Adequate iron chelation therapy for at least six months improves survival in transfusion-dependent patients with lower risk myelodysplastic syndromes

作者：Delforge Michel; Selleslag Dominik; Beguin Yves; Triffet Agnes; Mineur Philippe; Theunissen Koen; Graux Carlos; Trullemans Fabienne; Boulet Dominique; Van Eygen Koen; Noens Lucien; Van Steenweghen Steven; Lemmens Jan; Pierre Pascal; D' hondt Randal; Ferrant Augustin; Deeren Dries; Van De Velde Ann; Wynendaele Wim; Andre Marc; De Bock Robrecht; Efira Andre; Breems Dimitri; Deweweire Anne; Geldhof Kurt; Pluymers Wim; Harrington Amanda; MacDonald Karen

来源：Leukemia Research, 2014, 38(5): 557-563.

DOI：10.1016/j.leukres.2014.02.003

• Myelodysplastic syndromes

Background: Most patients with myelodysplastic syndromes (MDS) require transfusions at the risk of iron overload and associated organ damage, and death. Emerging evidence indicates that iron chelation therapy (ICT) could reduce mortality and improve survival in transfusion-dependent MDS patients, especially those classified as International Prognostic Scoring System (IPSS) Low or Intermediate-1 (Low/Int-1). %26lt;br%26gt;Methods: Follow-up of a retrospective study. Sample included 127 Low/Int-1 MDS patients from 28 centers in Belgium. Statistical analysis stratified by duration (%26gt;= 6 versus %26lt; 6 months) and quality of chelation (adequate versus weak). %26lt;br%26gt;Results: Crude chelation rate was 63% but 88% among patients with serum ferritin %26gt;= 1000 mu g/L. Of the 80 chelated patients, 70% were chelated adequately mainly with deferasirox (26%) or deferasirox following deferoxamine (39%). Mortality was 70% among non-chelated, 40% among chelated, 32% among patients chelated %26gt;= 6 m, and 30% among patients chelated adequately; with a trend toward reduced cardiac mortality in chelated patients. Overall, median overall survival (OS) was 10.2 years for chelated and 3.1 years for non-chelated patients (p %26lt; 0.001). For patients chelated %26gt;= 6 m or patients classified as adequately chelated, median OS was 10.5 years. Mortality increased as a function of average monthly transfusion intensity (HR = 1.08, p = 0.04) but was lower in patients receiving adequate chelation or chelation = 6 m( HR = 0.24, p %26lt; 0.001). %26lt;br%26gt;Conclusion: Six or more months of adequate ICT is associated with markedly better overall survival. This suggests a possible survival benefit of ICT in transfusion-dependent patients with lower-risk MDS.

• 出版日期2014-5