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Improving survival with deferiprone treatment in patients with thalassemia major: A prospective multicenter randomised clinical trial under the auspices of the Italian Society for Thalassemia and Hemoglobinopathies
- Publication Year :
- 2009
- Publisher :
- Elsevier, 2009.
-
Abstract
- 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 trial. Here, we performed a multicenter, long-term, randomised control trial that compared deferoxamine (DFO) versus DFP alone, sequential DFP-DFO, or combined DFP-DFO iron chelation treatments. The trial included 265 patients with thalassemia major, with 128 (48.3%) females and 137 (51.7%) males. No deaths occurred with the DFP-alone or the combined DFP-DFO treatments. One death occurred due to graft versus host disease (GVHD) in a patient that had undergone bone marrow transplantation; this patient was censored at the time of transplant. Only one death occurred with the DFP-DFO sequential treatment in a patient that had experienced an episode of heart failure one year earlier. Ten deaths occurred with the deferoxamine treatment. The main factors that correlated with an increase in the hazard ratio for death were: cirrhosis, arrhythmia, previous episode of heart failure, diabetes, hypogonadism, and hypothyroidism. In a Cox regression model, the interaction effect of sex and age was statistically significant (p-value
- Subjects :
- Male
Thalassemia
Kaplan-Meier Estimate
law.invention
chemistry.chemical_compound
Randomized controlled trial
law
Cause of Death
Neoplasms
Deferiprone
Prospective Studies
Child
Cause of death
Hazard ratio
Hematology
Middle Aged
Combined Modality Therapy
Survival Rate
Thalassemia, survival, chelation, treatment, trial, thalassemia major
Combination
Splenectomy
Molecular Medicine
Drug Therapy, Combination
Female
Adult
medicine.medical_specialty
Adolescent
Pyridones
Deferoxamine
Iron Chelating Agents
Young Adult
Drug Therapy
Internal medicine
medicine
Humans
Blood Transfusion
Chelation Therapy
Heart Failure
Kaplan-Meiers Estimate
Proportional Hazards Models
beta-Thalassemia
Molecular Biology
Survival rate
Survival analysis
business.industry
Proportional hazards model
Cell Biology
medicine.disease
Surgery
chemistry
business
Subjects
Details
- Language :
- English
- Database :
- OpenAIRE
- Accession number :
- edsair.doi.dedup.....c528499eeaa1c4acab63f412e90b29eb