EHA 2023 | Highlights Sikkelcelziekte & Thalassemie

Prof. dr. Bart Biemond volgde op de EHA 2023 met grote interesse onder andere het nieuws over Sikkelcelziekte en Thalassemie. In deze video geeft hij zijn persoonlijke hoogtepunten die op het congres in Frankfurt aan de orde kwamen. 

Hieronder vindt u de abstracts die hij bespreekt:

Sikkelcelziekte 

• Effect of Voxelotor on cerebral perfusion and cerebral oxygen metabolism in adult patients with sickle cell disease (The Coverage Study)

• Male patients with SCD have a higher risk of cerebrovascular disease, increased inflammatory markers, and poorer response to hydroxyurea

• Interim analysis of a phase 2 trial to assess the efficacy and safety of crizanlizumab in sickle cell disease patients with priapism (SPARTAN)

Thalassemie

• Transfusion independence after exagamglogene autotemcel in patients with transfusion-dependent beta-thalassemia (CLIMB-THAL-111)

• An updated follow-up of BRL-101, CRISPR-CAS9-MEDIATED gene editing of the BCL11A enhancer for transfusion-dependent betha-thalasse

• Safety and efficacy of RM-001 in patients with transfusion-dependent B-Thalassemia: early results from the ongoing of autologous HBG1/2 promotor-modified CD34+ hematopoietic stem and progenitor cells.

Bekijk hier het volledige overzicht van onze EHA 2023 journaals en interviews.