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On January 4, 2023, briquilimab, a monoclonal antibody targeting the CD117 receptor, was granted orphan drug designation by the European Commission, as a conditioning treatment prior to hematopoietic stem cell transplantation (HSCT).1 Briquilimab received orphan drug designation from the U.S Food and Drug Administration (FDA) in June 2022.1
As previously reported by the AML Hub, briquilimab is currently being investigated as a conditioning treatment prior to HSCT in patients with acute myeloid leukemia and myelodysplastic syndromes (MDS).1 Briquilimab was found to have a tolerable safety profile, with full donor chimerism in all 24 patients with acute myeloid leukemia or MDS. There were no reports of treatment-related significant adverse events, classical acute Grade II–IV graft-versus-host disease, or transplant mortality within 100 days. Briquilimab is also being assessed as a primary therapy for patients with low-risk MDS and as a conditioning therapy prior to HSCT for patients with severe combined immunodeficiency.1
Editorial theme | Genetic pathogenesis of MDS: Translating molecular advances into better diagnosis and prognosis of CCUS and MDS
The last 30 years of advances in molecular biology and high-throughput techniques have significantly impacted the...
The role of the Nlrp3 inflammasome in hematological pathologies
The Nlrp3 inflammasome has recently gained a lot of attention due to its reported involvement in various hematological pathologies. Mariusz Ratajczak and colleagues published in...
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