Successful control of central nervous system bleeding in two newborns with severe factor VII deficiency using rFVIIa administered via Port-a-Cath.
We report two cases of newborn infants (15 and 20 days old, respectively; both were full-term infants with normal vaginal deliveries) with severe factor VII (FVII) deficiencies (FVII <1%) who presented with convulsions and poor feeding. Brain sonography and computed tomography scan showed intracranial hemorrhage. Prothrombin time (PT) was severely prolonged, but partial thromboplastin time, platelet count, and bleeding time were normal. FVII assay showed that both infants had FVII levels of less than 1%. Treatment with recombinant activated factor VII (rFVIIa; NovoSeven(((R))), Novo Nordisk, Bagsvaerd, Denmark) was deemed necessary. We started rFVIIa therapy with 20 microg/kg administered intravenously (IV) every 6 hours for 2 to 3 days. This treatment schedule resulted in normalization of PT, control of convulsions, and reduction in central nervous system (CNS) bleeding. Although prophylaxis is currently an off-label use of rFVIIa, the patients then received one IV bolus dose of 20 microg/kg every other day as a prophylaxis regimen, but the symptoms recurred in one patient a few days later. Therefore, we increased the dose to 30 microg/kg IV every 4 hours, and this led to control of convulsions and CNS bleeding. This patient received rFVIIa at a dose of 30 microg/kg every other day via Port-a-Cath (Bistric ATH, France) due to poor venous access. His parents were educated in the use of the Port-a-Cath, and the patient received home treatment with rFVIIa (30 microg/kg IV every other day and then twice per week) for 6 months. He had a normal neurological status at this time. Unfortunately, at the age of 6 months, this infant died from severe post-traumatic CNS bleeding when he was 60 km away from our center. The second patient, who also received prophylactic rFVIIa at a dose of 20 microg/kg every other day with gradual prolongation of the dose interval to once per week, is currently in good condition with a mild neurological deficit caused by repeated CNS bleedings that occurred when she was newborn, before her first presentation to our clinic. She is now 36 months old and on a 3-year rFVIIa prophylactic regimen, which has provided good results to date.
Department of Pediatrics, Hematology Research Center, Shiraz University of Medical Sciences, Shiraz, Iran. karimim@sums.ac.ir
Mehran
M
Karimi
D005168:Factor VII Deficiency / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy*; D015942:Factor VIIa / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007231:Infant, Newborn; D020300:Intracranial Hemorrhages / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use
D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa