1. Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitors
- Author
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Alessandro Gringeri, Erik Berntorp, Kaan Kavakli, Riitta Lassila, Hyejin Jo, Angiola Rocino, Bulent Zulfikar, Jerzy Windyga, Marusia Valentina Uscatescu, Claude Negrier, Paolo Cortesi, Bülent Antmen, Cindy A. Leissinger, Chiara Biasoli, Lorenzo G. Mantovani, Massimo Morfini, Wolfgang Schramm, Margit Serban, Shannon L. Carpenter, Çukurova Üniversitesi, Leissinger, C, Gringeri, A, Antmen, B, Berntorp, E, Biasoli, C, Carpenter, S, Cortesi, P, Jo, H, Kavakli, K, Lassila, R, Morfini, M, Neǵrier, C, Rocino, A, Schramm, W, Serban, M, Uscatescu, M, Windyga, J, Zul̈fikar, B, and Mantovani, L
- Subjects
Adult ,Male ,Health Services Research, Adolescent ,medicine.medical_specialty ,Adolescent ,MED/42 - IGIENE GENERALE E APPLICATA ,Hemorrhage ,Hemophilia A ,Drug Administration Schedule ,Statistics, Nonparametric ,Joint disease ,Young Adult ,Anti-Inhibitor Coagulant Complex ,Internal medicine ,medicine ,Humans ,Prospective Studies ,Young adult ,Prospective cohort study ,Child ,Aged ,Bleeding episodes ,Cross-Over Studies ,Factor VIII ,business.industry ,Medicine (all) ,General Medicine ,Cross-Over Studie ,Middle Aged ,Crossover study ,Treatment period ,Blood Coagulation Factors ,Surgery ,Target dose ,Prospective Studie ,Child, Preschool ,Female ,business ,Blood Coagulation Factor ,Human - Abstract
BACKGROUND: Patients with severe hemophilia A and factor VIII inhibitors are at increased risk for serious bleeding complications and progression to end-stage joint disease. Effective strategies to prevent bleeding in such patients have not yet been established. METHODS: We enrolled patients with hemophilia A who were older than 2 years of age, had high-titer inhibitors, and used concentrates known as bypassing agents for bleeding in a prospective, randomized, crossover study comparing 6 months of anti-inhibitor coagulant complex (AICC), infused prophylactically at a target dose of 85 U per kilogram of body weight (±15%) on 3 nonconsecutive days per week, with 6 months of on-demand therapy (AICC at a target dose of 85 U per kilogram [±15%] used for bleeding episodes). The two treatment periods were separated by a 3-month washout period, during which patients received on-demand therapy for bleeding. The primary outcome was the number of bleeding episodes during each 6-month treatment period. RESULTS: Thirty-four patients underwent randomization; 26 patients completed both treatment periods and could be evaluated per protocol for the efficacy analysis. As compared with on-demand therapy, prophylaxis was associated with a 62% reduction in all bleeding episodes (P
- Published
- 2011