Publications by authors named "P Pavel"

Article Synopsis
  • Gene addition and editing therapies for β-thalassemia, particularly betibeglogene autotemcel (beti-cel), show promise as potentially curative options, with real-world studies demonstrating their effectiveness and safety.* -
  • Out of 15 patients evaluated for treatment with beti-cel, 8 were treated post-busulfan conditioning, achieving transfusion independence within 8 to 59 days, while experiencing some acute toxicity typical of the conditioning regimen.* -
  • Monitoring of patients revealed several unexpected side effects, including endocrine dysfunction, posttreatment depression and anxiety in one patient, and fatigue impacting quality of life in another, underscoring the need for careful management of these complications.*
View Article and Find Full Text PDF

Exposure to toxic elements in drinking water, such as arsenic (As) and fluoride (F), starts at gestation and has been associated with memory and learning deficits in children. Studies in which rodents underwent mechanistic single exposure to As or F showed that the neurotoxic effects are associated with their capacity to disrupt redox balance, mainly by diminishing glutathione (GSH) levels, altering glutamate disposal, and altering glutamate receptor expression, which disrupts synaptic transmission. Elevated levels of As and F are common in groundwater worldwide.

View Article and Find Full Text PDF

Background: Third-generation chimeric antigen receptor (CAR)-engineered T cells (CARTs) might improve clinical outcome of patients with B cell malignancies. This is the first report on a third-generation CART dose-escalating, phase-1/2 investigator-initiated trial treating adult patients with refractory and/or relapsed (r/r) acute lymphoblastic leukemia (ALL).

Methods: Thirteen patients were treated with escalating doses of CD19-directed CARTs between 1 × 10 and 50 × 10 CARTs/m.

View Article and Find Full Text PDF

Clonal hematopoiesis (CH) is an age-related condition driven by stem and progenitor cells harboring recurrent mutations linked to myeloid neoplasms. Currently, potential effects on hematopoiesis, stem cell function and regenerative potential under stress conditions are unknown. We performed targeted DNA sequencing of 457 hematopoietic stem cell grafts collected for autologous stem cell transplantation (ASCT) in myeloma patients and correlated our findings with high-dimensional longitudinal clinical and laboratory data (26,510 data points for blood cell counts/serum values in 25 days around transplantation).

View Article and Find Full Text PDF

Atopic dermatitis (AD) is a complex disease characterized by chronic recurring eczema and pruritus. In addition, patients with AD display increased cutaneous and systemic levels of oxidative damage markers, whose source remains elusive. In this study, we investigated oxidative and mitochondrial stress in AD epidermis.

View Article and Find Full Text PDF