title: One-year results from a prospective randomized trial comparing phlebotomy with deferasirox for the treatment of iron overload in pediatric patients with thalassemia major following curative stem cell transplantation.
abstract: Iron overload is well documented in patients with β-thalassemia major, and patients who have undergone hematopoietic stem cell transplantation (HSCT) remain at risk as a result of pre- and immediate post-HSCT transfusions.
  
  
  
  This is a prospective, randomized, 1-year clinical trial that compares the efficacy and safety of the once-daily oral iron chelator deferasirox versus phlebotomy for the treatment of iron overload in children with β-thalassemia major following HSCT.
  
  
  
  Patients (aged 12.4 years) received deferasirox (n = 12, 10 mg/kg/day starting dose) or phlebotomy (n = 14, 6 ml/kg/2 weeks) for 1 year. In two and five patients, deferasirox dose was increased to 15 and 20 mg/kg/day, respectively. Magnetic resonance imaging (MRI)-assessed liver iron concentration (LIC) decreased with deferasirox (mean 12.5 ± 10.1 to 8.5 ± 9.3 mg Fe/g dry weight [dw]; P = 0.0005 vs. baseline) and phlebotomy (10.2 ± 6.8 to 8.3 ± 9.2 mg Fe/g dw; P = 0.05). LIC reductions were greater with deferasirox than with phlebotomy for patients with baseline serum ferritin 1,000 ng/ml or higher (-8.1 ± 1.5 vs. -3.5 ± 5.7 mg Fe/g dw; P = 0.048). Serum ferritin and non-transferrin-bound iron also decreased significantly. In two patients with severe cardiac siderosis, a clinically relevant improvement in myocardial T2* was seen, following phlebotomy and deferasirox therapy (n = 1 each). Adverse effects with deferasirox were skin rash, gastrointestinal upset, and increased liver function tests (all n = 1), while those for phlebotomy were difficulty with venous access (n = 4) and distress during procedure (n = 1). Parents of 13/14 children receiving phlebotomy wished to switch to deferasirox, with 1/14 being satisfied with phlebotomy.
  
  
  
  Deferasirox treatment or phlebotomy reduces iron burden in pediatric patients with β- thalassemia major post-HSCT, with a manageable safety profile.
authors: Lebanese American University and University Medical Center Rizk Hospital.|Rafik Hariri University Hospital, Beirut, Lebanon.
authors: Adlette
authors: A
authors: Inati
authors: Rafik Hariri University Hospital, Beirut, Lebanon.
authors: Mario
authors: M
authors: Kahale
authors: Zahraa University Hospital, Beirut, Lebanon.
authors: Nada
authors: N
authors: Sbeiti
authors: Universitá di Milano, Ca Granda Foundation IRCCS, Milan, Italy.
authors: Maria Domenica
authors: MD
authors: Cappellini
authors: American University of Beirut, Beirut, Lebanon.
authors: Ali T
authors: AT
authors: Taher
authors: Chronic Care Center, Hazmieh, Lebanon.
authors: Suzanne
authors: S
authors: Koussa
authors: Chronic Care Center, Hazmieh, Lebanon.
authors: Therese A
authors: TA
authors: Nasr
authors: American University of Beirut, Beirut, Lebanon.
authors: Khaled M
authors: KM
authors: Musallam
authors: University of Texas MD Anderson Cancer Center, Houston, Texas.
authors: Hussein A
authors: HA
authors: Abbas
authors: University College London, London, UK.
authors: John B
authors: JB
authors: Porter
mesh_terms: D000293:Adolescent; D001565:Benzoates / Q000627:therapeutic use*; D002648:Child; D002675:Child, Preschool; D003131:Combined Modality Therapy; D000077588:Deferasirox; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects*; D006801:Humans; D007502:Iron Chelating Agents / Q000627:therapeutic use*; D019190:Iron Overload / Q000175:diagnosis / Q000209:etiology / Q000628:therapy*; D008297:Male; D018962:Phlebotomy / Q000379:methods*; D011379:Prognosis; D011446:Prospective Studies; D014230:Triazoles / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000628:therapy*
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D000077588:Deferasirox
keywords: deferasirox; hematopoietic stem cell transplant; phlebotomy; randomized controlled trial; thalassemia