Managing the Pediatric Patient and the Adolescent/Adult Transition

ABSTRACT

Healthcare management of the developing child with hemophilia presents several difficulties. Determining the optimum medication dose is complicated by the difficulty of performing pharmacokinetic studies in children and the fact that extrapolation from data collected in adults is generally unsatisfactory. Even when pediatric pharmacokinetic data are available, accumulating evidence suggests that there can be wide interindividual differences; age and size are important contributing factors. Achieving the venous access necessary for blood factor administration is frequently difficult in the small child. Preliminary results suggest that an arteriovenous fistula may be an appropriate option in some patients. Finally, there is the question of whether prophylactic therapy should be discontinued as patients enter adulthood. This article provides insights for managing this challenging patient population.

Pediatric patient - adolescent/adult transition - venous access - prophylactic therapy

REFERENCES

• 1 Kearns G L, Abdel-Rahman S M, Alander S W et al.. Developmental pharmacology-drug disposition, action, and therapy in infants and children.  N Engl J Med. 2003;  349 1157-1167
• 2 Blanchette V. Advate Pediatric Study interim results: Pharmacokinetics in young children versus adults. Paper presented at: 15th Annual Hemophilia Research Study Update January 21-24, 2005 San Juan, Puerto Rico;
• 3 Tarantino M D, Collins P W, Hay C R et al.. Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A.  Haemophilia. 2004;  10 428-437
• 4 Villar A, Aronis S, Morfini M et al.. Pharmacokinetics of activated recombinant coagulation factor VII (NovoSeven) in children vs. adults with haemophilia A.  Haemophilia. 2004;  10 352-359
• 5 Poon M C, Lillicrap D, Hensman C, Card R, Scully M F. Recombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance study.  Thromb Haemost. 2002;  87 431-435
• 6 Valentino L A, Ewenstein B, Navickis R J, Wilkes M M. Central venous access devices in haemophilia.  Haemophilia. 2004;  10 134-146
• 7 Ewenstein B M, Valentino L A, Journeycake J M et al.. Consensus recommendations for use of central venous access devices in haemophilia.  Haemophilia. 2004;  10 629-648
• 8 Valentino L A, Kapoor M. Central venous access devices in patients with hemophilia.  Expert Rev Med Devices. 2005;  2 699-711
• 9 Price V E, Carcao M D, Connolly B et al.. A prospective, longitudinal study of central venous catheter-related deep venous thrombosis in boys with hemophilia.  J Thromb Haemost. 2004;  2 737-742
• 10 Journeycake J M, Quinn C T, Miller K L, Zajac J L, Buchanan G R. Catheter-related deep venous thrombosis in children with hemophilia.  Blood. 2001;  98 1727-1731
• 11 Ettingshausen C E, Kurnik K, Schobess R et al.. Catheter-related thrombosis in children with hemophilia A: evidence of a multifactorial disease.  Blood. 2002;  99 1499-1500
• 12 Kherlakian G M, Roedersheimer L R, Arbaugh J J, Newmark K J, King L R. Comparison of autogenous fistula versus expanded polytetrafluoroethylene graft fistula for angioaccess in hemodialysis.  Am J Surg. 1986;  152 238-243
• 13 Winsett O E, Wolma F J. Complications of vascular access for hemodialysis.  South Med J. 1985;  78 513-517
• 14 Santagostino E, Gringeri A, Berardinelli L et al.. Long-term safety and feasibility of arteriovenous fistulae as vascular accesses in children with haemophilia: a prospective study.  Br J Haematol. 2003;  123 502-506
• 15 Santagostino E. Challenges and approaches to venous access. Paper presented at: 15th Annual Hemophilia Research Study Update January 23, 2005 San Juan, Puerto Rico;
• 16 Fischer K, van der Bom J G, Mauser-Bunschoten E P et al.. Endogenous clotting factor activity and long-term outcome in patients with moderate haemophilia.  Thromb Haemost. 2000;  84 977-980
• 17 Carcao M D, Aledort L. Prophylactic factor replacement in hemophilia.  Blood Rev. 2004;  18 101-113
• 18 Nilsson I M, Berntorp E, Lofqvist T, Pettersson H. Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B.  J Intern Med. 1992;  232 25-32
• 19 Kreuz W, Escuriola-Ettingshausen C, Funk M, Schmidt H, Kornhuber B. When should prophylactic treatment in patients with haemophilia A and B start?-The German experience.  Haemophilia. 1998;  4 413-417
• 20 Berntorp E, Boulyjenkov V, Brettler D et al.. Modern treatment of haemophilia.  Bull World Health Organ. 1995;  73 691-701
• 21 Fischer K, Van Der Bom J G, Prejs R et al.. Discontinuation of prophylactic therapy in severe haemophilia: incidence and effects on outcome.  Haemophilia. 2001;  7 544-550
• 22 van Dijk K, Fischer K, van der Bom J G et al.. Can long-term prophylaxis for severe haemophilia be stopped in adulthood? Results from Denmark and the Netherlands.  Br J Haematol. 2005;  130 107-112
• 23 van den Berg M. If and when and how to stop prophylaxis. Paper presented at: 15th Annual Hemophilia Research Study Update January 21-24, 2005 San Juan, Puerto Rico;