Session: 322. Hemophilia A and B: Clinical and Epidemiological: Poster III
Hematology Disease Topics & Pathways:
Research, Clinical Practice (Health Services and Quality), Clinical Research, Real-world evidence
Methods: We conducted a retrospective study on children with Hemophilia A at Beijing Children's Hospital. Data were collected from March 2023 to July 2024 for patients who underwent EMI treatment. Laboratory tests performed included TEG, FVIII-like activity, and EMI concentration during the loading period (just prior to the first maintenance dose) and throughout the maintenance period. FVIII-like activity was measured using the human-derived chromogenic substrate method, and EMI drug concentration were measured using the modified one-stage assay. We used Spearman's rank correlation analysis to explore relationships among TEG parameters, EMI concentration, and FVIII-like activity.
Results: Forty-four Hemophilia A patients without inhibitor, treated with EMI, were included in the study. Their median age was 4 years (range 0.1-11), with a Body Mass Index of 16.1 (range 12.4-25.7). Of these, seven had moderate and thirty-seven had severe hemophilia. During the loading period, 24 patients received a median weekly EMI dose of 2.7 mg/kg (range 2.1-2.3). Median EMI concentration was 53.6 μg/ml (range 31.8-79.9), with median FVIII-like activity at 17.4 IU/dl (range 15.4-35.4). Spearman's rank correlation analysis revealed a strong positive correlation between FVIII-like activity and EMI concentration (r=0.6491, p=0.0006). In the maintenance period, 40 patients received treatment with a median monthly EMI dose of 5.2 mg/kg (range 1.6-6.4). Their median EMI concentration was 52.6 μg/ml (range 17.8-73.8), and median FVIII-like activity was 16.2 IU/dl (range 4.3-33.3). A continued strong correlation persisted between FVIII-like activity and EMI concentration (r=0.6636, p<0.0001). EMI concentration negatively correlated with the R-value of TEG (r=-0.4112, p=0.0127), and FVIII-like activity showed correlations with both the R-value (r=-0.4497, p=0.0059) and Clotting Index (CI) of TEG (r=0.3338, p=0.0466). Thirty-seven patients (92.5%) had CI within the normal range (-3 to 3), but three patients (7.5%) displayed CI outside the normal range, with values of -5.7, -3.4, and -3.3, and corresponding R values of 9.2, 10.2, and 10.3 minutes. Their respective FVIII-like activity were 10.5 IU/dl, 10.4 IU/dl, and 14.6 IU/dl.
Conclusion: Our study presents the characteristics of EMI concentration and FVIII-like activity in Chinese pediatric hemophilia patients. It finds that prolonged R-values and reduced CI-values may indicate lower FVIII-like activity, suggesting a risk of bleeding. These findings imply that R and CI values of TEG could serve as important predictors of bleeding risk, offering valuable insights for monitoring the coagulation status during EMI treatment.
Disclosures: No relevant conflicts of interest to declare.
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