Deferasirox in MDS patients with transfusion-caused iron overload—a phase-II study - Archive ouverte HAL Accéder directement au contenu
Article Dans Une Revue Annals of Hematology Année : 2008

Deferasirox in MDS patients with transfusion-caused iron overload—a phase-II study

Résumé

Blood transfusions represent a main component of supportive care in myelodysplastic syndromes (MDS). To avoid organ damage caused by transfusion-dependent iron overload, an adequate iron chelation therapy is required. Recently, a new oral iron chelator deferasirox (ICL670, Exjade®) has become available. A study was conducted to demonstrate the efficacy and tolerability of deferasirox in transfusion-dependent iron-overloaded patients with MDS. The efficacy of deferasirox was monitored by changes in serum ferritin, bone marrow iron, and liver iron concentration (LIC), as determined by T2*-weighted magnetic resonance imaging. Twelve patients with MDS of different subtypes (median age 76 years, range 53–91) were enrolled. Deferasirox administered in a once-daily dose of 20–30 mg/kg for 12 months was effective in reducing median ferritin concentration from 1,515 µg/L (range 665–6,900) to 413 µg/L (range 105–3,052). Within the first 4 weeks of treatment before the continuous decline of ferritin levels, the values markedly rose in eight of 12 patients. The median LIC declined from 315 to 230 µmol/g ( = 0.02) at the end of study, accompanied by a reduction of bone marrow siderosis. The most common adverse events were mild and transient gastrointestinal disturbances, skin rash, nonprogressive transient increases in serum creatinine and urine β2-microglobulin, and a temporary reduction of the creatinine clearance. The renal parameters normalized after end of treatment. No hematologic toxicities were observed. Deferasirox proved to be effective in transfusion-dependent iron overload in MDS by mobilizing iron deposits in liver and at least stabilizing iron stores in bone marrow.
Fichier principal
Vignette du fichier
PEER_stage2_10.1007%2Fs00277-008-0588-3.pdf (211.47 Ko) Télécharger le fichier
Origine : Fichiers produits par l'(les) auteur(s)
Loading...

Dates et versions

hal-00477975 , version 1 (30-04-2010)

Identifiants

Citer

Georgia Metzgeroth, Dietmar Dinter, Beate Schultheis, Alexandra Dorn-Beineke, Kira Lutz, et al.. Deferasirox in MDS patients with transfusion-caused iron overload—a phase-II study. Annals of Hematology, 2008, 88 (4), pp.301-310. ⟨10.1007/s00277-008-0588-3⟩. ⟨hal-00477975⟩

Collections

PEER
239 Consultations
469 Téléchargements

Altmetric

Partager

Gmail Facebook X LinkedIn More