Abstract Number: PB0701
Meeting: ISTH 2022 Congress
Background: RUNX1 is a master regulator of hematopoiesis. Pathogenic germline RUNX1 variants cause Familial Platelet Disorder with associated Myeloid Malignancy (FPDMM), which is characterized by abnormal bleeding and a 35%-45% lifetime risk of hematologic malignancy. The ISTH Bleeding Assessment Tool (BAT) questionnaire quantifies bleeding symptoms but has not been studied in patients with germline RUNX1 variants.
Aims: To evaluate the agreement between self-administered and clinician-administered BAT questionnaires in patients with deleterious RUNX1 germline variants.
Methods: Thirty-eight patients consented on the NIH IRB-approved protocol 19-HG-0059 completed the questionnaire. The clinician administered the questionnaire without knowledge of the self-administered questionnaire results. The McNemar test assesses how similarly these two versions agreed in classifying abnormal or normal status per criteria. A Bland–Altman plot of differences vs. averages compares the self- and clinician-administered questionnaire score results.
Results: Self- and clinician-administered BAT score classifications of normal or abnormal agreed for 31 of 38 (82%) patients, with a p-value of 0.12 (Table 1). Six patients self-classified their symptoms as abnormal, in contrast to the clinician’s classification as normal. One patient self-classified their symptoms as normal, in contrast to the clinician’s classification as abnormal. Comparison of the self- and clinician-administered BAT scores shows that all but one of the differences are between or very close to the lower and upper limits of agreement, with no distinctive pattern by sex (Figure 1).
Conclusion(s): In patients with germline RUNX1 mutations, self- and clinician- administered ISTH-BAT questionnaire score classifications agreed at a high rate. For most discordant classifications, patients self-classified their bleeding symptoms as more severe than did the clinician. In the future, a larger sample size will improve the statistical confidence in the rate of agreement, and the BAT can be further analyzed with data on platelet count and function in this unique patient population.
To cite this abstract in AMA style:Andrews E, Choo-Wosoba H, Kalsi S, Merguerian M, Ring M, Davis J, Steinberg S, Liu P, Cunningham L. Comparison between Self-Administered and Clinician-Administered ISTH Bleeding Assessment Tool in RUNX1 Patient Population [abstract]. https://abstracts.isth.org/abstract/comparison-between-self-administered-and-clinician-administered-isth-bleeding-assessment-tool-in-runx1-patient-population/. Accessed February 21, 2024.
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