Conference scheduling — A personalized approach

Relevance. Today, in preventive medicine, climatic conditions that have a pathological effect on the functional state of a person are increasingly being updated. the occurrence of exacerbations of many diseases can be causally associated with various weather conditions. Aim: to develop the main tasks for improving the organization of medical care for weather-dependent patients with diseases of the cardiovascular system. Material and methods. The assessment of personnel, material and technical support and the main performance indicators of an outpatient clinic was carried out on the example of the Voronezh city polyclinic No. 18 to develop the main tasks for improving the organization of medical care for weather-dependent patients with diseases of the cardiovascular system. Results. The main personnel problem is the low staffing of district therapists and specialists of a narrow service. One of the priorities for reducing the burden on medical hospitals is the organization of inpatient replacement medical care on the basis of outpatient clinics. The indicators for the implementation of state guarantees for the outpatient network for 2018, which were fully implemented, are given. The analysis of the planned load performance by polyclinic specialists is presented. Cardiological and neurological services carry out measures to reduce the risk of exacerbations of diseases with cerebral atherosclerosis, hypertension, and major neurological nosologies. Conclusion. Improving the organization of medical care for weather-dependent patients with cardiovascular diseases are: informing patients about the sources of specialized medical weather forecasts in the region, organizing the work of the medical prevention office, implementing an interdepartmental approach to providing health care to the most vulnerable groups of the population.

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Proteomics of Pancreatic Neuroendocrine Tumors: A Systematic Review

Protein and Peptide Letters ◽

10.2174/0929866527666200504122116 ◽

2020 ◽

Vol 27 (12) ◽

pp. 1276-1287

Author(s):

Brigida Anna Maiorano ◽

Giovanni Schinzari ◽

Sabrina Chiloiro ◽

Felicia Visconti ◽

Domenico Milardi ◽

...

Keyword(s):

Systematic Review ◽

Neuroendocrine Tumors ◽

Prognostic Markers ◽

State Of The Art ◽

Therapeutic Targets ◽

The State ◽

Pancreatic Neuroendocrine Tumors ◽

Rare Tumors ◽

Personalized Approach ◽

Future Management

Pancreatic neuroendocrine tumors (PanNETs) are rare tumors having usually an indolent behavior, but sometimes with unpredictable aggressiveness. PanNETs are more often non-functioning (NF), unable to produce functioning hormones, while 10-30% present as functioning (F) - PanNETs, such as insulinomas , gastrinomas , and other rare tumors. Diagnostic and prognostic markers, but also new therapeutic targets, are still lacking. Proteomics techniques represent therefore promising approaches for the future management of PanNETs. We conducted a systematic review to summarize the state of the art of proteomics in PanNETs. A total of 9 studies were included, focusing both on NF- and F-PanNETs. Indeed, proteomics is useful for the diagnosis, the prognosis and the detection of therapeutic targets. However, further studies are required. It is also warranted to standardize the analysis methods and the collection techniques, in order to validate proteins with a relevance in the personalized approach to PanNETs management.

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New Strategies for Clinical Trials in Autism Spectrum Disorder

Reviews on Recent Clinical Trials ◽

10.2174/1574887115666201120093634 ◽

2020 ◽

Vol 15 ◽

Author(s):

Rini Pauly ◽

Catherine A. Ziats ◽

Ludovico Abenavoli ◽

Charles E. Schwartz ◽

Luigi Boccuto

Keyword(s):

Autism Spectrum Disorder ◽

Clinical Trials ◽

Autism Spectrum ◽

Spectrum Disorder ◽

Genetic Profiling ◽

Omics Technologies ◽

Molecular Abnormalities ◽

Sequencing Technologies ◽

Linear Relationships ◽

Personalized Approach

Background: Autism spectrum disorder (ASD) is a complex neurodevelopmental condition that poses several challenges in terms of clinical diagnosis and investigation of molecular etiology. The lack of knowledge on the pathogenic mechanisms underlying ASD has hampered the clinical trials that so far have tried to target ASD behavioral symptoms. In order to improve our understanding of the molecular abnormalities associated with ASD, a deeper and more extensive genetic profiling of targeted individuals with ASD was needed. Methods: The recent availability of new and more powerful sequencing technologies (third-generation sequencing) has allowed to develop novel strategies for characterization of comprehensive genetic profiles of individuals with ASD. In particular, this review will describe integrated approaches based on the combination of various omics technologies that will lead to a better stratification of targeted cohorts for the design of clinical trials in ASD. Results: In order to analyze the big data collected by assays such as whole genome, epigenome, transcriptome, and proteome, it is critical to develop an efficient computational infrastructure. Machine learning models are instrumental to identify non-linear relationships between the omics technologies and therefore establish a functional informative network among the different data sources. Conclusion: The potential advantage provided by these new integrated omics-based strategies is to better characterize the genetic background of ASD cohorts, identify novel molecular targets for drug development, and ultimately offer a more personalized approach in the design of clinical trials for ASD.

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Complications in Idiopathic Pulmonary Fibrosis: Focus on Their Clinical and Radiological Features

Diagnostics ◽

10.3390/diagnostics10070450 ◽

2020 ◽

Vol 10 (7) ◽

pp. 450

Author(s):

Federica Galioto ◽

Stefano Palmucci ◽

Giovanna M. Astuti ◽

Ada Vancheri ◽

Giulio Distefano ◽

...

Keyword(s):

Idiopathic Pulmonary Fibrosis ◽

Pulmonary Fibrosis ◽

Lung Disease ◽

Integrated Approach ◽

Correct Treatment ◽

Radiological Features ◽

Palliative Care Services ◽

Pictorial Essay ◽

Personalized Approach ◽

Fibrotic Lung Disease

Idiopathic pulmonary fibrosis (IPF) is a fibrotic lung disease with uncertain origins and pathogenesis; it represents the most common interstitial lung disease (ILD), associated with a pathological pattern of usual interstitial pneumonitis (UIP). This disease has a poor prognosis, having the most lethal prognosis among ILDs. In fact, the progressive fibrosis related to IPF could lead to the development of complications, such as acute exacerbation, lung cancer, infections, pneumothorax and pulmonary hypertension. Pneumologists, radiologists and pathologists play a key role in the identification of IPF disease, and in the characterization of its complications—which unfortunately increase disease mortality and reduce overall survival. The early identification of these complications is very important, and requires an integrated approach among specialists, in order to plane the correct treatment. In some cases, the degree of severity of patients having IPF complications may require a personalized approach, based on palliative care services. Therefore, in this paper, we have focused on clinical and radiological features of the complications that occurred in our IPF patients, providing a comprehensive and accurate pictorial essay for clinicians, radiologists and surgeons involved in their management.

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AB0267 HOW EFFECTIVE IS PAIN MANAGEMENT IN THE PATIENTS’ OPINION? DATA FROM THE COMPAS STUDY

Annals of the Rheumatic Diseases ◽

10.1136/annrheumdis-2021-eular.2806 ◽

2021 ◽

Vol 80 (Suppl 1) ◽

pp. 1160.1-1160

Author(s):

E. Pogozheva ◽

A. Karateev ◽

V. Amirdzhanova

Keyword(s):

Pain Management ◽

Pain Therapy ◽

Significant Proportion ◽

Connective Tissue Diseases ◽

Underlying Disease ◽

Additional Treatment ◽

Results Of Treatment ◽

Analgesic Therapy ◽

Folk Remedies ◽

Personalized Approach

Objectives:to evaluate the effectiveness and satisfaction of pain management in patients with rheumatic diseases (RD) according to a survey in the COMPAS (Quality of Pain Management according to Patients with Arthritis and Back pain) study.Methods:the survey involved 1040 patients with RD (rheumatoid arthritis-40.6%, osteoarthritis -32.1%, spondyloarthritis-10.6%, connective tissue diseases-8.6% of patients). 76.8% were women, the mean age was 55.8±14.0 years. 35.7% of patients continued to work in their specialty, 31.6% had various degrees of disability. The effectiveness of pain therapy was evaluated by the patient in the last month preceding the survey on a 5-point scale, where 1 - no effect and 5-excellent effect. Patients ‘ satisfaction with treatment, possible reasons for the lack of effectiveness of pain therapy and the use of additional treatment tools were also evaluated.Results:as therapy for the underlying disease, 40% of patients received conventional disease modifying antirheumatic drugs, 33.1% - glucocorticoids, 7.2% - biological agents and 15.2% - symptomatic slow-acting drugs in osteoarthritis. At the same time, 68% of patients needed additional analgesic therapy with nonsteroidal anti-inflammatory drugs (NSAIDs). Slightly less than half of the surveyed patients (46.9%) noted a moderate effect of analgesic therapy, 22.7% - a low effect and 5% - no effect, 23.7% rated the effectiveness of therapy as good and only 1.7% - as excellent. At the same time, only 15.6% of patients were completely satisfied with the result of NSAIDs, 64% were partially satisfied with the treatment and 20.4% were completely dissatisfied. As the reason of insufficient effectiveness of NSAIDs, most often (34.3%) patients named fear of adverse events associated with taking drugs, 19.4% - weak drugs, 15.3% - insufficient attention of doctors to complaints, 6.6% - poor diagnosis of the causes of pain. Others found it difficult to answer or were completely satisfied with the treatment. 40% of patients used additional methods, most often chiropractic (12.3%), acupuncture (4.8%), physiotherapy (12.7%) and folk remedies (7.4%).Conclusion:A significant proportion of patients with RD don’t have adequate pain control. Only 25.4% of patients rate the result of treatment as good and excellent, and even fewer patients (15.6%) are completely satisfied with the results of therapy. Thus, a personalized approach to analgesic therapy is necessary, taking into account the expectations of patients regarding the results of treatment.Disclosure of Interests:None declared

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LGG-18. EVEROLIMUS TREATMENT IN PEDIATRIC PATIENTS AFFECTED BY LOW-GRADE GLIOMAS (pLGG) NON-TSC, BRAF v600-WT

Neuro-Oncology ◽

10.1093/neuonc/noaa222.400 ◽

2020 ◽

Vol 22 (Supplement_3) ◽

pp. iii369-iii369

Author(s):

Antonella Cacchione ◽

Evelina Miele ◽

Maria Chiara Lodi ◽

Andrea Carai ◽

Giovanna Stefania Colafati ◽

...

Keyword(s):

Adverse Event ◽

Disease Progression ◽

Mapk Pathway ◽

Brain Mri ◽

Mammalian Target Of Rapamycin ◽

Low Grade ◽

Duration Of Treatment ◽

Tumor Biopsy ◽

Low Grade Gliomas ◽

Personalized Approach

Abstract BACKGROUND MAPK pathway is the hallmark of pediatric low grade gliomas (pLGGs); hyperactivation of mTOR (mammalian target of rapamycin) might be a suitable biomarker for therapeutic response. We investigated the feasibility of Everolimus, mTOR inhibitor, in patients affected by pLGGs. METHODS Patients 1 to 18 years old, diagnosed with pLGG, with a positive tumor biopsy for mTOR/phospho-mTOR and radiological and / or clinical disease progression, treated at Bambino Gesù Children’s Hospital in Rome were evaluated. Tumor DNA methylation analysis was performed in 10 cases. Exclusion criteria included: Tuberous Sclerosis patients, Sub Ependymal Giant Astrocytoma. Everolimus was administered orally at a dose of 2.5 mg or 5 mg daily based on body weight. Patients were evaluated with brain MRI every 4, 8 and 12 months after treatment start and every six months thereafter. RESULTS 16 patients were enrolled from September 2014 and 2019. The median age was 7.5 years old. All patients had at least one adverse event. Events rated as severe (grade 3/4) were reported in 6 patients. Stomatitis was the most frequent adverse event. One patient discontinued treatment due to grade 4 toxicity (ulcerative stomatitis and fatigue). The median duration of treatment was 21 months (4–57 months). Brain MRI evaluations have showed disease stability in 11 patients, partial response in 2 patients and disease progression in 3 patients. CONCLUSIONS Everolimus has proven to be well tolerated and effective treatment in terms of disease stability in patients with pLGGs. It’s also an excellent example of chemo-free personalized approach.

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Personalized Genetic Diagnosis of Congenital Heart Defects in Newborns

Journal of Personalized Medicine ◽

10.3390/jpm11060562 ◽

2021 ◽

Vol 11 (6) ◽

pp. 562

Author(s):

Olga María Diz ◽

Rocio Toro ◽

Sergi Cesar ◽

Olga Gomez ◽

Georgia Sarquella-Brugada ◽

...

Keyword(s):

Congenital Heart Disease ◽

Heart Disease ◽

Congenital Heart Defects ◽

Congenital Malformations ◽

Congenital Heart ◽

Heart Diseases ◽

Heart Defects ◽

Genetic Diagnosis ◽

Single Nucleotide Variants ◽

Personalized Approach

Congenital heart disease is a group of pathologies characterized by structural malformations of the heart or great vessels. These alterations occur during the embryonic period and are the most frequently observed severe congenital malformations, the main cause of neonatal mortality due to malformation, and the second most frequent congenital malformations overall after malformations of the central nervous system. The severity of different types of congenital heart disease varies depending on the combination of associated anatomical defects. The causes of these malformations are usually considered multifactorial, but genetic variants play a key role. Currently, use of high-throughput genetic technologies allows identification of pathogenic aneuploidies, deletions/duplications of large segments, as well as rare single nucleotide variants. The high incidence of congenital heart disease as well as the associated complications makes it necessary to establish a diagnosis as early as possible to adopt the most appropriate measures in a personalized approach. In this review, we provide an exhaustive update of the genetic bases of the most frequent congenital heart diseases as well as other syndromes associated with congenital heart defects, and how genetic data can be translated to clinical practice in a personalized approach.

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The art and science of drug titration

Therapeutic Advances in Drug Safety ◽

10.1177/2042098620958910 ◽

2020 ◽

Vol 11 ◽

pp. 204209862095891

Author(s):

Aisling R. Caffrey ◽

Eric P. Borrelli

Keyword(s):

Real World ◽

Therapeutic Index ◽

Clinical Expertise ◽

Coordination Of Care ◽

Patient Centered ◽

Narrow Therapeutic Index ◽

Drug Dosing ◽

Scientific Principles ◽

Suboptimal Adherence ◽

Personalized Approach

A “one-size-fits-all” approach has been the standard for drug dosing, in particular for agents with a wide therapeutic index. The scientific principles of drug titration, most commonly used for medications with a narrow therapeutic index, are to give the patient adequate and effective treatment, at the lowest dose possible, with the aim of minimizing unnecessary medication use and side effects. The art of drug titration involves the interplay of scientific drug titration principles with the clinical expertise of the healthcare provider, and an individualized, patient-centered partnership between the provider and the patient to review the delicate balance of perceived benefits and risks from both perspectives. Drug titration may occur as up-, down-, or cross-titration depending on whether the goal is to reach or maintain a therapeutic outcome, decrease the risk of adverse effects, or prevent withdrawal/discontinuation syndromes or recurrence of disease. Drug titration introduces additional complexities surrounding the conduct of clinical trials and real-world studies, confounding our understanding of the true effect of medications. In clinical practice, wide variations in titration schedules may exist due to a lack of evidence and consensus on titration approaches that achieve an optimal benefit-harm profile. Further, drug titration may be challenging for patients to follow, resulting in suboptimal adherence and may require increased healthcare-related visits and coordination of care amongst providers. Despite the challenges associated with drug titration, it is a personalized approach to drug dosing that blends science with art, and with supportive real-world outcomes-based evidence, can be effective for optimizing pharmacotherapeutic outcomes and improving drug safety.

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