Abstract Number: PB0528
Meeting: ISTH 2021 Congress
Background: The International Society of Thrombosis and Haemostasis Bleeding Assessment Tool (ISTH-BAT) was developed to objectively quantify bleeding symptoms in patients with a bleeding disorder. Quantitative data on of the bleeding phenotype in patients with non-severe hemophilia is currently lacking. The use of the ISTH-BAT may provide insight into the extent of reported bleeding in this population.
Aims: To describe the bleeding phenotype in patients with non-severe hemophilia according to the ISTH-BAT.
Methods: The DYNAMO study is a multicenter international cohort study that includes patients with non-severe hemophilia A and B (baseline FVIII/FIX 2-35 IU/dL) aged 12-55 years. Data were collected from medical files and participants completed questionnaires including the ISTH-BAT. ISTH-BAT scores were compared between factor levels, severities and hemophilia type. Abnormal scores were defined as ≥4. Medical ethical approval and written consent was obtained (ClinicalTrials.gov: NCT03623295).
Results: We report preliminary data on 106 patients (89 HA, 17 HB) who completed the questionnaire (response rate 60%). The median age was 38 years (IQR 25-50) and the median factor level was 11 IU/dL (IQR 5-16). The median ISTH-BAT score in our population was 9 (IQR 4-15), with 82% of our population reporting an abnormal bleeding score. Patients with moderate and mild hemophilia had median ISTH-BAT scores of 13 (IQR 9-16) and 7 (IQR 4-13) respectively. An inverse association was observed between the ISTH-BAT scores and the baseline factor level (Figure 1).
ISTH-BAT scores were similar between hemophilia A and B. Joint bleeds, muscle bleeds and bruises were scored most frequently: 59%, 63% and 60% respectively (Figure 2), with 54% and 49% of patients reporting sub-scores ≥ 3 for joint and muscle bleeds respectively.
Conclusions: Abnormal bleeding scores were seen in 82% of the patients, and the median ISTH-BAT score of 9, demonstrating a considerable burden of disease in patients with non-severe hemophilia.
To cite this abstract in AMA style:Kloosterman F, Zwagemaker A, Gouw SC, Bagot CN, Brons P, Cnossen M, Collins P, Jackson S, Meijer K, Kruip MJHA, Leebeek FWG, Laros-van Gorkom B, Male C, Pabinger I, Shapiro S, Coppens M, van der Bom JG, Fijnvandraat K, DYNAMO Study Group . Assessment of the Bleeding Phenotype in Patients with Non-severe Hemophilia According to the ISTH Bleeding Assessment Tool (BAT) [abstract]. Res Pract Thromb Haemost. 2021; 5 (Suppl 2). https://abstracts.isth.org/abstract/assessment-of-the-bleeding-phenotype-in-patients-with-non-severe-hemophilia-according-to-the-isth-bleeding-assessment-tool-bat/. Accessed December 8, 2021.
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