title: Efficacy and safety of a novel combination of two oral chelators deferasirox/deferiprone over deferoxamine/deferiprone in severely iron overloaded young beta thalassemia major patients.
abstract: OBJECTIVE
  Minimal data are available on the combined two oral iron chelators in β-thalassemia major (β-TM). Comparison of safety, efficacy, compliance, treatment satisfaction, and quality of life (QoL) of two regimens: deferiprone (DFP) and deferoxamine (DFO) versus DFP and deferasirox (DFX) were studied.
  
  
  METHODS
  A prospective randomized trial (NCT01511848) was conducted on 96 young β-TM patients with severe iron overload. Patients were randomized to receive either DFP with DFO (arm 1) or DFP and DFX (arm 2). Efficacy endpoints were the difference between two groups in the change of serum ferritin (SF), liver iron concentration (LIC), cardiac MRI, and quality of life (QoL).
  
  
  RESULTS
  In both arms, SF and LIC at 12 months were significantly lower, and geometric mean cardiac T2* was higher compared to baseline. On regression analysis of change in each studied variable against time, significant difference between slopes of the two groups regarding cardiac T2* (P = 0.001 with more improvement in DFP/DFX patients) was found with no significant difference in the slopes of SF and LIC (P = 0.218 and 0.340).
  
  
  CONCLUSIONS
  Both iron chelation combination regimens were equally effective in reducing iron overload and improving QoL.DFP/DFX combination proved superior in improving cardiac T2*, treatment compliance, and patients satisfaction with no greater adverse events.
authors.affiliation: Department of Pediatrics, Ain Shams University, Cairo, Egypt.
authors.forename: Mohsen S
authors.initials: MS
authors.lastname: Elalfy
authors.affiliation: Department of Pediatrics, Ain Shams University, Cairo, Egypt.
authors.forename: Amira M
authors.initials: AM
authors.lastname: Adly
authors.affiliation: Sultan Qaboos University Hospital, Muscat, Oman.
authors.forename: Yasser
authors.initials: Y
authors.lastname: Wali
authors.affiliation: Sultan Qaboos University Hospital, Muscat, Oman.
authors.forename: Samir
authors.initials: S
authors.lastname: Tony
authors.affiliation: Department of Radiology, Ain Shams University, Cairo, Egypt.
authors.forename: Ahmad
authors.initials: A
authors.lastname: Samir
authors.affiliation: Department of Pediatrics, Ain Shams University, Cairo, Egypt.
authors.forename: Yasmine I
authors.initials: YI
authors.lastname: Elhenawy
mesh_terms: D000293:Adolescent; D001565:Benzoates / Q000008:administration & dosage* / Q000009:adverse effects; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D000077543:Deferiprone; D003676:Deferoxamine / Q000008:administration & dosage* / Q000009:adverse effects; D004359:Drug Therapy, Combination; D005260:Female; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D019190:Iron Overload / Q000188:drug therapy* / Q000209:etiology; D008297:Male; D011728:Pyridones / Q000008:administration & dosage* / Q000009:adverse effects; D012720:Severity of Illness Index; D014230:Triazoles / Q000008:administration & dosage* / Q000009:adverse effects; D017086:beta-Thalassemia / Q000150:complications / Q000188:drug therapy*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D011728:Pyridones; D014230:Triazoles; D000077543:Deferiprone; D003676:Deferoxamine; D000077588:Deferasirox
keywords: Cardiac MRI; combined oral iron chelators; liver iron; quality of life; thalassemia major