A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome
Abstract Background There is uncertainty regarding the safety and effectiveness of direct oral anticoagulant agents in patients with antiphospholipid syndrome (APS). We performed a multicenter feasibility study to examine our ability to identify and obtain consent from eligible APS patients and to o...
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| Format: | Article |
| Language: | English |
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BMC
2020-04-01
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| Series: | Pilot and Feasibility Studies |
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| Online Access: | http://link.springer.com/article/10.1186/s40814-020-00594-1 |
| _version_ | 1817994972842950656 |
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| author | Kimberly Legault Mark Blostein Marc Carrier Susan Khan Sam Schulman Sudeep Shivakumar Cynthia Wu Mark A. Crowther |
| author_facet | Kimberly Legault Mark Blostein Marc Carrier Susan Khan Sam Schulman Sudeep Shivakumar Cynthia Wu Mark A. Crowther |
| author_sort | Kimberly Legault |
| collection | DOAJ |
| description | Abstract Background There is uncertainty regarding the safety and effectiveness of direct oral anticoagulant agents in patients with antiphospholipid syndrome (APS). We performed a multicenter feasibility study to examine our ability to identify and obtain consent from eligible APS patients and to obtain 95% adherence with daily rivaroxaban administration, in order to inform and power a larger study. Clinical outcomes of bleeding and thrombosis were also collected. Methods APS patients with prior venous thromboembolism (VTE) were recruited over 2 years (Oct 2014–Sept 2016) and followed for ≥ 1 year. Patients were assessed clinically every 3 months and had pill counts performed every 6 months. Numbers of patients fulfilling study criteria, as well as those consenting to participate, were tracked, and percentage adherence based on pill counts was recorded. These data were compared against the feasibility endpoints. Rates of thrombosis and bleeding were calculated. Criterion for feasibility was obtaining consent from 135 of 150 identified APS patients over 2 years. Results Ninety-six eligible patients were identified, and 14 declined participation. Eighty-two patients were followed for a mean of 19 months, representing 129.8 patient-years. Average rivaroxaban adherence was 95.0%. During follow-up, there were 4 thromboembolic events (2 cerebrovascular and 2 VTE). There were no episodes of major bleeding. Conclusions Adequately powered comparative trials using patient-important outcomes in APS are unlikely to be successful due to inability to recruit sufficient numbers of study subjects. This study does not reveal a higher than expected risk of recurrent thromboembolic disease compared to historical cohorts; however, this is an uncontrolled study in relatively low-risk APS patients. Trial registration The study was registered with clinicaltrials.gov, identifier NCT02116036 , April 16, 2014. |
| first_indexed | 2024-04-14T02:00:14Z |
| format | Article |
| id | doaj.art-8e75d6afce2846d8b7d6c62a71986b26 |
| institution | Directory Open Access Journal |
| issn | 2055-5784 |
| language | English |
| last_indexed | 2024-04-14T02:00:14Z |
| publishDate | 2020-04-01 |
| publisher | BMC |
| record_format | Article |
| series | Pilot and Feasibility Studies |
| spelling | doaj.art-8e75d6afce2846d8b7d6c62a71986b262022-12-22T02:18:52ZengBMCPilot and Feasibility Studies2055-57842020-04-01611710.1186/s40814-020-00594-1A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndromeKimberly Legault0Mark Blostein1Marc Carrier2Susan Khan3Sam Schulman4Sudeep Shivakumar5Cynthia Wu6Mark A. Crowther7Department of Medicine, McMaster UniversityDepartment of Medicine, McGill UniversityDepartment of Medicine, University of OttawaDepartment of Medicine, McGill UniversityDepartment of Medicine, McMaster UniversityDepartment of Medicine, Dalhousie UniversityDepartment of Medicine, University of AlbertaDepartment of Medicine, McMaster UniversityAbstract Background There is uncertainty regarding the safety and effectiveness of direct oral anticoagulant agents in patients with antiphospholipid syndrome (APS). We performed a multicenter feasibility study to examine our ability to identify and obtain consent from eligible APS patients and to obtain 95% adherence with daily rivaroxaban administration, in order to inform and power a larger study. Clinical outcomes of bleeding and thrombosis were also collected. Methods APS patients with prior venous thromboembolism (VTE) were recruited over 2 years (Oct 2014–Sept 2016) and followed for ≥ 1 year. Patients were assessed clinically every 3 months and had pill counts performed every 6 months. Numbers of patients fulfilling study criteria, as well as those consenting to participate, were tracked, and percentage adherence based on pill counts was recorded. These data were compared against the feasibility endpoints. Rates of thrombosis and bleeding were calculated. Criterion for feasibility was obtaining consent from 135 of 150 identified APS patients over 2 years. Results Ninety-six eligible patients were identified, and 14 declined participation. Eighty-two patients were followed for a mean of 19 months, representing 129.8 patient-years. Average rivaroxaban adherence was 95.0%. During follow-up, there were 4 thromboembolic events (2 cerebrovascular and 2 VTE). There were no episodes of major bleeding. Conclusions Adequately powered comparative trials using patient-important outcomes in APS are unlikely to be successful due to inability to recruit sufficient numbers of study subjects. This study does not reveal a higher than expected risk of recurrent thromboembolic disease compared to historical cohorts; however, this is an uncontrolled study in relatively low-risk APS patients. Trial registration The study was registered with clinicaltrials.gov, identifier NCT02116036 , April 16, 2014.http://link.springer.com/article/10.1186/s40814-020-00594-1Antiphospholipid syndromeFeasibility studiesHemorrhageRivaroxabanThromboembolism |
| spellingShingle | Kimberly Legault Mark Blostein Marc Carrier Susan Khan Sam Schulman Sudeep Shivakumar Cynthia Wu Mark A. Crowther A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome Pilot and Feasibility Studies Antiphospholipid syndrome Feasibility studies Hemorrhage Rivaroxaban Thromboembolism |
| title | A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome |
| title_full | A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome |
| title_fullStr | A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome |
| title_full_unstemmed | A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome |
| title_short | A single-arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome |
| title_sort | single arm feasibility cohort study of rivaroxaban in antiphospholipid syndrome |
| topic | Antiphospholipid syndrome Feasibility studies Hemorrhage Rivaroxaban Thromboembolism |
| url | http://link.springer.com/article/10.1186/s40814-020-00594-1 |
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