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showing 10 items of 798 documents

GERMLINE PROKINETICIN RECEPTOR 2 (PROKR2) VARIANTS ASSOCIATED WITH CENTRAL HYPOGONADISM CAUSE DIFFERENTAL MODULATION OF DISTINCT INTRACELLULAR PATHWA…

2013

INTRODUCTION: Defects of prokineticin pathway affect the neuroendocrine control of reproduction, but their role in the pathogenesis of central hypogonadism remains undefined, and the functional impact of the missense PROKR2 variants has been incompletely characterized. MATERIAL AND METHODS: In a series of 246 idiopathic central hypogonadism patients, we found three novel (p.V158I, p.V334M, and p.N15TfsX30) and six already known (p.L173R, p.T260M, p.R268C, p.V274D, p.V331M, and p.H20MfsX23) germline variants in the PROKR2 gene. We evaluated the effects of seven missense alterations on two different prokineticin receptor 2 (PROKR2)-dependent pathways: inositol phosphate-Ca(2+) (Gq coupling) a…

MaleKallmann syndromeEndocrinology Diabetes and MetabolismClinical BiochemistryInositol Phosphatemedicine.disease_causeBiochemistryHypogonadotropic hypogonadismGermlineReceptors G-Protein-CoupledCohort StudiesEndocrinologySettore MED/38 - Pediatria Generale E SpecialisticaAdolescent; Adult; Child; Cohort Studies; Cyclic AMP; Female; Genetic Association Studies; Humans; Hypogonadism; Inositol Phosphates; Male; Middle Aged; Mutation Missense; Receptors G-Protein-Coupled; Receptors Peptide; Signal Transduction; Young Adult; Germ-Line MutationReceptorsCyclic AMPmutations; Kallmann syndrome; septo-optic dysplasiaMissense mutationReceptorChildMutationMiddle AgedProkineticinPeptideFemaleHumanSignal TransductionAdultmedicine.medical_specialtyReceptors PeptideAdolescentAdolescent Adult Child Cohort Studies Cyclic AMP; metabolism Female Genetic Association Studies Germ-Line Mutation Humans Hypogonadism; epidemiology/genetics Inositol Phosphates; metabolism Male Middle Aged Missense Receptors; G-Protein-Coupled; genetics Receptors; Peptide; genetics Signal Transduction; genetics Young AdultInositol PhosphatesMutation MissenseGenetic Association StudieBiologyG-Protein-CoupledYoung AdultGermline mutationInternal medicinesepto-optic dysplasiamedicineHumansGenetic Association StudiesGerm-Line MutationHypogonadismBiochemistry (medical)Kallmann syndromeProkineticin receptor 2medicine.diseasePROKR2 hypogonadism prokineticinmutationsAdolescent; Adult; Child; Cohort Studies; Cyclic AMP; Female; Genetic Association Studies; Humans; Hypogonadism; Inositol Phosphates; Male; Middle Aged; Mutation; Missense; Receptors; G-Protein-Coupled; Peptide; Signal Transduction; Young Adult; Germ-Line MutationEndocrinologyMutationCohort StudieMissense
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Impaired GH secretion in patients with SHOX deficiency and efficacy of recombinant human GH therapy.

2012

<b><i>Background/Aims:</i></b> Mutations of the short stature homeobox-containing <i>(SHOX)</i> gene on the pseudoautosomal region of the sex chromosomes cause short stature. GH treatment has been recently proposed to improve height in short patients with SHOX deficiency. The aim of this study was to evaluate GH secretion and analyze growth and safety of recombinant human GH (rhGH) therapy in short children and adolescents with SHOX deficiency. <b><i>Patients and Design:</i></b> We studied 16 patients (10 females; 9.7 ± 2.9 years old; height –2.46 ± 0.82 standard deviation score, SDS) with SHOX deficiency. All subjects underwent au…

MaleLanger-Giedion SyndromeEndocrinology Diabetes and MetabolismSHOX deficiencyPseudoautosomal regionMadelung deformityLer Weill syndromelaw.inventionEndocrinologySettore MED/38 - Pediatria Generale E SpecialisticaShort Stature Homeobox ProteinGH treatmentShort Stature Homeobox ProteinlawSHOX DeficiencyChildGrowth DisordersHuman Growth HormoneGrowth hormone secretionRecombinant ProteinsGHRecombinant Human GHChild PreschoolRecombinant DNAFemalemedicine.symptomSHOX Deficiencymedicine.medical_specialtyAdolescentNoseOsteochondrodysplasiasShort statureFingersInternal medicinemedicineHumansLéri–Weill dyschondrosteosisGeneLeri-Weill dyschondrosteosiHomeodomain Proteinsbusiness.industrymedicine.diseaseBody HeightSHOX Deficiency; Ler Weill syndrome; Recombinant Human GHShort statureEndocrinologyGrowth HormonePediatrics Perinatology and Child HealthbusinessHair DiseasesSHOX
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Children with special health care needs attending emergency department in Italy: analysis of 3479 cases

2020

Abstract Background Although children with special health care needs (CSHCN) represent a minority of the population, they go through more hospitalizations, more admissions to the Emergency Department (ED), and receive a major number of medical prescriptions, in comparison to general pediatric population. Objectives of the study were to determine the reasons for admission to the ED in Italian CSHCN, and to describe the association between patient’s demographic data, clinical history, and health services requirements. Methods Ad hoc web site was created to collect retrospective data of 3479 visits of CSHCN to the ED in 58 Italian Hospitals. Results Seventy-two percent of patients admitted to …

MaleMetabolic diseaseHospitalization rateCongenital skeletal conditionHospitalization rateChildren with special health care needs; Congenital skeletal condition; Emergency department; Hospitalization rate; Isolated CNS malformation; Metabolic diseases; Multiple AED therapy; Neuromuscular diseases; Syndromic disorders; True isolated microcephaly0302 clinical medicineClinical historyMedicineChildeducation.field_of_studyNeuromuscular diseaseSettore MED/38Disabled ChildrenHospitalizationNeuromuscular diseasesSettore MED/38 - PEDIATRIA GENERALE E SPECIALISTICAItalyChild PreschoolFemaleChildren with special health care needEmergency Service Hospitalmedicine.medical_specialtyAdolescentPopulationTriage CodeChildren with special health care needsSyndromic disordersChildren with special health care needs03 medical and health sciencesPharmacotherapy030225 pediatricsHumansMedical prescriptioneducationRetrospective StudiesHealth Services Needs and DemandSyndromic disorderEmergency departmentTrue isolated microcephalybusiness.industryResearchInfant NewbornInfantMetabolic diseases030208 emergency & critical care medicineEmergency departmentChildren with special health care needs Congenital skeletal conditionsEmergency department Hospitalization rate Isolated CNS malformation Metabolic diseases Multiple AED therapy Neuromuscular diseases Syndromic disorders True isolated microcephalyFamily medicineChronic DiseaseMultiple AED therapyIsolated CNS malformationbusinessFacilities and Services UtilizationItalian Journal of Pediatrics
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tst1-positive ST22-MRSA-IVa in healthy Italian preschool children

2013

A survey was performed in May 2013 to assess methicillin-resistant Staphylococcus aureus (MRSA) nasal colonization in healthy children attending 26 municipal daycare centres in Palermo, Italy. Of the 500 children, ten (2 %) tested positive. Eight MRSA isolates were tst1-positive ST22-MRSA-IVa, spa t223; the other two isolates were identified as ST1-IVa and ST398-V, respectively. tst1-positive ST22-MRSA, spa t223 has been previously identified only in the Middle Eastern area.

MaleMethicillin-Resistant Staphylococcus aureusMicrobiology (medical)Pediatricsmedicine.medical_specialtySettore MED/07 - Microbiologia E Microbiologia ClinicaGenotypeMEDLINEmedicine.disease_causeSettore MED/42 - Igiene Generale E ApplicataMolecular typingSettore MED/38 - Pediatria Generale E SpecialisticaGenotypemedicineHumansHealthy childrenNasal colonizationSicilyMolecular EpidemiologyMolecular epidemiologyMiddle Eastern areabusiness.industryData CollectionGeneral MedicineStaphylococcal Infectionsbiochemical phenomena metabolism and nutritionbacterial infections and mycosesST22-MRSA-IVaMolecular TypingInfectious DiseasesStaphylococcus aureusChild PreschoolCarrier StateDaycare centerFemalebusiness
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Methicillin-resistant Staphylococcus aureus colonization: a three-year prospective study in a neonatal intensive care unit in Italy.

2014

Background: Methicillin resistant Staphylococcus aureus (MRSA) is a major etiological agent of infection in neonatal intensive care units (NICUs). Routes of entry of this organism can be different and the transmission pathway complex. Colonized neonates are the main endogenous reservoir. Methods and Results: We conducted a prospective three-year study on MRSA colonization recruiting 722 neonates admitted between 2009 and 2012. Nasal swabs were cultured weekly and MRSA isolates were submitted to molecular typing. The annual incidence density of acquisition of MRSA ranged from a maximum of 20.2 cases for 1000 patient-days during the first year to a minimum of 8.8 cases in the second one to ra…

MaleMethicillin-Resistant Staphylococcus aureusSettore MED/07 - Microbiologia E Microbiologia ClinicaPediatricsmedicine.medical_specialtyMeticillinNeonatal intensive care unitInfectious Disease ControlClinical Research DesignEpidemiologyBacterial diseasesPrevalenceColony Count Microbiallcsh:MedicineDrug resistanceSettore MED/42 - Igiene Generale E Applicatamedicine.disease_causePediatricsInfectious Disease EpidemiologySettore MED/01 - Statistica MedicaSettore MED/38 - Pediatria Generale E SpecialisticaIntensive careIntensive Care Units NeonatalNosocomial infectionsmedicineHumansColonizationProspective Studieslcsh:ScienceMolecular EpidemiologyMultidisciplinarybusiness.industrylcsh:RInfant NewbornOutbreakStaphylococcal Infectionsbacterial infections and mycosesMethicillin-resistant Staphylococcus aureusMolecular TypingMethicillin resistant Staphylococcus aureus neonatal intensive care unitMedicineInfectious diseasesFemalelcsh:QNeonatologybusinessmedicine.drugResearch ArticlePLoS ONE
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Recurrent missense variant in the nuclear export signal of FMR1 associated with FXS-like phenotype including intellectual disability, ASD, facial abn…

2021

Fragile X syndrome (FXS; MIM 300624) is an X-linked genetic disorder characterized by physical abnormalities associated with intellectual disability and a wide spectrum of neurological and psychiatric impairments. FXS occurs more frequently in males, 1 in 5000 males and 1 in 8000 females accounting for 1-2% of overall intellectual disability (ID). In more than 99% of patients, FXS results from expansions of a CGG triplet repeat (>200 in male) of the FMR1 gene. In the last years an increasing number, albeit still limited, of FXS subjects carrying FMR1 mutations including deletions, splicing errors, missense, and nonsense variants was reported. Nevertheless, the studies concerning the func…

MaleNuclear Export SignalsSettore M-PSI/02 - Psicobiologia E Psicologia FisiologicaAutism Spectrum DisorderMutation MissenseGeneral MedicineFMR1 point mutationSettore MED/39 - Neuropsichiatria InfantileFragile X Mental Retardation ProteinPhenotypeSettore MED/38 - Pediatria Generale E SpecialisticaIntellectual DisabilityAutism spectrum disorders ASDSettore M-PSI/08 - Psicologia ClinicaGeneticsHumansIntellectual disability IDFemaleNuclear export signal NES.Genetics (clinical)Fragile X syndrome
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The prognostic value of biological markers in paediatric Hodgkin lymphoma

2015

Abstract Background Many biological and inflammatory markers have been proposed as having a prognostic value at diagnosis of Hodgkin lymphoma (HL), but very few have been validated in paediatric patients. We explored the significance of these markers in a large population of 769 affected children. Patients and methods By using the database of patients enrolled in A.I.E.O.P. (Associazione Italiana di Emato-Oncologia Pediatrica) trial LH2004 for paediatric HL, we identified 769 consecutive patients treated with curative intent from 1st June 2004 to 1st April 2014 with ABVD (doxorubicin, bleomycin, vinblastine, and dacarbazine), or hybrid COPP/ABV (cyclophosphamide, vincristine, prednisone, pr…

MaleOncologyCancer ResearchPathologyTime FactorsDatabases Factualmedicine.medical_treatmenthodgkin lymphoma; paediatric; prognostic factorHodgkin lymphoma; Paediatric; Prognostic factor; Adolescent; Age Factors; Antineoplastic Combined Chemotherapy Protocols; Biomarkers Tumor; Child; Child Preschool; Databases Factual; Disease Progression; Disease-Free Survival; Female; Ferritins; Hodgkin Disease; Humans; Infant; Infant Newborn; Italy; Kaplan-Meier Estimate; Leukocyte Count; Male; Multivariate Analysis; Neoplasm Staging; Platelet Count; Predictive Value of Tests; Proportional Hazards Models; Retrospective Studies; Risk Factors; Time Factors; Treatment Outcome; Blood Platelets; EosinophilsKaplan-Meier EstimateProcarbazineLeukocyte Countchemistry.chemical_compound0302 clinical medicineRisk FactorsPrednisoneAntineoplastic Combined Chemotherapy ProtocolsChildPrognostic factorTumorAge FactorsHodgkin DiseaseVinblastineTreatment OutcomeSettore MED/38 - PEDIATRIA GENERALE E SPECIALISTICAItalyOncologyPaediatricChild Preschool030220 oncology & carcinogenesisDisease ProgressionFemalemedicine.drugBlood Plateletsmedicine.medical_specialtyVincristineAdolescentDacarbazineBleomycinDisease-Free SurvivalDatabases03 medical and health sciencesPredictive Value of TestsInternal medicineBiomarkers TumormedicineHumansPreschoolFactualNeoplasm StagingProportional Hazards ModelsRetrospective StudiesChemotherapyPlatelet Countbusiness.industryInfant NewbornInfantNewbornEosinophilschemistryABVDFerritinsMultivariate AnalysisbusinessBiomarkersHodgkin lymphoma030215 immunologyEuropean Journal of Cancer
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Clinical Significance of Rare Copy Number Variations in Epilepsy A Case-Control Survey Using Microarray-Based Comparative Genomic Hybridization

2012

Objective To perform an extensive search for genomic rearrangements by microarray-based comparative genomic hybridization in patients with epilepsy. Design Prospective cohort study. Setting Epilepsy centers in Italy. Patients Two hundred seventy-nine patients with unexplained epilepsy, 265 individuals with nonsyndromic mental retardation but no epilepsy, and 246 healthy control subjects were screened by microarray-based comparative genomic hybridization. Main Outcomes Measures Identification of copy number variations (CNVs) and gene enrichment. Results Rare CNVs occurred in 26 patients (9.3%) and 16 healthy control subjects (6.5%) (P = .26). The CNVs identified in patients were larger (P = …

MaleOncologyendocrine system diseasesMicroarrayGene DosagePreschool Cohort Studies Computational Biology Diagnostic and Statistical Manual of Mental Disorders EpilepsyBioinformaticsPolymerase Chain ReactionFluorescence Intellectual DisabilityCohort StudiesEpilepsySettore MED/38 - Pediatria Generale E SpecialisticaGene DuplicationProspective StudiesCopy-number variationAge of OnsetChildProspective cohort studyIn Situ Hybridization Fluorescenceepidemiology/genetics Nucleic Acid Hybridization Polymerase Chain Reaction Prospective Studies Young AdultGene RearrangementNucleic Acid HybridizationMiddle AgedControl subjectsMagnetic Resonance ImagingDiagnostic and Statistical Manual of Mental Disordersgenetics Female Gene Deletion Gene Dosage Gene Duplication Gene Rearrangement Genome-Wide Association Study Humans In Situ HybridizationItalyRare Copy Number Variations EpilepsyChild PreschoolFemaleepidemiology/genetics ItalyAdultmedicine.medical_specialtyAdolescentBiologyYoung AdultAdolescent Adult Age of Onset Aged Child ChildArts and Humanities (miscellaneous)Intellectual DisabilityInternal medicinemental disordersmedicineHumansIn patientClinical significanceepidemiology Magnetic Resonance Imaging Male Microarray Analysis Middle Aged Nervous System DiseaseAgedEpilepsyComputational BiologyMicroarray Analysismedicine.diseaseSettore MED/03 - Genetica MedicaNeurology (clinical)Nervous System DiseasesGene DeletionGenome-Wide Association StudyComparative genomic hybridization
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Point of view of the Italians pediatric scientific societies about the pediatric care during the COVID-19 lockdown: What has changed and future prosp…

2020

Abstract Background The coronavirus disease 2019 (COVID-19) is currently rare in children and they seem to have a milder disease course and better prognosis than adults. However, SARS-Cov-2 pandemic has indirectly caused problems in pediatric medical assistance. In view of this we wanted to draw a picture of what happened during health emergency and analyze future prospects for restarting. Methods We involved the Italian pediatric scientific societies institutionally collected in the Italian Federation of Associations and Scientific Societies of the Pediatric Area (FIARPED); We sent a questionnaire to all scientific societies about the pediatric care activity during the COVID-19 emergency a…

MaleOutcome AssessmentPediatricsPatient Care PlanningSurveys and QuestionnairesPandemicOutcome Assessment Health CareAmbulatory CareMedicineViralChildChildrenSocieties MedicalPediatricEmergency Servicelcsh:RJ1-570children; COVID-19; pediatric assistance; telemedicineSettore MED/38TelemedicineHospitalizationOutpatient visitsItalySettore MED/38 - PEDIATRIA GENERALE E SPECIALISTICAQuarantineContinuity of careFemalePediatric careCoronavirus InfectionsEmergency Service HospitalHumanAdultmedicine.medical_specialtyTelemedicineCoronavirus disease 2019 (COVID-19)Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)Pneumonia ViralDisease courseHospitalMedicalHumansPandemicsInfection ControlPediatric assistancePandemicbusiness.industryCoronavirus InfectionCOVID-19lcsh:PediatricsPneumoniaHealth CareFamily medicineChildren; COVID-19; Pediatric assistance; Telemedicine; Adult; Ambulatory Care; Child; Coronavirus Infections; Delivery of Health Care; Emergency Service Hospital; Female; Hospitalization; Humans; Infection Control; Italy; Male; Outcome Assessment Health Care; Pandemics; Patient Care Planning; Pediatrics; Pneumonia Viral; Quarantine; Societies Medical; Telemedicine; Surveys and QuestionnairesbusinessSocietiesDelivery of Health Care
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Clinical features and follow-up in patients with 22q11.2 deletion syndrome

2014

Objective To investigate the clinical manifestations at diagnosis and during follow-up in patients with 22q11.2 deletion syndrome to better define the natural history of the disease. Study design A retrospective and prospective multicenter study was conducted with 228 patients in the context of the Italian Network for Primary Immunodeficiencies. Clinical diagnosis was confirmed by cytogenetic or molecular analysis. Results The cohort consisted of 112 males and 116 females; median age at diagnosis was 4 months (range 0 to 36 years 10 months). The diagnosis was made before 2 years of age in 71% of patients, predominantly related to the presence of heart anomalies and neonatal hypocalcemia. In…

MalePediatrics22q11.2 deletionDelayed DiagnosisTime FactorsChromosomes Human Pair 22Developmental Disabilitiesdigeorge syndromeSex FactorSeverity of Illness IndexRetrospective StudieDiGeorge syndromeEarly DiagnosiAge FactorProspective StudiesNeonatal hypocalcemiaProspective cohort studyChildmedicine.diagnostic_testDelayed Diagnosi22q11.2 deletion; Primary immune disordersAge Factorsdel 22qMIMAbnormalities Multiple; Adolescent; Adult; Age Factors; Child; Child Preschool; Chromosomes Human Pair 22; Delayed Diagnosis; Developmental Disabilities; DiGeorge Syndrome; Early Diagnosis; Female; Follow-Up Studies; Genetic Testing; Humans; Infant; Infant Newborn; Male; Monitoring Physiologic; Prospective Studies; Retrospective Studies; Risk Assessment; Severity of Illness Index; Sex Factors; Time Factors; Young Adult; Disease ProgressionChild PreschoolCohortDisease ProgressionPrimary immune disordersFemaleAbnormalitiesMultipleAbnormalities Multiple; Adolescent; Adult; Age Factors; Child; Child Preschool; Chromosomes Human Pair 22; Delayed Diagnosis; Developmental Disabilities; DiGeorge Syndrome; Early Diagnosis; Female; Follow-Up Studies; Genetic Testing; Humans; Infant; Infant Newborn; Male; Monitoring Physiologic; Prospective Studies; Retrospective Studies; Risk Assessment; Severity of Illness Index; Sex Factors; Time Factors; Young Adult; Disease Progression; Pediatrics Perinatology and Child HealthHumanAdultmedicine.medical_specialtyTime FactorAdolescentMonitoringDevelopmental DisabilitieItalian Association of Pediatric Haematology and OncologyContext (language use)Risk AssessmentChromosomesFollow-Up StudieYoung AdultSex FactorsSeverity of illnessmedicineDiGeorge SyndromeHumansAbnormalities MultipleGenetic Testing22q11DS; 22q11.2 deletion syndrome; AIEOP; Italian Association of Pediatric Haematology and Oncology; MIM; Mendelian Inheritance in Man22q11DSPreschoolPhysiologicdigeorge syndrome; del 22qGenetic testingMonitoring PhysiologicRetrospective StudiesSettore MED/38 - Pediatria Generale e Specialisticabusiness.industryMendelian Inheritance in ManInfant NewbornInfantRetrospective cohort studymedicine.diseaseNewbornAIEOPProspective StudieEarly Diagnosis22q11.2 deletion syndromePediatrics Perinatology and Child HealthPair 22businessFollow-Up Studies
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