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Published on: September 5, 2016
Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician's guide
1Leeds Teaching Hospitals NHS Trust, St James Hospital, Beckett Street, Leeds LS1 3EX, UK.
Abstract:
Paroxysmal nocturnal haemoglobinuria (PNH), an ultra-orphan disease with a prevalence of 15.9 per million in Europe, is a life-threatening disorder, characterized by haemolysis, bone marrow failure and thrombosis. Patients with PNH prior to the availability of eculizumab had a median survival of between 10 and 22 years, with thrombosis accounting for 22-67% of deaths. 29-44% of patients had at least one thrombosis. This paper provides a clinician's guide to the diagnosis, management and complications of PNH, with an emphasis on thrombosis.
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