December 10, 2018

Emicizumab data confirm early promise

Updated data on the use of emicizumab in children with haemophilia A who have inhibitors were presented at the 60th American Society of Hematology Annual Meeting in San Diego.

Manufacturer Roche says its latest analysis of the HAVEN 2 trial in children under 12 years old shows that after a median of 12 weeks of weekly treatment, 77% of children experienced no bleeds requiring treatment. Treated bleeds were reduced by 99% compared with historical treatment with bypassing agents, so that 23% of children experienced between one and three treated bleeds during the study period.

The study also evaluated the efficacy of less frequent dosing: 90% of children treated every two weeks and 60% of those treated every four weeks experienced no bleeds requiring treatment.

A review of evidence for the safety of emicizumab describes three cases of thrombotic microangiopathy and two of thrombotic events associated with combined treatment with emicizumab and activated prothrombin complex concentration, either alone or together with activated recombinant factor FVII (Expert Opin Drug Saf 2018 Nov 21. doi: 10.1080/14740338.2019.1551356). It notes concern about the use of emicizumab in combination with aPCC and about the management of patients with breakthrough bleeding.