Background: Pain is a prevalent, frequent, and often persistent symptom among children with acute lymphoblastic leukemia (ALL). Despite its high prevalence, pain has remained understudied, and no evidence-based recommendations exist for how best to assess and treat pain in this population. Without proper assessment, clinical efforts to improve pain management in pediatric ALL will be ineffective.
View Article and Find Full Text PDFThe Engraft Learning Health Network (LHN) aims to improve outcomes for patients undergoing transplant and cellular therapy (TCT) through a collaborative, data-driven approach. Engraft brings together diverse stakeholders, including clinicians, patients, caregivers, and institutions, to standardize best practices and accelerate the dissemination of innovations in TCT care. By establishing a multicenter, real-world clinical registry focused on rapid-cycle quality improvement (QI) and implementation research, Engraft seeks to reduce variability in clinical practice to improve TCT outcomes across centers.
View Article and Find Full Text PDFStem Cell Res Ther
December 2024
Background: Sickle cell disease (SCD) and β-thalassemia patients with elevated gamma globin (HBG1/G2) levels exhibit mild or no symptoms. To recapitulate this natural phenomenon, the most coveted gene therapy approach is to edit the regulatory sequences of HBG1/G2 to reactivate them. By editing more than one regulatory sequence in the HBG promoter, the production of fetal hemoglobin (HbF) can be significantly increased.
View Article and Find Full Text PDFPol II-mediated transcription is essential for eukaryotic life. While loss of transcription is thought to be universally lethal, the associated mechanisms promoting cell death are not yet known. Here, we show that death following loss of Pol II is not caused by dysregulated gene expression.
View Article and Find Full Text PDFBackground: Standard treatment for symptomatic chronic subdural hematoma (CSDH) is a burr-hole evacuation. However, in patients in whom surgical evacuation carries a very high risk, we do not have an established practice guideline.
Objective: To analyze the outcome of symptomatic CSDH treated only by tranexamic acid.