1. Sequential alternating deferiprone and deferoxamine treatment compared to deferiprone monotherapy: main findings and clinical follow-up of a large multicenter randomized clinical trial in -thalassemia major patients
- Author
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Pietro Violi, R. Malizia, Domenico Giuseppe D'Ascola, Alessia Pepe, Alberto Morabito, Saveria Campisi, Gaetano Restivo Pantalone, Maria Antonietta Romeo, Calogera Gerardi, Michele Rizzo, Alessandra Quota, Christian Gluud, Aurelio Maggio, Paolo Cianciulli, Gennaro D'Amico, Francesco Gagliardotto, Aldo Filosa, Carmelo Magnano, Crocetta Argento, Paolo Rigano, Luciano Prossomariti, Vincenzo Caruso, Carmelo Fidone, Angela Vitrano, Liana Cuccia, Marcello Capra, Pantalone, GR, Maggio, A, Vitrano, A, Capra, M, Cuccia, L, Gagliardotto, F, Filosa, A, Romeo, MA, Magnano, C, Caruso, V, Argento, C, Gerardi, C, Campisi, S, Violi, P, Malizia, R, Cianciulli, P, Rizzo, M, D'Ascola, DG, Quota, A, Prossomariti, L, Fidone, C, Rigano, P, Pepe, A, D'Amico, G, Morabito, A, and Gluud, C
- Subjects
Adult ,Male ,medicine.medical_specialty ,Adolescent ,Pyridones ,Thalassemia ,Clinical Biochemistry ,Deferoxamine ,Iron Chelating Agents ,Gastroenterology ,Drug Administration Schedule ,law.invention ,chemistry.chemical_compound ,Young Adult ,Randomized controlled trial ,law ,Internal medicine ,Medicine ,Humans ,Deferiprone ,Adverse effect ,Genetics (clinical) ,Survival analysis ,business.industry ,Biochemistry (medical) ,Serum ferritin level ,beta-Thalassemia ,Hematology ,Iron chelation therapy ,medicine.disease ,Chelation Therapy ,Treatment Outcome ,chemistry ,Drug Therapy, Combination ,Female ,business ,Thalassemia, Iron overload, Iron chelation therapy, Deferiprone (L1), Deferroxamine (DFO) ,medicine.drug ,Follow-Up Studies - Abstract
In β-thalassemia major (β-TM) patients, iron chelation therapy is mandatory to reduce iron overload secondary to transfusions. Recommended first line treatment is deferoxamine (DFO) from the age of 2 and second line treatment after the age of 6 is deferiprone (L1). A multicenter randomized open-label trial was designed to assess the effectiveness of long-term alternating sequential L1-DFO versus L1 alone iron chelation therapy in β-TM patients. Deferiprone 75 mg/kg 4 days/week and DFO 50 mg/kg/day for 3 days/week was compared with L1 alone 75 mg/kg 7 days/week during 5-year follow-up. A total of 213 thalassemia patients were randomized and underwent intention-to-treat analysis. Statistically, a decrease of serum ferritin levels was significantly higher in alternating sequential L1-DFO patients compared with L1 alone patients (p = 0.005). Kaplan-Meier survival analysis for the two chelation treatments did not show statistically significant differences (log-rank test, p = 0.3145). Adverse events and costs were comparable between the groups. Alternating sequential L1-DFO treatment decreased serum ferritin concentration during a 5-year treatment by comparison to L1 alone, without significant differences of survival, adverse events or costs. These findings were confirmed in a further 21-month follow-up. These data suggest that alternating sequential L1-DFO treatment may be useful for some β-TM patients who may not be able to receive other forms of chelation treatment.
- Published
- 2011