The recommended indication is for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria
with haemolysis with clinical symptoms indicative of high disease activity, and also for adult patients who are clinically stable after having been treated with Soliris (eculizumab) for at least the past six months.
Whilst I am aware its use is indicated in atypical haemolytic uraemic syndrome, I understood it is a monoclonal antibody targeted against the C5 component of the complement system, and its action on complement is the mechanism of action in this and other disorders, such as paroxysmal nocturnal haemoglobinuria
of Cases % Megaloblastic anaemia 18 23 Acute leukaemia- lymphoblastic 9 11 Myelodysplastic syndrome 6 8 Lymphoma 6 8 Hypoplastic marrow 6 8 Hypersplenism 3 4 Acute leukaemia- myeloid 3 4 Multiple myeloma 3 4 Aplastic anaemia 3 4 Plasmacytosis 2 2 Chronic granulomatous lesion 2 2 Myelofibrosis 2 2 Leukaemia/Lymphoma 1 1 Macrophage activation syndrome 1 1 Marrow necrosis 1 1 Paroxysmal nocturnal haemoglobinuria
1 1 No definite opinion 13 16 Table 2.
The product is claimed to be a highly innovative, longer-acting anti-C5 antibody that inhibits terminal complement, which is being evaluated to treat patients with paroxysmal nocturnal haemoglobinuria
Richards et al., "The management of pregnancy in paroxysmal nocturnal haemoglobinuria
on long term eculizumab," British Journal of Haematology, vol.
Paroxysmal nocturnal haemoglobinuria
: clinical manifestations, haematology, and nature of the disease.
John Duggan, 33, was initially refused Soliris by HSE bosses to fight Paroxysmal Nocturnal Haemoglobinuria
with which he was diagnosed in 2010.
The event at Banney Royd raised cash for treatment of Paroxysmal Nocturnal Haemoglobinuria
(PNH) at St James's Hospital, Leeds.
Fujimoto et al., "Establishment of a human T-cell line with deficient surface expression of glycosylphosphatidylinositol (GPI)-anchored proteins from a patient with paroxysmal nocturnal haemoglobinuria
," The British Journal of Haematology, vol.
 Hypercoagulation disorders such as systemic lupus erythematosus, antiphospholipid syndrome, the presence of factor V Leiden, paroxysmal nocturnal haemoglobinuria
, hyperhomocysteinaemia, protein C and S deficiency, and heparin-induced thrombocytopenia are also all reported as risk factors for ovarian vein thrombosis.
Flow cytometry may be considered to investigate for an abnormal clone in the case of paroxysmal nocturnal haemoglobinuria
and may be used on bone marrow samples to further evaluate the cells.