European principles of inhibitor management in patients with haemophilia

P L F Giangrande,B O’Mahony,A Batorova,C Hermans,M Bedford,P De Kleijn,K Jansone,J Blatný

doi:10.1186/s13023-018-0800-z

Abstract

BackgroundIn spite of recent major advances in the understanding and treatment of inhibitor development in patients with haemophilia, multidisciplinary management of many of these patients remains suboptimal and highly heterogenous across Europe.MethodsFollowing a series of multidisciplinary meetings and a review of the literature, the European haemophilia community of health professionals and patients jointly defined practical optimum standards for ensuring and harmonizing treatment and care for patients with an inhibitor.ResultsTen complementary principles for the management of inhibitors in haemophilia have been developed, emphasizing the importance and benefits of a centralized, multidisciplinary, expert and holistic approach.ConclusionsThis document will serve as a benchmark to improve the multidisciplinary and practical management of patients with inhibitor. Implementation and adherence to each of these principles should have a major positive impact on the management and outcomes of patients developing an inhibitor.

Highlights

In spite of recent major advances in the understanding and treatment of inhibitor development in patients with haemophilia, multidisciplinary management of many of these patients remains suboptimal and highly heterogenous across Europe
Haemophilia A and B are inherited bleeding disorders caused by deficiencies of coagulation factors VIII (FVIII) and IX (FIX) respectively [1]
Inhibitor development is an immunological reaction of the body to exogenous factor VIII or IX

Summary

Introduction

In spite of recent major advances in the understanding and treatment of inhibitor development in patients with haemophilia, multidisciplinary management of many of these patients remains suboptimal and highly heterogenous across Europe. Haemophilia A and B are inherited bleeding disorders caused by deficiencies of coagulation factors VIII (FVIII) and IX (FIX) respectively [1]. Inhibitor development is an immunological reaction of the body to exogenous factor VIII or IX. It is the most serious complication in haemophilia, following the effective elimination of the risk of transmission of blood borne pathogens such as HIV or hepatitis C. The presence of circulating neutralizing antibodies against FVIII or FIX inactivates infused coagulation factor proteins, impairing their clinical efficacy [2]. Inhibitors are classified into low (5BU)

Methods

Discussion

Conclusion