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Treating rheumatic patients with a malignancy.

Management of patients with inflammatory rheumatic disease and a history of (or even a current) malignant disease poses some particular challenges. As direct evidence of the risk of (recurrent or de novo) malignancy in patients with a history of malignant disease is scarce, such a risk may be estimated indirectly from the principal carcinogenicity of the respective drug to be used or (also indirectly) from cancer reactivation data from the transplant literature. In general, cancer risk is increased in patients receiving combination immunosuppressive treatment, but the risk in patients receivin

The dark side of immunotherapy.

Immunotherapy has broadened the therapeutic scope and response for many cancer patients with drugs that are generally of higher efficacy and less toxicity than prior therapies. Multiple classes of immunotherapies such as targeted antibodies and immune checkpoint inhibitors (ICI), cell-based immunotherapies, immunomodulators, vaccines, and oncolytic viruses have been developed to help the immune system target and destroy malignant tumors. ICI targeting programmed cell death protein-1 (PD-1) or its ligand (PD-L1) are among the most effective immunotherapy agents and are a major focus of current

Hypophosphatasia: Results of a Country-Wide Selective Screening Program Using NGS Technology as a First-Tier Test.

Hypophosphatasia (HPP) is a rare hereditary metabolic disorder caused by nucleotide variants (NVs) in the ALPL gene, leading to a deficiency of tissue-nonspecific alkaline phosphatase (TNSALP). HPP has a variable clinical presentation (such as impaired mineralization of bones and teeth, respiratory dysfunctions, muscle weakness, muscle and bone pain, growth deficiency, nephrocalcinosis, seizures, encephalopathy, intracranial hypertension, and intellectual disability) overlapping with other disorders which often delays a correct diagnosis. In this study, we present the results of a two-center (

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… Notably, low ALP levels might be observed in case of many other conditions, namely celiac disease, Cushing syndrome, hypothyroidism, multiple myeloma, cardiac bypass surgery, Clofibrate therapy, osteogenesis imperfecta (OI) type 2, pernicious anemia, massive transfusion, milk-alkali syndrome, profound …

The burden and predictors of venous thromboembolic diseases in patients with multiple primary malignancies.

BackgroundVenous thromboembolism (VTE) represents a considerable burden on cancer patients' survival and quality of life, but this burden varies based on the patient's baseline characteristics and cancer-related factors. Although solid evidence on the predictors and effect of VTE in cancer patients exists.AimTo evaluate VTE rate, morbidity, and mortality to develop parameters that could predict VTEs and their associated mortality in patients with multiple primary malignancies (MPMs).Method and resultsThis was a retrospective cohort study that took place at King Abdulaziz Medical City, Riyadh,

Japan Atherosclerosis Society (JAS) Guidelines for Prevention of Atherosclerotic Cardiovascular Diseases 2022.

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Pathology Competencies in Medical Education and Educational Cases: Update 2023.

Pathology is a core component of medical school curricula because understanding the pathogenesis of the disease is foundational both for diagnostic efficiency and optimal use of ancillary resources in patient care. The Pathology Competencies for Medical Education (PCME) were developed as a national resource of expectations of pathology knowledge for medical students. The PCME are composed of three competencies: disease mechanisms and processes, organ system pathology, and diagnostic pathology and therapeutic pathology. The learning goals and learning objectives of the PCME that were first publ

Society for Immunotherapy of Cancer (SITC) clinical practice guideline on immune effector cell-related adverse events.

Immune effector cell (IEC) therapies offer durable and sustained remissions in significant numbers of patients with hematological cancers. While these unique immunotherapies have improved outcomes for pediatric and adult patients in a number of disease states, as 'living drugs,' their toxicity profiles, including cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), differ markedly from conventional cancer therapeutics. At the time of article preparation, the US Food and Drug Administration (FDA) has approved tisagenlecleucel, axicabtagene ciloleuc

Causes, clinical findings and therapeutic options in chylomicronemia syndrome, a special form of hypertriglyceridemia.

The prevalence of hypertriglyceridemia has been increasing worldwide. Attention is drawn to the fact that the frequency of a special hypertriglyceridemia entity, named chylomicronemia syndrome, is variable among its different forms. The monogenic form, termed familial chylomicronemia syndrome, is rare, occuring in 1 in every 1 million persons. On the other hand, the prevalence of the polygenic form of chylomicronemia syndrome is around 1:600. On the basis of the genetical alterations, other factors, such as obesity, alcohol consumption, uncontrolled diabetes mellitus and certain drugs may sign

Cancer Therapy-Induced Encephalitis.

Encephalitis associated with cancer therapies is a rare but serious complication that can significantly impact patients' quality of life and it requires prompt identification and management. Over the past two decades, immunotherapy-particularly immune checkpoint inhibitors-has become a cornerstone of cancer treatment, with up to half of metastatic cancer patients in economically developed countries now receiving these therapies. The widespread adoption of immunotherapy has led to improved survival rates and long-term remissions, even in patients with advanced metastatic disease. However, as im

Quantifying hope: an EU perspective of rare disease therapeutic space and market dynamics.

Rare diseases, affecting millions globally, pose a significant healthcare burden despite impacting a small population. While approximately 70% of all rare diseases are genetic and often begin in childhood, diagnosis remains slow and only 5% have approved treatments. The UN emphasizes improved access to primary care (diagnostic and potentially therapeutic) for these patients and their families. Next-generation sequencing (NGS) offers hope for earlier and more accurate diagnoses, potentially leading to preventative measures and targeted therapies. In here, we explore the therapeutic landscape fo

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… Some of the key indications targeted by ODD drugs are multiple myeloma, cystic fibrosis, spinal muscular atrophy, endogenous Cushing syndrome, hereditary angioedema and acute myeloid leukemia ( Figure 3F ). …

Neurotoxicity of Immunotherapy: Immune Checkpoint Inhibitor-Related Encephalitis vs. Immune Effector Cell-Associated Neurotoxicity Syndrome.

Immune checkpoint inhibitors and engineered T-cell therapies such as chimeric antigen receptor T-cell (CAR-T) cells and bispecific T-cell engagers (BiTEs) have revolutionized oncology care, and with them came two neurologic syndromes that look deceptively alike at the bedside with confusion, seizures, and encephalopathy: immune checkpoint inhibitor-related encephalitis (irEncephalitis) and immune effector cell-associated neurotoxicity syndrome (ICANS). Several differential observations between the two syndromes motivated this review: 1) although loss of immune tolerance likely drives irEncepha

Навигатор биомаркеров человека.

Биомаркеры играют ключевую роль в современных исследованиях медицины, особенно в ранней диагностике заболеваний и персонализированном лечении. Несмотря на то, что предыдущие исследования систематически обзоры маркеров в различных областях заболеваний, интегрированная система, которая связывает основные физиологические системы, охватывает множество органов и охватывает широкий спектр заболеваний, все еще отсутствует. В контексте современных вызовов здоровья, характеризующихся высокой распространенностью хронических заболеваний и широкими сопутствующими заболеваниями, создание панорамной системы

Глубокая стимуляция мозга в гипоталамической области: систематический обзор.

КОЛИЧЕСТВО: Глубокая стимуляция мозга (БРС) успешно используется для лечения циклопатии, включая движение, беспокойство и поведенческие расстройства. Гипоталамус является важнейшим интеграционным центром для многих периферийных и центральных путей, связанных с сердечно - сосудистыми, метаболическими и поведенческими функциями, и представляет собой потенциальную цель нейромодуляции в лечебных условиях. Проведение систематического обзора, изучение гипоталамических целей в ДБС, их признаков и основных клинических результатов.МЕТОДЫ: База данных PubMed, Scopus и Web of Science была подвергнута обы

Bone fragility and osteoporosis in children and young adults.

Osteoporosis is a metabolic bone disorder which increases fragility fracture risk. Elderly individuals, especially postmenopausal women, are particularly susceptible to osteoporosis. Although rare, osteoporosis in children and young adults is becoming increasingly evident, highlighting the need for timely diagnosis, management and follow-up. Early-onset osteoporosis is defined as the presence of a low BMD (Z-score of ≤ -2.0 in individuals aged < 20 years; T-score of ≤ -2.5 in those aged between 20 to 50 years) accompanied by a clinically significant fracture history, or the presence of low-ene

Диагностика и лечение ИГАН: стероиды, Будесонид, или, возможно, и то, и другое?

Нефропатия Ига (IgAN), наиболее часто встречающаяся в мире первичная гломерулонефрит, характеризуется мезангиальным залежем Ига и различными гистологическими повреждениями, как описано в Оксфордской системе классификации. Несмотря на хорошо описываемыеfour-Гипотеза, все еще существует множество менее или неописываемых механизмов, которые участвуют в болезнетворном патогенезе, например B-клеток, который, как представляется, инициируется различными антигенами в кишечном микробиоте. Диагноз болезни в настоящее время основан на результатах биопсии почек, поскольку чувствительность и специфичность

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… . | More side effects were recorded in this group (infections, weight gain, and Cushing syndrome). 42 (2nd group) | Oral administration of prednisone | Oral administration of 0.8–1.0 mg/kg of prednisone per day with a maximum daily dosage of 70 mg (full-dose treatment). | 6 months (this treatment was …

The Role of Drug Repurposing in the Development of Novel Antimicrobial Drugs: Non-Antibiotic Pharmacological Agents as Quorum Sensing-Inhibitors.

Background: The emergence of multidrug-resistant organisms (MDROs) is a global public health issue, severely hindering clinicians in administering appropriate antimicrobial therapy. Drug repurposing is a drug development strategy, during which new pharmacological applications are identified for already approved drugs. From the viewpoint of the development of virulence inhibitors, inhibition of quorum sensing (QS) is a promising route because various important features in bacterial physiology and virulence are mediated by QS-dependent gene expression. Methods: Forty-five pharmacological agents,

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… Examples of the success of drug repurposing include the use of thalidomide (morning sickness → multiple myeloma, erythema nodosum leprosum), minoxidil (hypertension → alopecia), ketoconazole (antifungal drug → Cushing syndrome), aspirin (analgesia → colorectal cancer), and sildenafil (angina pectoris …

Approach to the Patient With Bone Fracture: Making the First Fracture the Last.

The global burden of osteoporosis and osteoporotic fractures will increase significantly as we enter a rapidly aging population. Osteoporotic fractures lead to increased morbidity, mortality, and risk of subsequent fractures if left untreated. However, studies have shown that the majority of patients who suffer an osteoporotic fracture are not investigated or treated for osteoporosis, leading to an inexcusable "osteoporosis care gap." Systematic and coordinated models of care in secondary fracture prevention known as fracture liaison services (FLS) have been established to streamline and impro

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… A targeted physical examination should examine for evidence of vertebral fractures (eg, thoracic kyphosis, focal vertebral tenderness, increased wall-to-occiput distance > 0 cm, height loss) and causes of secondary osteoporosis (eg, hyperthyroidism, Cushing syndrome, hypogonadism). Table 1. …

Protective Effects of Omega-3 Fatty Acids in Cancer-Related Complications.

Omega-3 polyunsaturated fatty acids (PUFAs) are considered immunonutrients and are commonly used in the nutritional therapy of cancer patients due to their ample biological effects. Omega-3 PUFAs play essential roles in cell signaling and in the cell structure and fluidity of membranes. They participate in the resolution of inflammation and have anti-inflammatory and antinociceptive effects. Additionally, they can act as agonists of G protein-coupled receptors, namely, GPR40/FFA1 and GPR120/FFA4. Cancer patients undergo complications, such as anorexia-cachexia syndrome, pain, depression, and p

Bone Health for Gynaecologists.

Osteoporosis, the most common bone disorder, profoundly impacts women's health, especially during postmenopausal phases. Characterised by diminished bone mineral density (BMD), it increases the risk of fractures, affecting mobility, quality of life, and potentially mortality. The present review analyses the intricate interactions among physiological alterations, diseases, and medications that lead to bone mineral density reduction in women. It underscores the importance of gynaecologists in the prevention, diagnosis, and management of osteoporosis via early risk assessment, suitable hormone tr

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… Genetic diseases | Idiopathic juvenile osteoporosis Osteogenesis imperfecta Osteoporosis—pseudoglioma syndrome (OPPG) Hereditary enzyme deficiencies Turner syndrome Endocrine diseases | Hyperthyroidism Hypothyroidism Diabetes mellitus Hypogonadism Hyperparathyroidism Cushing syndrome Growth hormone deficiency …

Descriptive Analysis of Adverse Events Reported for New Multiple Myeloma Medications Using FDA Adverse Event Reporting System (FAERS) Databases from 2015 to 2022.

BackgroundNew multiple myeloma (MM) medications have revolutionized the treatment landscape, but they are also associated with a range of adverse events (AEs). This study aims to provide a comprehensive overview of AEs reported for four new MM medications: daratumumab, ixazomib, elotuzumab, and panobinostat.MethodsThis study uses a descriptive retrospective approach to analyze the FDA Adverse Event Reporting System (FAERS) from 2015 to 2022. It includes variables like medication names, report details, patient demographics, adverse events, and reporter types. The initial dataset consists of ove