The effect of a provisional diagnosis on intern diagnostic reasoning: a mixed methods study.

Objectives: Competency in diagnostic reasoning is integral to medical training and patient safety. Situativity theory highlights the importance of contextual factors on learning and performance, such as being informed of a provisional diagnosis prior to a patient encounter. This study aims to determine how being informed of a provisional diagnosis affects an intern's approach to diagnostic reasoning.

Clonazepam repurposing in patients through conventional RCT and N-of-1 trials: an experimental strategy for orphan disease development.

Background: Clinical trials for rare disorders have unique challenges due to low prevalence, patient phenotype variability and high expectations. These challenges are highlighted by our study on clonazepam in patients, a common cause of intellectual disability. Previous studies on Arid1b-haploinsufficient mice showed positive effects of clonazepam on various cognitive aspects.

Phase 1b/2 study of orally administered pexidartinib in combination with radiation therapy and temozolomide in patients with newly diagnosed glioblastoma.

Background: Glioblastoma (GBM) has a median survival of <2 years. Pexidartinib (PLX3397) is a small-molecule inhibitor of CSF1R, KIT, and oncogenic FTL3, which are implicated in GBM treatment resistance. Results from glioma models indicate that combining radiation therapy (RT) and pexidartinib reduces radiation resistance.

BCMA-directed CAR T-cell Therapy in patients with multiple myeloma and CNS involvement.

We investigated BCMA-directed CART in patients with relapsed or refractory multiple myeloma (RRMM) and CNS involvement. Ten patients who received either ide-cel (n=6) or cilta-cel (n=4) were included in this analysis. Patients had brain/cranial nerve and/or spinal cord involvement/leptomeningeal disease evident on either MRI (100%) and/or CSF (40%).

First-trimester screening and small for gestational age in twin pregnancies: a single center cohort study.

Objective: This study aimed to investigate the association between maternal factors and first-trimester biophysical and biochemical markers with small for gestational age (SGA) neonates in twin pregnancies (TwPs).

Methods: Single-center retrospective cohort study of TwPs followed from January 2010 to December 2022 at a tertiary perinatal center, Portugal. Maternal and pregnancy characteristics, mean arterial pressure, pregnancy-associated plasma protein-A (PAPP-A), β-human chorionic gonadotropin (β-HCG), and uterine artery pulsatility index (UtA-PI) were analyzed.