{Reference Type}: Journal Article
{Title}: How we('ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future.
{Author}: Risitano AM;Peffault de Latour R;
{Journal}: Br J Haematol
{Volume}: 0
{Issue}: 0
{Year}: Aug 2021 5
{Factor}: 8.615
{DOI}: 10.1111/bjh.17753
{Abstract}: Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by complement-mediated intravascular haemolysis, severe thrombophilia and bone marrow failure. While for patients with bone marrow failure the treatment follows that of immune-mediated aplastic anaemia, that of classic, haemolytic PNH is based on anti-complement medication. The anti-C5 monoclonal antibody eculizumab has revolutionized treatment, resulting in control of intravascular haemolysis and thromboembolic risk, with improved long-term survival. Novel strategies of complement inhibition are emerging. New anti-C5 agents reproduce the safety and efficacy of eculizumab, with improved patient convenience. Proximal complement inhibitors have been developed to address C3-mediated extra-vascular haemolysis and seem to improve haematological response.