title: Subcutaneous protein C concentrate in the management of severe protein C deficiency--experience from 12 centres.
abstract: Since the first description of subcutaneous protein C concentrate as treatment for severe protein C deficiency in 1996, further cases have been reported but there is no uniform approach to this form of treatment. In order to assess the safety and effectiveness of subcutaneous protein C concentrate and suggest recommendations for future use, patients who had received subcutaneous protein C concentrate were identified from the literature, by contacting the manufacturers and by personal communication. Treatment details were available from 14 cases. Apart from one case where the infusion interval was inadvertently increased, no thrombotic events occurred even when doses were subsequently reduced. Initially, a trough protein C level of >0·25 iu/ml should be aimed for. Subsequently, a smaller dose of subcutaneous protein C concentrate, especially if taken with an oral anticoagulant, may be protective maintenance treatment. The treatment was well tolerated with few side effects. Subcutaneous protein C concentrate on its own or combined with an oral anticoagulant appears to be safe and effective as maintenance treatment of severe protein C deficiency. A major advantage is the avoidance of central venous access devices. The incidence of neurodevelopmental handicap was high with blindness affecting the majority of patients.
authors.affiliation: Department of Paediatrics, Bradford Royal Infirmary, Bradford, UK.
authors.forename: Adrian
authors.initials: A
authors.lastname: Minford
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Behnisch
authors.forename: Paul
authors.initials: P
authors.lastname: Brons
authors.forename: Michele
authors.initials: M
authors.lastname: David
authors.forename: Natividad
authors.initials: N
authors.lastname: Gomez Gomez
authors.forename: Hans-Joerg
authors.initials: HJ
authors.lastname: Hertfelder
authors.forename: Anne
authors.initials: A
authors.lastname: Kruempel
authors.forename: Karin
authors.initials: K
authors.lastname: Kurnik
authors.forename: Mary
authors.initials: M
authors.lastname: Mathias
authors.forename: Antonio
authors.initials: A
authors.lastname: Molines Honrubia
authors.forename: Paul
authors.initials: P
authors.lastname: Monagle
authors.forename: Mary
authors.initials: M
authors.lastname: Morgan
authors.forename: Ulrike
authors.initials: U
authors.lastname: Nowak-Göttl
authors.forename: Martin
authors.initials: M
authors.lastname: Olivieri
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007223:Infant; D055104:Infusions, Subcutaneous; D008297:Male; D008875:Middle Aged; D011486:Protein C / Q000008:administration & dosage* / Q000009:adverse effects / Q000378:metabolism; D020151:Protein C Deficiency / Q000097:blood / Q000188:drug therapy*; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D011486:Protein C
keywords: blindness; protein C; protein C concentrate; protein C deficiency; subcutaneous infusion