Searching for monogenic autoimmune etiology in patients with type 1 diabetes onset under 30 months of age.

Introduction: The most frequent form of diabetes in pediatric patients is polygenic autoimmune diabetes (T1D), but single-gene variants responsible for autoimmune diabetes have also been described. Both disorders share clinical features, which can lead to monogenic forms being misdiagnosed as T1D. However, correct diagnosis is crucial for therapeutic choice, prognosis and genetic counseling.

Fitness, Body Composition, and Health Behaviors in Long-Term Survivors of Adolescent and Young Adult Cancers.

To investigate cardiorespiratory fitness (CRF), body composition, health behaviors, and health-related quality of life (HRQoL) in adolescent and young adult cancer survivors (AYA-CS) compared with age-matched counterparts without a cancer diagnosis. This cross-sectional study recruited participants aged 15-25 years at the time of their cancer diagnosis and ≥ 5 years post-treatment. Participants completed cardiopulmonary exercise testing, dual-energy X-ray absorptiometry, food diaries, physical activity (PA), fatigue, and HRQoL questionnaires.

Prediction of Radiation Therapy Induced Cardiovascular Toxicity from Pretreatment CT Images in Patients with Thoracic Malignancy via an Optimal Biomarker Approach.

Rationale And Objectives: Cardiovascular toxicity is a well-known complication of thoracic radiation therapy (RT), leading to increased morbidity and mortality, but existing techniques to predict cardiovascular toxicity have limitations. Predictive biomarkers of cardiovascular toxicity may help to maximize patient outcomes.

Methods: The machine learning optimal biomarker (OBM) method was employed to predict development of cardiotoxicity (based on serial echocardiographic measurements of left ventricular ejection fraction and longitudinal strain) from computed tomography (CT) images in patients with thoracic malignancy undergoing RT.

Comparative impact of tertiary lymphoid structures and tumor-infiltrating lymphocytes in cholangiocarcinoma.

Background: Cholangiocarcinoma is a challenging malignancy with limited responses to conventional therapies, particularly immune checkpoint inhibitor therapy. Tumor-infiltrating lymphocytes (TILs) and tertiary lymphoid structures (TLSs) are key components of the tumor microenvironment (TME) and have been implicated in the immune response to cancer. However, the role and difference of TLSs and TILs in patients with cholangiocarcinoma remains unclear.

Conduction system metastasis in a patient with carcinoma oesophagus.

A young man in his 30s presented to us with multiple episodes of syncope and exertional dyspnoea for the last 2 weeks. He was diagnosed with squamous cell carcinoma of the lower one-third of the oesophagus in 2021 for which he was treated with neoadjuvant chemoradiotherapy, followed by McKeown oesophagectomy. At 2-year follow-up, he had developed a soft tissue swelling in the scalp, which was diagnosed as a tumour recurrence and radiotherapy was initiated.

Clinical significance of post-chemoradiotherapy 2-[F]FDG PET/CT response in locally advanced nasopharyngeal carcinoma: A real-world study.

Purpose: To investigate the prognostic value of post-chemoradiotherapy 2-[F]FDG PET/CT in locally advanced nasopharyngeal carcinoma (LANPC) and develop an accurate prognostic model based on the 2-[F]FDG PET/CT results.

Methods: 900 LANPC patients who underwent pretreatment and post-chemoradiotherapy 2-[F]FDG PET/CT from May 2014 to August 2022 were included in the study. We divided the patients into two distinct cohorts for the purpose of our study: a training cohort comprising 506 individuals, included from May 2008 to April 2020, and a validation cohort consisting of 394 individuals, included from May 2020 to August 2022.

A year in review: new treatments and expanded indications in dermatology in 2024.

Methods: A literature search was conducted on Drugs@FDA: FDA-Approved Drugs for the year 2024 to identify new dermatologic treatments.

Results: In 2024, the FDA approved seven new dermatologic therapies and expanded the indications for seven current therapies. These therapies treat conditions such as atopic dermatitis, hidradenitis suppurativa, prurigo nodularis, molluscum contagiosum, and alopecia areata, among others.

Safety of interleukin inhibitors in patients with plaque psoriasis and history of neoplasms: a multicenter retrospective study - IL PSO (Italian landscape psoriasis).

Interleukin (IL) inhibitors are increasingly used in the management of moderate-to-severe plaque psoriasis. However, their use in patients with a history of cancer is debated. We conducted a multicenter retrospective study across nine Italian Dermatology Units to assess the real-world effectiveness and safety of IL inhibitors (IL-23, IL-17, IL-12/23) in 136 oncological patients with moderate-to-severe plaque psoriasis.

Impact of Immunotherapy on the Care Patterns and Outcomes of Patients with Advanced Hepatocellular Carcinoma.

Background: Modern immunotherapy with checkpoint inhibitors revolutionized cancer treatment and outcomes. This study aims to demonstrate how immunotherapy has impacted the national landscape of systemic treatment and palliative care in advanced hepatocellular carcinoma (HCC).

Methods: Retrospective cohort selecting patients from the U.

Prospective Validation of CAR-HEMATOTOX and a simplified version predict Survival in Patients with Large B-Cell Lymphoma treated with anti-CD19 CAR T-cells: data from CART-SIE study.

Background: Anti-CD19 CAR T-cells have revolutionized outcomes in relapsed/refractory large B-cell lymphomas. Long-term follow-up underscored the role of hematological toxicity in non-relapse mortality, largely driven by infections, leading to the development of the CAR-HEMATOTOX (HT) score for predicting neutropenia. The European scientific community (EHA/EBMT) later reached a consensus, defining a new entity: immune effector cell-associated hematotoxicity (ICAHT).

Long-Term Cognitive Outcomes in Adult Patients Receiving Chimeric Antigen Receptor T-Cell Therapies.

Background: CAR T-cell therapy (CAR-T) is leading to durable responses in patients with cancer but there is concern that cytokine release syndrome (CRS) and neurotoxicity may impact survivors' cognitive function. We assessed long-term cognitive function in CAR-T recipients and examine factors associated with change in cognition over time.

Methods: We assessed perceived cognition (Functional Assessment of Cancer Therapy - Cognition) and neurocognitive performance (standardized neuropsychological battery) in adult patients prior to receiving CAR-T and at 6 month follow-up.

Comparison of Acute and Late toxicities with Daily vs. Every-Other-Day Five Fraction Partial Breast Radiation Therapy.

Purpose/objective: Partial breast irradiation (PBI) delivered with intensity modulated radiation therapy (IMRT) over five fractions every other day (QOD) represents an attractive, convenient method of delivering adjuvant radiation therapy for well selected patients without compromising oncologic or toxicity outcomes. Condensing this regimen to a week of treatment through consecutive daily delivery may further increase patient convenience, though comparison of toxicities between daily and QOD regimens are limited.

Materials/methods: We retrospectively reviewed 507 patients from an institutional registry undergoing PBI for DCIS or early-stage breast cancer (T1-2N0/x) from 2015 to 2022.

Preparation and characterization of polyvinyl alcohol and chitosan composite film with cassia oil encapsulated in β-cyclodextrin and its application in fresh banana.

In this study, composite films were developed by encapsulating cassia oil (CO) with β-cyclodextrin through a microencapsulation technique and incorporating it into a chitosan (CS), polyvinyl alcohol (PVA) and glycerol matrix. The primary objective of the film was to inhibit bacterial growth on the surface of fresh bananas and extend their shelf life. Characterization methods were employed to evaluate the physical properties and functionality of the composite films.

A fatal case of enterovirus A71-induced meningoencephalitis following allogenic hematopoietic stem cell transplantation.

Enterovirus A71 (EV-A71) is a major pathogen responsible for hand, foot, and mouth disease (HFMD) in infants and children. EV-A71 infection represents an epidemic in the Asia-Pacific region, and can cause serious central nervous system (CNS) infections in immunocompromised patients that can result in paralysis, disability, or death. There have been few reports in the literature concerning EV-A71 CNS infections after allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adult patients.

GRP78: A New Promising Candidate in Colorectal Cancer Pathogenesis and Therapy.

Colorectal cancer (CRC) is a significant global health challenge, marked by varying incidence and mortality rates across different regions. The pathogenesis of CRC involves multiple stages, including initiation, promotion, progression, and metastasis, influenced by genetic and epigenetic factors. The chaperone protein glucose-regulated protein 78 (GRP78), crucial in regulating the unfolded protein response (UPR) during endoplasmic reticulum (ER) stress, plays a pivotal role in CRC pathogenesis.

Gastric cancer mesenchymal stem cells upregulate PD-1 expression on the CD8 T cells by regulating the PI3K/AKT pathway.

Gastric cancer mesenchymal stem cells (GC-MSCs) are a crucial component of the gastric cancer microenvironment, exerting a pivotal influence on the formation of a suppressive immune microenvironment and the progression of gastric cancer. In this study, we utilized GC-MSCs to co-culture peripheral blood mononuclear cells (PBMCs) obtained from both gastric cancer patients and healthy individuals in a proportionate manner by direct cell-to-cell contact. Our findings reveal that co-culture of GC-MSCs with PBMCs led to a notable reduction in CD8 T cells percentages and an increase in surface PD-1 expression levels on CD8 T cells.

Immunological characteristics of peripheral T cells as prognostic markers for Camrelizumab and Apatinib combination therapy in advanced squamous non-small-cell lung cancer.

Purpose: To determine the characteristic changes of peripheral blood T cells and identify potential biomarkers that associated with the clinical efficacy of combined immunotherapy and anti-angiogenic therapy in patients with advanced squamous non-small cell lung cancer (NSCLC).

Methods: We performed a comprehensive immunological assessment of peripheral blood mononuclear cell samples from advanced squamous NSCLC patients before and after combination of immunotherapy (Camrelizumab) and anti-angiogenic therapy (Apatinib) using spectral flow cytometry. Correlations between these immunological features and clinical efficacy were analyzed.

Neoadjuvant chemotherapy in locally advanced colon cancer: A systematic review with proportional meta-analysis.

Neoadjuvant chemotherapy is suggested in locally advanced colon cancer. Data on improved long-term oncological outcomes are lacking, which hampers the implementation in clinical practice. This systematic review provides an overview of the benefits and drawbacks of neoadjuvant chemotherapy in patients with locally advanced colon cancer.

The Association of Epstein-Barr Virus Donor and Recipient Serostatus With Outcomes After Kidney Transplantation : A Retrospective Cohort Study.

Background: Prior studies indicate that 1% to 4% of Epstein-Barr virus (EBV)-seronegative recipients of EBV-seropositive donor (EBV D+/R-) kidneys develop posttransplant lymphoproliferative disorder (PTLD). However, these estimates are based on limited data that lack granularity.

Objective: To determine the associations between pretransplant EBV D+/R- and recipient EBV-seropositive status (R+) and the outcomes of PTLD and graft and patient survival among adult kidney transplant recipients.

Perspectives on Swedish Regulations for Online Record Access Among Adolescents With Serious Health Issues and Their Parents: Mixed Methods Study.

Background: With the increasing implementation of patient online record access (ORA), various approaches to access to minors' electronic health records have been adopted globally. In Sweden, the current regulatory framework restricts ORA for minors and their guardians when the minor is aged between 13 and 15 years. Families of adolescents with complex health care needs often desire health information to manage their child's care and involve them in their care.

Clinical Activity of Mitogen-Activated Protein Kinase Inhibitors in Patients With MAP2K1 (MEK1)-Mutated Metastatic Cancers.

Purpose: MAP2K1/MEK1 mutations are potentially actionable drivers in cancer. MAP2K1 mutations have been functionally classified into three groups according to their dependency on upstream RAS/RAF signaling. However, the clinical efficacy of mitogen-activated protein kinase (MAPK) pathway inhibitors (MAPKi) for MAP2K1-mutant tumors is not well defined.

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.

Highly Optimized CNS Penetrant Inhibitors of EGFR Exon20 Insertion Mutations.

Despite recent advances in the inhibition of EGFR (epidermal growth factor receptor), there remains a clinical need for new EGFR Exon20 insertion (Ex20Ins) inhibitors that spare EGFR WT. Herein, we report the discovery and optimization of two chemical series leading to ether and biaryl as potent, selective, and brain-penetrant inhibitors of Ex20Ins mutants. Building on our earlier discovery of alkyne which allowed access to CNS property space for an Ex20Ins inhibitor, we utilized structure-based design to move to lower lipophilicity and lower CL compounds while maintaining a WT selectivity margin.

Small molecule modulators of B56-PP2A restore 4E-BP function to suppress eIF4E-dependent translation in cancer cells.

Dysregulated eIF4E-dependent translation is a central driver of tumorigenesis and therapy resistance. eIF4E binding proteins (4E-BP1/2/3) are major negative regulators of eIF4E-dependent translation that are inactivated in tumors through inhibitory phosphorylation or downregulation. Previous studies have linked PP2A phosphatase(s) to activation of 4E-BP1.