August 7, 2020 – Bristol Myers Squibb (BMS) and Acceleron have developed a brochure called “Your Guide to Starting REBLOZYL”. This brochure provides background information about ineffective erythropoiesis in thalassemia, an overview of the clinical trial findings for REBLOZYL/luspatercept in adults with transfusion-dependent beta thalassemia, a listing of the benefits and side effects of REBLOZYL […]
July 16, 2020 – The University of California San Francisco (UCSF) has been conducting a clinical trial of in utero hematopoietic stem cell transplantation for fetuses that have been diagnosed with Alpha Thalassemia Major. They are now considering the possibility of expanding this trial to also include fetuses which have been diagnosed with Beta Thalassemia […]
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 […]
June 22, 2020 – If you were unable to join us for last Thursday’s webinar entitled “Overview of Novel Thalassemia Treatments” by Dr. Sujit Sheth of the New York Presbyterian Hospital/Weill Cornell Medical Center, the archived webinar can now be accessed online at: https://www.cdc.gov/ncbddd/blooddisorders/webinar.html
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 […]
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 […]
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 […]
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 […]
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 […]
