Updated guidance for efficacy and safety outcomes for clinical trials in venous thromboembolism in children: communication from the ISTH SSC Subcommittee on Pediatric and Neonatal Thrombosis and Hemostasis

Whitworth, Hilary; Amankwah, Ernest K.; Betensky, Marisol; Castellucci, Lana A; Cuker, Adam; Goldenberg, Neil A.; Male, Christoph; Rinzler, Elliot; Zia, Ayesha; Raffini, Leslie

doi:10.1016/j.jtha.2023.03.004

Cited by 5 publications

(3 citation statements)

References 28 publications

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“…In our analysis, we considered this minor bleeding. The ISTH Subcommittee on Pediatric and Neonatal Thrombosis and Hemostasis published a practice guideline defining these outcomes as “patient important bleeding, no intervention” [ 15 ] and specifically as “bleeding for which a patient seeks care but there is no change in management.” This type of bleeding event is important to consider in clinical studies; although these events do not require a change in management, they are bothersome to families and patients and impact the quality of life. As such, this bleeding classification should be included in prospective studies in this population.…”

Section: Discussionmentioning

confidence: 99%

Procedural outcomes in children with mild type 1 von Willebrand disease

Heery,

Zimowski,

Mason

et al. 2024

Research and Practice in Thrombosis and Haemostasis

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Section: Discussionmentioning

confidence: 99%

Procedural outcomes in children with mild type 1 von Willebrand disease

Heery,

Zimowski,

Mason

et al. 2024

Research and Practice in Thrombosis and Haemostasis

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“…VTE progression and symptomatic recurrence while receiving anticoagulation were adjudicated by chart and imaging review by the corresponding author. 14 Bleeding and thrombosis progression/recurrence were expected to occur in this medically complex patient cohort. A detailed review of events included (i) appropriate anticoagulation monitoring (eg, anti-Xa levels for those on enoxaparin therapy), (ii) platelet transfusions for thrombocytopenia, and (iii) interruption of anticoagulation during surgical/invasive procedures.…”

Section: Methodsmentioning

confidence: 99%

Improving Anticoagulation Care for Pediatric Oncology Patients: A Quality Improvement Initiative

Rodriguez,

Mitchell,

Stanek

et al. 2024

Pediatric Quality &Amp; Safety

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Background: Cancer is associated with increased venous thromboembolism in children. Risk factors for venous thromboembolism in this cohort include using central venous catheters, mass effect from underlying malignancy, chemotherapy, and surgery. Anticoagulation management in this cohort is challenging, given recurrent episodes of thrombocytopenia, the need for invasive procedures, and coagulopathy. A quality improvement (QI) initiative was developed to improve hematology consultation services and provide documentation of an individualized anticoagulation care plan for this high-risk cohort. Methods: Through the use of QI methods, interviews of stakeholders, expert consensus, and review of baseline data, a multidisciplinary team was organized, and key drivers relevant to improving access to hematology consultations and documentation of individualized anticoagulation care plans were identified. We used a Plan-Do-Study-Act model to improve hematology consultations and documentation of anticoagulation care plan (process measure). Outcome measures were bleeding and thrombosis recurrence/progression. Results: Seventeen patients with oncologic and venous thromboembolism diagnoses were included as baseline data. Slightly over half of these patients [53% (n = 9)] had a hematology consultation, and 7 (43.8%) had documentation of an anticoagulation care plan. After implementing QI methods, all 34 patients (100%) received hematology consultations and documentation of an anticoagulation care plan, and this measure was sustained for 1 year. Bleeding and thrombosis rates were similar in the baseline and post-QI cohorts. Conclusions: QI interventions proved effective in sustaining access to hematology consultations and providing anticoagulation care plans for patients with concomitant improved anticoagulation plan documentation for patients.

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“…Patients were analyzed using IBM SPSS statistical software, and a descriptive statistical analysis was conducted. Efficacy and safety outcomes were defined per the International Society on Thrombosis and Hemostasis (ISTH) scientific and standardization subcommittee on pediatric and neonatal thrombosis and hemostasis updated 2023 definitions 27 . A major change in the definitions is that heavy menstrual bleeding (HMB) can now be included in any category if the criteria are met based on severity.…”

Section: Methodsmentioning

confidence: 99%

Real‐world experience of direct oral anticoagulant use in a single pediatric center

Valenti,

Sabo,

Hyde

et al. 2024

Pediatric Blood & Cancer

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BackgroundPediatric venous thromboembolism has increased by 130%–200%, specifically in hospitalized children, and direct oral anticoagulants (DOACs) offer several therapeutic advantages.MethodsThis study aims to evaluate the real‐world epidemiological and outcome data from a retrospective review of pediatric patients treated with DOACs from January 1, 2013 to December 31, 2022. In this single‐center, IRB‐approved study, 65 patients were identified and analyzed using SPSS statistical software, and a descriptive statistical analysis was conducted.ResultsOf the 65 patients, 37% were on apixaban, 61.5% were on rivaroxaban, and 1.5% were on dabigatran. Per the 2023 ISTH outcome definitions, one (2%) patient had a major bleeding episode, six (9%) had clinically relevant non‐major bleeding, three (5%) patients had patient‐important heavy menstrual bleeding (HMB), and one (1.5%) patient had minor bleeding. Seven (19%) of 37 postmenarchal patients had evidence of HMB. Six (9.2%) patients had recurrent venous thromboembolism while on a DOAC (one was on apixaban, and five were on rivaroxaban) and were transitioned to other forms of anticoagulation.ConclusionThus, bleeding rates after DOAC therapy are comparable to previous DOAC trials, as well as other anticoagulants in pediatrics. HMB is an important outcome measure and should continue to be investigated. This study reports a higher rate of recurrent thrombosis (9.2%) compared to other trials. However, this observation may be attributed to patients who had ongoing risk factors, as well as a longer duration of study follow‐up. Additional multicentered outcome studies evaluating DOAC use in children are needed to determine long‐term recurrence and HMB risks.

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Updated guidance for efficacy and safety outcomes for clinical trials in venous thromboembolism in children: communication from the ISTH SSC Subcommittee on Pediatric and Neonatal Thrombosis and Hemostasis

Cited by 5 publications

References 28 publications

Procedural outcomes in children with mild type 1 von Willebrand disease

Procedural outcomes in children with mild type 1 von Willebrand disease

Improving Anticoagulation Care for Pediatric Oncology Patients: A Quality Improvement Initiative

Real‐world experience of direct oral anticoagulant use in a single pediatric center

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