263 results match your criteria: "Dmitry Rogachev National Medical Research Center of Pediatric Hematology[Affiliation]"

: Empirical antibacterial therapy for febrile neutropenia reduces mortality due to Gram-negative blood stream infections (BSIs). Pediatric guidelines recommend monotherapy with an antipseudomonal beta-lactam or a carbapenem and to add a second anti-Gram-negative agent in selected situations. We evaluated the changes in the proportions of resistance of beta-lactam monotherapies vs.

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  • The study examined lipid and carbohydrate metabolism as well as adipokines and growth factors in three mouse models with different metabolic disorders: alimentary obesity, leptin-resistant obesity, and diabetes mellitus.
  • In the alimentary obesity model, mice showed moderate liver fat, enlarged adipose tissue, and elevated levels of glucose, adiponectin, and cholesterol.
  • In the leptin-resistant model, severe tissue issues were noted, along with high blood sugar and leptin but low triglycerides and certain growth factors.
  • The diabetes mellitus model revealed a decrease in insulin-producing cells and lower levels of important hormones like adiponectin and leptin, along with high insulin levels.
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Personalization of a computational systems biology model of blood platelet calcium signaling.

Biomed Khim

December 2024

Center for Theoretical Problems of Physico-Chemical Pharmacology, Russian Academy of Sciences, Moscow, Russia; Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Moscow, Russia.

Anuclear blood cells, platelets, are the basis for the formation of blood clots in human vessels. While antiplatelet therapy is most often used after ischemic events, there is a need for its personalization due to the limited effectiveness and risks of bleeding. Previously, we developed a series of computational models to describe intracellular platelet signaling and a set of experimental methods to characterize the platelets of a given patient.

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Ionic liquids (ILs) are structurally tunable salts with applications ranging from chemical synthesis to batteries, novel materials and medicine. Despite their potential, the toxicity of ILs poses significant environmental and biological challenges. This study introduces a comprehensive dataset of cytotoxicity of 1227 ILs, compiled from 151 research papers and encompassing 3837 data entries.

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The molecular classification of endometrial cancer developed by The Cancer Genome Atlas project (TCGA, 2013) is currently actively used in gynecological oncology. According to it, endometrial carcinoma is divided into four molecular subtypes: -mutated, MMR deficient (dMMR), -aberrant and unspecified. Endometrial cancer samples belonging to the dMMR and -mutant types are characterized by specific genetic profiles reflecting the hyper- and ultramutant phenotypes of the tumor.

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Unlabelled: Rhabdomyosarcomas (RMS) are one of the most common types of sarcomas in children and adolescents. The alveolar RMS subgroup is of particular interest because in some cases, the translocation of the and genes is combined with an amplification of the corresponding hybrid gene. According to literature data, the frequency of the translocation is 70-90% and the translocation 10-30%.

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Unlabelled: Tumor tissue samples are necessary for histological diagnosis. Biopsy is associated with certain difficulties, especially in neuro-oncology. An alternative approach (liquid biopsy) is currently being developed.

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Timing matters: diurnal spine length variation in pediatric patients during radiotherapy.

Radiat Oncol

November 2024

Department of Radiation Oncology, Cancer Center Amsterdam, Amsterdam UMC, Amsterdam University Medical Center, University of Amsterdam, Amsterdam, The Netherlands.

Background: During the day-night cycle, gravity and applied stress to the body mass and spine causes a decrease in body height, which is restored overnight. This diurnal spine length variation has not yet been quantified during radiotherapy. Therefore, we aimed to quantify diurnal spine length variation on cone beam CTs (CBCTs) of pediatric patients (< 18 years) who underwent radiotherapy.

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Recent advancements in experimental and computational methods for RNA secondary structure detection have revealed the crucial role of RNA structural elements in diverse molecular processes within living cells. It has been demonstrated that the secondary structure of the entire viral genome is often responsible for performing crucial functions in the viral life cycle and also influences virus evolution. To investigate the role of viral RNA secondary structure, alongside experimental techniques, the use of bioinformatics tools is important for analyzing various secondary structure patterns, including hairpin loops, internal loops, multifurcations, external loops, bulges, stems, and pseudoknots.

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  • Activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) is a rare genetic disease that disrupts the immune system, causing various symptoms that usually begin in childhood.
  • In a phase III clinical trial, a drug called leniolisib was shown to effectively reduce lymph node swelling and increase naïve B cell levels in both adolescents and adults with APDS compared to those given a placebo.
  • The study found that leniolisib was well-tolerated across age groups and suggests it could be a promising treatment option for managing APDS by addressing the underlying cause rather than just the symptoms.
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  • * A pilot screening program in Russia analyzed 202,908 newborns, finding 157 with trisomy 21, indicating a birth prevalence of 1:1,284, and explored their lymphocyte levels (TREC and KREC).
  • * Results showed reduced TREC values in DS newborns, similar to extremely preterm infants, and significant KREC differences from the general newborn population, highlighting the need for deeper research into immune development in DS and better support from healthcare teams.
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The increasing need to understand and control the environmental impact of chemical processes has revealed the challenge in efficient evaluation of toxicity of the vast number of chemical compounds and their varying effects on biological systems. In this study, we introduce "Build-a-bio-Strip", a novel online service designed to carry out a quick initial analysis of the toxic impact of chemical processes. This platform enables users to automatically generate toxicity characteristics of chemical reactions using their own data on cytotoxicity or median lethal doses of the substances involved or computational predictions based on SMILES strings.

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Purpose: Posterior fossa tumors account for half of all childhood brain tumors, prompting the search for effective and affordable interventions to combat the neurocognitive and motor sequelae of the tumor and its treatment. The main aim of this pilot study was to evaluate the feasibility and effects of sensorimotor training incorporating cognitive tasks for a group of pediatric survivors of posterior fossa tumors.

Materials And Methods: A total of 48 participants (M= 12.

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Here we explored new 1,5-disubstituted pyrrolidin-2-ones 1, 2 and 5-aryl-3,3,4,5-tetrahydropyrrolo[1,2-]quinoline-1(2)-ones 3 as inhibitors of tubulin polymerization. We evaluated their effects on microtubule dynamics and on the proliferation of A549 cells, using flow cytometry-based cell cycle analysis. The results were verified with phase-contrast microscopy in three cancer cell lines: A549, HeLa and MCF-7.

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  • A 13-year-old child was found to have a rare epithelioid neoplasm located in the pterygopalatine fossa, which featured a specific genetic fusion (PANX3::GLI1) and MDM2 amplification.
  • The identified gene fusion contributes to the tumor's signs of bone-forming differentiation, which complicates its diagnosis.
  • This case is the first of its kind to report these genetic features and adds to the understanding of rare tumors involving GLI1 rearrangements.
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Pediatric low-hypodiploidy B-cell acute lymphoblastic leukemia (LH-ALL) with variants has been proposed to be considered a manifestation of Li-Fraumeni syndrome (LFS). However, our study demonstrates that of the majority the pathogenic variants in the TP53 gene are somatic (70.5%), and only 12.

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  • A male newborn was found to have low TREC values, indicating T cell lymphopenia, and genetic testing revealed a mutation linked to Ectrodactyly-Ectodermal Dysplasia-Cleft lip/palate syndrome (EEC).
  • This case underscores the importance of further research on the immune system issues related to mutations in the TP63 gene, suggesting that patients with such mutations should undergo thorough immunological assessments.
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DICER1-Related Tumor Predisposition: Identification of At-risk Individuals and Recommended Surveillance Strategies.

Clin Cancer Res

December 2024

International Pleuropulmonary Blastoma/DICER1 Registry, Children's Minnesota, Minneapolis, Minnesota.

Article Synopsis
  • DICER1-related tumor predisposition is linked to an increased risk of both benign and malignant tumors, prompting the need for guidelines on testing and imaging for affected individuals.
  • A study enrolled participants from various registries and identified 713 individuals with germline DICER1 variants, leading to the diagnosis of multiple cases of pleuropulmonary blastoma (PPB) and ovarian Sertoli-Leydig cell tumors (SLCT).
  • The findings suggest that early imaging and surveillance can help detect PPB and may lower the risk of advanced disease, leading to recommendations for earlier ovarian surveillance beginning at the detection of DICER1 variants.
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  • Mesenchymal stromal cells (MSCs) have significant potential in regenerative medicine but face challenges in classification, particularly for those from the pancreas; this study aims to better characterize pancreatic islet-derived MSCs (hPD-MSCs).
  • The hPD-MSCs were isolated from donor islets and analyzed through various methods, confirming they possess key MSC features and can differentiate into fat and bone cells, with unique gene expression patterns compared to MSCs from other tissues.
  • The findings reveal that hPD-MSCs share similarities with pancreatic stellate cells and identify specific genes related to pancreatic and neural functions, enhancing the understanding of their therapeutic potential.
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  • - This study compares the outcomes of Central Liver Segments Resection (CLSR) and Extended Hepatectomy (EH) in children with liver tumors, noting that CLSR is less common due to its technical complexity yet is successful in adults.
  • - The research involved analyzing data from 14 patients who underwent CLSR and 44 who had EH, focusing on factors like liver remnant volume, blood loss, operation time, and postoperative complications.
  • - Results showed that while CLSR preserves more liver tissue and has similar intraoperative and postoperative outcomes compared to EH, it takes longer to perform; both methods have comparable complication rates.
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Peptides from heptad repeat (HR1 and HR2) regions of gp41 are effective inhibitors of HIV-1 entry that block the fusion of viral and cellular membranes, but the generation of antibodies highly specific for these peptides is challenging. We have previously described a mouse hybridoma that recognizes MT-C34-related peptides derived from HR2. It was used for the selection of HIV-1-resistant CD4 lymphocytes engineered to express the MT-C34 peptide via a CRISPR/Cas9-mediated knock-in into the locus.

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Purpose: This study aimed to evaluate the ability of 18 F-FDG PET/CT metabolic parameters to predict the histological response to neoadjuvant chemotherapy in patients with osteosarcoma and Ewing sarcoma.

Patients And Methods: This systematic review and network meta-analysis adhered to the PRISMA-NMA and Cochrane guidelines. Electronic databases were searched from January 2008 to January 2024; this search was supplemented by snowballing methods.

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Cisplatin is one of the most well-known anti-cancer drugs and has demonstrated efficacy against numerous tumor types for many decades. However, a key challenge with cisplatin, as with any chemotherapeutic agent, is the development of resistance with a resultant loss of efficacy. This resistance is often associated with metabolic alterations that allow insensitive cells to divide and survive under treatment.

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Hematopoietic stem cell transplantation for CTLA-4 insufficiency across Europe: A European Society for Blood and Marrow Transplantation Inborn Errors Working Party study.

J Allergy Clin Immunol

December 2024

Paediatric Haematopoietic Stem Cell Transplant Unit, Great North Children's Hospital, Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom; Translational and Clinical Research Institute, Newcastle University, Newcastle upon Tyne, United Kingdom.

Background: Cytotoxic T-lymphocyte antigen 4 (CTLA-4) insufficiency causes a primary immune regulatory disorder characterized by lymphoproliferation, dysgammaglobulinemia, and multiorgan autoimmunity including cytopenias and colitis.

Objective: We examined the outcome of hematopoietic stem cell transplantation (HSCT) for CTLA-4 insufficiency and study the impact of pre-HSCT CTLA-4 fusion protein (CTLA-4-Ig) therapy and pre-HSCT immune dysregulation on survival and immunologic outcome.

Methods: This was a retrospective study of HSCT for CTLA-4 insufficiency and 2q33.

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Allogeneic Hematopoietic Stem Cell Transplantation in Immunodeficiency-Centromeric Instability-Facial Dysmorphism (ICF) Syndrome: an EBMT/ESID Inborn Errors Working Party Study.

J Clin Immunol

August 2024

Willem-Alexander Children's Hospital, Department of Pediatrics, Division of Pediatric Immunology, Hematology and Stem Cell Transplantation, Leiden University Medical Center, Leiden, The Netherlands.

Immunodeficiency-Centromeric instability-Facial dysmorphism (ICF) syndrome is an inborn error of immunity characterized by progressive immune dysfunction and multi-organ disease usually treated with antimicrobial prophylaxis and immunoglobulin substitution. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative treatment, but data on outcome are scarce. We provide a detailed description of disease characteristics and HSCT outcome in an international cohort of ICF syndrome patients.

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