title: Efficacy and Safety of Combined Oral Chelation With Deferiprone and Deferasirox in Children With β-Thalassemia Major: An Experience From North India.
abstract: A combination of desferrioxamine with either deferiprone (DFP) or deferasirox (DFX) for patients with β-thalassemia major who do not achieve negative iron balance with monotherapy has been studied widely. However, poor compliance resulting from the need for parentral administration of desferrioxamine and its cost necessicitates combining 2 oral chelators.
  
  
  
  A prospective study was conducted in patients with transfusion-dependent β-thalassemia major in a tertiary care center over 2 years. Patients on either DFP or DFX who were not improving on monotherapy over a long period and persistently maintaining serum ferritin >2500 µg/L were enrolled. Efficacy was assessed by serum ferritin levels assessed at 12 months and 2 years. Complete blood counts and liver and kidney function tests were monitored to assess the safety of the combination of drugs.
  
  
  
  In total, 33 patients with a mean age of 12.67 years (7.5 to 17.5 y) and a mean ferritin of 4835.2394±1443.85 µg/L formed the study cohort.In total, 28 patients completed the 1-year study period; and 12 patients completed 2 years. Mean serum ferritin reduction at 1 and 2 years was 34.99%±18.13% (range, -34.36% to 56.17%) and 44.67%±13.78% (range, 22.17% to 62.74%), respectively. The combination therapy was well tolerated.
  
  
  
  Combined oral chelation with DFP and DFX has better efficacy than either drug used alone. The combination of drugs was well tolerated and no new adverse effects were observed.
authors.affiliation: *Department of Pediatrics, Division of Hematology and Oncology, Kalawati Saran Children's Hospital and Lady Hardinge Medical College Departments of †Pathology ‡Ophthalmology, Lady Hardinge Medical College, New Delhi.
authors.forename: Nupur
authors.initials: N
authors.lastname: Parakh
authors.forename: Jagdish
authors.initials: J
authors.lastname: Chandra
authors.forename: Sunita
authors.initials: S
authors.lastname: Sharma
authors.forename: Bhavna
authors.initials: B
authors.lastname: Dhingra
authors.forename: Rajesh
authors.initials: R
authors.lastname: Jain
authors.forename: DeoNath
authors.initials: D
authors.lastname: Mahto
mesh_terms: D000293:Adolescent; D001565:Benzoates / Q000627:therapeutic use*; D001803:Blood Transfusion; D002648:Child; D000077588:Deferasirox; D000077543:Deferiprone; D003676:Deferoxamine / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007194:India; D007502:Iron Chelating Agents; D007677:Kidney Function Tests; D008111:Liver Function Tests; D008297:Male; D011446:Prospective Studies; D011728:Pyridones / Q000627:therapeutic use*; D062606:Tertiary Care Centers; D014230:Triazoles / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000188:drug therapy*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D011728:Pyridones; D014230:Triazoles; D000077543:Deferiprone; D005293:Ferritins; D003676:Deferoxamine; D000077588:Deferasirox