In addition to the long list of abstracts dedicated to malignant haematology, EHA 2019 also featured several sessions dedicated to benign haematological abnormalities. Results from the Northstar clinical trial program highlighted the efficacy of LentiGlobin gene therapy in transfusion-dependant β-thalassaemia. The BELIEVE study showed that Luspatercept treatment was associated with increased HbF in patients with red blood cell (RBC) transfusion dependent (TD) β-thalassaemia in both responders and non-responders. With respect to paroxysmal nocturnal haemoglobinuria, ravulizumab (a novel C5 complement inhibitor) and ACH-4471 (an inhibitor of factorD, a highly specific serine protease that cleaves factor B) proved to be promising new drugs. Finally, long term inhibition of complement C1s with the humanized monoclonal antibody sutimlimab can be a new strategy in treating patients with cold agglutinin disease.
(BELG J HEMATOL 2019;10(5):220–3)