title: Side effects of Deferasirox Iron Chelation in Patients with Beta Thalassemia Major or Intermedia.
abstract: OBJECTIVE
  Chelating agents remain the mainstay in reducing the iron burden and extending patient survival in homozygous beta-thalassemia but adverse and toxic effects may increase with the institution and long term use of this essential therapy. This study aimed to estimate the incidence of deferasirox (DFX) side effects in patients with thalassemia major or intermedia.
  
  
  METHODS
  A retrospective study of 72 patients (mean age: 20.3±0.9 yrs; 36 male, 36 female) with thalassemia major or intermedia treated at Sultan Qaboos University Hospital, Oman, was performed to assess the incidence of side effects related to deferasirox over a mean of 16.7 month follow-up period.
  
  
  RESULTS
  Six patients experienced rashes and 6 had gastro-intestinal upset. DFX was discontinued in 18 patients for the following reasons: persistent progressive rise(s) in serum creatinine (7 patients; 40% mean serum creatinine rise from baseline), feeling unwell (2), severe diarrhea (1), pregnancy (1), death unrelated to chelator (2) and rise in serum transaminases (2). Three patients were reverted to desferoxamine and deferiprone combination therapy as DFX was no longer biochemically effective after 18 months of therapy. There was no correlation between baseline serum ferritin and serum creatinine or a rise in serum creatinine. Cardiac MRI T2* did not change with DFX therapy. However, there was an improvement in liver MRI T2* (p=0.013).
  
  
  CONCLUSIONS
  Renal side effects related to deferasirox appear to be higher than those reported in published clinical trials. Further larger studies are required to confirm these findings.
authors.affiliation: Department of Hematology, Sultan Qaboos University Hospital, Muscat, Sultanate of Oman.
authors.forename: Murtadha
authors.initials: M
authors.lastname: Al-Khabori
authors.forename: Sunil
authors.initials: S
authors.lastname: Bhandari
authors.forename: Mohammed
authors.initials: M
authors.lastname: Al-Huneini
authors.forename: Khalil
authors.initials: K
authors.lastname: Al-Farsi
authors.forename: Vinodh
authors.initials: V
authors.lastname: Panjwani
authors.forename: Shahina
authors.initials: S
authors.lastname: Daar
keywords: Chelator; Kidney function; Thalassemia; Toxicity