Another oral thrombin inhibitor for stroke prevention
in atrial fibrillation?
John W. Eikelboom
1
; Jeffrey I. Weitz
1,2
1
Department of Medicine, McMaster University, Hamilton, Ontario, Canada;
2
Department of Biochemistry and Biomedical Sciences and Henderson Research Centre, Hamilton,
Ontario, Canada
Atrial fibrillation (AF), the most common
arrhythmia, is associated with a five-fold
increase in the risk of stroke (1). Fur-
thermore, cardio-embolic strokes in AF pa-
tients tend to be severe, and are often fatal
or associated with serious morbidity. Vit-
amin K antagonists, such as warfarin, pro-
duce about a 64% reduction in the risk of
stroke in AF patients (2), but have numer-
ous limitations that curtail their uptake and
diminish their effectiveness. These limi-
tations include a slow onset of action, a
variable dose requirement, reflecting com-
mon genetic polymorphisms that influence
their metabolism, and an unpredictable
anticoagulant response because of differ-
ences in dietary vitamin K intake and nu-
merous drug-drug interactions, which in-
crease or decrease the anticoagulant effect
(3). With the anticoagulant response so
variable, routine monitoring is necessary to
ensure that the international normalised
ratio (INR) is therapeutic because over-
anticoagulation is associated with an in-
creased risk of haemorrhage, whereas
under-anticoagulation increases the risk of
thrombosis. Such monitoring is inconven-
ient for patients and physicians and costly
for the healthcare system. Because of the
complexity of management, only about
one-half of eligible patients with AF receive
warfarin, and among those who receive
such treatment, the INR is frequently out-
side of the therapeutic range (4). The large
unmet need caused by the limitations of
warfarin has prompted the development of
new oral anticoagulants with potential ad-
vantages over existing agents.
Ximelagatran, the first oral thrombin
inhibitor, was effective for stroke preven-
tion in patients with AF, but was withdrawn
in 2006 because of potential hepatic toxic-
ity (5). The effectiveness of ximelagatran
prompted development of a second gener-
ation of oral thrombin inhibitors. The Ran-
domized Evaluation of Long-Term Anti-
coagulant Therapy (RE-LY) trial demon-
strated the effectiveness of dabigatran etex-
ilate, the first of this new generation, for
stroke prevention in AF, endorsing throm-
bin as an appropriate target for new anti-
coagulants. Two doses of dabigatran etex-
ilate were compared with warfarin in this
trial; the higher dose regimen (150 mg
twice-daily) was associated with signifi-
cantly fewer intracranial bleeds and strokes
compared with warfarin, whereas the lower
dose (110 mg twice-daily) was associated
with significantly less major bleeding than
warfarin and similar efficacy (6).
AZD0837 is the second of the new gen-
eration of oral thrombin inhibitors. A fol-
low-up of ximelagatran, AZD0837 is a pro-
drug of AR-H067637, a selective and re-
versible inhibitor of free and fibrin-bound
thrombin (7). Cytochrome P450
isoenzymes in the liver convert AZD0837 to
AR-H069927, an intermediate that under-
goes further metabolism to AR-H067637,
the active form. In healthy individuals,
AR-H067637 has a plasma half-life of 9–14
hours and the drug is cleared in the urine
and faeces. An immediate-release prepara-
tion was the first formulation of AZD0837
evaluated in clinical trials; a more recent
extended-release formulation enables
once-daily dosing.
In this issue of Thrombosis and Haemos-
tasis, Olsson et al. (8) report the results of a
phase II, randomised, controlled, dose-
finding study evaluating the safety and tol-
Correspondence to:
Dr. Jeffrey Weitz
Henderson Research Centre
711 Concession Street
Hamilton, Ontario
L8V 1C3, Canada
Tel.: +1 905 574 8550, Fax: +1 905 575 2646
E-mail: jweitz@thrombosis.hhscr.org
Received: December 10, 2009
Accepted: December 10, 2009
Prepublished online: January 13, 2010
doi:10.1160/TH09-12-0828
Thromb Haemost 2010; 103: 481–483
Editorial Focus
erability of the immediate-release formu-
lation of AZD0837 in 250 patients with AF
who had at least one risk factor for stroke.
Patients were allocated to a three month
course of AZD0837, at doses of 150 or 350
mg twice daily, or warfarin, dose-adjusted
to achieve an INR of 2–3. There was blind-
ing to AZD0837 dose, but the comparison
with warfarin was open-label. More than
90% of patients included in the study had
previously received warfarin and 60–71%
of patients allocated to the warfarin arm
had INR values within the target range be-
tween study day 12 and 90. Consistent with
its anticoagulant effects, AZD0837 pro-
duced a dose-dependent prolongation of
the activated partial thromboplastin time
and thrombin clotting time and suppres-
sion of the endogenous thrombin poten-
tial. Reductions in the levels of D-dimer
and prothrombin fragment 1.2 with
AZD0837 were similar to those with warfa-
rin.
Although rates of adverse events were
similar with AZD0837 and warfarin, serious
adverse events and adverse events leading to
treatment discontinuation were more com-
mon in patients randomised to the higher-
dose AZD0837 arm than they were in those
assigned to lower-dose AZD0837 or to war-
farin. Gastrointestinal complaints, such as
nausea and diarrhea, were the most frequent
adverse events leading to AZD0837 discon-
tinuation. Arrhythmic or ischaemic cardiac
events occurred in 7% of patients who re-
ceived AZD0837 at a dose of 350 mg twice-
daily compared with 1% of those given
lower-dose AZD0837 and in none who re-
ceived warfarin. Both doses of AZD0837
were associated with a 10% increase in
serum creatinine, but creatinine levels re-
turned to normal within days of stopping
treatment. This phenomenon has been at-
tributed to AZD0837-induced inhibition of
the tubular secretion of creatinine (9). There
were no strokes during the study and no dif-
ferences in the rates of major bleeding
481 © Schattauer 2010
Thrombosis and Haemostasis 103.3/2010
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