Hairy cell leukemia (HCL) is a chronic B-cell lymphoproliferative disorder that accounts for 2% of all leukemia. Recent identification of the recurrent V600E BRAF mutation in a majority of HCL patients has led some teams to evaluate the clinical potential of vemurafenib, a BRAF V600 specific inhibitor in a limited number of refractory HCL patients. Recently, we published the case of an HCL patient successfully treated with a low dose of vemurafenib. Eight months after the ending of treatment this patient relapsed. We present here the successful retreatment of this patient with a second line of vemurafenib. Our data suggest for the first time that vemurafenib at the dose of 240 mg once a day could be sufficient to maintain a complete hematological remission after an initial induction treatment with low-dose vemurafenib (2 × 240 mg) daily without inducing major toxicity.
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http://dx.doi.org/10.18632/oncoscience.111 | DOI Listing |
Int J Mol Sci
January 2025
Clinica Medica "Augusto Murri", Department of Precision and Regenerative Medicine and Ionian Area (DiMePrev-J), University of Bari Aldo Moro, 70124 Bari, Italy.
The integrity of esophageal epithelial cells in patients with gastroesophageal reflux disease (GERD) or GERD-like symptoms is the first mechanism of protection to decrease the sensitivity to gastric reflux and heartburn symptoms. We investigated the protective effects of Poliprotect (PPRO), a CE-marked medical device, on esophageal epithelial integrity using in vitro and ex vivo models. In vitro, the protective effects of PPRO were tested on Caco-2 cells.
View Article and Find Full Text PDFEndocr Pract
January 2025
University of Colorado Anschutz Medical Campus, Barbara Davis Center for Diabetes, 1775 Aurora Court, Aurora, CO 80045, USA. Electronic address:
Objectives: Type 1 diabetes (T1D) in pregnancy is challenging. This study explores how assisted hybrid closed-loop (HCL) therapy versus sensor-augmented pump therapy (SAPT) impacts quality of life (QoL) in pregnancy.
Methods: We interviewed 22 of 24 participants randomized to HCL therapy or SAPT in the Pregnancy Intervention with a Closed-Loop System study.
Cell Rep Med
January 2025
Department of Lymphoma and Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX, USA; Department of Experimental Therapeutics, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. Electronic address:
Multiple myeloma is a clonal plasma cell (PC) dyscrasia that arises from precursors and has been studied utilizing approaches focused on CD138 cells. By combining single-cell RNA sequencing (scRNA-seq) with scB-cell receptor sequencing (scBCR-seq), we differentiate monoclonal/neoplastic from polyclonal/normal PCs and find more dysregulated genes, especially in precursor patients, than we would have by analyzing bulk PCs. To determine whether this approach can identify oncogenes that contribute to disease pathobiology, mitotic arrest deficient-2 like-1 (MAD2L1) and S-adenosylmethionine synthase isoform type-2 (MAT2A) are validated as targets with drug-like molecules that suppress myeloma growth in preclinical models.
View Article and Find Full Text PDFSci Rep
January 2025
Stroke Unit, Department of Neurology, Universitat de Lleida, IRBLleida, Hospital Universitari Arnau de Vilanova de Lleida, Avda Rovira Roure, 80, Lleida, 25198, Spain.
Evaluating scales to detect large vessel occlusion (LVO) could aid in considering early referrals to a thrombectomy-capable center in the prehospital stroke code setting. Nevertheless, they entail a significant number of false positives, corresponding to intracranial hemorrhages (ICH). Our study aims to identify easily collectible variables for the development of a scale to differentiate patients with ICH from LVO.
View Article and Find Full Text PDFAm J Cardiovasc Dis
December 2024
Prince Sultan Cardiac Centre, Heart Function Unit, Department of Adult Cardiology Riyadh, Saudi Arabia.
Background: Transthyretin cardiac amyloidosis (ATTRCA) is a prevalent disease, and it can be associated with heart failure (HF), left ventricle hypertrophy (LVH), atrial fibrillation (AF), and aortic stenosis (AS).
Aim: The study aims to detect the prevalence of ATTRCA in the symptomatic AS population.
Method: A single-center prospective study screening for ATTRCA in patients diagnosed with symptomatic severe AS undergoing aortic valve (AV) intervention.
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