Hsieh, Matthew M.Bonner, MelissaPierciey, Francis John JrUchida, NaoyaRottman, JamesDemopoulos, LauraSchmidt, ManfredKanter, JulieWalters, Mark C.Thompson, Alexis A.
...
Ability to accurately attribute adverse events post–gene therapy is required to describe the benefit-risk of these novel treatments. A SCD patient developed myelodysplastic syndrome post-LentiGlobin treatment; we show how insertional oncogenesis was excluded as the cause.
Monitoring factor VIII (FVIII) activity has traditionally been complicated by discrepancies between assays for the various sorts of FVIII molecules. The advent of novel nonfactor therapies (emicizumab, fitusiran, and anti-tissue factor pathway inhibitor antibodies) in hemophilia A poses a new level of difficulty on the laboratory monitoring of thes...
Since November 2018, Blood Advances has published American Society of Hematology (ASH) clinical practice guidelines on venous thromboembolism, immune thrombocytopenia, and sickle cell disease. More ASH guidelines on these and other topics are forthcoming. These guidelines have been developed using consistent processes, methods, terminology, and pre...
Quirino-Teixeira, Anna CecílliaRozini, Stephane VicenteBarbosa-Lima, GiselleCoelho, Diego RodriguesCarneiro, Pedro HenriqueMohana-Borges, RonaldoBozza, Patrícia T.Hottz, Eugenio D.
