds in patients with congenital haemophilia A and inhibitors receiving emicizumab prophylaxis: Review of the real‐world evidence

Kenet,

Fujii

2024

Haemophilia

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BackgroundEmicizumab is used as a subcutaneous prophylaxis for prevention of bleeding episodes in patients with haemophilia A (HA) with and without inhibitors. While low bleeding rates were observed in clinical trials, patients still experience breakthrough bleeds (BTBs) with emicizumab in the real‐world. Current guidelines recommend use of recombinant activated factor VII (rFVIIa) for treatment of BTBs in patients with inhibitors. Due to thrombotic events observed in the HAVEN 1 study, activated prothrombin complex concentrate (aPCC) should be used with caution.ObjectivesThe objective of this review is to identify and discuss real‐world data on the frequency of BTBs and the safety of concomitant rFVIIa use in patients with inhibitors on emicizumab prophylaxis.MethodsA search of the following databases was conducted on 15 July 2022: BIOSIS Previews®, Current Contents Search®, Embase®, MEDLINE®. Search terms included ‘real world’, ‘haemophilia A’, and ‘emicizumab’.Results and conclusionsEleven relevant publications were identified (seven original research articles and four congress abstracts). The frequency of BTBs specifically for HA patients with inhibitors was described in three publications with 5%–56% patients on emicizumab reporting ≥1 bleeding episode. Treatment of these BTBs appeared to be managed according to relevant guidelines. Importantly, no thrombotic complications occurred during concomitant rFVIIa use. Due to the nature of real‐world studies, direct comparison of the results between studies is limited. However, real‐world data show that BTBs in inhibitor patients during emicizumab prophylaxis can be safely treated with rFVIIa.

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Section: Resultsmentioning

confidence: 99%