Paediatric haemophilia with inhibitors: existing management options, treatment gaps and unmet needs |
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| Authors: | E. SANTAGOSTINO,M. MORFINI&dagger ,G. K.-H. AUERSWALD&Dagger ,G. M. BENSON§ ,S. Z. &# ALEK¶ ,T. LAMBERT,P. SALAJ&dagger &dagger ,V. JIMENEZ-YUSTE&Dagger &Dagger , R. C. R. LJUNG§ § |
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| Affiliation: | Angelo Bianchi Bonomi Haemophilia and Thrombosis Centre, IRCCS Maggiore Hospital, Mangiagalli and Regina Elena Foundation, University of Milan, Milan, Italy;;Agency for Hemophilia –Reference Center for Inherited Bleeding Disorders of Tuscany, Department of Emergency and Reception, Azienda Ospedaliero Universitaria Careggi, Florence, Italy;;Klinikum Bremen-mitte, Professor Hess Children's Hospital, Bremen, Germany;;Northern Ireland Haemophilia Comprehensive Care Centre, Belfast, Northern Ireland;;National Haemophilia Centre, University Hospital Centre, Rebro, Croatia;;Hemophilic Care Center, Bicêtre AP-HP Hospital and Facultéde Médecine Paris XI, Paris, France;;Institute of Hematology and Blood Transfusion, Prague, Czech Republic;;Haematology Department, Hospital Universitario La Paz, Madrid, Spain;;and Department of Paediatrics and Coagulation Disorders, University Hospital Malmö, Malmö, Sweden |
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| Abstract: | Summary. Development of inhibitors is a severe complication of haemophilia posing many management challenges. While a long-term goal in inhibitor patients is eradication of inhibitors through immune tolerance induction, bypassing agents such as recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrate (aPCC) are essential for control of bleeding episodes. Paediatric patients with haemophilia and inhibitors are at particular risk of recurrent haemarthroses, and management of these patients should seek to avoid joint damage and support the child's full social and physical development. Current options for management of bleeding complications include on-demand treatment of acute bleeding episodes, secondary prophylaxis to avoid recurrent bleeds and surgery to treat affected joints. There is also a rationale for adopting prophylactic approaches to prevent bleeding in inhibitor patients, allowing this group similar opportunities for protection against arthropathy development as are given to non-inhibitor patients. This paper, based on a roundtable meeting of haematology experts at the first Zürich Haemophilia Forum in May 2008, reviews the current evidence supporting more intense and prophylactic approaches to manage bleeding risk in paediatric haemophilia patients with inhibitors, and highlights the need for investigations of primary prophylaxis in this vulnerable patient group, to support best long-term outcome. |
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| Keywords: | activated prothrombin complex concentrate haemophilia inhibitors paediatric prophylaxis recombinant activated factor VII |
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