title: Patient-reported outcomes of deferasirox (Exjade, ICL670) versus deferoxamine in sickle cell disease patients with transfusional hemosiderosis. Substudy of a randomized open-label phase II trial.
abstract: OBJECTIVE
  There is increasing evidence demonstrating the value of transfusions in sickle cell disease (SCD). However, resultant iron overload can be life threatening if untreated. Chelation therapy with deferoxamine requires parenteral infusions that can negatively impact quality of life and adherence to treatment.
  
  
  METHODS
  As part of a phase II trial, SCD patient-reported outcomes were evaluated. One hundred and ninety-five patients were randomized (2:1) to receive oral deferasirox (5-30 mg/kg/day) or deferoxamine (20-50 mg/kg, 5 days per week); 121 had previously received deferoxamine.
  
  
  RESULTS
  At each time point, significantly more patients who had previously received deferoxamine were 'satisfied/very satisfied' with deferasirox, or found treatment to be 'convenient/very convenient' compared with deferoxamine (p < 0.001). In these patients, fewer hours were lost from daily activities with deferasirox than deferoxamine treatment. Most patients (77%) preferred deferasirox, and more were willing to continue taking deferasirox than deferoxamine at end-of-study (84 vs. 11%, respectively).
  
  
  CONCLUSIONS
  Patients with SCD are therefore more satisfied with deferasirox, which has a lower impact on daily activities than deferoxamine. Given the high levels of satisfaction, it is likely that quality of life will be improved. These results also suggest that treatment adherence with deferasirox may be better than with deferoxamine, which should lead to improved long-term outcomes.
authors.affiliation: Children's Hospital and Research Center, Oakland, CA 94609, USA. evichinsky@mail.cho.org
authors.forename: Elliott
authors.initials: E
authors.lastname: Vichinsky
authors.forename: Zahra
authors.initials: Z
authors.lastname: Pakbaz
authors.forename: Onyinye
authors.initials: O
authors.lastname: Onyekwere
authors.forename: John
authors.initials: J
authors.lastname: Porter
authors.forename: Paul
authors.initials: P
authors.lastname: Swerdlow
authors.forename: Thomas
authors.initials: T
authors.lastname: Coates
authors.forename: Peter
authors.initials: P
authors.lastname: Lane
authors.forename: Beatrice
authors.initials: B
authors.lastname: Files
authors.forename: Brigitta U
authors.initials: BU
authors.lastname: Mueller
authors.forename: Lena
authors.initials: L
authors.lastname: Coïc
authors.forename: Gian Luca
authors.initials: GL
authors.lastname: Forni
authors.forename: Roland
authors.initials: R
authors.lastname: Fischer
authors.forename: Peter
authors.initials: P
authors.lastname: Marks
authors.forename: Diana
authors.initials: D
authors.lastname: Rofail
authors.forename: Linda
authors.initials: L
authors.lastname: Abetz
authors.forename: Jean-Francois
authors.initials: JF
authors.lastname: Baladi
mesh_terms: D000041:Absenteeism; D000293:Adolescent; D000328:Adult; D000755:Anemia, Sickle Cell / Q000150:complications / Q000523:psychology / Q000628:therapy*; D001565:Benzoates / Q000627:therapeutic use*; D015913:Chelation Therapy / Q000523:psychology* / Q000706:statistics & numerical data; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D003676:Deferoxamine / Q000627:therapeutic use*; D005260:Female; D006486:Hemosiderosis / Q000188:drug therapy* / Q000209:etiology / Q000523:psychology; D006801:Humans; D007501:Iron; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D017060:Patient Satisfaction / Q000706:statistics & numerical data; D011795:Surveys and Questionnaires; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000627:therapeutic use*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D003676:Deferoxamine; D000077588:Deferasirox