Direct oral anticoagulants in patients with severe inherited thrombophilia: Real-world data from a tertiary care center

Inherited thrombophilia (IT) predisposes individuals to venous thromboembolism (VTE) and increases the risk for first event VTE as well as for recurrent VTE. Outcomes in patients with IT treated with direct oral anticoagulants (DOACs) or vitamin K antagonists (VKAs), remain mostly underexplored. This retrospective study analyzed VTE patients with severe IT treated with DOACs at a large tertiary center. The MDClone platform was used for data extraction. Main outcomes were rates of VTE recurrence and major bleeding while on treatment with either DOAC or VKAs. A total of 160 patients with IT were included. The median age was 44.3 and 56.9 % were female. Unprovoked VTE was the most common presentation, accounting for 45.0 % of cases, followed by events provoked by estrogen exposure (21.9 %) and other minor triggers (16.9 %). DOACs were exclusively administered in 82 patients (51.2 %), whereas 78 (48.7 %) received vitamin Kantagonists (VKAs), of whom 40 were later switched to DOACs. Over a median of 5.2 years follow-up, VTE recurrence was observed in 12.5 %, and associated with higher Charlson comorbidity scores. Patients with unprovoked VTE exhibited the highest recurrence rates (20.8 %). In multivariate analysis recurrence rates were unaffected by gender, age at initial VTE event, comorbidity, thrombophilia subtype, or anticoagulant type. Incidence of major bleeding was low and was also similar across anticoagulant groups. DOACs and VKAs provide comparable outcomes in patients with IT in terms of VTE recurrence and bleeding risk. • This retrospective study analyzed VTE management and outcomes in 160 patients with severe IT treated with anticoagulants. • On treatment VTE recurrence was observed in 12.5 % of patients and 20.8 % of patients with unprovoked VTE. • DOACs showed comparable efficacy to VKAs, with similar rates of major bleeding. • Multivariate analysis did not reveal significant predictors of VTE recurrence.