title: Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician's guide.
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.
authors.affiliation: Leeds Teaching Hospitals NHS Trust, St James Hospital, Beckett Street, Leeds LS1 3EX, UK.
authors.forename: Morag
authors.initials: M
authors.lastname: Griffin
authors.affiliation: Leeds Teaching Hospitals NHS Trust, Leeds, UK.
authors.forename: Talha
authors.initials: T
authors.lastname: Munir
keywords: haemoglobinuria; haemolytic anaemia; paroxysmal nocturnal; thrombosis; ultra-orphan disease