title: Prospective evaluation of patient-reported outcomes during treatment with deferasirox or deferoxamine for iron overload in patients with beta-thalassemia.
abstract: BACKGROUND
  Iron chelation therapy (ICT) with deferoxamine (DFO), the current standard for the treatment of iron overload in patients with transfusion-dependent disorders such as beta-thalassemia, requires regular subcutaneous or intravenous infusions. This can lead to reduced quality of life and poor adherence, resulting in increased morbidity and mortality in iron-overloaded patients with beta-thalassemia. Deferasirox is an orally administered iron chelator that has been approved for use in the United States, Switzerland, and other countries.
  
  
  OBJECTIVE
  This analysis was conducted to compare patient-reported outcomes (PROs) during receipt of DFO infusions or once-daily oral therapy with deferasirox (ICL670).
  
  
  METHODS
  PROs were prospectively evaluated as part of a randomized, Phase III study comparing the efficacy and safety profile of DFO 20 to 60 mg/kg per day with those of deferasirox 5 to 30 mg/kg per day in patients (age > or =2 years) with beta-thalassemia who were receiving regular transfusions and had a liver iron concentration of > or =2 mg/g dry weight. PRO questionnaires were completed by patients or a parent or legal guardian at baseline, week 4, week 24, and end of study (EOS). Patients assessed their level of satisfaction with study treatment (very satisfied, satisfied, neutral, dissatisfied, or very dissatisfied) and rated its convenience (very convenient, convenient, neutral, inconvenient, or very inconvenient). Time lost from normal activities due to ICT in the previous 4 weeks was recorded using a single global assessment. At week 4, patients who had previous experience with DFO were asked to indicate their preference for treatment (ICT received before the study, ICT received during the study, no preference, or no response) and the reason for that preference. At EOS, all patients were asked if they would be willing to continue using the ICT they had received during the study. All study analyses were performed in all patients who received at least 1 dose of study medication.
  
  
  RESULTS
  Five hundred eighty-six patients (304 females, 282 males; age range, 2-53 years) received treatment with DFO (n = 290) or deferasirox (n = 296). Significantly more patients treated with deferasirox reported being very satisfied or satisfied with treatment compared with those treated with DFO (week 4: 92.0% vs 50.4%, respectively; week 24: 89.6% vs 44.0%; EOS: 85.1% vs 38.7%; all, P < 0.001). At the same time points, the majority of those treated with deferasirox reported that treatment was very convenient or convenient compared with those treated with DFO (95.5% vs 21.3%, 91.7% vs 17.4%, and 92.7% vs 11.3%, respectively; all, P < 0.001). Among patients who had previously taken DFO and were randomized to receive deferasirox during the study, 96.9% reported a preference for deferasirox over DFO. At EOS, the proportion of patients indicating a willingness to continue study therapy was significantly greater in those receiving deferasirox than in those receiving DFO (85.8% vs 13.8%; P < 0.001).
  
  
  CONCLUSIONS
  In this study, patient-reported satisfaction and convenience were significantly higher for the once-daily, oral ICT deferasirox than for DFO infusions. Among patients who had received DFO before the study, the majority indicated a preference for deferasirox over DFO. Most patients receiving deferasirox indicated that they would be willing to continue taking it. These results suggest that deferasirox had a positive impact on patients' daily lives.
authors: Fondazione Policlinico IRCCS, Universitd di Milano, Milan, Italy. Electronic address: maria.cappellini@unimi.it.
authors: Maria Domenica
authors: MD
authors: Cappellini
authors: Centre National des Greffes de la Moelle Osseuse, Tunis, Tunisia.
authors: Mohamed
authors: M
authors: Bejaoui
authors: Istanbul University, Istanbul Medical Faculty, Istanbul, Turkey.
authors: Leyla
authors: L
authors: Agaoglu
authors: University College London, London, United Kingdom.
authors: John
authors: J
authors: Porter
authors: Children's Hospital of Los Angeles, Los Angeles, California, USA.
authors: Thomas
authors: T
authors: Coates
authors: Stan ford Hospital, Stanford, California, USA.
authors: Michael
authors: M
authors: Jeng
authors: Ospedale Regionale delle Microcitemie e Dipartimento di Scienze Mediche, Università di Cagliari, Cagliari, Italy.
authors: Maria Eliana
authors: ME
authors: Lai
authors: Ospedale Umberto 1st, Siracusa, Italy.
authors: Antonio
authors: A
authors: Mangiagli
authors: Charité Kinderklinik, Berlin, Germany.
authors: Gabriele
authors: G
authors: Strauss
authors: Hôpital Tenon, Paris, France.
authors: Robert
authors: R
authors: Girot
authors: Hospital Ramos Mejía, Buenos Aires, Argentina.
authors: Nora
authors: N
authors: Watman
authors: Hôpital Universitaire des Enfants Reine Fabiola, Brussels, Belgium.
authors: Alina
authors: A
authors: Ferster
authors: Centro de Hematología Sao Paulo, Sao Paulo, Brazil.
authors: Sandra
authors: S
authors: Loggetto
authors: Montreal Children's Hospital, Montreal, Canada.
authors: Sharon
authors: S
authors: Abish
authors: Universitaetsklinikum Ulm, Ulm, Germany.
authors: Holger
authors: H
authors: Cario
authors: General University Hospital of Patras, Patras, Greece.
authors: Nicolaos
authors: N
authors: Zoumbos
authors: Children's Hospital and Research Center at Oakland, Oakland, California, USA.
authors: Elliott
authors: E
authors: Vichinsky
authors: Novartis Pharma AG, Basel, Switzerland.
authors: Herbert
authors: H
authors: Opitz
authors: Novartis Pharma AG, Basel, Switzerland.
authors: Catherine
authors: C
authors: Ressayre-Djaffer
authors: Mapi Values Limited, Bollington, United Kingdom, USA.
authors: Linda
authors: L
authors: Abetz
authors: Mapi Values Limited, Bollington, United Kingdom, USA.
authors: Diana
authors: D
authors: Rofail
authors: Novartis Pharmaceuticals Corporation, Florham Park, NewJersey, USA.
authors: Jean-Francois
authors: JF
authors: Baladi
mesh_terms: D000293:Adolescent; D000328:Adult; D000931:Antidotes / Q000627:therapeutic use*; D001565:Benzoates / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D003676:Deferoxamine / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy*
chemical_list: D000931:Antidotes; D001565:Benzoates; D014230:Triazoles; D003676:Deferoxamine; D000077588:Deferasirox