title: Efficacy and safety of deferasirox in β-thalassemia major patients in Iran: a prospective study from a single referral center in Iran.
abstract: BACKGROUND
  Herein, the results of a prospective study evaluating the efficacy and safety of treatment with deferasirox are studied in iron-overloaded patients with β-thalassemia major during an 18-month trial.
  
  
  METHODS
  Thirty patients who were previously chelated with deferoxamine with/without deferiprone, and fulfilled the inclusion criteria were recruited. Patients received an initial dose of 10-30 mg/kg/day. Liver and cardiac MRI T2* were evaluated before and after the trial. In addition, serum ferritin level was assessed every 3 months. Primary endpoint was regarded as significant improvement in the severity of liver and cardiac iron overload in severe and moderate cases, in addition to improvement or maintenance of the grade of severity in patients with mild iron overload or normal iron accumulation. Therapy was considered effective if primary endpoint was met in >50%.
  
  
  RESULTS
  Liver MRI values improved significantly (P = .002), achieving a 73.33% success rate. A successful outcome regarding myocardial iron overload was observed in 80%. Finally, an overall of 66.66% of patients met the success criteria. Secondary endpoint, regarded as safety and tolerability was reached by 93.33%. The most common adverse events were skin rash and gastrointestinal disturbance. A dose between 30 and 40 mg/kg/day, tailored to each patient was considered the optimal dose.
  
  
  CONCLUSIONS
  Deferasirox proved as an efficient and safe chelating agent in our patients, specifically in mild to moderate iron overloaded patients.
authors.affiliation: 1Pediatric Congenital Hematologic Disorders Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran.
authors.forename: Samin
authors.initials: S
authors.lastname: Alavi
authors.forename: Maryam
authors.initials: M
authors.lastname: Ebadi
authors.forename: Farid
authors.initials: F
authors.lastname: Ghazizadeh
authors.forename: Mohammad Taghi
authors.initials: MT
authors.lastname: Arzanian
authors.forename: Bibishahin
authors.initials: B
authors.lastname: Shamsian
authors.forename: Fatemeh
authors.initials: F
authors.lastname: Abdollah Gorji
mesh_terms: D000293:Adolescent; D000328:Adult; D001565:Benzoates / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D015913:Chelation Therapy / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D003875:Drug Eruptions / Q000209:etiology; D005260:Female; D005293:Ferritins / Q000097:blood; D005767:Gastrointestinal Diseases / Q000139:chemically induced; D006801:Humans; D007492:Iran / Q000453:epidemiology; D007501:Iron / Q000032:analysis; D007502:Iron Chelating Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology / Q000473:pathology; D008099:Liver / Q000737:chemistry / Q000473:pathology; D008279:Magnetic Resonance Imaging; D008297:Male; D009206:Myocardium / Q000737:chemistry / Q000473:pathology; D011446:Prospective Studies; D013318:Stroke Volume; D065227:Transfusion Reaction; D016896:Treatment Outcome; D014230:Triazoles / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D055815:Young Adult; D017086:beta-Thalassemia / Q000150:complications* / Q000453:epidemiology / Q000628:therapy
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D007501:Iron; D000077588:Deferasirox