Benign disorders & Topics-in-Focus Hemaglobinopathies


Benign disorders

This year at the 24th EHA Congress, a lot of attention was paid to erythrocytes, iron and hepcidin in the various educational and scientific sessions. Prominently positioned were the presentations from Simón Méndez-Ferrer and Elizabeta Nemeth in Plenary Session I, and the oral presentation from Antonella Nai in the Presidential Symposium. Together with other speakers, they provided overviews on new developments related to the functioning of the normal and malignant bone marrow niche, iron homeostasis, identification of targets for treatment of aplastic anemia and experimental models for beta-thalassemia.

Topics-in-Focus Hemoglobinopathies (Sickle Cell Disease)

Since the kick-off of the Topics-in-Focus program in 2018, EHA and its partners have been furthering the programs related with sickle cell disease (SCD). Most notably, a web portal -- sicklecellnet.org  -- has been launched at EHA24. This will be a central point of information for all those interested in this disease.

At the EHA congress a number of presentations discussed several aspects of SCD, of which you can find a selection below.

Links to the library and multimedia

Click on the blocks below to read the full stories, view abstracts, presentations, webcasts and expert interviews.

Simón Méndez-Ferrer | Neuronal regulation of normal and malignant BM niches

Elizabeta Nemeth | Iron homeostasis in host defense and inflammation

Neal Young | Pathophysiology and therapeutic targets in acquired aplastic anemia: Inferences from immunosuppression and stem cell stimulation

Maria-Domenica Cappellini | LUSPATERCEPT increases fetal HEMOGLOBIN (HBF) levels in adult Β-thalassemia patients who require Red Blood Cell (RBC) transfusions

Antonella Nai | TFR2-HAPLOINSUFFICIENCY enhances the beneficial effect of TMPRSS6-ANTISENSE OLIGONUCLEOTIDE treatment in beta-thalassemia mice

Diego Moretti | Novel approaches to oral iron treatment

Kofi Anie | Impact of social stigma on coping with SCD

Jo Howard | Results from the randomized PLACEBO-CONTROLLED Phase 3 HOPE trial of VOXELOTOR in adults and adolescents with sickle cell disease

Franco Locatelli | Safety and efficacy of LENTIGLOBIN gene therapy in patients with transfusion-dependent B-thalassaemia and Non-B0/B0 genotypes in the Phase 3 NORTHSTAR-2 study

Wei Wang | A fully human bispecific antibody functionally rescues factor VIII deficiency EX VIVO