Direct oral anticoagulants in patients with severe inherited thrombophilia: a single-center cohort study

We investigated the safety and efficacy of direct oral anticoagulants (DOACs) in patients with venous thromboembolism (VTE) associated with severe inherited thrombophilia. In this single-center cohort study, we enrolled 56 consecutive VTE patients with severe inherited thrombophilia, defined as the presence of antithrombin (n = 18), protein C (n = 12) and protein S (n = 12) deficiencies, homozygous Factor V Leiden (n = 3) and prothrombin G20210AA (n = 4) mutations, or combined defects (n = 7). During a median follow-up of 44.5 (IQR 30-52.5) months, rivaroxaban was used in 30 (53.6%), apixabanin 14 (25%), and dabigatran in 12 (21.4%) subjects. Recurrent nonfatal VTE was observed in 5 (8.9%) patients (2.4 per 100 patient-years), treated with rivaroxaban (n = 4) and apixaban (n = 1). Major bleeding and clinically relevant non-major bleeding (CRNMB) occurred in 2 (3.5%) and 4 (7%) subjects, respectively (0.96 per 100 patient-years and 1.92 per 100 patient-years, respectively), including 4 patients on rivaroxaban. The event-free survival analysis showed that the use of rivaroxaban was associated with increased risk of recurrent VTE or bleeding, compared with apixaban or dabigatran (HR 2.76, 95% CI 1.26-3.92,p = 0.039). In conclusion, the results of our cohort study indicate that full-dose dabigatran or apixaban are effective and safe in patients with severe inherited thrombophilia.

Automated summary

The study showed that full-dose dabigatran or apixaban are effective and safe treatment options for patients with severe inherited thrombophilia and venous thromboembolism. Rivaroxaban was associated with an increased risk of recurrent VTE or bleeding compared to apixaban or dabigatran.