Preventing bleeds by treatment: new era for haemophilia changing the paradigm

Abstract

Introduction: Coagulation products have allowed patients with severe haemophilia to lead a normal life. This is, however, only true for patients who received an early diagnosis and could start with primary prophylaxis. The absence of a positive family history for haemophilia, in the majority of children with severe haemophilia, postpones the age that treatment can be started. This makes general awareness of the clinical presentation important and a proper diagnosis a prerequisite for progress. The long delay between joint bleeding and overt arthropathy has been an important factor in the delay of implementation of primary prophylaxis. After the development of guidelines on 'how to treat', implementation of the advised practice is needed. Data collection of current treatment regimens in haemophilia centres will support the further optimization of the care for persons with haemophilia and further optimize treatment guidelines. Episodic ('on demand') therapy as a treatment strategy for severe haemophilia needs reconsideration.

Conclusion: In an era where clotting factor concentrates are abundant and gene therapy a reality, all patients with severe haemophilia should be offered a strategy of bleeding prevention.

Keywords: episodic treatment; on demand treatment; prophylaxis; severe haemophilia.