Inhibitor development in previously untreated patients with severe haemophilia: A comparison of included patients and outcomes between a clinical study and a registry‐based study

Abstract

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FVIII tolerance in the Emicizumab era: Why yes?

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when estimating the risk of inhibitor development 3 and more than 70% of mutations responsible for severe forms of HA are associated with a high risk of developing it. 2 This fact is supported by other subsequent observations such as that of Jonker et al 4 which cite 3 factors especially associated with the risk of inhibitor development: a positive family history of inhibitors, a high-risk F8 genotype, and intensive treatment at first exposure.

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“…The need for intensive treatment in these first exposures to FVIII due to intercurrent bleeding that may occur while the patient is on emicizumab® prophylaxis confers a high risk of inhibitor development. 4 In fact, in the HAVEN-7 study, which evaluates the efficacy and safety of emicizumab® prophylaxis in patients with severe AH, aged ≤12 months, 2 patients developed inhibitors against FVIII in the context of bleeding that required coadministration of CFVIII for resolution. 5 The risk of inhibitor development in PUPs (previously untreated patients) with severe AH 1 is about 30%; 79% appear in the first 20 exposures (SD) to CFVIII, although the risk persists until at least 75 SD.…”