Effect of Oral Nintedanib vs Placebo on Epistaxis in Hereditary Hemorrhagic Telangiectasia: The EPICURE Multicenter Randomized Double-Blind Trial

Medicine, Hematology,
Hereditary Hemorrhagic Telangiectasia   Epistaxis   Anti-angiogenic   Nintedanib   Anemia   Tyrosine Kinase Inhibitors  

The Effect of Oral Nintedanib vs Placebo on Epistaxis in Hereditary Hemorrhagic Telangiectasia: The EPICURE Multicenter Randomized Double-Blind Trial Academic Background Hereditary Hemorrhagic Telangiectasia (HHT) is a rare genetic disorder characterized by vascular malformations, particularly capillary dilatations. Epistaxis (nosebleeds) is the mo...

Genetic and Pharmacological Targeting of mTORC1 in Mouse Models of Arteriovenous Malformation Expose Non-Cell Autonomous Signalling in HHT

Medicine, Hematology, Life Sciences, Cell Biology, Biochemistry, Immunology, Genetics,
Hereditary Hemorrhagic Telangiectasia   Arteriovenous Malformation   Endothelial Cells   mTORC1   Signaling Pathways   Genetic Targeting   Pharmacological Treatment  

Genetic and Pharmacological Targeting of mTORC1 in Mouse Models of Arteriovenous Malformation Expose Non-Cell Autonomous Signalling in HHT Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder caused by mutations leading to the loss of function in either Activin Receptor-Like Kinase 1 (ACVRL1) or Endoglin (ENG), which act as receptors a...

BRD4 Inhibitor Reduces Exhaustion and Blocks Terminal Differentiation in CAR-T Cells by Modulating BATF and EGR1

Medicine, Hematology, Oncology, Life Sciences, Cell Biology, Biochemistry, Immunology,
CAR-T cell exhaustion   BRD4 inhibitor   single-cell RNA sequencing   transcriptional factors   acute myeloid leukemia  

Acute myeloid leukemia (AML) is the most common type of leukemia in adults. Although most AML patients achieve complete remission after standard chemotherapy, refractory and relapsed disease remain a significant challenge. Over the past decade, immunotherapy has been widely applied in cancer treatment, particularly chimeric antigen receptor T-cell ...

Single or Double Induction with 7+3 Containing Standard or High-Dose Daunorubicin for Newly Diagnosed AML: The Randomized DAUNODouble Trial by the Study Alliance Leukemia

Medicine, Hematology, Oncology,
Acute Myeloid Leukemia   Induction Therapy   Randomized Controlled Trial   Daunorubicin   Survival Rate  

Academic Background Acute Myeloid Leukemia (AML) is a malignant blood disorder, with the core of its treatment being induction chemotherapy, typically involving a combination of cytarabine and anthracyclines (such as daunorubicin). Although this treatment regimen has been in use since the 1980s, questions regarding the optimal dose of daunorubicin ...

Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia: Efficacy and Safety

Medicine, Hematology, Oncology,
Revumenib   KMT2A-rearranged   acute leukemia   relapsed   refractory   safety   efficacy  

Academic Background Acute leukemia is a malignant hematologic disease caused by genetic mutations in hematopoietic cells, characterized by a block in differentiation during hematopoiesis and uncontrolled cellular proliferation. Among these, KMT2A gene rearrangements (KMT2A-rearranged, KMT2Ar) occur in up to 10% of acute leukemias in children and ad...

Revumenib Revises the Treatment Landscape for KMT2A-R Leukemia

Medicine, Hematology, Oncology,
Revumenib   KMT2A-R leukemia   menin inhibitors   gene expression   clinical trial   leukemia cell differentiation   drug resistance  

Revumenib Alters the Treatment Landscape for KMT2A-Rearranged Leukemia Academic Background Leukemia is a malignant disease caused by abnormalities in the hematopoietic system, with KMT2A-rearranged (KMT2A-r) leukemia being a common subtype, particularly in pediatric and adult acute myeloid leukemia (AML). Rearrangements of the KMT2A gene (lysine me...