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The German sickle cell disease registry reveals a surprising risk of acute splenic sequestration and an increased transfusion requirement in patients with compound heterozygous sickle cell disease HbS/β-thalassaemia and no or low HbA expression.
Allard P, Tagliaferri L, Weru V, Cario H, Lobitz S, Grosse R, Bleeke M, Oevermann L, Hakimeh D, Jarisch A, Kopp-Schneider A, Kulozik AE, Kunz JB; German Sickle Cell Disease Study Group; Lassay L. Allard P, et al. Eur J Haematol. 2024 Oct;113(4):501-509. doi: 10.1111/ejh.14259. Epub 2024 Jun 30. Eur J Haematol. 2024. PMID: 38946051
Gene Therapy in Transfusion-Dependent Non-β0/β0 Genotype β-Thalassemia: First Real-World Experience of Beti-cel.
Mirza A, Ritsert ML, Tao G, Thakar H, Lobitz S, Heine S, Koscher L, Dürken M, Schmitt A, Schmitt M, Pavel P, Laier S, Jakoby D, Greil J, Kunz JB, Kulozik A. Mirza A, et al. Among authors: ritsert ml. Blood Adv. 2024 Oct 17:bloodadvances.2024014104. doi: 10.1182/bloodadvances.2024014104. Online ahead of print. Blood Adv. 2024. PMID: 39418614