Real‐World Clinical Data and Thrombin Generation of Efanesoctocog Alfa Prophylaxis in Pediatric Patients With Haemophilia A
Jessica Garcia, Ayesha ZiaABSTRACT
Background
Clinical trials have shown efficacy and safety of efanesoctocog alfa prophylaxis in children and adults, however, real‐world data on the efficacy and safety remains scarce.
Objectives
To report real‐world data on the effectiveness, immunogenicity, safety, pharmacokinetics and thrombin generation in pediatric persons with haemophilia A receiving efanesoctocog alfa.
Methods
We performed a retrospective chart review of pediatric males with haemophilia A on efanesoctocog alfa prophylaxis at Children's Health in Dallas, Texas.
Results/Conclusion
Twenty two pediatric patients with haemophilia A (mean age of 13 years old (4–17)) switched or started efanesoctocog alfa prophylaxis. Over a median (IQR) duration of 15.5 (10–26) months, two patients experienced minor side effects. No inhibitors were detected. The mean trough FVIII level was 15 IU/dL (7–29; SD 6) and FVIII peak 137 IU/dL (92–207; SD 28), mean trough ETP 396, SD 180 nanomolar‐minutes (nM.min), and peak ETP 980, SD 219 nM.min, mean trough PT 38, SD 23 nM, and mean peak PT 161, SD 65. There were no spontaneous breakthrough bleeding events; six patients (5 played sports) developed a traumatic bleeding event. Our real‐world data shows that efanesoctocog alfa was overall well tolerated, effective, and favourable immunogenicity in pediatric patients. Cohorts with longer follow‐up, further measures of haemostasis, joint health surveillance with imaging and quality of life measurements should be included in future reports.