Deferasirox in iron-overloaded patients with transfusion-dependent myelodysplastic syndromes: Results from the large 1-year EPIC study.
The prospective 1-year EPIC study enrolled 341 patients with myelodysplastic syndromes (MDS); although baseline iron burden was >2500ng/mL, approximately 50% were chelation-naïve. Overall median serum ferritin decreased significantly at 1 year (p=0.002). Decreases occurred irrespective of whether patients were chelation-naïve or previously chelated; changes were dependent on dose adjustments and ongoing iron intake. Sustained reductions in labile plasma iron were observed. Discontinuation rate (48.7%) and adverse event profile were consistent with previously reported deferasirox data in MDS. Alanine aminotransferase levels decreased significantly; change correlated significantly with reduction in serum ferritin (p<0.0001). This large dataset prospectively confirms the efficacy and well characterizes the safety profile of deferasirox in MDS.
Gattermann
Norbert
N
Heinrich-Heine-University, Düsseldorf, Germany. gattermann@med.uni-duesseldorf.de
Finelli
Carlo
C
Porta
Matteo Della
MD
Fenaux
Pierre
P
Ganser
Arnold
A
Guerci-Bresler
Agnes
A
Schmid
Mathias
M
Taylor
Kerry
K
Vassilieff
Dominique
D
Habr
Dany
D
Domokos
Gabor
G
Roubert
Bernard
B
Rose
Christian
C
D000293:Adolescent; D000328:Adult; D000368:Aged; D000410:Alanine Transaminase / Q000097:blood; D001565:Benzoates / Q000009:adverse effects / Q000627:therapeutic use*; D001803:Blood Transfusion; D002648:Child; D002675:Child, Preschool; D000077588:Deferasirox; D005260:Female; D005293:Ferritins / Q000097:blood; D006801:Humans; D007502:Iron Chelating Agents / Q000009:adverse effects / Q000627:therapeutic use*; D019190:Iron Overload / Q000150:complications / Q000188:drug therapy*; D008297:Male; D008875:Middle Aged; D009190:Myelodysplastic Syndromes / Q000150:complications*; D014230:Triazoles / Q000009:adverse effects / Q000627:therapeutic use*; D055815:Young Adult
D001565:Benzoates; D007502:Iron Chelating Agents; D014230:Triazoles; D005293:Ferritins; D000410:Alanine Transaminase; D000077588:Deferasirox