This article is an overview of thrombosis in paroxysmal nocturnal haemoglobinuria (PNH), including its pathogenesis, disease burden, diagnosis, and treatment. Expert commentary by Associate Professor Glen Kennedy provides a clinical practice perspective on this feared and difficult-to-treat condition.
Discussion of the treatment of thrombosis in PNH includes the role of the complement inhibitor, eculizumab (Soliris®). In Australia, eculizumab is a prescription medication that is indicated and
funded for the treatment of PNH. Subsidised access to eculizumab for the treatment of PNH is available through the Life Saving Drugs Programme (LSDP), which is a supply arrangement outside of the Pharmaceutical Benefits Scheme. A patient must continually meet the LSDP funding conditions in order to be eligible to receive access to subsidised treatment for PNH through the LSDP.
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