title: Deferasirox effectively decreases iron burden in patients with double heterozygous HbS/β-thalassemia.
abstract: Iron overload is present in several cases of double heterozygous sickle-cell/beta-thalassemia (HbS/β-thal). Deferasirox is an orally administered iron chelator which is effective on iron overloaded patients with transfusion-dependent anemia. The aim of this study was to investigate the efficacy and safety of deferasirox on HbS/β-thal patients with iron overload. We evaluated 31 adult patients with HbS/β-thal (14M/17F; median age 41 years) who had serum ferritin levels >1,000 ng/mL and who were sporadically transfused. Total iron burden was monitored by measuring serum ferritin levels before and monthly after starting deferasirox, while liver iron concentration and cardiac iron burden were measured by magnetic resonance imaging (MRI) T2 and T2* parameters at baseline and 12 months after deferasirox treatment. Deferasirox managed to reduce the mean serum ferritin levels after 12 months of treatment from 1,989 ± 923 to 1,008 ± 776 ng/mL (P < 0.001). This reduction was accompanied by a significant improvement on MRI T2* of the liver (from 3.9 ± 3.2 to 5.8 ± 3.1 ms; P < 0.01) and by a comparable improvement of biochemical parameters of liver function. Mild nausea and diarrhea of grade 1/2 were reported in 25% of patients within the first month of treatment, but did not re-occur. These data indicate that deferasirox provided effective control of iron levels (mainly of the liver) in minimally transfused patients with HbS/β-thal, without significant adverse events, at similar doses to those studied widely for the treatment of patients with thalassemia syndromes.
authors.affiliation: Thalassemia Unit, Laikon General Hospital, 16 Sevastoupoleos Street, 11526, Athens, Greece. ersi_voskaridou@yahoo.com
authors.forename: Ersi
authors.initials: E
authors.lastname: Voskaridou
authors.forename: Eleni
authors.initials: E
authors.lastname: Plata
authors.forename: Marousa
authors.initials: M
authors.lastname: Douskou
authors.forename: Anastasia
authors.initials: A
authors.lastname: Sioni
authors.forename: Efrosini
authors.initials: E
authors.lastname: Mpoutou
authors.forename: Dimitrios
authors.initials: D
authors.lastname: Christoulas
authors.forename: Maria
authors.initials: M
authors.lastname: Dimopoulou
authors.forename: Evangelos
authors.initials: E
authors.lastname: Terpos
mesh_terms: D000328:Adult; D000755:Anemia, Sickle Cell / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000235:genetics; D001565:Benzoates / Q000494:pharmacology / Q000627:therapeutic use*; D000077588:Deferasirox; D015536:Down-Regulation / Q000187:drug effects; D005260:Female; D006451:Hemoglobin, Sickle / Q000235:genetics*; D006579:Heterozygote; D006801:Humans; D007501:Iron / Q000378:metabolism; D007502:Iron Chelating Agents / Q000494:pharmacology / Q000627:therapeutic use; D019190:Iron Overload / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000235:genetics; D008099:Liver / Q000000981:diagnostic imaging / Q000187:drug effects / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D011859:Radiography; D014230:Triazoles / Q000494:pharmacology / Q000627:therapeutic use*; D017086:beta-Thalassemia / Q000150:complications / Q000000981:diagnostic imaging / Q000188:drug therapy* / Q000235:genetics
chemical_list: D001565:Benzoates; D006451:Hemoglobin, Sickle; D007502:Iron Chelating Agents; D014230:Triazoles; D007501:Iron; D000077588:Deferasirox