PB0611 - Real-world evidence (RWE) on direct oral anticoagulants (DOACs) in pediatric patients: a systematic review of the literature

Background: The DOAC pediatric investigational programs have generated some of highest quality evidence supporting its use in pediatric thromboembolism (TE). However, there is a need to address many clinically relevant questions regarding their use in children.

Aims: To summarize the RWE on patient, TE events and treatment characteristics, as well as outcomes of children receiving a DOAC for TE treatment or prophylaxis.

Methods: A systematic search of MEDLINE, EMBASE, and the Cochrane Library from 2008 to December 2023 was performed. Eligible publications included observational studies reporting on the RWE of a DOAC in patients ≤21 years old with one outcome of interest: 1) patient, thrombus or treatment characteristics, or 2) efficacy or safety outcomes. Clinical/interventional trials, studies including patients >21 years of age with outcomes not reported by age group, and studies including different anticoagulant agents with outcomes not reported by type of agent were excluded. Descriptive statistics were used to summarize baseline characteristics and outcomes.

Results: Nineteen studies were included (Figure 1): twelve cohorts (retrospective = 11), six case series and one registry. A total of 2,476 patients ≤ 21 years old were included (Table 1). Median age ranged from 3.5 – 20.5 years with most patients in the 12-17 years age group (70%); 41% were female. The most common co-morbidity was heart disease (12%). The most frequent TE event was venous thromboembolism (VTE, 46%). Rivaroxaban was the most frequently used DOAC (47%), followed by apixaban (32%). Development of VTE or arterial thrombosis was reported in 2.3% and 0.35% of patients respectively, while the frequency of any bleeding, major bleeding and clinically-relevant non-major bleeding was 11%, 0.4 % and 2.3% respectively.

Conclusion(s): We report on the RWE of the use of DOACs in patients ≤ 21 years old.