The FDA has approved leniolisib (Joenja) for treating activated phosphoinositide 3-kinase delta syndrome in pediatric patients aged 4 to 11 years.
Leniolisib was initially approved for patients aged 12 years and older in 2023, targeting a rare genetic disorder affecting the immune system.
APDS can cause recurrent infections and enlargement of lymph nodes, tonsils, and spleen, potentially leading to airway and gastrointestinal obstruction.
Efficacy for older patients was demonstrated in a 12-week study showing reduced lymph node size and improved naïve B-cell counts with leniolisib.
Common adverse effects in younger patients include abdominal pain, respiratory infections, diarrhea, and headache; leniolisib is contraindicated in severe liver impairment.
Objective: To inform about the FDA approval of floretyrosine F 18 (Pixclara) for PET imaging in glioma. Approach: FDA Approval: The FDA approved floretyrosine F
Objective: To assess the role of carotid artery stenting (CAS) in patients with severe asymptomatic carotid stenosis and its effectiveness alongside intensive m
CREST-2 provides evidence on carotid artery stenting (CAS) for severe asymptomatic carotid stenosis, emphasizing operator experience and patient selection.
The study included two trials comparing CAS plus intensive medical management to medical management alone, showing lower primary outcome rates for stenting.
Operator credentialing in CREST-2 was selective, with only 9% approved on first review, highlighting the importance of training and recent experience.
Standardized techniques in CAS included dual antiplatelet therapy and embolic protection, reflecting advancements in procedural safety and outcomes.
CREST-2 findings support individualized treatment pathways, balancing medical therapy and revascularization based on patient-specific factors.
Objective: To investigate the current practice of HPV-specific testing in oropharyngeal squamous cell carcinoma among head and neck pathologists in the UK and I
A survey of UK and Ireland head and neck pathologists revealed limited in-house access to HPV-specific testing for oropharyngeal squamous cell carcinoma.
While 91% of respondents had access to HPV-specific testing, only 25% had it available in-house, leading to longer turnaround times.
The median turnaround time for HPV-specific testing was reported as 14 days, compared to 2 days for P16 immunohistochemistry.
Most pathologists requested HPV-specific testing based on clinical suspicion, with 36% reporting this practice.
Pathologists indicated that HPV-specific testing improves diagnostic accuracy, but cost and turnaround times are significant barriers.
Objective: To discuss the resurgence of measles in the U.S. linked to declining vaccination rates. Approach: Vaccination Coverage: The increase in measles cases
This systematic review and meta-analysis evaluated the impact of prolonged inhaled corticosteroid use on growth in children with asthma.
Thirteen studies met the inclusion criteria, with six providing data for quantitative analysis on linear growth velocity.
The primary analysis showed no significant reduction in linear growth velocity with ICS treatment, but sensitivity analysis indicated a potential small decrease.
Among school-age children, a reduction in growth velocity was observed, while evidence on adult height was limited and suggested a small decrease.
The findings warrant cautious interpretation, considering the established benefits of inhaled corticosteroids in asthma management.
The study examined the relationship between the neutrophil percentage-to-albumin ratio (NPAR) and 90-day mortality in patients with acute-on-chronic liver failure (ACLF).
Among 104 patients, 29 (27.9%) died within 90 days, with NPAR significantly higher in the non-survivor group compared to survivors (P < 0.001).
Log-transformed NPAR was associated with mortality in univariate analysis, showing an odds ratio of 2.67 for each 1-unit increase (P = 0.041).
After adjusting for the MELD score, the association between NPAR and 90-day mortality was no longer statistically significant (adjusted OR: 1.66, P = 0.484).
The study suggests that larger studies are needed to evaluate whether NPAR provides prognostic information beyond established severity scores.
Objective: To propose a multi-agent framework that enhances the quality of AI-generated medical assessment questions by mimicking established collaborative item
The study evaluated small molecules targeting the YAP–TEAD interaction in mesothelioma cell models with altered Hippo pathway activity.
Computational screening of approximately 917,000 compounds identified candidates for experimental assessment of their antiproliferative effects.
The validated TEAD inhibitor K-975 showed dose-dependent antiproliferative effects in some mesothelioma cell lines, while cisplatin decreased viability in all.
None of the computationally nominated compounds demonstrated measurable antiproliferative activity at the tested concentrations.
The findings highlight a limited concordance between computational nominations and actual cell-based activity in targeting the YAP–TEAD interface.