Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomised clinical trial

Angela Vitrano, Paolo Rigano, Luciano Prossomariti, Calogera Gerardi, Alessia Pepe, Carmelo Fidone, Francesco Gagliardotto, Crocetta Argento, Angela Vitrano, Pietro Violi, Alessandra Quota, Michele Rizzo, Domenico Giuseppe D'Ascola, Liana Cuccia, Carmelo Magnano, Roberto Malizia, Paolo Cianciulli, Saveria Campisi, Aldo Filosa, Vincenzo CarusoChristian Gluud, Alberto Morabito, Marcello Capra, Aurelio Maggio, Maria Antonietta Romeo, Gennaro D'Amico

Risultato della ricerca: Article

58 Citazioni (Scopus)

Abstract

A multicentre randomized open-label trial was designed to assess theeffectiveness 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 subcutaneousinfusion (8–12 h) at 50 mg/kg per day for the remaining 3 d/week wascompared with DFP alone at 75 mg/kg, administered 7 d/week during a5-year follow-up. The main outcome measures were differences betweenmultiple observations of serum ferritin concentrations. Secondary outcomeswere survival analysis, adverse events, and costs. Consecutive thalassaemiapatients (275) were assessed for eligibility; 213 of these were randomized andunderwent intention-to-treat analysis. The decrease of serum ferritin levelsduring the treatment period was statistically significant higher in sequentialDFP–DFO patients compared with DFP-alone patients (P = 0.005). Kaplan–Meier survival analysis for the two chelation treatments did not show anystatistically significant differences (log-rank test, P = 0.3145). Adverseevents and costs were comparable between the groups. The trial resultsshow that sequential DFP–DFO treatment compared with DFP alonesignificantly decreased serum ferritin concentration during treatment for5 years without significant differences regarding survival, adverse events,or costs.
Lingua originaleEnglish
pagine (da-a)245-254
Numero di pagine10
RivistaBritish Journal of Haematology
Volume2
Stato di pubblicazionePublished - 2009

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Isoflurophate
Deferoxamine
beta-Thalassemia
Randomized Controlled Trials
Ferritins
Survival Analysis
Costs and Cost Analysis
Serum
Intention to Treat Analysis
Therapeutics
Outcome Assessment (Health Care)
deferiprone
Survival

All Science Journal Classification (ASJC) codes

  • Hematology

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Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomised clinical trial. / Vitrano, Angela; Rigano, Paolo; Prossomariti, Luciano; Gerardi, Calogera; Pepe, Alessia; Fidone, Carmelo; Gagliardotto, Francesco; Argento, Crocetta; Vitrano, Angela; Violi, Pietro; Quota, Alessandra; Rizzo, Michele; D'Ascola, Domenico Giuseppe; Cuccia, Liana; Magnano, Carmelo; Malizia, Roberto; Cianciulli, Paolo; Campisi, Saveria; Filosa, Aldo; Caruso, Vincenzo; Gluud, Christian; Morabito, Alberto; Capra, Marcello; Maggio, Aurelio; Romeo, Maria Antonietta; D'Amico, Gennaro.

In: British Journal of Haematology, Vol. 2, 2009, pag. 245-254.

Risultato della ricerca: Article

Vitrano, A, Rigano, P, Prossomariti, L, Gerardi, C, Pepe, A, Fidone, C, Gagliardotto, F, Argento, C, Vitrano, A, Violi, P, Quota, A, Rizzo, M, D'Ascola, DG, Cuccia, L, Magnano, C, Malizia, R, Cianciulli, P, Campisi, S, Filosa, A, Caruso, V, Gluud, C, Morabito, A, Capra, M, Maggio, A, Romeo, MA & D'Amico, G 2009, 'Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomised clinical trial', British Journal of Haematology, vol. 2, pagg. 245-254.
Vitrano, Angela ; Rigano, Paolo ; Prossomariti, Luciano ; Gerardi, Calogera ; Pepe, Alessia ; Fidone, Carmelo ; Gagliardotto, Francesco ; Argento, Crocetta ; Vitrano, Angela ; Violi, Pietro ; Quota, Alessandra ; Rizzo, Michele ; D'Ascola, Domenico Giuseppe ; Cuccia, Liana ; Magnano, Carmelo ; Malizia, Roberto ; Cianciulli, Paolo ; Campisi, Saveria ; Filosa, Aldo ; Caruso, Vincenzo ; Gluud, Christian ; Morabito, Alberto ; Capra, Marcello ; Maggio, Aurelio ; Romeo, Maria Antonietta ; D'Amico, Gennaro. / Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomised clinical trial. In: British Journal of Haematology. 2009 ; Vol. 2. pagg. 245-254.
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abstract = "A multicentre randomized open-label trial was designed to assess theeffectiveness 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 subcutaneousinfusion (8–12 h) at 50 mg/kg per day for the remaining 3 d/week wascompared with DFP alone at 75 mg/kg, administered 7 d/week during a5-year follow-up. The main outcome measures were differences betweenmultiple observations of serum ferritin concentrations. Secondary outcomeswere survival analysis, adverse events, and costs. Consecutive thalassaemiapatients (275) were assessed for eligibility; 213 of these were randomized andunderwent intention-to-treat analysis. The decrease of serum ferritin levelsduring the treatment period was statistically significant higher in sequentialDFP–DFO patients compared with DFP-alone patients (P = 0.005). Kaplan–Meier survival analysis for the two chelation treatments did not show anystatistically significant differences (log-rank test, P = 0.3145). Adverseevents and costs were comparable between the groups. The trial resultsshow that sequential DFP–DFO treatment compared with DFP alonesignificantly decreased serum ferritin concentration during treatment for5 years without significant differences regarding survival, adverse events,or costs.",
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author = "Angela Vitrano and Paolo Rigano and Luciano Prossomariti and Calogera Gerardi and Alessia Pepe and Carmelo Fidone and Francesco Gagliardotto and Crocetta Argento and Angela Vitrano and Pietro Violi and Alessandra Quota and Michele Rizzo and D'Ascola, {Domenico Giuseppe} and Liana Cuccia and Carmelo Magnano and Roberto Malizia and Paolo Cianciulli and Saveria Campisi and Aldo Filosa and Vincenzo Caruso and Christian Gluud and Alberto Morabito and Marcello Capra and Aurelio Maggio and Romeo, {Maria Antonietta} and Gennaro D'Amico",
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T1 - Long-term sequential deferiprone-deferoxamine versus deferiprone alone for thalassemia major patients: a randomised clinical trial

AU - Vitrano, Angela

AU - Rigano, Paolo

AU - Prossomariti, Luciano

AU - Gerardi, Calogera

AU - Pepe, Alessia

AU - Fidone, Carmelo

AU - Gagliardotto, Francesco

AU - Argento, Crocetta

AU - Vitrano, Angela

AU - Violi, Pietro

AU - Quota, Alessandra

AU - Rizzo, Michele

AU - D'Ascola, Domenico Giuseppe

AU - Cuccia, Liana

AU - Magnano, Carmelo

AU - Malizia, Roberto

AU - Cianciulli, Paolo

AU - Campisi, Saveria

AU - Filosa, Aldo

AU - Caruso, Vincenzo

AU - Gluud, Christian

AU - Morabito, Alberto

AU - Capra, Marcello

AU - Maggio, Aurelio

AU - Romeo, Maria Antonietta

AU - D'Amico, Gennaro

PY - 2009

Y1 - 2009

N2 - A multicentre randomized open-label trial was designed to assess theeffectiveness 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 subcutaneousinfusion (8–12 h) at 50 mg/kg per day for the remaining 3 d/week wascompared with DFP alone at 75 mg/kg, administered 7 d/week during a5-year follow-up. The main outcome measures were differences betweenmultiple observations of serum ferritin concentrations. Secondary outcomeswere survival analysis, adverse events, and costs. Consecutive thalassaemiapatients (275) were assessed for eligibility; 213 of these were randomized andunderwent intention-to-treat analysis. The decrease of serum ferritin levelsduring the treatment period was statistically significant higher in sequentialDFP–DFO patients compared with DFP-alone patients (P = 0.005). Kaplan–Meier survival analysis for the two chelation treatments did not show anystatistically significant differences (log-rank test, P = 0.3145). Adverseevents and costs were comparable between the groups. The trial resultsshow that sequential DFP–DFO treatment compared with DFP alonesignificantly decreased serum ferritin concentration during treatment for5 years without significant differences regarding survival, adverse events,or costs.

AB - A multicentre randomized open-label trial was designed to assess theeffectiveness 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 subcutaneousinfusion (8–12 h) at 50 mg/kg per day for the remaining 3 d/week wascompared with DFP alone at 75 mg/kg, administered 7 d/week during a5-year follow-up. The main outcome measures were differences betweenmultiple observations of serum ferritin concentrations. Secondary outcomeswere survival analysis, adverse events, and costs. Consecutive thalassaemiapatients (275) were assessed for eligibility; 213 of these were randomized andunderwent intention-to-treat analysis. The decrease of serum ferritin levelsduring the treatment period was statistically significant higher in sequentialDFP–DFO patients compared with DFP-alone patients (P = 0.005). Kaplan–Meier survival analysis for the two chelation treatments did not show anystatistically significant differences (log-rank test, P = 0.3145). Adverseevents and costs were comparable between the groups. The trial resultsshow that sequential DFP–DFO treatment compared with DFP alonesignificantly decreased serum ferritin concentration during treatment for5 years without significant differences regarding survival, adverse events,or costs.

KW - Chelation

KW - clinical trials

KW - iron overload.

KW - red blood cell disorders

KW - thalassaemia

UR - http://hdl.handle.net/10447/62339

M3 - Article

VL - 2

SP - 245

EP - 254

JO - British Journal of Haematology

JF - British Journal of Haematology

SN - 0007-1048

ER -