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In the largest study of pediatric hemophilia A with inhibitors to date, emicizumab
prophylaxis prevented or reduced bleeds substantially and was well tolerated, updated
analysis of the multicenter, open-label, phase III HAVEN 2 trial reports.
G
uy Young, MD, of the Children's Hospital of
Los Angeles, and University of Southern
California Keck School of Medicine, explained
that emicizumab is a bispecific humanized mon-
oclonal antibody administered subcutaneously.
Emicizumab bridges factors IXa and X to restore
the function of missing factor VIIIa.
Emicizumab is being developed to prevent bleeds
in patients with hemophilia A with and without
inhibitors.
In 2017, an interim analysis of the HAVEN 2 study
(n=20) in patients age 2–12 years (data cut-off
in October of 2016) showed that subcutaneous,
once-weekly emicizumab prophylaxis prevented
or reduced bleeds successfully, provided clinically
meaningful reductions in the annualized bleed rate
vs prior bypassing agent treatment, and was well
tolerated.
At ASH, Dr. Young presented an updated, much
larger (40 additional patients, 60 total) analy-
sis of efficacy, safety, and pharmacokinetics of
once-weekly subcutaneous emicizumab prophy-
laxis in pediatric hemophilia A with inhibitors.
“I chose to participate in this study,” Dr. Young
told Elsevier’s
PracticeUpdate
, “because I felt
this medication could offer a great benefit for my
inhibitor patients who were continuing to suffer
with repeated bleeding events and the associated
pain and morbidity. In addition, our center makes
every effort to bring innovative therapies to our
patients as soon as possible.”
The study enrolled children with hemophilia A with
inhibitors age 2–12 years (or 12–17 years of age
in those weighing <40 kg), and is enrolling those
<2 years of age treated previously with bypassing
agents to emicizumab prophylaxis for ≥52 weeks.
Efficacy analyses included annual bleed rate and
bleed reduction vs annual bleed rate on prior
bypassing agent treatment from a prospective
non-interventional study. Health-related quality
of life, aspects of caregiver burden, and safety
parameters were also assessed.
Weekly Subcutaneous
Emicizumab a New
Standard of Care in
Hemophilia Management
PRACTICEUPDATE CONFERENCE SERIES • ASH 2017
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