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Review
. 2017 Mar;8(3):119-126.
doi: 10.1177/2040620716681748. Epub 2016 Dec 21.

Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician's guide

Morag Griffin  1 , Talha Munir  2
Affiliations
Review

Management of thrombosis in paroxysmal nocturnal hemoglobinuria: a clinician's guide

Morag Griffin et al. Ther Adv Hematol. 2017 Mar.

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.

Keywords: haemoglobinuria; haemolytic anaemia; paroxysmal nocturnal; thrombosis; ultra-orphan disease.

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Conflict of interest statement

Conflict of interest statement: Dr Griffin has received honoraria, travel and accommodation support from Alexion Pharmaceuticals. Dr Munir has received honoraria, and travel/accommodation support from Alexion Pharmaceuticals

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