Kimberly Rath, PharmD, is a freelance health writer who previously worked as a clinical pharmacist with Elevance Health for a decade. She has also worked for Amerigroup and Sentara Group.
AAV5 Gene Therapy for Severe Hemophilia A Successfully Enables FVIII Production
The phase 3 GENEr8-1 study demonstrated that valoctocogene roxaparvovec, an AAV5-vectored gene therapy, effectively enabled endogenous FVIII production in adults with severe hemophilia A without developing clinically meaningful FVIII inhibitors.
Early BP Reduction Shows No Benefit in Acute Stroke, but Outcomes May Vary by Stroke Type
A study found that prehospital blood pressure (BP) reduction had no impact on functional outcomes among patients with undifferentiated acute stroke, but that it may decrease the odds of poor outcomes following hemorrhagic stroke.
Decitabine, Venetoclax Combo Boosts Survival in Patients With AML and MDS
Study findings demonstrate that combining decitabine with venetoclax enhances molecular response rates in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS), correlating strongly with improved clinical outcomes and overall survival.
Atypical Marker Expression in T-Cell Acute Lymphoblastic Leukemia/Lymphoma
Two unique case reports highlight the diagnostic challenges and critical importance of comprehensive immunophenotyping in cases of T-cell acute lymphoblastic leukemia/lymphoma that present without typical immaturity markers but with rare γδ T-cell receptor expression.
Valoctocogene Roxaparvovec Dramatically Reduces Bleeding in Hemophilia A
New study findings demonstrate that valoctocogene roxaparvovec, a gene therapy for severe hemophilia A, significantly reduces bleeding episodes and dependency on factor VIII infusions over a 3-year period, while maintaining a favorable safety profile.
New Strategy Shows Promise for CD7-Positive Leukemia/Lymphoma
This study introduces a novel "all-in-one" strategy combining CD7 chimeric antigen receptor T-cell therapy with haploidentical hematopoietic stem-cell transplantation, showing significant potential to improve survival and reduce toxicities in patients with relapsed or refractory CD7-positive hematologic cancers.