Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassaemia major patients: a randomized clinical trial.
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.
Maggio
Aurelio
A
U.O.C. Ematologia II con Talassemia, A.O. V. Cervello, Palermo, Italy. aureliomaggio@virgilio.it
Vitrano
Angela
A
Capra
Marcello
M
Cuccia
Liana
L
Gagliardotto
Francesco
F
Filosa
Aldo
A
Romeo
Maria Antonietta
MA
Magnano
Carmelo
C
Caruso
Vincenzo
V
Argento
Crocetta
C
Gerardi
Calogera
C
Campisi
Saveria
S
Violi
Pietro
P
Malizia
Roberto
R
Cianciulli
Paolo
P
Rizzo
Michele
M
D'Ascola
Domenico Giuseppe
DG
Quota
Alessandra
A
Prossomariti
Luciano
L
Fidone
Carmelo
C
Rigano
Paolo
P
Pepe
Alessia
A
D'Amico
Gennaro
G
Morabito
Alberto
A
Gluud
Christian
C
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
D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine