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Beta-thalassemia
TLDR
Prognosis for individuals with beta-thalassemia has improved substantially in the last 20 years following recent medical advances in transfusion, iron chelation and bone marrow transplantation therapy, however, cardiac disease remains the main cause of death in patients with iron overload.
Survival and Complications in Thalassemia
TLDR
In conclusion, rates of survival and complication‐free survival continue to improve, due to better treatment strategies, and new complications are appearing in long‐term survivors.
Liver iron concentrations and urinary hepcidin in beta-thalassemia.
TLDR
In thalassemia intermedia, high erythropoietic drive causes severe hepcidin deficiency, which results in hyperabsorption of dietary iron, but also in iron depletion of macrophages, lowering their secretion of ferritin and, consequently, serumferritin levels.
Randomized phase II trial of deferasirox (Exjade, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload.
TLDR
Deferasirox showed a plasma elimination half-life of 8-16 hours, supporting its once-daily administration, and was well tolerated and showed similar efficacy to DFO 40 mg/kg in terms of decreases in LIC.
Pregnancy and β-thalassemia: an Italian multicenter experience
TLDR
Provided that a multidisciplinary team is available, pregnancy is possible, safe and usually has a favorable outcome in patients with thalassemia, and in women with hypog onadotropic hypogonadism, gonadal function is usually intact and fertility is usually retrievable.
Second international round robin for the quantification of serum non-transferrin-bound iron and labile plasma iron in patients with iron-overload disorders
TLDR
The possibility of using non-transferrin-bound iron and labile plasma iron measures as clinical indicators of overt iron overload and/or of treatment efficacy would largely depend on the rigorous validation and standardization of assays.
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia
TLDR
In a phase 1/2 clinical trial, gene therapy with autologous hematopoietic stem cells significantly reduced transfusion requirement in adults and children with transfusion dependent ß-thalassemia.
Combined therapy with deferiprone and desferrioxamine in thalassemia major.
TLDR
Continuous deferiprone treatment with intermittent administration of subcutaneous desferrioxamine is a practical and effective procedure to decrease severe iron overload in patients with thalassemia major.
Onset of cardiac iron loading in pediatric patients with thalassemia major
TLDR
Assessment of cardiac T2* assessments from 77 thalassemia major patients between the ages of 2.5 and 18 years found optimal timing of cardiac iron screening by magnetic resonance imaging should be determined according to age and transfusional burden rather than indices of iron overload.
A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia.
TLDR
The percentage of patients with transfusion-dependent β-thalassemia who had a reduction in transfusion burden was significantly greater in the luspatercept group than in the placebo group, and few adverse events led to the discontinuation of treatment.
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