The use of direct oral anticoagulants in inherited thrombophilia

To review the use of the direct oral anticoagulant (DOAC) agents in inherited thrombophilia based on the literature. MEDLINE, International Pharmaceutical Abstracts, and Google Scholar searches (1970-May 2016) were conducted for case reports, case series, retrospective cohorts, or clinical trials using the key words: protein C deficiency, protein S deficiency, antithrombin deficiency, activated protein C resistance, Factor V Leiden, hypercoagulable, NOACs, dabigatran, apixaban, rivaroxaban, betrixaban, edoxaban, Xa inhibitor, direct thrombin inhibitor. Results were limited to English-only articles. Clinical studies evaluating the use of DOACs for hypercoagulable states related to inherited thrombophilia were selected and evaluated. Thrombophilia, a predisposition to thrombosis, manifests predominantly as venous thromboembolism. Causes of inherited thrombophilia include antithrombin deficiency, deficiencies of proteins C and S, and Factor V Leiden mutation. Many patients with thrombophilia receive anticoagulant therapy for primary or secondary prevention of VTE, historically either warfarin or a heparin product. DOAC's have been considered as potential alternatives to traditional agents based on their pharmacologic activity. Case reports and a post-hoc analysis of a clinical trial have indicated positive results in patients with inherited thrombophilia and VTE. Positive results have been reported for the use of DOACs in inherited thrombophilia. Further robust studies are needed for definitive decision making by clinicians.