title: Feasibility of prophylaxis and immune tolerance induction regimens in haemophilic children using fully implantable central venous catheters.
abstract: Venous access represents the major barrier to the feasibility of prophylaxis and immune tolerance induction (ITI) in haemophilic children. Ports improve treatment feasibility, but their duration is limited by infectious complications. This study aimed at evaluating whether or not ports allow haemophilic children to maintain the treatment regimen in the long term. Children were prospectively followed-up and underwent port removal either for complications or transition to peripheral veins. Of 27 ports (17 used for prophylaxis and 10 for ITI), 25 were removed after a median of 3.3 years. Inhibitor children showed a younger age at port insertion (P = 0.02), an earlier occurrence of infections (P = 0.006) at a higher rate (P = 0.00001) and an earlier removal for infection (P = 0.05) than non-inhibitor patients. Daily port use was associated with earlier infections at a higher rate compared to less frequent use (P = 0.02). Port removal after a median of 0.8 years prevented ITI completion in 50% of children, while it hampered the maintenance of prophylaxis in 27% of patients. This study showed that ports improved the feasibility of prophylaxis in the majority of non-inhibitor children, while they were not suitable for inhibitor children who require a prolonged ITI regimen with daily infusions.
authors.affiliation: Department of Medicine and Medical Specialities, Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre, IRCCS Maggiore Hospital, Mangiagalli & Regina Elena Foundation and University of Milan, Milan, Italy.
authors.forename: Maria Elisa
authors.initials: ME
authors.lastname: Mancuso
authors.forename: Pier Mannuccio
authors.initials: PM
authors.lastname: Mannucci
authors.forename: Angelo
authors.initials: A
authors.lastname: Sartori
authors.forename: Andrea
authors.initials: A
authors.lastname: Agliardi
authors.forename: Elena
authors.initials: E
authors.lastname: Santagostino
mesh_terms: D001424:Bacterial Infections / Q000209:etiology; D002405:Catheterization, Central Venous / Q000009:adverse effects / Q000295:instrumentation*; D002408:Catheters, Indwelling / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D003029:Coagulants / Q000008:administration & dosage*; D020878:Device Removal; D004812:Epidemiologic Methods; D004866:Equipment Contamination; D004868:Equipment Failure; D005164:Factor IX / Q000008:administration & dosage / Q000276:immunology; D005169:Factor VIII / Q000008:administration & dosage / Q000276:immunology; D005500:Follow-Up Studies; D006395:Hemarthrosis / Q000517:prevention & control; D006467:Hemophilia A / Q000188:drug therapy*; D002836:Hemophilia B / Q000188:drug therapy*; D006801:Humans; D007108:Immune Tolerance; D007223:Infant; D008297:Male; D019990:Perioperative Care / Q000379:methods; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000276:immunology
chemical_list: D003029:Coagulants; D011994:Recombinant Proteins; D005169:Factor VIII; D005164:Factor IX