Year: 2020

TIF Position Statement on COVID-19 and Thalassemia Published in the European Journal of Hematology

June 25, 2020 – The Thalassaemia International Federation’s (TIF’s) position statement on COVID-19 and Thalassemia has just been published in the European Journal of Haematology. You can access the position statement here:….pdf The article notes that “The optimization of blood use will help safeguard blood supplies during the pandemic…One particular aspect of optimizing blood […]

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Study Shows Outcomes of COVID-19 Infection in Italian Patients with Thalassemia

June 16, 2020 – Dr. Maria-Domenica Cappellini of the University of Milan, Italy, recently presented the following data showing the outcomes of COVID-19 infection in a cohort of 11 patients with thalassemia from Northern Italy.  These 11 Italian thalassemia patients experienced relatively mild to moderate COVID-19 disease. All the patients had thalassemia-related comorbidities. 53.5% of […]

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A Majority of Patients Achieve Transfusion Independence in Phase 3 Trials of LentiGlobin

June 12, 2020 – Bluebird bio has reported the findings from its Phase 3 clinical trial program of betibeglogene autotemcel (beti-cel; formerly LentiGlobin™ for β-thalassemia). These show that pediatric, adolescent and adult patients with a range of genotypes of transfusion-dependent β-thalassemia are able to achieve and maintain transfusion independence with hemoglobin (Hgb) levels that are near-normal (≥10.5 […]

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Two Patients Become Transfusion Independent After Gene Editing Therapy

June 12, 2020 – CRISPR Therapeutics and Vertex Pharmaceuticals have announced early results from their CLIMB Thal-111 Study in which transfusion-dependent beta thalassemia patients are treated with a gene editing therapy called CTX001. CTX001 is a therapy that involves CRISPR/Cas9 gene editing of a patient’s own hematopoietic stem cells (HSCs) in order to reactivate fetal hemoglobin […]

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