THromboprophylaxis In Sickle Cell Disease with central venous catheters (THIS): an internal pilot randomised controlled trial protocol.

Introduction: Individuals with sickle cell disease (SCD) and central venous catheters (CVC) are at high risk for venous thromboembolism (VTE). Minimal data exist regarding the use of anticoagulation as thromboprophylaxis of VTE in this demographic, and as a result, clinical equipoise exists. Prophylactic dose rivaroxaban, a direct oral anticoagulant, is efficacious and safe as thromboprophylaxis in other demographics, and may be an optimal agent in SCD with CVC. Prior to conducting a full clinical trial to assess rivaroxaban as thromboprophylaxis in SCD with CVC, a pilot study is needed to gauge its feasibility.
Methods and Analysis: THromboprophylaxis In Sickle Cell Disease pilot trial is an investigator-initiated, multicentre, double-blinded, randomised controlled trial (RCT) assessing if it is feasible and safe to conduct an adequately powered RCT comparing rivaroxaban to matching placebo as thromboprophylaxis in those with SCD and CVC. Fifty adult patients with SCD and CVC will be randomised to receive either rivaroxaban 10 mg daily or matching placebo for the duration of the CVC in situ for up to 1 year. After randomisation, follow-up visits will occur every 3 months. The primary outcomes pertain to the feasibility of a full trial and include numbers of eligible and recruited participants. Exploratory outcomes include overall incidence of VTE and bleeding complications, as well as quality of life. If the full trial is feasible, blinding will be maintained and patients in the pilot study will be included in the full trial.
Ethics and Dissemination: The trial was initially approved by the University Health Network Research Ethics Board (REB) in Toronto, Canada. All sites will obtain approval from their respective REB prior to commencement of study activities. Study results will be disseminated through presentations at medical conferences and peer-reviewed publications.
Trial Registration Number: NCT05033314.
Competing Interests: Competing interests: SF has received research funding from the Canadian Hematology Society, and PFIZER; Honoraria from Novartis; and Consultancy fees from Novo Nordisk and Vertex. PB has received research funding from Bluebird, NOVARTIS, ROCHE Fabre Foundation and ADDMEDICA; Consultancy fees from AGIOS, EMMAUS, GBT, ROCHE, HEMANEXT, Bluebird, NOVARTIS, and ADDMEDICA; lecture fees from Jazz Pharma, NOVARTIS, and ADDMEDICA; is the cofounder of INNOVHEM, is on steering committees for NOVARTIS, ROCHE, ADDMEDICA, and PFIZER. KHMK has received research funding from AGIOS and PFIZER; Consulting fees from Alexion Pharmaceuticals, AGIOS, Bristol Myers Squibb, Forma Therapeutics, Pfizer, Novo Nordisk, and Vertex; Honoraria for speaking from AGIOS, and Bristol Myers Squibb; and is a member of a Data Safety Monitoring Board or Advisory Board for Bioverativ/Sanofi/Sangamo. The other authors have no competing interests to declare.
(© Author(s) (or their employer(s)) 2024. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)