R Kulkarni1, R J Presley2, J M Lusher3, A D Shapiro4, J C Gill5, M Manco-Johnson6, M A Koerper7, T C Abshire8, D DiMichele9, W K Hoots10, P Mathew11, D J Nugent12, S Geraghty13, B L Evatt2, J M Soucie2. 1. Department of Pediatrics and Human Development, Michigan State University, East Lansing, MI, USA. 2. Division of Blood Disorders Centers for Disease Control and Prevention, Atlanta, GA, USA. 3. Department of Pediatric Hematology, Children's Hospital of Michigan Wayne State Medical Center, Detroit, MI, USA. 4. Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA. 5. Comprehensive Center for Bleeding Disorders, Blood Center of Wisconsin, Milwaukee, WI, USA. 6. Department of Pediatrics, University of Colorado, Aurora, CO, USA. 7. UCSF Medical Center, San Francisco, CA, USA. 8. Blood Center of Wisconsin, Milwaukee, WI, USA. 9. Division of Blood Diseases and Resources, Bethesda, MD, USA. 10. National Heart Lung and Blood Institute, National Institutes of Health, Bethesda, MD, USA. 11. Department of Pediatric Hematology/Oncology, University of New Mexico, Albuquerque, NM, USA. 12. Center for Inherited Blood Disorders, Children's Hospital of Orange County, Orange, CA, USA. 13. University of Colorado Denver, Aurora, CO, USA.
Abstract
AIM: To describe the prevalence and complications in babies ≤2 years with haemophilia. METHODS: We used a standardized collection tool to obtain consented data on eligible babies aged ≤2 years with haemophilia enrolled in the Centers for Disease Control and Prevention Universal Data Collection System surveillance project at US Hemophilia Treatment Centers (HTCs). RESULTS: Of 547 babies, 82% had haemophilia A, and 70% were diagnosed within one month of birth. Diagnosis was prompted by known maternal carrier status (40%), positive family history (23%), bleeding (35%) and unknown 2%; 81% bled during the first two years. The most common events were bleeding (circumcision, soft tissue, oral bleeding) and head injury. There were 46 episodes of intracranial haemorrhage (ICH) in 37 babies (7%): 18 spontaneous, 14 delivery related, 11 traumatic, 2 procedure related and 1 unknown cause. Of the 176 central venous access devices (CVADs) in 148 (27%) babies, there were 137 ports, 22 surgically inserted central catheters and 20 peripherally inserted central catheters. Ports had the lowest complication rates. Inhibitors occurred in 109 (20%) babies who experienced higher rates of ICH (14% vs. 5%; P = 0.002), CVAD placement (61% vs. 19%; P < 0.001) and CVAD complications (44% vs. 26%; P < 0.001). The most common replacement therapy was recombinant clotting factor concentrates. CONCLUSION: Bleeding events in haemophilic babies ≤2 years were common; no detectable difference in the rates of ICH by the mode of delivery was noted. Neonatal factor exposure did not affect the inhibitor rates. Minor head trauma, soft tissue and oropharyngeal bleeding were the leading indications for treatment.
AIM: To describe the prevalence and complications in babies ≤2 years with haemophilia. METHODS: We used a standardized collection tool to obtain consented data on eligible babies aged ≤2 years with haemophilia enrolled in the Centers for Disease Control and Prevention Universal Data Collection System surveillance project at US Hemophilia Treatment Centers (HTCs). RESULTS: Of 547 babies, 82% had haemophilia A, and 70% were diagnosed within one month of birth. Diagnosis was prompted by known maternal carrier status (40%), positive family history (23%), bleeding (35%) and unknown 2%; 81% bled during the first two years. The most common events were bleeding (circumcision, soft tissue, oral bleeding) and head injury. There were 46 episodes of intracranial haemorrhage (ICH) in 37 babies (7%): 18 spontaneous, 14 delivery related, 11 traumatic, 2 procedure related and 1 unknown cause. Of the 176 central venous access devices (CVADs) in 148 (27%) babies, there were 137 ports, 22 surgically inserted central catheters and 20 peripherally inserted central catheters. Ports had the lowest complication rates. Inhibitors occurred in 109 (20%) babies who experienced higher rates of ICH (14% vs. 5%; P = 0.002), CVAD placement (61% vs. 19%; P < 0.001) and CVAD complications (44% vs. 26%; P < 0.001). The most common replacement therapy was recombinant clotting factor concentrates. CONCLUSION:Bleeding events in haemophilic babies ≤2 years were common; no detectable difference in the rates of ICH by the mode of delivery was noted. Neonatal factor exposure did not affect the inhibitor rates. Minor head trauma, soft tissue and oropharyngeal bleeding were the leading indications for treatment.
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