Karen L Zimowski1, Catherine E McGuinn2, Yasmina L Abajas3, Corinna L Schultz4, Shipra Kaicker2, Glaivy Batsuli1. 1. Aflac Cancer and Blood Disorders Center of Children's Healthcare of Atlanta and Department of Pediatrics, Emory University, Atlanta, Georgia, USA. 2. Division of Pediatric Hematology and Oncology, Weill Cornell Medicine, New York, New York, USA. 3. Pediatric Hematology/Oncology, University of North Carolina, Chapel Hill, North Carolina, USA. 4. Nemours Center for Cancer & Blood Disorders, Nemours/AI duPont Hospital for Children, Wilmington, Delaware, USA.
Abstract
BACKGROUND: Congenital factor X deficiency (FXD) is a rare bleeding disorder that often presents with severe bleeding in the neonatal period. Long-term prophylaxis with infusions of FX-containing products is recommended in patients with FXD and a personal or family history of severe bleeding. A plasma-derived FX concentrate (pdFX) is approved for on-demand and prophylactic therapy in adults and children with FXD. The safety and efficacy of pdFX has been demonstrated in patients <12 years of age, yet limited data exist regarding its use in infants. PATIENTS/ METHODS: This retrospective case series details clinical experience using pdFX in four neonates with moderate and severe FXD across four institutions. RESULTS AND CONCLUSIONS: All four patients presented in the first week of life with severe bleeding. Following treatment of the acute bleed, prophylactic pdFX was initiated at an average of 29 days of life and a dose of 69 IU/kg every 48 hours. Incremental recovery (IR) in three infants averaged 1.42 IU/dL per IU/kg (min-max: 1.06-1.67 IU/dL per IU/kg). One patient experienced thrombotic complications in the setting of sepsis. After a median follow-up of 26.5 months, no patient has experienced breakthrough bleeding episodes. Our study supports the use of pdFX in neonates and infants and suggests that higher pdFX dosing of 70 to 80 IU/kg every 48 hours based on the smallest available vial size is feasible. Because of variability in IR, close monitoring of FX activity should be used to guide dosing in this age group.
BACKGROUND:Congenital factor X deficiency (FXD) is a rare bleeding disorder that often presents with severe bleeding in the neonatal period. Long-term prophylaxis with infusions of FX-containing products is recommended in patients with FXD and a personal or family history of severe bleeding. A plasma-derived FX concentrate (pdFX) is approved for on-demand and prophylactic therapy in adults and children with FXD. The safety and efficacy of pdFX has been demonstrated in patients <12 years of age, yet limited data exist regarding its use in infants. PATIENTS/ METHODS: This retrospective case series details clinical experience using pdFX in four neonates with moderate and severe FXD across four institutions. RESULTS AND CONCLUSIONS: All four patients presented in the first week of life with severe bleeding. Following treatment of the acute bleed, prophylactic pdFX was initiated at an average of 29 days of life and a dose of 69 IU/kg every 48 hours. Incremental recovery (IR) in three infants averaged 1.42 IU/dL per IU/kg (min-max: 1.06-1.67 IU/dL per IU/kg). One patient experienced thrombotic complications in the setting of sepsis. After a median follow-up of 26.5 months, no patient has experienced breakthrough bleeding episodes. Our study supports the use of pdFX in neonates and infants and suggests that higher pdFX dosing of 70 to 80 IU/kg every 48 hours based on the smallest available vial size is feasible. Because of variability in IR, close monitoring of FX activity should be used to guide dosing in this age group.
Authors: Jeanette Payne; Glaivy Batsuli; Andrew D Leavitt; Mary Mathias; Catherine E McGuinn Journal: Haemophilia Date: 2022-05-02 Impact factor: 4.263