title: Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial.
abstract: A multicentre randomized open-label trial was designed to assess the effectiveness of long-term sequential deferiprone-deferoxamine (DFO-DFP) versus DFP alone to treat thalassaemia major (TM). DFP at 75 mg/kg, divided into three oral daily doses, for 4 d/week and DFO by subcutaneous infusion (8-12 h) at 50 mg/kg per day for the remaining 3 d/week was compared with DFP alone at 75 mg/kg, administered 7 d/week during a 5-year follow-up. The main outcome measures were differences between multiple observations of serum ferritin concentrations. Secondary outcomes were survival analysis, adverse events, and costs. Consecutive thalassaemia patients (275) were assessed for eligibility; 213 of these were randomized and underwent intention-to-treat analysis. The decrease of serum ferritin levels during the treatment period was statistically significant higher in sequential DFP-DFO patients compared with DFP-alone patients (P = 0.005). Kaplan-Meier survival analysis for the two chelation treatments did not show any statistically significant differences (long-rank test, P = 0.3145). Adverse events and costs were comparable between the groups. The trial results show that sequential DFP-DFO treatment compared with DFP alone significantly decreased serum ferritin concentration during treatment for 5 years without significant differences regarding survival, adverse events, or costs.
authors.affiliation: U.O.C. Ematologia II con Talassemia, A.O. V. Cervello, Palermo, Italy. aureliomaggio@virgilio.it
authors.forename: Aurelio
authors.initials: A
authors.lastname: Maggio
authors.forename: Angela
authors.initials: A
authors.lastname: Vitrano
authors.forename: Marcello
authors.initials: M
authors.lastname: Capra
authors.forename: Liana
authors.initials: L
authors.lastname: Cuccia
authors.forename: Francesco
authors.initials: F
authors.lastname: Gagliardotto
authors.forename: Aldo
authors.initials: A
authors.lastname: Filosa
authors.forename: Maria Antonietta
authors.initials: MA
authors.lastname: Romeo
authors.forename: Carmelo
authors.initials: C
authors.lastname: Magnano
authors.forename: Vincenzo
authors.initials: V
authors.lastname: Caruso
authors.forename: Crocetta
authors.initials: C
authors.lastname: Argento
authors.forename: Calogera
authors.initials: C
authors.lastname: Gerardi
authors.forename: Saveria
authors.initials: S
authors.lastname: Campisi
authors.forename: Pietro
authors.initials: P
authors.lastname: Violi
authors.forename: Roberto
authors.initials: R
authors.lastname: Malizia
authors.forename: Paolo
authors.initials: P
authors.lastname: Cianciulli
authors.forename: Michele
authors.initials: M
authors.lastname: Rizzo
authors.forename: Domenico Giuseppe
authors.initials: DG
authors.lastname: D'Ascola
authors.forename: Alessandra
authors.initials: A
authors.lastname: Quota
authors.forename: Luciano
authors.initials: L
authors.lastname: Prossomariti
authors.forename: Carmelo
authors.initials: C
authors.lastname: Fidone
authors.forename: Paolo
authors.initials: P
authors.lastname: Rigano
authors.forename: Alessia
authors.initials: A
authors.lastname: Pepe
authors.forename: Gennaro
authors.initials: G
authors.lastname: D'Amico
authors.forename: Alberto
authors.initials: A
authors.lastname: Morabito
authors.forename: Christian
authors.initials: C
authors.lastname: Gluud
mesh_terms: D000284:Administration, Oral; D000293:Adolescent; D000328:Adult; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage* / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005260:Female; D005293:Ferritins / Q000097:blood; D005500:Follow-Up Studies; D006801:Humans; D055104:Infusions, Subcutaneous; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000627:therapeutic use; D053208:Kaplan-Meier Estimate; D008297:Male; D011728:Pyridones / Q000008:administration & dosage* / Q000627:therapeutic use; D013789:Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000401:mortality; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine