title: Deferiprone versus deferoxamine in sickle cell disease: results from a 5-year long-term Italian multi-center randomized clinical trial.
abstract: Blood transfusion and iron chelation currently represent a supportive therapy to manage anemia, vasculopathy and vaso-occlusion crises in Sickle-Cell-Disease. Here we describe the first 5-year long-term randomized clinical trial comparing Deferiprone versus Deferoxamine in patients with Sickle-Cell-Disease. The results of this study show that Deferiprone has the same effectiveness as Deferoxamine in decreasing body iron burden, measured as repeated measurements of serum ferritin concentrations on the same patient over 5-years and analyzed according to the linear mixed-effects model (LMM) (p=0.822). Both chelators are able to decrease, significantly, serum ferritin concentrations, during 5-years, without any effect on safety (p=0.005). Moreover, although the basal serum ferritin levels were higher in transfused compared with non-transfused group (p=0.031), the changes over time in serum ferritin levels were not statistically significantly different between transfused and non-transfused cohort of patients (p=0.389). Kaplan-Meier curve, during 5-years of study, suggests that Deferiprone does not alter survival in comparison with Deferoxamine (p=0.38). In conclusion, long-term iron chelation therapy with Deferiprone was associated with efficacy and safety similar to that of Deferoxamine. Therefore, in patients with Sickle-Cell-Disease, Deferiprone may represent an effective long-term treatment option.
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy.
authors.forename: Giusi
authors.initials: G
authors.lastname: Calvaruso
authors.affiliation: Dipartimento di Scienze Economiche, Aziendali e Statistiche, Università di Palermo, Palermo, Italy.
authors.forename: Angela
authors.initials: A
authors.lastname: Vitrano
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy.
authors.forename: Rosario
authors.initials: R
authors.lastname: Di Maggio
authors.affiliation: Division of Hematology/Cardeza Foundation, Department of Medicine, Jefferson Medical College, Thomas Jefferson University, Philadelphia, PA, USA.
authors.forename: Samir
authors.initials: S
authors.lastname: Ballas
authors.affiliation: Center of Excellence in Sickle Cell Disease, Department of Pediatrics, Pathology and Laboratory Medicine, Boston Medical Center, Boston, MA, USA.
authors.forename: Martin H
authors.initials: MH
authors.lastname: Steinberg
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy.
authors.forename: Paolo
authors.initials: P
authors.lastname: Rigano
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy.
authors.forename: Massimiliano
authors.initials: M
authors.lastname: Sacco
authors.affiliation: Department of Hematology, The Royal London Hospital, London, United Kingdom.
authors.forename: Paul
authors.initials: P
authors.lastname: Telfer
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy.
authors.forename: Disma
authors.initials: D
authors.lastname: Renda
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy.
authors.forename: Rita
authors.initials: R
authors.lastname: Barone
authors.affiliation: Unita'Operativa Complessa Ematologia II, A.O.R. Villa Sofia-V. Cervello, Palermo, Italy. Electronic address: md.amaggio@gmail.it.
authors.forename: Aurelio
authors.initials: A
authors.lastname: Maggio
mesh_terms: D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000097:blood / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D001803:Blood Transfusion; D002648:Child; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use*; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007501:Iron / Q000097:blood; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy* / Q000401:mortality / Q000473:pathology; D007558:Italy; D016014:Linear Models; D008297:Male; D008875:Middle Aged; D011728:Pyridones / Q000627:therapeutic use*; D016019:Survival Analysis
chemical_list: D007502:Iron Chelating Agents; D011728:Pyridones; D000077543:Deferiprone; D005293:Ferritins; D007501:Iron; D003676:Deferoxamine
keywords: Chelation in Sickle-Cell-Disease; DFP versus DFO in Sickle-Cell-Disease; Effectiveness of Deferiprone in Sickle-Cell-Disease