Cost Conversations to Mitigate the Effects of Financial Toxicity in Oncology: Current State, Opportunities, and Barriers.

The direct and indirect financial burden of cancer care, from medication costs to lost wages, results in financial toxicity for patients. Despite the growing recognition of financial toxicity as a problem for patients, there are few solutions available at the point of care. Structured cost conversations between oncologists and patients to help identify financial toxicity and intervene early when it is recognized have been posited as a patient facing intervention.

Spontaneous regression of metastatic clear cell renal cell carcinoma: A report of a rare case and a review of the literature.

Renal cell carcinoma (RCC) is the seventh most common cancer in the United States; clear cell RCC (ccRCC) is the most common subtype. We report a case of spontaneous regression of metastatic ccRCC and discuss possible underlying mechanisms informed by a literature review. While regression of metastatic RCC has been described following nephrectomy or treatment of the primary tumor, spontaneous regression is rare.

Financial toxicity in early-phase cancer clinical trial participants.

Background: Little is known about financial toxicity in early-phase clinical trial (EP-CT) participants. This study sought to describe financial toxicity in EP-CT participants and assess associations with patient characteristics and patient-reported outcomes (PROs).

Methods: Prospectively enrolled EP-CT participants from were followed from April 2021 through January 2023.

National survey of patient perspectives on cost discussions among recipients of copay assistance.

Background: Individuals with cancer and other medical conditions often experience financial concerns from high costs-of-care and may utilize copay assistance programs (CAP). We sought to describe CAP recipients' experiences/preferences for cost discussions with clinicians.

Methods: We conducted a national, cross-sectional electronic-survey from 10/2022 to 11/2022 of CAP recipients with cancer or autoimmune conditions to assess patient perspectives on cost discussions.

Oncology drug pricing: potential Medicare savings on cancer-directed and supportive care medications through the Mark Cuban cost plus drug model.

Prescription drug costs within oncology remain a challenge for many patients with cancer. The Mark Cuban Cost Plus Drug Company (MCCPDC) launched in 2022, aiming to provide transparently priced medications at reduced costs. In this study, we sought to describe the potential impact of MCCPDC on Medicare Part-D oncology spending related to cancer-directed (n = 7) and supportive care (n = 26) drugs.

B cell focused transient immune suppression protocol for efficient AAV readministration to the liver.

Adeno-associated virus (AAV) vectors are used for correcting multiple genetic disorders. Although the goal is to achieve lifelong correction with a single vector administration, the ability to redose would enable the extension of therapy in cases in which initial gene transfer is insufficient to achieve a lasting cure, episomal vector forms are lost in growing organs of pediatric patients, or transgene expression is diminished over time. However, AAV typically induces potent and long-lasting neutralizing antibodies (NAbs) against capsid that prevents re-administration.

A novel class of self-complementary AAV vectors with multiple advantages based on cceAAV lacking mutant ITR.

Self-complementary AAV vectors (scAAV) use a mutant inverted terminal repeat (mITR) for efficient packaging of complementary stranded DNA, enabling rapid transgene expression. However, inefficient resolution at the mITR leads to the packaging of monomeric or subgenomic AAV genomes. These noncanonical particles reduce transgene expression and may affect the safety of gene transfer.

Care Delivery Interventions for Individuals with Cancer: A Literature Review and Focus on Gastrointestinal Malignancies.

Background: Gastrointestinal malignancies represent a particularly challenging condition, often requiring a multidisciplinary approach to management in order to meet the unique needs of these individuals and their caregivers.

Purpose: In this literature review, we sought to describe care delivery interventions that strive to improve the quality of life and care for patients with a focus on gastrointestinal malignancies.

Conclusion: We highlight patient-centered care delivery interventions, including patient-reported outcomes, hospital-at-home interventions, and other models of care for individuals with cancer.

TLR9-independent CD8 T cell responses in hepatic AAV gene transfer through IL-1R1-MyD88 signaling.

Upon viral infection of the liver, CD8 T cell responses may be triggered despite the immune suppressive properties that manifest in this organ. We sought to identify pathways that activate responses to a neoantigen expressed in hepatocytes, using adeno-associated viral (AAV) gene transfer. It was previously established that cooperation between plasmacytoid dendritic cells (pDCs), which sense AAV genomes by Toll-like receptor 9 (TLR9), and conventional DCs promotes cross-priming of capsid-specific CD8 T cells.

Losing vigilance in diagnosing pulmonary alveolar microlithiasis: A report on four cases.

Pulmonary alveolar microlithiasis (PAM) is a rare chronic lung disease characterized by calcium and phosphate deposition in the alveolar lumen throughout the parenchyma of both lungs, with predominance in the middle and lower lung fields. It is caused by mutations in the recessive gene, , on the autosomal chromosome. In this article, we characterize four cases of PAM and analyze the loss of diagnostic vigilance in two of them.

Clinical Characteristics and Outcomes of Long COVID-19 Hospitalized Children in Vietnam.

A cross-sectional study was conducted on 205 pediatric patients, including 150 post-COVID-19 patients and 55 noninfected patients. The study identified 10 common respiratory symptoms in post-COVID-19 patients, with significant differences in clinical symptoms between the 2 groups. Post-COVID-19 pediatric patients had a lower lymphocyte count and a higher rate of pneumonia diagnosis, which can persist for up to 16 weeks after discharge.

β Cell microRNAs Function as Molecular Hubs of Type 1 Diabetes Pathogenesis and as Biomarkers of Diabetes Risk.

MicroRNAs (miRNAs) are small non-coding RNAs that play a crucial role in modulating gene expression and are enriched in cell-derived extracellular vesicles (EVs). We investigated whether miRNAs from human islets and islet-derived EVs could provide insight into β cell stress pathways activated during type 1 diabetes (T1D) evolution, therefore serving as potential disease biomarkers. We treated human islets from 10 cadaveric donors with IL-1β and IFN-γ to model T1D .

Pharmacist-Led Interventions to Reduce Drug-Related Problems in Prescribing for Pediatric Outpatients in a Developing Country: A Randomized Controlled Trial.

Objective: To evaluate a pharmacist-led intervention's effectiveness in reducing drug-related problems (DRPs ( related to prescriptions for pediatric outpatients.

Methods: We conducted a randomized controlled trial. We recruited and randomly assigned 31 physicians to control or intervention groups.

Chemical modification of AAV9 capsid with N-ethyl maleimide alters vector tissue tropism.

Although more adeno-associated virus AAV-based drugs enter the clinic, vector tissue tropism remains an unresolved challenge that limits its full potential despite that the tissue tropism of naturally occurring AAV serotypes can be altered by genetic engineering capsid vie DNA shuffling, or molecular evolution. To further expand the tropism and thus potential applications of AAV vectors, we utilized an alternative approach that employs chemical modifications to covalently link small molecules to reactive exposed Lysine residues of AAV capsids. We demonstrated that AAV9 capsid modified with N-ethyl Maleimide (NEM) increased its tropism more towards murine bone marrow (osteoblast lineage) while decreased transduction of liver tissue compared to the unmodified capsid.

Breaking membrane barriers to neutralize E. coli and K. pneumoniae virulence with PEGylated branched polyethylenimine.

Bacterial infections caused by Gram-negative pathogens, such as those in the family Enterobacteriaceae, are among the most difficult to treat because effective therapeutic options are either very limited or non-existent. This raises serious concern regarding the emergence and spread of multi-drug resistant (MDR) pathogens in the community setting; and thus, creates the need for discovery efforts and/or early-stage development of novel therapies for infections. Our work is directed towards branched polyethylenimine (BPEI) modified with polyethylene glycol (PEG) as a strategy for targeting virulence from Gram-negative bacterial pathogens.

Cryptic resolution sites in the vector plasmid lead to the heterogeneities in the rAAV vectors.

Recombinant adeno-associated virus (rAAV) vectors carry a cassette of interest retaining only the inverted terminal repeats (ITRs) from the wild-type virus. Conventional rAAV production primarily uses a vector plasmid as well as helper genes essential for AAV replication and packaging. Nevertheless, plasmid backbone related contaminants have been a major source of vector heterogeneity.

Genomic Designs of rAAVs Contribute to Pathological Changes in the Livers and Spleens of Mice.

Recombinant AAV (rAAV) gene therapy is being investigated as an effective therapy for several diseases including hemophilia B. Reports of liver tumor development in certain mouse models due to AAV treatment and genomic integration of the rAAV vector has raised concerns about the long-term safety and efficacy of this gene therapy. To investigate whether rAAV treatment causes cancer, we utilized two mouse models, inbred C57BL/6 and hemophilia B Balb/C mice (HemB), to test if injecting a high dose of various rAAV8 vectors containing or lacking hFIX transgene, a Poly-A sequence, or the CB or TTR promoter triggered liver fibrosis and/or cancer development over the course of the 6.

Tracheal bronchus and associated pathologies detected by multidetector-row computed tomography in the Vietnamese population.

Tracheal bronchus (TB) is a very rare condition, which is often associated with some other pathologies. This study was designed to characterize the morphology of tracheal bronchus and associated pathologies in Vietnamese individuals using multidetector-row computed tomography (MDCT). From August 2016 to February 2021, 16, 64-, and 128-detector-row computed tomography scanners were used to perform chest scans of 3663 patients, of whom 32 had tracheal bronchus and associated pathologies.

Fast and high-throughput LC-MS characterization, and peptide mapping of engineered AAV capsids using LC-MS/MS.

Adeno-associated virus (AAV) has emerged as a leading platform for gene therapy. With the skyrocketing rate of AAV research and the prevalence of many new engineered capsids being investigated in preclinical and clinical trials, capsid characterization plays a vital role in serotype confirmation and quality control. Further, peptide mapping the capsid proteins might inevitably be a future requirement by regulatory agencies since it is a critical step in good manufacturing practice (GMP) for biotherapeutic characterization.

Subgenomic particles in rAAV vectors result from DNA lesion/break and non-homologous end joining of vector genomes.

Recombinant adeno-associated virus (rAAV) vectors have been developed for therapeutic treatment of genetic diseases. Current rAAV vectors administered to affected individuals often contain vector DNA-related contaminants. Here we present a thorough molecular analysis of the configuration of non-standard AAV genomes generated during rAAV production using single-molecule sequencing.

Medication Adherence in Vietnamese Patients with Cardiovascular and Endocrine-Metabolic Diseases.

(1) Background: COVID-19 has significantly affected the quality of life and the medication adherence of patients with chronic diseases. Attitudes towards the disease and preventive measures are the things that need to be considered for patient adherence to medication during the COVID-19 pandemic. We aimed to evaluate the rate and compare the medication adherence and the impact of the COVID-19 pandemic on medication adherence in Vietnamese patients with cardiovascular and endocrine−metabolic diseases.

The Application of Light-Assisted Drying to the Thermal Stabilization of Nucleic Acid Nanoparticles.

Cold-chain storage can be challenging and expensive for the transportation and storage of biologics, especially in low-resource settings. Nucleic acid nanoparticles (NANPs) are an example of new biological products that require refrigerated storage. Light-assisted drying (LAD) is a new processing technique to prepare biologics for anhydrous storage in a trehalose amorphous solid matrix at ambient temperatures.

Dimerization of 600 Da branched polyethylenimine improves β-lactam antibiotic potentiation against antibiotic-resistant Staphylococcus epidermidis and Pseudomonas aeruginosa.

Antibiotic resistance is a growing concern in the medical field. Drug-susceptible infections are often treated with β-lactam antibiotics, which bind to enzymes known as penicillin-binding proteins (PBPs). When the PBPs are disabled, the integrity of the cell wall is compromised, leading to cell lysis.