N. Elbert PhD

Gene therapy for beta thalassemia and sickle cell disease in Europe and the United States

SUMMARY

Following the lead of the United States (US) Food and Drug Administration (FDA), the European Medicines Agency (EMA) approved gene-editing therapy with exagamglogene autotemcel in early 2024 for the treatment of patients with transfusion-dependent β-thalassemia or severe sickle cell disease. A year earlier, the FDA granted marketing authorisation for gene therapy with lovotibeglogene autotemcel in patients with severe sickle cell disease. This article describes the results of the clinical trials on the basis of which these new treatments were authorised for marketing.

(BELG J HEMATOL 2024;15(8):313–4)

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BeQuinT: Past and future activities

In 2011, the Belgian health authorities established the Belgian Quality in Transfusion (BeQuinT) committee to improve the quality of transfusion practice and hemovigilance in Belgian Hospitals. Following the international shift from a product-centred to a patient-centred focus, the scope of BeQuinT was enlarged in 2018 by supporting the implementation of Patient Blood Management (PBM) and measuring the results through indicators. During the 38^th General Annual Meeting of the Belgian Hematology Society (BHS), anaesthesiologist Sarah Lessire (CHU UCL Namur, Namur) presented an overview of the past and future activities of BeQuinT.

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