Abstract Number: PB2247
Meeting: ISTH 2020 Congress
Theme: Venous Thromboembolism and Cardioembolism » Thrombophilia
Background: Severe hereditary thrombophilia increases the risk to develop venous thromboembolism (VTE) even recurrent.
Aims: To evaluate efficacy and safety profile of direct oral anticoagulants (DOACs) in patients with VTE and severe thrombophilia.
Methods: Consecutive adult patients with a first acute VTE between 2014-2019 and confirmed severe thrombophilia treated with DOACs for at least 3 months were enrolled. Severe thrombophilia patients treated for at least 3 months with heparin or vitamin K antagonists [VKA]) between 2007-2019 acted as controls. Endpoints were incidence rate (IR) of VTE recurrence, bleeding complications, post-thrombotic syndrome (PTS) in the two cohorts.
Results: We enrolled 72 cases (mean age 48 ± 16 years, F 51%, protein [P]C 13, PS 23, antithrombin 10, homozygous factor V Leiden (FVL) or prothrombin mutation 11, compound heterozygous 13, FVL pseudohomozygous 1, antithrombin resistence 1) and 82 controls (mean age 44 ± 18 years, F 53%, PC 24, PS 26, antithrombin 13, homozygous FVL or prothrombin mutation 6, compound heterozygous 11, FVL pseudohomozygous 2). 21 patients initially treated with VKA and then with DOACs (PC 4, PS 5, antithrombin 3, homozygous FVL 2, compound heterozygous 3, FVL pseudohomozygous 3, antithrombin resistance 1) were also considered. Among cases, 3% were treated with dabigatran, 70% with rivaroxaban, 19% with apixaban and 8% with edoxaban. We documented 3 (3.2%) VTE recurrences in cases and 4 (3.8%) in controls, with IR of 2.23 per 100 p-y and 2.16 per 100 p-y, respectively, and RR 1.03 [95%CI 0.19-4.98]. We found 7 (7.5%) minor or clinically relevant bleeding complications in cases and 3 (2.9%) in controls, IR 5.2 per 100 p-y and 1.6 per 100 p-y, respectively, RR 3.21 [95%CI 0.84-15.2]. Similar incidence of post-thrombotic syndrome was observed between groups.
Conclusions: DOACs showed an efficacy and safety profile comparable with heparin/VKA in severe thrombophilic patients. A slight increase in menorrhagia was observed.
| Thrombophilia and DOACs | Thrombophilia and heparin/AVK | |
| VTE recurrence | Deep vein thrombosis in homozygous FVL during rivaroxaban | Deep vein thrombosis in compound heterozygous during warfarin |
| Superficial vein thrombosis in protein C deficiency during rivaroxaban | Splanchnic vein thrombosis in protein S deficiency during heparin | |
| Deep vein thrombosis in FVL pseudohomozygous during apixaban | Deep vein thrombosis in protein C deficiency during warfarin | |
| Superficial vein thrombosis in protein S deficiency during heparin | ||
| Bleeding | 1) Hospitalization for menorrhagia (clinically relevant) 2) Hospitalization for metrorrhagia (clinically relevant) 3) Menorrhagia (minor) | 2 patients with recurrent conjunctival hemorrhage (minor) |
| Iron deficiency anemia with the need for transfusion support (clinically relevant) | Hematuria (minor) | |
| Hospitalization for hematuria (clinically relevant) | ||
| Hemorrhoids hematochezia (minor) | ||
| Gingivorrhagia and hematuria (minor) |
[VTE recurrence according to thrombophilia type and bleeding complications]
To cite this abstract in AMA style:
Campello E, Simion C, Spiezia L, Tormene D, Camporese G, Saggiorato G, Dalla Valle F, Simioni P. Efficacy and Safety Profile of Direct Oral Anticoagulants in Consecutive Patients with Severe Hereditary Thrombophilia: A Single-centre Cohort Study [abstract]. Res Pract Thromb Haemost. 2020; 4 (Suppl 1). https://abstracts.isth.org/abstract/efficacy-and-safety-profile-of-direct-oral-anticoagulants-in-consecutive-patients-with-severe-hereditary-thrombophilia-a-single-centre-cohort-study/. Accessed September 29, 2023.« Back to ISTH 2020 Congress
ISTH Congress Abstracts - https://abstracts.isth.org/abstract/efficacy-and-safety-profile-of-direct-oral-anticoagulants-in-consecutive-patients-with-severe-hereditary-thrombophilia-a-single-centre-cohort-study/