Outcomes of real-world patients with non-valvular atrial fibrillation on
anticoagulants ineligible for Phase III trials of direct oral
anticoagulants
Abstract
Aims: To compare the outcomes of real-world Japanese patients with
nonvalvular atrial fibrillation who are ineligible for phase III trials
of direct oral anticoagulants with those of eligible patients. Methods:
In retrospective cohort design, consecutively registered patients with
nonvalvular atrial fibrillation who had taken warfarin were followed up
and assessed eligibility of patients for phase III trials of direct oral
anticoagulants. The effects of the ineligibility of patients on outcomes
were estimated using Cox proportional hazards models to calculate the
hazard ratio (HR) and 95% confidence interval. Results: We registered
7826 Japanese patients with nonvalvular atrial fibrillation from 71
hospitals. Nearly half (48.2%, n=3772) of these patients were
ineligible for phase III trials of direct oral anticoagulants, mainly
because of low CHADS2 scores (26.4%), renal dysfunction (9.5%), anemia
(6.4%), and chronic treatment with nonsteroidal anti-inflammatories
(4.0%). After excluding patients with a CHADS2 score <2
(n=2064, 26.4%) from total ineligible patients, the remaining
ineligible patients (n=1708) exhibited significantly greater risks of
major bleeding (unadjusted hazard ratio 2.00, 95% confidence interval
1.63–2.44, p<0.0001), stroke/systemic embolism (unadjusted
hazard ratio 1.53, 95% confidence interval 1.17–1.98, p=0.0016), and
all-cause mortality (unadjusted hazard ratio 2.84, 95% confidence
interval 2.36–3.43, p<0.0001) compared to the eligible
patients. Conclusions: The benefits and risks of direct oral
anticoagulants suggested by phase III trials may not necessarily apply
to patients ineligible for Phase III trials. This gap between evidence
and practice is an issue in the real-world safety and efficacy of
anticoagulants.