Improved Leukemia-Free and Overall Survival in Patients with Myelodysplastic Syndrome Receiving Iron Chelation Therapy: A Subgroup Analysis.

Myelodysplastic syndrome (MDS) is characterized by ineffective hematopoiesis and risk of progression to acute myeloid leukemia (AML). Many MDS patients (pts) require red blood cell (RBC) transfusions, risking iron overload (IOL)-related organ dysfunction. We previously showed in a multivariate analysis of 178 pts, improved survival in 18 pts with low and int-1 IPSS risk MDS and IOL receiving iron chelation therapy (ICT), and now examine the effect of ICT on AML-free survival (LFS). The effect of ICT on cytopenias and RBC transfusion requirements (TR); was also examined. To control for possible bias favoring ICT pts, a subgroup analysis was performed. Each of 18 ICT pts had a non-ICT control pt (CP) selected and these groups are matched for gender; neutrophil count (NC), platelet count (PLTC) and hemoglobin (Hb) at diagnosis (Dx); MDS subtype; no of cytopenias, karyotype; IPSS score; ECOG Performance Status; no of serious infections; initial ferritin level (FL); total RBC units (U) received; primary MDS treatment (Rx); and duration of follow-up (FU; all p=NS). Median age in ICT pts was 64 (range 32–70) years (y) vs. 78 (39–81)y for CP (p=0.04). Features of the 178 pts are previously reported. In a univariate analysis (n=178), factors significant for LFS were: MDS subtype; IPSS risk; increased FL; total RBCU transfused; ≥1 serious infection; and receipt of ICT (all p 9 /L NC; initial (236±32 and 104±25) and FU (164±24 and 82±20) x10 9 /L PLTC; or initial (2.6±0.3 and 3.0±0.5) and FU (4.2±0.5 and 4.3±0.9) RBC-TR (no RBCU/4wk; all p=NS). In conclusion, LFS and OS in MDS pts with IOL receiving ICT were improved compared to non-ICT control pts matched for baseline features. These results support findings in the larger cohort of MDS pts and suggest there may be a beneficial effect of ICT on AML transformation and OS. Prospective studies of ICT in MDS pts are warranted.