Role of miRNAs as epigenetic regulators of immune checkpoints in lung cancer immunity.

The advent of immunotherapy in cancer has provided new avenues in the treatment of many malignancies at various stages. Specifically, immune checkpoint inhibitors (ICIs) have transformed the field of lung cancer treatment. However, since some tumors can evade the immune system, not all patients respond properly.

Safety and efficacy of nipocalimab in adults with generalised myasthenia gravis (Vivacity-MG3): a phase 3, randomised, double-blind, placebo-controlled study.

Background: Given burdensome side-effects and long latency for efficacy with conventional agents, there is a continued need for generalised myasthenia gravis treatments that are safe and provide consistently sustained, long-term disease control. Nipocalimab, a neonatal Fc receptor blocker, was associated with dose-dependent reductions in total IgG and anti-acetylcholine receptor (AChR) antibodies and clinically meaningful improvements in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale in patients with generalised myasthenia gravis in a phase 2 study. We aimed to assess the safety and efficacy of nipocalimab in a phase 3 study.

Comprehensive Metabolomic Profiling in Adults with X-Linked Hypophosphatemia: A Case-Control Study.

Background: X-linked hypophosphatemia (XLH) is a rare disorder characterized by elevated levels of fibroblast growth factor 23 (FGF-23), leading to hypophosphatemia and complications in diagnosis due to its clinical heterogeneity. Metabolomic analysis, which examines metabolites as the final products of cellular processes, is a powerful tool for identifying in vivo biochemical changes, serving as biomarkers of pathological abnormalities, and revealing previously uncharted metabolic pathways.

Methods: A multicenter cross-sectional case-control study of adult patients diagnosed with XLH was conducted.

Clinical profile of an unselected population with heart failure treated with vericiguat in real life: differences with the VICTORIA trial.

Introduction: Vericiguat, an oral stimulator of soluble guanylate cyclase, reduces cardiovascular mortality and hospitalisations in patients with heart failure (HF) and reduced ejection fraction, as demonstrated in the VICTORIA trial. This study assessed the real-world use of vericiguat.

Material And Methods: This cross-sectional, prospective and multicenter registry (VERISEC) included 776 patients from 43 centres in Spain between December 2022 and October 2023.

Ex vivo intravascular methylene blue instillation in oesophagectomy and gastrectomy specimens and its implication in tumour histopathological assessment.

Surgical resection and lymphadenectomy are the mainstay of curative treatment for oesophagogastric cancer. In this study we evaluate the results of intravascular methylene blue injection into oesophagectomy and gastrectomy specimens as a tool to increase lymph node detection. A prospective and descriptive study was run on 24 patients (11 oesophagus, 13 stomach cases).

Survey of legislative frameworks and national recommendations governing paediatric maintenance haemodialysis in Europe.

Background: The application of international recommendations for paediatric maintenance haemodialysis (HD) could be strengthened by national laws or written recommendations. Our aim was therefore to describe the national rules governing paediatric maintenance HD in European countries.

Methods: A national representative, approved by the president of each paediatric nephrology society, was contacted in all 42 European countries to complete two online questionnaires.

Detection of clinically relevant variants in the gene below 10% allelic frequency: A multicenter study by ERIC, the European Research Initiative on CLL.

In chronic lymphocytic leukemia, the reliability of next-generation sequencing (NGS) to detect variants ≤10% allelic frequency (low-VAF) is debated. We tested the ability to detect 23 such variants in 41 different laboratories using their NGS method of choice. The sensitivity was 85.

[Translated article] Prospective observational follow-up study of psychoactive drug treatment initiated in the intensive care unit.

Objective: The treatment and prevention of delirium in the intensive care unit (ICU) have gained significant importance in patient care in recent years. Some studies have linked delirium with increased risks of mortality, prolonged hospital stay, and more days of mechanical ventilation. This study aims to analyse the use of psychotropic drugs initiated in the ICU and their continuation upon hospital discharge, as well as to evaluate their contribution to polypharmacy and associated adverse clinical effects.

Comprehensive Clinical and Genetic Characterization of a Spanish Cohort of 22 Patients With Bainbridge-Ropers Syndrome.

Bainbridge-Ropers Syndrome (BRPS) is a genetic condition resulting from truncating variants in the ASXL3 gene. The clinical features include neurodevelopmental and language impairments, behavioral issues, hypotonia, feeding difficulties, and distinctive facial features. In this retrospective study, we analyzed 22 Spanish individuals with BRPS, aiming to perform a detailed clinical and molecular description and establish a genotype-phenotype correlation.

Is lipedema a progressive disease?

Background: It is not yet known whether lipedema is a progressive chronic disease or not. The aim of this paper is to describe the long-term changes in the volume of the lower limbs.

Methods: The primary endpoint of this prospective cohort study of patients with lipedema was the percentage of volume change (PVC) during follow up.

Improve the quality of end-of-life in cancer patients using social representations of nutrition.

Introduction: The problems related to nutrition generate great concern in palliative cancer patients and their caregivers. Literature has analyzed the psychological and social problems that nutrition causes. From patient-centered orientation, there are protocols for nutritional care.

Inter-Rater Disagreements in Applying the Montreal Classification for Crohn's Disease: The Five-Nations Survey Study.

Background: The Montreal classification has been widely used in Crohn's disease since 2005 to categorize patients by the age of onset (A), disease location (L), behavior (B), and upper gastrointestinal tract and perianal involvement. With evolving management paradigms in Crohn's disease, we aimed to assess the performance of gastroenterologists in applying the Montreal classification.

Methods: An online survey was conducted among participants at an international educational conference on inflammatory bowel diseases.

Olutasidenib in combination with azacitidine induces durable complete remissions in patients with relapsed or refractory mIDH1 acute myeloid leukemia: a multicohort open-label phase 1/2 trial.

Background: Olutasidenib is a potent, selective, oral, small molecule inhibitor of mutant IDH1 (mIDH1) which induced durable remissions in high-risk, relapsed/refractory (R/R) mIDH1 AML patients in a phase 1/2 trial. We present a pooled analysis from multiple cohorts of the phase 1/2 trial of patients with R/R AML who received combination olutasidenib and azacitidine therapy.

Methods: Adult patients with mIDH1 AML received 150 mg olutasidenib twice daily plus standard-of-care azacitidine (OLU + AZA) and were evaluated for response and safety.

Characteristics, outcomes and treatment patterns in acute myeloid leukemia patients 60 years or older in Colombia: a RENEHOC-PETHEMA study.

There is a limited information available on the clinical characteristics, treatment patterns and outcomes on older patients diagnosed with Acute Myeloid Leukemia (AML) in Latin-America. This multicenter retrospective study analyzed 269 patients over 60 years of age diagnosed with AML in Colombia, using data from RENEHOC-PETHEMA registry, from 2009 to 2023. The median age at diagnosis was 70 years (Range:60-98), 55% were men, 61% had an ECOG < 2, and 75.

Thoracoabdominal chordoma in a pediatric patient. A rare entity.

Introduction: Chordoma is a rare, slow-growing notochordal neoplasm typical of adults. Less than 5% of the cases occur in children, where they are located at the skull base. Treatment involves surgical resection with or without radiotherapy.

Distal Access Catheter Improves Balloon Guide and Stent Retriever Thrombectomy Outcomes in Nonagenarians.

Background And Purpose: The safety and effectiveness of endovascular techniques in elderly patients with large vessel occlusion (LVO) remain controversial. We investigated the angiographic and clinical outcomes of nonagenarians treated with different endovascular techniques using a balloon guide catheter (BGC), distal aspiration catheter (DAC), and/or stent retriever (SR).

Methods: We analyzed the data from the Registry of Combined versus Single Thrombectomy Techniques (ROSSETTI) of consecutive nonagenarian patients with anterior circulation LVO and compared the outcomes of those treated with BGC+noDAC+SR (101-group), BGC+DAC+SR (111-group), and noBGC+DAC+SR (011-group).

Growth, Safety and Tolerance in Infants Fed Rice Protein Hydrolysate Formula: The GRITO Randomised Controlled Trial.

: Hydrolysed rice formula (HRF) is tolerated by >90% of children with cow's milk protein allergy (CMPA). However, concerns have been raised about potential suboptimal growth in infants fed HRF compared to those fed an extensively hydrolysed milk protein formula (eHF). : To compare growth, safety and tolerance acquisition in infants with CMPA when fed HRF versus eHF.

Withdrawal of antitumour necrosis factor in inflammatory bowel disease patients in remission: a randomised placebo-controlled clinical trial of GETECCU.

Background And Objectives: Primary objectives: to compare the rates of sustained clinical remission at 12 months in patients treated with antitumour necrosis factor (anti-TNF) and immunomodulators who withdraw anti-TNF treatment versus those who maintain it.

Secondary Objectives: to evaluate the effect of anti-TNF withdrawal on relapse-free time, endoscopic and radiological activity, safety, quality of life and work productivity; and to identify predictive factors for relapse.

Design: Prospective, quadruple-blind, multicentre, randomised, controlled trial.

Machine Learning Algorithms in Controlled Donation After Circulatory Death Under Normothermic Regional Perfusion: A Graft Survival Prediction Model.

Background: Several scores have been developed to stratify the risk of graft loss in controlled donation after circulatory death (cDCD). However, their performance is unsatisfactory in the Spanish population, where most cDCD livers are recovered using normothermic regional perfusion (NRP). Consequently, we explored the role of different machine learning-based classifiers as predictive models for graft survival.

Artificial intelligence in rheumatology research: what is it good for?

Artificial intelligence (AI) is transforming rheumatology research, with a myriad of studies aiming to improve diagnosis, prognosis and treatment prediction, while also showing potential capability to optimise the research workflow, improve drug discovery and clinical trials. Machine learning, a key element of discriminative AI, has demonstrated the ability of accurately classifying rheumatic diseases and predicting therapeutic outcomes by using diverse data types, including structured databases, imaging and text. In parallel, generative AI, driven by large language models, is becoming a powerful tool for optimising the research workflow by supporting with content generation, literature review automation and clinical decision support.

Leishmaniasis in Patients With Inflammatory Bowel Disease: A National Multicenter Study of GETECCU.

Background: Leishmaniasis (LI) is a vector-borne illness caused by a protozoan of the genus Leishmania. Data on the features of LI in patients with inflammatory bowel disease (IBD) are scarce.

Aim: To describe the characteristics of patients with IBD who present with leishmaniasis, infection outcomes and the risk factors associated with developing visceral leishmaniasis (VL).

Clinical features, mutation spectrum and factors related to reaching molecular diagnosis in a cohort of patients with distal myopathies.

Background: Distal myopathies (MPDs) are heterogeneous diseases of complex diagnosis whose prevalence and distribution in specific populations are unknown.

Methods: Demographic, clinical, genetic, neurophysiological, histopathological and muscle imaging characteristics of a MPDs cohort from a neuromuscular reference center were analyzed to study their epidemiology, features, genetic distribution and factors related to diagnosis.

Results: The series included 219 patients (61% were men, 94% Spanish and 41% sporadic cases).

Mortality in patients with giant cell arteritis in Spain: results from the ARTESER registry.

Objectives: To compare mortality rates between GCA patients and the general population in Spain, and to identify associated factors influencing mortality.

Methods: ARTESER, a multicenter registry by the Spanish Society of Rheumatology, includes GCA patients from June 2013 to March 2019. Demographic, clinical, imaging, histological and mortality data were collected retrospectively.

Pharmacogenetics of Neoadjuvant MAP Chemotherapy in Localized Osteosarcoma: A Study Based on Data from the GEIS-33 Protocol.

Osteosarcoma is a rare disease, but it is the most frequent malignant bone tumor. Primary treatment consists of preoperative MAP (methotrexate (MTX), doxorubicin and cisplatin) chemotherapy followed by surgery and adjuvant chemotherapy. Pathological response to preoperative chemotherapy is one of the most important prognostic factors, but molecular biomarkers are lacking.