title: Quality of life, clinical effectiveness, and satisfaction in patients with beta thalassemia major and sickle cell anemia receiving deferasirox chelation therapy.
abstract: OBJECTIVE
  There is a need to remove excess iron with iron chelation therapy (ICT) to avoid the serious clinical sequelae associated with iron overload in patients with beta thalassemia major (BTM) and sickle cell anemia (SCA). Due to the effects of the diseases and their treatments, ICT is still a major reason for unsatisfactory compliance. The aim of this single-center observational study was to evaluate the quality of life, clinical effectiveness, and satisfaction in pediatric and adult patients with BTM and SCA receiving deferasirox (DFX) chelation therapy.
  
  
  METHODS
  In this study, 37 pediatric and 35 adult patients with BTM or SCA receiving DFX for at least 6 months participated. Upon receipt of Informed Consent Form, Case Report Form, Demographic Data Collection Form, Child Health Questionnaire-Parent Form, Life Quality Survey Short Form-36, and ICT Satisfaction Survey were used to obtain data for the effectiveness of ICT and parameters that may affect compliance to treatment and life quality of the participants.
  
  
  RESULTS
  As a main index for the effectiveness of DFX chelation therapy, serum ferritin levels were higher than the normal values in the patients receiving DFX. The increased ferritin levels were also associated with hematological and biochemical abnormalities. Our findings regarding quality of life and satisfaction with DFX chelation therapy indicated that the patients with BTM or SCA had lower scores. Overall, problems with treatment regimen and side effects appeared to be common causes of poor compliance to DFX chelation therapy.
  
  
  CONCLUSIONS
  Our findings suggest that health care providers should be aware of the importance of monitoring iron load with timely initiation of DFX chelation therapy and ongoing adjustments to chelation regimens and/or transfusion methods to decrease hospitalizations and improve compliance to ICT of the patients with BTM and SCA.
authors.affiliation: Department of Pharmacology, Faculty of Pharmacy, Mersin University, Mersin, Turkey.
authors.forename: Sefika Pinar
authors.initials: SP
authors.lastname: Senol
authors.affiliation: Department of Internal Medicine, Faculty of Medicine, Mersin University, Mersin, Turkey.
authors.forename: Eyup Naci
authors.initials: EN
authors.lastname: Tiftik
authors.affiliation: Department of Pediatric Hematology, Faculty of Medicine, Mersin University, Mersin, Turkey.
authors.forename: Selma
authors.initials: S
authors.lastname: Unal
authors.affiliation: Department of Internal Medicine, Faculty of Medicine, Mersin University, Mersin, Turkey.
authors.forename: Aydan
authors.initials: A
authors.lastname: Akdeniz
authors.affiliation: Department of Biostatistics, Faculty of Medicine, Mersin University, Mersin, Turkey.
authors.forename: Bahar
authors.initials: B
authors.lastname: Tasdelen
authors.affiliation: Department of Pharmacology, Faculty of Pharmacy, Mersin University, Mersin, Turkey.
authors.forename: Bahar
authors.initials: B
authors.lastname: Tunctan
keywords: Beta thalassemia major; compliance; deferasirox chelation therapy; sickle cell anemia