title: Long-term subcutaneous protein C replacement in neonatal severe protein C deficiency.
abstract: We describe here the case of a boy who presented 2 days after birth with purpura fulminans on his feet and scalp. Laboratory investigations revealed signs of disseminated intravascular coagulation. An underlying coagulation disorder was suspected, and therapy with recombinant tissue plasminogen activator, fresh-frozen plasma, and unfractionated heparin was started. On the basis of plasma protein C activity and antigen levels of 0.02 and 0.03 IU/mL, respectively, after administration of fresh-frozen plasma, a diagnosis of severe protein C deficiency was established, and therapy with intravenous protein C concentrate (Ceprotin [Baxter, Deerfield, IL]) was started. Because of difficulties with venous access, we switched to subcutaneous administration after 6 weeks. The precise dosing schedule for subcutaneously administered protein C concentrate is unknown. In the literature, a trough level of protein C activity at >0.25 IU/mL is recommended to prevent recurrent thrombosis. During 1 year of follow-up our patient frequently had protein C activity levels at <0.25 IU/mL. Clinically, however, there was no recurrent thrombosis, and we kept the dosage unchanged. This report highlights 2 important points: (1) subcutaneously administered protein C concentrate is effective in treating severe protein C deficiency; and (2) in accordance with previous studies, after the acute phase trough levels of protein C activity at >0.25 IU/mL may not be necessary to prevent recurrent thrombosis. However, further research on the dosing, efficacy, and safety of protein C concentrate for prophylaxis and treatment of severe protein C deficiency is needed.
authors.affiliation: Department of Pediatrics, Radboud University Nijmegen Medical Centre, PO Box 9101, 6500 HB Nijmegen, Netherlands.
authors.forename: Ellen H M
authors.initials: EH
authors.lastname: de Kort
authors.forename: Sabine L A G
authors.initials: SL
authors.lastname: Vrancken
authors.forename: Arno F J
authors.initials: AF
authors.lastname: van Heijst
authors.forename: Mathijs
authors.initials: M
authors.lastname: Binkhorst
authors.forename: Marcel P J M
authors.initials: MP
authors.lastname: Cuppen
authors.forename: Paul P T
authors.initials: PP
authors.lastname: Brons
mesh_terms: D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005500:Follow-Up Studies; D006801:Humans; D007231:Infant, Newborn; D007262:Infusions, Intravenous; D055104:Infusions, Subcutaneous; D008134:Long-Term Care; D008297:Male; D011486:Protein C / Q000627:therapeutic use*; D020151:Protein C Deficiency / Q000150:complications / Q000175:diagnosis* / Q000188:drug therapy* / Q000235:genetics; D055665:Purpura Fulminans / Q000188:drug therapy* / Q000209:etiology / Q000503:physiopathology; D018570:Risk Assessment; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D011486:Protein C