Emergent Carotid Stenting During Thrombectomy in Tandem Occlusions Secondary to Dissection: A STOP-CAD Secondary Study.

The optimal endovascular management of cervical carotid dissection causing tandem occlusion remains uncertain. We investigated the impact of emergent carotid stenting during endovascular treatment (EVT) for acute ischemic stroke (AIS) in patients with tandem occlusion secondary to cervical carotid artery dissection. This was a secondary analysis of patients treated with EVT for AIS due to occlusive carotid artery dissection and tandem occlusion included in the retrospective international Antithrombotic Treatment for Stroke Prevention in Cervical Artery Dissection (STOP-CAD) study.

Value contribution of leniolisib in the Treatment of Activated PI3Kδ syndrome (APDS) in Spain using Multi-Criteria Decision Analysis (MCDA).

Background: Activated phosphoinositide 3-kinase (PI3K) δ Syndrome (APDS) is an ultra-rare, potentially life-threatening disease that lacks approved treatments in Spain. This study aimed to apply Multi-Criteria Decision Analysis (MCDA) to assess the value of the first pharmacological treatment for APDS in Spain.

Methods: A multidisciplinary group of 8 experts evaluated the selective PI3Kδ inhibitor leniolisib against Standard of Care (SoC).

Acute appendicitis and its treatment: a historical overview.

Purpose: Acute appendicitis (AA) is the leading cause of acute abdomen worldwide, with an incidence of 90-100 cases per 100,000 individuals annually and a lifetime risk of 7-12%. Despite its prevalence, historical accounts of AA are limited, particularly when compared to conditions like haemorrhoids, likely due to the appendix's internal location. This article traces the historical evolution of AA treatment from ancient times to the present, highlighting key contributions.

Posterior fossa ring-enhancing lesions in the adult immunocompetent host: illustrative cases, systematic review, and proposed diagnostic algorithm.

Purpose: Posterior fossa ring-enhancing lesions (PFREL) in the adult immunocompetent hosts pose a diagnostic challenge. We aimed to evaluate the spectrum of PFREL etiologies and propose a diagnostic algorithm.

Methods: This study involved a retrospective analysis of PFREL cases from our institution (January 2023 to April 2024) and a systematic literature review conducted using Embase and PubMed databases following the PRISMA 2020 guidelines.

New Artisse intrasaccular device for intracranial aneurysm treatment: short term clinical and angiographic result from the prospective registry INSPIRE-A.

Background: Intrasaccular devices have broadened treatment options for wide necked aneurysms. This study presents the preliminary experience with the Artisse 2.0 device.

Anaphylaxis definition, overview, and clinical support tool: 2024 consensus report.

Background: The 2006 National Institute of Allergy and Infectious Disease/Food Allergy and Anaphylaxis Network (NIAID/FAAN) anaphylaxis criteria are widely used in clinical care and research. In 2020, the World Allergy Organization (WAO) published modified criteria that have not been uniformly adopted. Different criteria contribute to inconsistent care and research outcomes.

Development and validation of the Neuro-Score: a specific scale to detect and monitor cognitive impairment in kidney or liver transplant recipients.

We created and validated the Neuro-Score, a specific scale to detect and monitor cognitive impairment, including mild stages, in kidney or liver transplant recipients. A qualitative study was conducted to define a preliminary set of 62 items. Item reduction was performed using exploratory factor analysis.

Sequence variants in HECTD1 result in a variable neurodevelopmental disorder.

Dysregulation of genes encoding the homologous to E6AP C-terminus (HECT) E3 ubiquitin ligases has been linked to cancer and structural birth defects. One member of this family, the HECT-domain-containing protein 1 (HECTD1), mediates developmental pathways, including cell signaling, gene expression, and embryogenesis. Through GeneMatcher, we identified 14 unrelated individuals with 15 different variants in HECTD1 (10 missense, 3 frameshift, 1 nonsense, and 1 splicing variant) with neurodevelopmental disorders (NDDs), including autism, attention-deficit/hyperactivity disorder, and epilepsy.

Cost-effectiveness of robotic compared with laparoscopic rectal resection. Results from the Spanish prospective national trial ROBOCOSTES.

Objective: The costs related to robotic surgery are known to be greater than those associated with laparoscopy. However, the potential for better outcomes of the former could lead to a cost-effectiveness advantage. The aim of this study is therefore to highlight the difference in cost-effectiveness between robotic and laparoscopic rectal resection.

A comparative analysis of risk stratification tools in systemic sclerosis-associated pulmonary arterial hypertension: a EUSTAR analysis.

Objectives: The 2022 European Society of Cardiology and European Respiratory Society (ESC/ERS) Guidelines for pulmonary arterial hypertension (PAH) recommend risk stratification to optimize management. However, the performance of generic PAH risk stratification tools in patients with systemic sclerosis (SSc)-associated PAH remains unclear. Our objective was to identify the most accurate approach for risk stratification at SSc-PAH diagnosis.

SPIRIT-DEFINE explanation and elaboration: recommendations for enhancing quality and impact of early phase dose-finding clinical trials protocols.

Unlabelled: Transparent and accurate reporting in early phase dose-finding (EPDF) clinical trials is crucial for informing subsequent larger trials. The SPIRIT statement, designed for trial protocol content, does not adequately cover the distinctive features of EPDF trials. Recent findings indicate that the protocol contents in past EPDF trials frequently lacked completeness and clarity.

Identification of strengths and weaknesses of the healthcare system for persons living with rare diseases in Catalonia (Spain), and recommendations to improve its comprehensive attention: the "acERca las enfermedades raras" project.

Background: Rare diseases (RDs) are a heterogeneous group of complex and low-prevalence conditions in which the time to establish a definitive diagnosis is often too long. In addition, for most RDs, few to no treatments are available and it is often difficult to find a specialized care team.

Objectives: The project "acERca las enfermedades raras" (in English: "bringing RDs closer") is an initiative primary designed to generate a consensus by a multidisciplinary group of experts to detect the strengths and weaknesses in the public healthcare system concerning the comprehensive care of persons living with a RD (PLWRD) in the region of Catalonia, Spain, where a Network of Clinical Expert Units (Xarxa d'Unitats de Expertesa Clínica or XUEC) was created and is being implemented since 2015.

Nedosiran in pediatric patients with PH1 and relatively preserved kidney function, a phase 2 study (PHYOX8).

Background: Primary hyperoxaluria type 1 (PH1) is an autosomal recessive disorder with dysregulated glyoxylate metabolism in the liver. Oxalate over-production leads to renal stones, progressive kidney damage and renal failure, with potentially life-threatening systemic oxalosis. Nedosiran is a synthetic RNA interference therapy, designed to reduce hepatic lactate dehydrogenase (LDH) to decrease oxalate burden in PH.

Nivolumab plus ipilimumab versus nivolumab in microsatellite instability-high metastatic colorectal cancer (CheckMate 8HW): a randomised, open-label, phase 3 trial.

Background: CheckMate 8HW prespecified dual primary endpoints, assessed in patients with centrally confirmed microsatellite instability-high or mismatch repair-deficient status: progression-free survival with nivolumab plus ipilimumab compared with chemotherapy as first-line therapy and progression-free survival with nivolumab plus ipilimumab compared with nivolumab alone, regardless of previous systemic treatment for metastatic disease. In our previous report, nivolumab plus ipilimumab showed superior progression-free survival versus chemotherapy in first-line microsatellite instability-high or mismatch repair-deficient metastatic colorectal cancer in the CheckMate 8HW trial. Here, we report results from the prespecified interim analysis for the other primary endpoint of progression-free survival for nivolumab plus ipilimumab versus nivolumab across all treatment lines.

Usefulness of serum neurofilament light chain in chronic inflammatory demyelinating polyradiculoneuropathy.

Background: The development of new biomarkers is essential to improve diagnostic accuracy and guide treatment decisions in chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The aim of this study was to investigate the utility of the serum neurofilament light chain (sNfL) level as a marker for disability and response to immunomodulatory treatment in patients with CIDP.

Methods: This prospective, single-center, observational study included 38 patients with CIDP: 19 treatment-naive (CIDP-I) patients assessed before and after the initiation of immunomodulatory therapy and 19 stable patients on maintenance immunoglobulins (CIDP-M).

Emerging treatments for sarcoma: from 2024 onward.

Introduction: Sarcomas are a rare and diverse group of mesenchymal-origin solid tumors, constituting only 1% of adult malignancies and classified into soft tissue and bone sarcomas. For localized disease, surgery and radiotherapy remain the cornerstone treatments. However, systemic options for advanced stages are limited, with an overall survival of approximately 20 months.

Nebulized aminoglycosides for ventilator-associated pneumonia: Methodological considerations and lessons from experimental studies.

Aminoglycosides are concentration-dependent antibiotics exerting a bactericidal effect when concentrations at the site of infection are equal to or greater than 5 times the minimum inhibitory concentrations (MIC). When administered intravenously, they exhibit poor lung penetration and high systemic renal and ototoxicity, imposing to restrict their administration to 5 days. Experimental studies conducted in anesthetized and mechanically ventilated sheep and pigs provide evidence that high doses of nebulized aminoglycosides induce a rapid and potent bacterial killing in the infected lung parenchyma.

Occluding mucous airway plugs in patients with obstructive lung diseases: a new treatable trait?

https://bit.ly/4gyJHFW.

Patterns, timing and predictors of recurrence following pancreaticoduodenectomy for pancreatic ductal adenocarcinoma: an international multicentre retrospective cohort study.

Background: Most patients undergoing pancreaticoduodenectomy (PD) for pancreatic ductal adenocarcinoma (PDAC) develop recurrence. No previous studies have investigated predictors of local-only recurrence following PD for PDAC. Our study aimed to determine timing, pattern and predictors of any-site and local-only recurrence following PD for PDAC.

Endovascular Thrombectomy for Extracranial Internal Carotid Artery Occlusions With Large Ischemic Strokes: Insights From the SELECT2 Trial.

Background And Objectives: Although previous trials have established the efficacy and safety of endovascular thrombectomy (EVT) in large ischemic core strokes, most of them excluded patients with extracranial internal carotid artery (e-ICA) occlusion. We aimed to compare outcomes in patients with e-ICA occlusion and large ischemic core infarcts treated with EVT vs medical management (MM).

Methods: This was a secondary analysis of the SELECT2 trial, a randomized controlled trial conducted at 31 international sites.

Elective Discontinuation of Larotrectinib in Pediatric Patients With TRK Fusion Sarcomas and Related Mesenchymal Tumors.

Larotrectinib is a highly selective tropomyosin receptor kinase (TRK) inhibitor with efficacy in children with TRK fusion tumors. We evaluated patient outcomes after elective discontinuation of larotrectinib in the absence of disease progression in a protocol-defined wait-and-see subset analysis of eligible patients where treatment resumption with larotrectinib was allowed if disease progressed. We also assessed the safety and efficacy of larotrectinib in all pediatric patients with sarcoma.

Desensitization With Imlifidase for HLA-Incompatible Deceased Donor Kidney Transplantation: A Delphi International Expert Consensus.

Highly sensitized (HS) patients in need of kidney transplantation (KTx) typically spend a longer time waiting for compatible kidneys, are unlikely to receive an organ offer, and are at increased risk of antibody-mediated rejection (AMR). Desensitization using imlifidase, which is more rapid and removes total body immunoglobulin G (IgG) to a greater extent than other methods, enables transplantation to occur between HLA-incompatible (HLAi) donor-recipient pairs and allows patients to have greater access to KTx. However, when the project was launched there was limited data and clinical experience with desensitization in general and with imlifidase specifically.

Primary Visceral Ewing Sarcoma (Gastric and Bladder) with KIT Expression: A Report of Two Patients and a Differential Diagnostic Approach.

Extra-skeletal Ewing sarcoma is a rare tumor, most commonly occurring in the paravertebral region, extremities, retroperitoneum, and very rarely in visceral locations. We present two primary visceral Ewing sarcomas confirmed at the molecular level: one located in the stomach and the other in the bladder. Both tumors demonstrated strong and diffuse membranous CD99 expression, accompanied by variable KIT immunoreactivity.