Search results for "Thalassemia Major"

showing 6 items of 16 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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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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Pattern of cognitive impairment in beta-thalassemia major

2000

Settore MED/26 - Neurologiacognitive beta-thalassemia major
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Effects of thalassemia major on bone mineral density in late adolescence

2003

Management of thalassemia major has shown substantial clinical and prognostic improvement, suggesting the need for major attention to quality of life. We studied bone health in 25 patients (13 males, 12 females; 15-23 years old) affected by β-thalassemia major. In all patients, bone mineral density (BMD), biochemical markers of bone and calcium metabolism (calcium, phosphate, magnesium, alkaline phosphatase, urinary calcium, 25-hydroxyvitamin D [25OH-D], 1,25-dihydroxyvitamin D [1,25(OH)2,D], parathyroid hormone [PTH]), hematological parameters and gonadal steroids status were assessed and related to each other and to auxological parameters (chronological, statural and bone ages, height, we…

Settore MED/38 - Pediatria Generale E SpecialisticaBone regulating hormoneBone makerBone mineral densityThalassemia majorAdolescence
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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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Potential myocardial iron content evaluation by magnetic resonance imaging in thalassemia major patients treated with Deferoxamine or Deferiprone dur…

2003

The purpose of this study was to evaluate if the variations of heart magnetic resonance imaging in beta-thalassemia major patients treated with Deferoxamine B mesylate (DF) or Deferiprone (L1) chelation therapy is a useful tool of the indirect myocardial iron content determination. For this reason, a prospective study was carried out. Seventy-two consecutive patients with beta-thalassemia major (35 treated with DF and 37 with L1) were studied. The main outcome results were laboratory parameters including determination of the liver iron concentration (LIC) and magnetic resonance imaging (MRI) of the heart and liver. The heart to muscle signal intensity ratios (HSIRs) were significantly incre…

thalassemia majormyocardial iron content
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