Daratumumab in Pediatric Relapsed/Refractory Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma: The DELPHINUS Study Academic Background Acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LL) are among the most common malignancies in children. Although the cure rate for newly diagnosed ALL and LL patients is relatively high, 10% to ...

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 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...

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 ...

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 ...

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 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...

Academic Background and Problem Acute lymphoblastic leukemia (ALL) is a malignant hematologic disease, particularly challenging to treat in older patients, with limited treatment options and poor outcomes. Traditional chemotherapy regimens yield long-term survival rates of only 20%-30% in elderly patients. Therefore, finding more effective and bett...

Multi-omics Analysis Reveals Predictors of Survival in African American AML Patients Background and Objectives Acute Myeloid Leukemia (AML) is a hematologic malignancy characterized by clonal expansion of myeloid progenitor cells driven by genomic alterations. Despite advances in genomic profiling, studies focusing on diverse ancestral populations ...

Selective Advantage of Clonal Hematopoietic Mutant Stem Cells in Aging and Inflammatory Responses Background and Research Motivation Clonal Hematopoiesis (CH) is an aging-related phenomenon in the hematopoietic system, where hematopoietic stem cells (HSCs) acquire specific somatic mutations and undergo expansion, causing a particular mutated cell l...