Search results for " thalassemia major"

showing 5 items of 5 documents

Improving survival with deferiprone treatment in patients with thalassemia major: A prospective multicenter randomised clinical trial under the auspi…

2009

The prognosis for thalassemia major has dramatically improved in the last two decades. However, many transfusion-dependent patients continue to develop progressive accumulation of iron. This can lead to tissue damage and eventually death, particularly from cardiac disease. Previous studies that investigated iron chelation treatments, including retrospective and prospective non-randomised clinical trials, suggested that mortality, due mainly to cardiac damage, was reduced or completely absent in patients treated with deferiprone (DFP) alone or a combined deferiprone-deferoxamine (DFP-DFO) chelation treatment. However, no survival analysis has been reported for a long-term randomised control …

MaleThalassemiaKaplan-Meier Estimatelaw.inventionchemistry.chemical_compoundRandomized controlled triallawCause of DeathNeoplasmsDeferiproneProspective StudiesChildCause of deathHazard ratioHematologyMiddle AgedCombined Modality TherapySurvival RateThalassemia survival chelation treatment trial thalassemia majorCombinationSplenectomyMolecular MedicineDrug Therapy CombinationFemaleDeferiproneAdultmedicine.medical_specialtyAdolescentPyridonesDeferoxamineIron Chelating AgentsYoung AdultDrug TherapyInternal medicinemedicineHumansBlood TransfusionAdolescent; Adult; Blood Transfusion; Cause of Death; Chelation Therapy; Child; Combined Modality Therapy; Deferoxamine; Drug Therapy; Combination; Female; Heart Failure; Humans; Iron Chelating Agents; Kaplan-Meiers Estimate; Male; Middle Aged; Neoplasms; Proportional Hazards Models; Prospective Studies; Pyridones; Splenectomy; Survival Rate; Young Adult; beta-ThalassemiaMolecular BiologySurvival rateKaplan-Meiers EstimateSurvival analysisProportional Hazards ModelsHeart Failurebusiness.industryProportional hazards modelbeta-ThalassemiaCell Biologymedicine.diseaseChelation TherapySurgerychemistrybusiness
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Myocardial fibrosis by delayed enhancement cardiovascular magnetic resonance and HCV infection in thalassemia major patients.

2010

Abstract Abstract 4265 Introduction. Delayed enhancement (DE) cardiac magnetic resonance (CMR) technique with intravenous administration of gadolinium (Gd) chelates contrast agents is the only validated non-invasive approach for detecting myocardial fibrosis (Mahrholdt H et al, Eur Heart J 2005). This technique has been confirmed safe in patients with hemoglobinopathies (Meloni A et al, Haematologica 2009). In thalassemia major (TM), myocardial fibrosis has been detected using the DE technique and a positive correlation with anti-HCV antibodies has been described (Pepe A et al, Heart 2009). However, HCV-induced cardiomyopathy is still controversial (Matsumori A et al. J Card Fail 2006). The…

medicine.medical_specialtyHepatitis C virusThalassemiaImmunologyCardiomyopathymedicine.disease_causeBiochemistryGastroenterologyGadobutrolPathogenesisInternal medicineMyocardial fibrosisMedicinemedicine.diagnostic_testbusiness.industryMyocardial fibrosis; HCV infection; Thalassemia MajorRetrospective cohort studyMagnetic resonance imagingCell BiologyHematologymedicine.diseaseSurgeryHCV infectionMyocardial fibrosisbusinessThalassemia Majormedicine.drug
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Evaluation of the efficacy of oral deferiprone in beta-thalassemia major by multislice multiecho T2*.

2006

Objectives: Oral deferiprone (L1) appears to be promising in the treatment of beta-thalassemia major (TM) patients. T2* magnetic resonance imaging (MRI) with a single measurement in the mid-ventricular septum was validated as a quantitative evaluation of myocardial iron overload. Previous studies suggested a marked heterogeneity of iron distribution in the myocardium. We set up a multislice multiecho T2* MRI for the detection of this heterogeneity. The aim of our study was to investigate differences between the L1 vs. the subcutaneous desferrioxamine (DF)-treated patients using this new approach.Methods: Thirty-six beta-TM patients (age 29 +/- 8 yr) underwent MRI. Eighteen patients received…

AdultMaleIron OverloadPyridonesCoefficient of variationDeferoxamineBETA THALASSEMIA MAJORchemistry.chemical_compoundMagneticsmultislice multiecho T2*MedicineHumansMultisliceDeferiproneReproducibilitymedicine.diagnostic_testbusiness.industryMyocardiumbeta-ThalassemiaBeta thalassemiaMagnetic resonance imagingHematologyGeneral MedicineMiddle Agedmedicine.diseaseMagnetic Resonance Imagingchelation treatmentmedicine.anatomical_structurechemistryVentricleFemalebusinessNuclear medicineDeferiproneEuropean journal of haematology
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Dual therapy with peg-interferon and ribavirin in thalassemia major patients with chronic HCV infection: Is there still an indication?

2016

Background: Iron overload and hepatitis C virus (HCV) infection together can lead to chronic liver damage in thalassemia major (TM) patients. Aims: We investigated viral, genetic, and disease factors influencing sustained virological response (SVR) after peg-interferon and ribavirin therapy in TM patients with HCV infection. Methods: We analyzed 230 TM patients with HCV infection (mean age 36.0 ± 6.3 years; 59.1% genotype 1; 32.2% genotype 2; 3.4% genotype 3; and 5.3% genotype 4; 28.7% carried CC allele of rs12979860 in IL28B locus; 79.6% had chronic hepatitis and 20.4% cirrhosis; 63.5% naive and 36.5% previously treated with interferon alone) treated in 14 Italian centers. Results: By mul…

Liver Cirrhosis0301 basic medicineMaleCirrhosisThalassemiaHepacivirusCirrhosis; Hepatitis C virus; IL28B polymorphisms; Iron liver overload; Peg-interferon; Ribavirin; Sustained virological response; Thalassemia major; Adult; Antiviral Agents; Drug Therapy Combination; Female; Heart Diseases; Hepacivirus; Hepatitis C Chronic; Humans; Interferon-alpha; Interleukins; Italy; Liver Cirrhosis; Logistic Models; Male; Multivariate Analysis; Polymorphism Single Nucleotide; Retrospective Studies; Ribavirin; Treatment Outcome; Viral Load; beta-Thalassemiamedicine.disease_causeGastroenterologychemistry.chemical_compound0302 clinical medicineRetrospective StudieThalassemia majorMultivariate AnalysiGastroenterologyBeta thalassemiaHepatitis CViral LoadSustained virological responseHeart DiseaseTreatment OutcomeItaly030211 gastroenterology & hepatologyDrug Therapy CombinationFemaleViral loadHumanAdultmedicine.medical_specialtyHeart DiseasesLogistic ModelHepatitis C virusLiver CirrhosiAlpha interferonIL28B polymorphismAntiviral AgentsPolymorphism Single Nucleotide03 medical and health sciencesIron liver overloadInternal medicineRibavirinmedicineHumansRetrospective StudiesAntiviral AgentCirrhosiHepaciviruPeg-interferonHepatologybusiness.industryInterleukinsRibavirinbeta-ThalassemiaInterferon-alphaHepatitis C ChronicInterleukinmedicine.diseaseLogistic Models030104 developmental biologychemistryMultivariate AnalysisImmunologyInterferonsbusinessHepatitis C viru
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Granulocyte–Colony Stimulating Factor plus Plerixafor in Patients with β-thalassemia Major Results in the Effective Mobilization of Primitive CD34+ C…

2017

Successful gene therapy for β-thalassemia requires optimal numbers of autologous gene-transduced hematopoietic stem and progenitor cells (HSPCs) with high repopulating capacity. Previous studies suggested superior mobilization in these patients by the combination of granulocyte–colony stimulating factor (G-CSF) plus plerixafor over single agents. We mobilized four adult patients using G-CSF+plerixafor to assess the intra-individual variation of the circulating CD34+ cells number and subtypes preand post-plerixafor administration. The procedure was well-tolerated and the target cell dose of ≥8×10 6 CD34+ cells/kg was achieved in three of them with one apheresis procedure. The addition of ple…

Mobilizationbusiness.industryCD34+ cells expression profilingCd34 cellsPlerixaforGenetic enhancementβ-thalassemia; CD34 cells expression profiling; G-CSF plerixafor mobilization; gene therapygene therapySettore MED/15 - Malattie Del SangueGranulocyte colony-stimulating factorSettore BIO/18 - Geneticagene therapy.β-thalassemiaGene expressionImmunologyCancer researchG-CSF+plerixafor mobilizationMedicineDiseases of the blood and blood-forming organsIn patientβ-thalassemia; CD34+ cells expression profiling; G-CSF+plerixafor mobilization; gene therapyRC633-647.5businessβ thalassemia majormedicine.drugThalassemia Reports
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