Month: June 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:  https://www.thalassemia.org/wp-content/uploads/2020/06/COVID-19-and-Thalassaemia-A-Position-Statement-of-the-Thalassaemia-Int….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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Agios Pharmaceuticals Provides Update on Phase 2 Study of Mitapivant in Non-Transfusion-Dependent Thalassemia

June 12, 2020 – Agios Pharmaceuticals has provided an update on the preliminary findings from their Phase 2 study of mitapivat in non-transfusion-dependent thalassemia at the European Hematology Association (EHA) virtual medical congress. Mitapivat activates the pyruvate kinase-R enzyme, which improves red blood cell energy metabolism. Mitapivat was shown to improve ineffective erythropoiesis and anemia […]

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Registration Is Open for CAF Online Mini-Conference

June 11, 2020 – Registration is now open for CAF’s 2020 Online Mini-Conference for Patients and Families. The online event will be held Saturday, July 11, 2020 from 12:00 noon to 6:00 pm Eastern time, 9:00 am – 3:00 pm Pacific. There will be several breaks between sessions. An agenda is listed on the registration […]

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World Blood Donor Day – June 14

June 9, 2020 – World Blood Donor Day is commemorated each June 14th in order to celebrate and thank blood donors, and to encourage more people to begin donating blood. This special day also helps to raise public awareness about the ongoing need for safe blood and lets people know how they can contribute. Many […]

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ASH Launches Research Collaborative COVID-19 Registry

June 5, 2020 – The American Society of Hematology (ASH) recently created a Research Collaborative (RC) COVID-19 Registry to collect de-identified data on patients with a positive COVID-19 diagnosis and a hematologic malignancy. This global Registry has now been expanded to accept data for COVID-19 patients with ANY hematologic condition, including thalassemia. Healthcare providers can […]

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