Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD) are severe monogenic diseases with potentially life-threatening manifestations. In recent years, the FDA approved several disease-modifying therapies for these patients, including like L-glutamine, voxelotor, crizanlizumab and luspatercept. During ASH 2020, updated results for some of these agents were presented in addition to data with other disease-modifying agents, including the E-selectin inhibitor rivipansel and the P-selectin inhibitor crizanlizumab. With respect to TDT, ASH 2020 featured the presentation of quality-of-life data with luspatercept in the pivotal BELIEVE trial. In the second part of this congress highlights article the focus shifts to gene editing techniques, which potentially hold the promise for a cure for patients with these genetic disorders.
(BELG J HEMATOL 2021;12(1):29-32)