CLINICAL NEWS
Emicizumab: A Safe HAVEN from Hemophilia A Inhibitors
Emicizumab reduced the annualized bleeding rate to zero, without increasing clotting risk, in children with
hemophilia A and inhibitors, according to results from the single-arm, phase III HAVEN 2 trial. “Before this
drug, we didn’t have very effective ways to prevent joint bleeding in these patients,” said lead study author
Guy Young, MD, adding that “[emicizumab] has been life-changing for the children I’ve treated.”
Sixty children (ages 1-12 years) received weekly prophylaxis with
subcutaneous emicizumab. After a median of nine weeks of
follow-up (range = 1-41 weeks),
57 patients (95 % ) experienced
no bleeding events requiring
treatment.
An earlier trial of emicizumab involving adult and adolescent patients raised safety concerns about on-
treatment risk of thromboembolic or thrombotic microangiography, but emicizumab was well-tolerated in
HAVEN 2, with no children experiencing these types of events, according to the researchers. In addition, no
patients developed inhibitors to the drug.
Source: Young G, Sidonio RF, Leisner R, et al. HAVEN 2: efficacy, safety and pharmacokinetics of once-weekly prophylactic emicizumab
(ACE910) in pediatric patients (<12 years) with hemophilia A with inhibitors: interim analysis of single-arm, multicenter, open-label, phase 3
study. Abstract #85. Presented at the 2017 American Society of Hematology Annual Meeting, December 11, 2017; Atlanta, GA.
Cancer-Related Clot Busters?
About 20 percent of patients with cancer develop a venous thromboembolism (VTE), and while
guidelines recommend low-molecular-weight heparin to prevent VTE recurrence, newer direct oral
anticoagulants (DOAC) may offer similar efficacy in a more convenient administration.
In a pilot trial, 406 patients who had cancer and VTE were
randomly assigned to receive either dalteparin or the
DOAC rivaroxaban.
After six months of treatment, rivaroxaban
more than halved the rate of VTE recurrence
(p values not reported):
4 % with rivaroxaban
11 % with dalteparin
However, results for secondary endpoints were mixed,
with rivaroxaban-treated patients experiencing more:
major bleeding events:
%
%
9 (4 ) vs. 6 (3 )
clinically relevant non-major bleeding:
%
%
28 (13 ) vs. 5 (2 )
“[Our data] indicate that DOACs are potentially safe in cancer patients,” said lead study author
Annie Young, PhD. “We need to be looking at different groups of people and different types of
bleeds in more detail, so that we can choose the best treatment for each patient.”
Source: Young A, Marshall A, Thirlwall J, et al. Anticoagulation therapy in selected cancer patients at risk of recurrence of venous
thromboembolism: results of the Select-D pilot trial. Abstract #625. Presented at the 2017 American Society of Hematology
Annual Meeting, December 11, 2017; Atlanta, GA.
For more in-depth coverage of the 2017 ASH Annual Meeting,
turn to our “On Location” section on page 26.
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