Abstract | PURPOSE: PATIENTS AND METHODS: Eligible patients had serum ferritin ≥ 1,000 μg/L and had received ≥ 20 units of RBCs with ongoing transfusion requirements. The starting dose of deferasirox was 20 mg/kg/d, with dose escalation up to 40 mg/kg/d permitted. RESULTS: A total of 176 patients were enrolled, and 173 patients received therapy. Median serum ferritin decreased 23% in the 53% of patients who completed 12 months of treatment (n = 91), 36.7% in patients who completed 2 years (n = 49), and 36.5% in patients who completed 3 years (n = 33) despite continued transfusion requirement. Reduction in serum ferritin significantly correlated with ALT improvement (P < .001). Labile plasma iron (LPI) was measured quarterly during the first year of the study. Sixty-eight patients (39.3%) had elevated LPI at baseline. By week 13, LPI levels normalized in all patients with abnormal baseline level. Fifty-one (28%) of 173 patients experienced hematologic improvement by International Working Group 2006 criteria; of these, only seven patients received growth factors or MDS therapy. Over the 3-year study, 138 (79.8%) of 173 patients discontinued therapy, 43 patients (24.8%) because of adverse events or disease progression and 23 patients (13.2%) because of abnormal laboratory values. The most common drug-related adverse events were gastrointestinal disturbances and increased serum creatinine. There were 28 deaths, none of which were considered related to deferasirox. CONCLUSION:
Deferasirox reduces serum ferritin and LPI in transfusion-dependent patients with MDS. A subset of patients had an improvement in hematologic and hepatic parameters.
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Authors | Alan F List, Maria R Baer, David P Steensma, Azra Raza, Jason Esposito, Noelia Martinez-Lopez, Carole Paley, John Feigert, Emmanuel Besa |
Journal | Journal of clinical oncology : official journal of the American Society of Clinical Oncology
(J Clin Oncol)
Vol. 30
Issue 17
Pg. 2134-9
(Jun 10 2012)
ISSN: 1527-7755 [Electronic] United States |
PMID | 22547607
(Publication Type: Clinical Trial, Phase II, Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Chemical References |
- Benzoates
- HFE protein, human
- Hemochromatosis Protein
- Histocompatibility Antigens Class I
- Iron Chelating Agents
- Membrane Proteins
- Triazoles
- Ferritins
- Iron
- Deferasirox
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Topics |
- Adult
- Aged
- Aged, 80 and over
- Benzoates
(pharmacology)
- Deferasirox
- Erythrocyte Transfusion
(methods)
- Female
- Ferritins
(blood)
- Hemochromatosis Protein
- Histocompatibility Antigens Class I
(genetics)
- Humans
- Iron
(blood)
- Iron Chelating Agents
(pharmacology)
- Male
- Membrane Proteins
(genetics)
- Middle Aged
- Mutation
- Myelodysplastic Syndromes
(blood, drug therapy)
- Prospective Studies
- Time Factors
- Treatment Outcome
- Triazoles
(pharmacology)
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