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Italian Real-Life Experience of Patients with Hereditary Transthyretin Amyloidosis Treated with Patisiran.

Hereditary transthyretin amyloidosis (ATTRv) is a multisystemic, rare, inherited, progressive and adult-onset disease, affecting the sensorimotor nerves, heart, autonomic function and other organs. The actual scenario of pharmaceutical approaches for ATTRv amyloidosis includes five main groups: TTR stabilizers, TTR mRNA silencers, TTR fibril disruptors, inhibitor of TTR fibril seeding and gene therapy. Patisiran is a small, double-stranded interfering RNA encapsulated in a lipid nanoparticle, able to penetrate into hepatocytes, where it selectively targets TTR mRNA, reducing TTR production. We

Frailty-adjusted therapy in Transplant Non-Eligible patients with newly diagnosed Multiple Myeloma (FiTNEss (UK-MRA Myeloma XIV Trial)): a study protocol for a randomised phase III trial.

INTRODUCTION: Multiple myeloma is a bone marrow cancer, which predominantly affects older people. The incidence is increasing in an ageing population.Over the last 10 years, patient outcomes have improved. However, this is less apparent in older, less fit patients, who are ineligible for stem cell transplant. Research is required in this patient group, taking into account frailty and aiming to improve: treatment tolerability, clinical outcomes and quality of life. METHODS AND ANALYSIS: Frailty-adjusted therapy in Transplant Non-Eligible patients with newly diagnosed Multiple Myeloma is a nati

Knowledge mapping and visualized analysis of research progress in onconephrology: a bibliometric analysis.

OBJECTIVES: Onconephrology is an expanding subspecialty focused on the management of cancer patients with renal injury. This study used a comprehensive bibliometric analysis to emphasize the need for cooperation between oncologists and nephrologists, exploring current trends and future research areas in onconephrology. METHODS: Relevant literature on onconephrology published between 1 January 2000 and 27 April 2024 was retrieved from the Science Citation Index Expanded of the Web of Science Core Collection, followed by manual screening. Bibliometric analyses were performed using CiteSpace, VO

Drug Delivery Approaches for the Treatment of Cervical Cancer.

Cervical cancer is a highly prevalent cancer that affects women around the world. With the availability of new technologies, researchers have increased their efforts to develop new drug delivery systems in cervical cancer chemotherapy. In this review, we summarized some of the recent research in systematic and localized drug delivery systems and compared the advantages and disadvantages of these methods.

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… Although gels are tolerated more than other dosage forms, they can easily leak out of the vagina [ 2 ]. 3.5. Cervical Patches Another approach to the cervical delivery of a cytotoxic agent is using bioadhesive cervical patches [ 113 ]. …

Effect of a Medicinal Agaricus blazei Murill-Based Mushroom Extract, AndoSan™, on Symptoms, Fatigue and Quality of Life in Patients with Ulcerative Colitis in a Randomized Single-Blinded Placebo Controlled Study.

BackgroundIngestion of AndoSan™, based on the mushroom Agaricus blazei Murill, has previously been shown to exhibit anti-inflammatory effects because of reduction of pro-inflammatory cytokines in healthy individuals and patients with ulcerative colitis. In this randomized single-blinded placebo controlled study we examined whether intake of AndoSan™ also resulted in clinical effects.Methods and findings50 patients with symptomatic ulcerative colitis were block-randomized and blinded for oral daily intake of AndoSan™ or placebo for the 21 days' experimental period. The patients reported scores

Exploring Cost-Effectiveness of the Comprehensive Geriatric Assessment in Geriatric Oncology: A Narrative Review.

The Comprehensive Geriatric Assessment (CGA) and the corresponding geriatric interventions are beneficial for community-dwelling older persons in terms of reduced mortality, disability, institutionalisation and healthcare utilisation. However, the value of CGA in the management of older cancer patients both in terms of clinical outcomes and in cost-effectiveness remains to be fully established, and CGA is still far from being routinely implemented in geriatric oncology. This narrative review aims to analyse the available evidence on the cost-effectiveness of CGA adopted in geriatric oncology,

Associations of PD-L1 expression status, tumor histology and patient ethnic background with immunotherapy outcomes in patients with advanced esophageal cancer.

BackgroundCurrently, immune checkpoint inhibitors (ICIs) have become a mainstay in treating advanced esophageal cancer (EC), yet the predictive significance of routinely collected clinicopathological characteristics for overall survival (OS) remains uncertain.MethodsWe searched EMBASE, MEDLINE, Cochrane Library, Web of Science, and ClinicalTrials.gov for phase III randomized controlled trials (RCTs). Eligible studies reported OS for the overall population and subgroups stratified by various clinicopathological features. Pooled hazard ratios (HRs) and their 95% confidence intervals (CIs) were c

Иммунозащитное воздействие жидкости Кангфуксина на систематическую токсичность, связанную с аутологической трансплантацией стволовых клеток в множестве миеломы.

Цель Это исследование позволило оценить эффективность жидкости Канфуксина с точки зрения аутологической токсичности трансплантации стволовых клеток у пациентов с множественной миеломой. 82 участников, разделенных на простую группу по криотерапии (контрольная группа), в состав которой входили: 43 пациентов с множественными миэломами, перенесших аутлогическую трансплантацию стволовых клеток до мая 2023, и жидкость Канфуксина + пероральная криотерапия (экспериментальная группа), включая 39 пациенты с множественными миэломами, которые после июня прошли автологическую трансплантацию стволовых клето

Многоомичные биомаркеры, связанные с множественным склерозом: от менделианской случайности до прогнозирования наркотиков.

В настоящее время лечение и профилактика множественных склерозов (МС) по-прежнему сталкиваются с серьезными проблемами. Одним из важнейших методов проведения исследований в рамках реализации новых терапевтических стратегий стал менделийский анализ случайности (МСР). Соответственно, мы предполагаем, что существует причинно-следственная связь между генетическими вариантами конкретных плазменных белков и MS с помощью механизмов MR и что ключевые терапевтические цели можно точно определить путем интеграции многомерных аналитических подходов. В этом исследовании мы разработали всеобъемлющую аналити

Exploration of the quantitative-effectiveness association between acupuncture temporal parameters and chemotherapy-induced peripheral neuropathy in cancer patients: a dose-response meta-analysis of randomized controlled trials.

BackgroundChemotherapy-induced peripheral neuropathy (CIPN) is one of the commonly reported symptoms impacting cancer survivors. This study evaluated and compared the effectiveness of acupuncture treatments for CIPN.MethodsWe searched six databases from their inception to August 2024 to identify eligible randomized controlled trials (RCTs). Primary outcome were pain scores. Secondary outcomes were quality of life including FACT/GOG-Ntx and EORTC QLQ-C30. The robust error meta-regression (REMR) method was used to evaluate the dose-response relationship across treatment parameters, including num

A Review of Repurposed Cancer Drugs in Clinical Trials for Potential Treatment of COVID-19.

The pandemic of the coronavirus disease 2019 (COVID-19) represents an unprecedented challenge to identify effective drugs for prevention and treatment. While the world's attention is focused on news of COVID-19 vaccine updates, clinical management still requires improvement. Due to the similarity of cancer-induced inflammation, immune dysfunction, and coagulopathy to COVID-19, anticancer drugs, such as Interferon, Pembrolizumab or Bicalutamide, are already being tested in clinical trials for repurposing, alone or in combination. Given the rapid pace of scientific discovery and clinical data ge

Health-related quality of life in Croatian general population and multiple myeloma patients assessed by the EORTC QLQ-C30 and EORTC QLQ-MY20 questionnaires.

Background The impact of disease and treatment on the patient's overall well-being and functioning is a topic of growing interest in clinical research and practice. The aim of this study is to obtain reference data on quality of life of Croatian general population. Further, we aim to assess the impact of the disease and its primary systemic treatment on their health related quality of life (HrQoL) in multiple myeloma (MM) patients. Patients and methods Participants for the first part of the study were randomly selected from adult Croatian population. In the clinical part of the study MM patien

Hepatocellular carcinoma stem cells: the current state of small molecule-based inhibitors.

Hepatocellular carcinoma (HCC) is the most common type of liver cancer, accounting for over 90% of all cases. Patients with advanced-stage HCC are referred to systemic treatment. Although some advances in HCC therapy have been made in recent years, the prognosis for patients remains poor due to drug resistance, tumor relapse, and metastasis, implying that overall survival remains a challenge. Many studies have shown that tumor-initiating stem cells, also known as cancer stem cells (CSCs), play essential roles in tumorigenesis, metastasis, and treatment resistance in HCC and that future cancer

Exosomes for angiogenesis induction in ischemic disorders.

Ischaemic disorders are leading causes of morbidity and mortality worldwide. While the current therapeutic approaches have improved life expectancy and quality of life, they are unable to "cure" ischemic diseases and instate regeneration of damaged tissues. Exosomes are a class of extracellular vesicles with an average size of 100-150 nm, secreted by many cell types and considered a potent factor of cells for paracrine effects. Since exosomes contain multiple bioactive components such as growth factors, molecular intermediates of different intracellular pathways, microRNAs and nucleic acids, t

Computational Drug Repurposing Across the Multiple Myeloma Spectrum: From MGUS to MM.

Background/objectivesMultiple myeloma (MM) is a challenging, B cell malignancy characterised by the uncontrolled proliferation of plasma cells within the bone marrow. Despite significant advances in treatment options nowadays, MM remains an incurable malignancy, with the majority of patients succumbing to the disease. MM develops from a pre-malignant state known as monoclonal gammopathy of unknown significance (MGUS), which then has the potential to evolve either into smouldering (asymptomatic) multiple myeloma (SMM) or into MM. Since novel drug discovery takes years to reach the clinic, drug

A Review on the Molecular Mechanisms of Action of Natural Products in Preventing Bone Diseases.

The drugs used for treating bone diseases (BDs), at present, elicit hazardous side effects that include certain types of cancers and strokes, hence the ongoing quest for the discovery of alternatives with little or no side effects. Natural products (NPs), mainly of plant origin, have shown compelling promise in the treatments of BDs, with little or no side effects. However, the paucity in knowledge of the mechanisms behind their activities on bone remodeling has remained a hindrance to NPs' adoption. This review discusses the pathological development of some BDs, the NP-targeted components, an

Multimodal imaging diagnosis of multiple myeloma with renal involvement: a case report.

BackgroundExtramedullary renal involvement secondary to multiple myeloma (MM) is extremely rare and prone to preoperative misdiagnosis. Its clinical and imaging manifestations have not been systematically summarized, leading to frequent preoperative misdiagnosis. To summarize the multimodal imaging characteristics and clinical diagnostic value of MM with renal involvement through a typical case report, so as to improve the understanding and early recognition rate of this disease.Case presentationA 67-year-old male patient with left lumbodorsal pain was examined by multimodal imaging. Ultrasoun

ITF6475, a New Histone Deacetylase 6 Inhibitor, Prevents Painful Neuropathy Induced by Paclitaxel.

Chemotherapy-induced peripheral neuropathy remains a significant side effect of cancer treatment, often requiring dose reductions or even discontinuation of therapy. Paclitaxel (PTX), a widely used chemotherapeutic agent for solid tumors, is particularly neurotoxic, and no effective treatment exists for paclitaxel-induced peripheral neuropathy (PIPN). Histone deacetylases (HDACs) are enzymes that remove acetyl groups from histone and non-histone proteins, including transcription factors and cytoskeletal components. This study evaluates the HDAC6 inhibitor ITF6475 for its potential to prevent P

Optimizing Autologous Stem Cell Transplantation in Multiple Myeloma: The Significance of Pre-Transplant Controlling Nutritional Status Score.

Nutritional status is an important prognostic factor in patients with multiple myeloma (MM). The Controlling Nutritional Status (CONUT) score has shown promise in predicting outcomes in various malignancies; however, its role in autologous stem cell transplantation (ASCT) in patients with MM remains unclear. This study aimed to evaluate the significance of pre-transplant CONUT scores in predicting post-transplant engraftment kinetics and early complications in patients with MM undergoing ASCT. This single-center, retrospective study analyzed 59 multiple myeloma patients who underwent ASCT betw

Projection of age-standardised mortality rates for 23 cancer types in India: a nationwide projection until 2030 using autoregressive integrated moving average (ARIMA) models.

ObjectivesTo project the future burden of cancer mortality in India by forecasting age-standardised mortality rates (ASMRs) for 23 major cancer types up to the year 2030, providing crucial evidence for long-term health planning and resource allocation.DesignA retrospective analysis and time-series forecasting study. Participants Aggregated, national-level cancer mortality data for the population of India from 2000 to 2019 were used.ParticipantsAggregated, national-level cancer mortality data for the population of India from 2000 to 2019 were used.MethodsAnnual ASMR data for 23 cancer types wer