ASH Clinical News ACN_4.14_Full Issue_web | Page 74

Literature Scan
(RR=0.32; 95% CI 0.20-0.51; p<0.001),”
the authors reported.
At enrollment, target joints (ma-
jor joints in which ≥3 bleeding events
occurred over a 24-week period) were
reported by 76 of 89 participants (85%)
who had been receiving episodic therapy
with FVIII and by 26 of 63 participants
(41%) who had been receiving pro-
phylactic therapy with FVIII. During
emicizumab treatment, these numbers
appeared to decrease, with three of
71 participants in groups A and B
reporting target joints.
“Emicizumab had a favorable
safety profile with no unexpected
safety signals,” the researchers added.
Among the 127 participants who
received emicizumab, a total of 543
adverse events (AEs) were reported,
the most common of which was low-
grade injection-site reaction (n=38;
25%). There were no patient deaths
during the study, and no thromboem-
bolism, thrombotic microangiopathy,
or inhibitor development events
occurred.
“Emicizumab
had a
favorable
safety profile
with no
unexpected
safety signals.”
—JOHNNY MAHLANGU,
MBBCh, MMed
There were 14 serious AEs, which
included: bleeding events (n=4), infec-
tion (n=3), musculoskeletal disorder
(n=3), cardiac disorder (n=1), loosen-
ing of an orthopedic device (n=1),
psychiatric disorder (n=1), and trauma
(n=1). However, none of these were
considered related to emicizumab
treatment.
As a secondary endpoint of the
analysis, participants completed the
100-point Haemophilia Quality of
Life Questionnaire for Adults (Haem-
A-QoL) every 12 weeks to measure
how emicizumab prophylaxis affected
their quality of life. Compared with
patients in group C, those in groups
A and B had higher Haem-A-QoL
scores (representing greater impair-
ment), although these comparisons
were not statistically significant.
72
ASH Clinical News
However, of the 95 eligible participants
who completed a treatment preference
survey, 89 (94%) preferred emicizumab,
the investigators noted.
A limitation of the analysis was the
relatively short follow-up period, which
precludes the ability to determine the
longer-term impact of emicizumab
prophylaxis in this patient population.
Dr. Mahlangu also noted that data from
“real-life experience with emicizumab is
important in validating these results.”
The authors report financial re-
lationships with Roche and Chugai
Pharmaceutical Co., which supported the
study.
REFERENCE
Mahlangu J, Oldenburg J, Paz-Priel I, et al. Emicizumab prophylaxis
in patients who have hemophilia A without inhibitors. N Engl J Med.
2018;379:811-22.
may be all you need to treat
her von Willebrand disease.
VONVENDI ® [von Willebrand factor (Recombinant)] is indicated for on-demand
treatment and control of bleeding episodes and for perioperative management of
bleeding in adult patients, with an option to be dosed independently of recombinant
factor VIII, according to patient need as determined by monitoring levels. 1
VONVENDIPRO.com
Comprehensive dosing information can be found within the VONVENDI full
Prescribing Information at VONVENDIPRO.com.
VONVENDI [von Willebrand factor (recombinant)]
Important Information
Indications
VONVENDI [von Willebrand factor (recombinant)] is a recombinant
von Willebrand factor (rVWF) indicated for use in adults (age 18 and older)
diagnosed with von Willebrand disease (VWD) for:
• On-demand treatment and control of bleeding episodes
• Perioperative management of bleeding
Detailed Important Risk Information
CONTRAINDICATIONS
Do not use VONVENDI in patients who have had life-threatening
hypersensitivity reactions to VONVENDI or its components (tri-sodium
citrate-dihydrate, glycine, mannitol, trehalose-dihydrate, polysorbate 80,
and hamster or mouse proteins).
WARNINGS AND PRECAUTIONS
Embolism and Thrombosis
Thromboembolic reactions, including disseminated intravascular coagulation,
venous thrombosis, pulmonary embolism, myocardial infarction, and stroke,
can occur, particularly in patients with known risk factors for thrombosis,
including low ADAMTS13 levels. Monitor for early signs and symptoms of
thrombosis such as pain, swelling, discoloration, dyspnea, cough, hemoptysis,
and syncope, and institute prophylaxis measures against thromboembolism
based on current recommendations.
In patients requiring frequent doses of VONVENDI in combination with
recombinant factor VIII, monitor plasma levels for FVIII:C activity because
sustained excessive factor VIII plasma levels can increase the risk of
thromboembolic events.
One out of 80 VWD subjects treated with VONVENDI in clinical trials
developed proximal deep vein thrombosis in perioperative period after
undergoing total hip replacement surgery.
©2018 Shire US Inc., 300 Shire Way, Lexington, MA 02421. All rights reserved. 1-800-828-2088.
SHIRE and the Shire Logo are registered trademarks of Shire Pharmaceutical Holdings Ireland
Limited or its affiliates. VONVENDI is a registered trademark of Baxalta Incorporated,
a wholly owned, indirect subsidiary of Shire plc.
Patented: see www.shire.com/legal-notice/product-patents
S41323 08/18
Hypersensitivity Reactions
Hypersensitivity reactions, including anaphylaxis, may occur. Symptoms
can include anaphylactic shock, generalized urticaria, angioedema, chest
tightness, hypotension, shock, lethargy, nausea, vomiting, paresthesia,
pruritus, restlessness, wheezing and/or acute respiratory distress.
Discontinue VONVENDI if hypersensitivity symptoms occur and administer
appropriate emergency treatment.
Neutralizing Antibodies (Inhibitors)
Inhibitors to von Willebrand factor and/or factor VIII can occur. If the
expected plasma levels of VWF activity (VWF:RCo) are not attained, perform
an appropriate assay to determine if anti-VWF or anti-factor VIII inhibitors
are present. Consider other therapeutic options and direct the patient to a
physician with experience in the care of either VWD or hemophilia A.
In patients with high levels of inhibitors to VWF or factor VIII, VONVENDI
therapy may not be effective and infusion of this protein may lead to
severe hypersensitivity reactions. Since inhibitor antibodies can occur
concomitantly with anaphylactic reactions, evaluate patients experiencing
an anaphylactic reaction for the presence of inhibitors.
ADVERSE REACTIONS
In clinical trials, the most common adverse reactions observed in ≥2% of
subjects (n=80) were generalized pruritus, vomiting, nausea, dizziness
and vertigo.
One subject treated with VONVENDI in perioperative setting developed
deep vein thrombosis after undergoing total hip replacement surgery.
Please see VONVENDI Brief Summary on the following page.
Reference: 1. VONVENDI [von Willebrand factor (recombinant)] Prescribing Information.