title: Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients.
abstract: The 1-year THALASSA study enrolled 166 patients with various non-transfusion-dependent thalassemia (NTDT) syndromes, degrees of iron burden and patient characteristics, and demonstrated the overall efficacy and safety of deferasirox in reducing liver iron concentration (LIC) in these patients. Here, reduction in LIC with deferasirox 5 and 10 mg/kg/day starting dose groups is shown to be consistent across the following patient subgroups-baseline LIC/serum ferritin, age, gender, race, splenectomy (yes/no), and underlying NTDT syndrome (β-thalassemia intermedia, HbE/β-thalassemia or α-thalassemia). These analyses also evaluated deferasirox dosing strategies for patients with NTDT. Greater reductions in LIC were achieved in patients dose-escalated at Week 24 from deferasirox 10 mg/kg/day starting dose to 20 mg/kg/day. Patients who received an average actual dose of deferasirox >12.5-≤17.5 mg/kg/day achieved a greater LIC decrease compared with the ≥7.5-≤12.5 mg/kg/day and >0-<7.5 mg/kg/day subgroups, demonstrating a dose-response efficacy. LIC reduction across patient subgroups was generally consistent with the primary efficacy analysis with a similar safety profile.
authors.affiliation: Hematology and Oncology, Department of Internal Medicine, American University of Beirut, Beirut, Lebanon. ataher@aub.edu.lb
authors.forename: Ali T
authors.initials: AT
authors.lastname: Taher
authors.forename: John B
authors.initials: JB
authors.lastname: Porter
authors.forename: Vip
authors.initials: V
authors.lastname: Viprakasit
authors.forename: Antonis
authors.initials: A
authors.lastname: Kattamis
authors.forename: Suporn
authors.initials: S
authors.lastname: Chuncharunee
authors.forename: Pranee
authors.initials: P
authors.lastname: Sutcharitchan
authors.forename: Noppadol
authors.initials: N
authors.lastname: Siritanaratkul
authors.forename: Renzo
authors.initials: R
authors.lastname: Galanello
authors.forename: Zeynep
authors.initials: Z
authors.lastname: Karakas
authors.forename: Tomasz
authors.initials: T
authors.lastname: Lawniczek
authors.forename: Dany
authors.initials: D
authors.lastname: Habr
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Ros
authors.forename: Yiyun
authors.initials: Y
authors.lastname: Zhang
authors.forename: M Domenica
authors.initials: MD
authors.lastname: Cappellini
mesh_terms: D000328:Adult; D001565:Benzoates / Q000008:administration & dosage* / Q000009:adverse effects; D000077588:Deferasirox; D004305:Dose-Response Relationship, Drug; D004311:Double-Blind Method; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000008:administration & dosage* / Q000009:adverse effects; D019190:Iron Overload / Q000097:blood / Q000188:drug therapy / Q000378:metabolism; D008099:Liver / Q000187:drug effects* / Q000378:metabolism*; D008297:Male; D013789:Thalassemia / Q000097:blood / Q000188:drug therapy* / Q000378:metabolism*; D014230:Triazoles / Q000008:administration & dosage* / Q000009:adverse effects; D055815:Young Adult
chemical_list: D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000077588:Deferasirox
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