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    Treatment investigated to prevent blood-clotting disorders

    A new subcutaneous therapy could offer a promising solution to a bleeding disorder in both children and adults.


    Promising study results

    The Haven 2 study was conducted in 19 children aged younger than 12 years for about 12 weeks. The FDA granted priority review to the Biologics License Application for emicizumab based on preliminary results from the Haven 2 study and final results from the Haven 1 study, which showed that treatment with emicizumab for 31 weeks substantially reduced bleeding in patients by 87% overall—with a 79% reduction in treated bleeds on emicizumab compared with prior bypassing agent (BPA) prophylaxis therapies. According to data from Genentech, 70.8% of patients who had previously received BPA prophylaxis had zero treated bleeds on emicizumab compared with just 12.5% who had zero bleeds on their prior BPA prophylaxis. According to the full Haven 1 study results published in the New England Journal of Medicine,1 the treatment was not only effective in reducing bleeding events overall in untreated hemophiliac patients, but it was also more effective than other episodic treatments. More than 100 patients with a mean age of 28 years and mostly severe forms of the disease participated in the study. During the study, the annualized bleeding rate in patients who used emicizumab was 2.9 events compared with 23.3 events in patients with no therapy. In total, 22 of the 35 patients who received emicizumab had zero bleeding events compared with just 1 of the 18 patients who received no prophylactic therapy, according to the study.

    In a noninterventional arm of the study, researchers compared bleeding rates in patients who used emicizumab prophylaxis compared with those who used other episodic BPAs as treatments. They found that the annualized bleeding rate was 1.7 events in the group treated with emicizumab prophylactically compared with 21.6 events in the groups treated with previous episodic BPAs.1

    In terms of safety, the study revealed that there were 198 adverse events reported among 103 study participants, but mild injection site reactions were most frequently reported.1 Twelve serious events occurred in 9 patients, and those were thrombotic microangiopathy in 2 participants, and cavernous sinus thrombosis and skin necrosis—superficial thrombophlebitis—in 1 participant each. These events occurred in participants who had received multiple infusions of activated prothrombin complex concentrate at the same time as emicizumab. These events either resolved or did not require anticoagulation, the study reported.

    The FDA is not expected to issue a final decision on the priority review of emicizumab until February 2018, but Young says he is hopeful about the clinical benefits of the treatment.

    Next: New antibody eases hemophilia treatment

    “I expect that we will be able to substantially reduce the morbidity for a subset of patients with hemophilia, those with inhibitors, and offer them a much more normal life than we can offer them now,” Young says. “I also expect that for the majority of patients, those without inhibitors, that we will be able to offer them a therapy that will be as effective as what we can offer now but to do so with a far less burdensome treatment that they can easily administer at home.”


    1. Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377(9):809-818.

    Rachael Zimlich, RN
    Rachael Zimlich is a freelance writer in Cleveland, Ohio. She writes regularly for Contemporary Pediatrics, Managed Healthcare ...


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