Improved Survival in Patients with Myelodysplastic Syndrome Receiving Iron Chelation Therapy

Abstract

Purpose

Patients with myelodysplastic syndrome (MDS) and iron overload (IOL) often receive iron chelation therapy (ICT); however, data on clinical outcomes are limited. We reviewed 178 patients with MDS to determine the effect of ICT on survival.

Patients and Methods

Data were collected by chart review and survival analysis performed. A subgroup analysis compared control patients with clinical features similar to patients who received ICT.

Results

French-American-British MDS subtypes for patients were as follows: refractory anemia (RA), n = 36; RA with ringed sideroblasts, n = 42; RA with excess blasts (RAEB), n = 28; RAEB in transformation or acute myeloid leukemia (AML), n = 16; chronic myelomonocytic leukemia, n = 25; other, n = 31. International Prognostic Scoring System (IPSS) scores were as follows: low risk, n = 44; intermediate-1 risk, n = 55; intermediate-2 risk, n = 17; high risk, n = 17. Eighteen patients received ICT; median duration was 21.6 months (range, 1.3-151 months). In univariate analysis (UVA), factors significant for overall survival (OS) were IPSS score; MDS subtype; number of red blood cell (RBC) units transfused; MDS treatment; elevated ferritin; clinical IOL; receiving ICT (P < .05 for all); and age (P = .01). In multivariate analysis (MVA), significant factors included IPSS score (P = .008; hazard ratio [HR], 2.2 [95% CI, 1.3-3.7]) receiving ICT (P = .02; HR, 0.2 [95% CI, 0.01-1.0]). For low/intermediate-1 risk IPSS score, 4-year OS was 64% for patients receiving ICT and 43% for patients not receiving ICT (P = .003). An MVA was performed, including number of cytopenias; blast count; karyotype; AML transformation; ≥ 1 serious infection (P < .05 in UVA for all) with MDS treatment; number of RBC units transfused; and clinical IO; receipt of iron chelation therapy determined that factors significant for OS were infection (P = .05; HR, 3.2 [95% CI, 0.97-10.4]) and ICT (P = .02). Improved OS was maintained in the subgroup analysis (P = .01; HR, 0.29 [95% CI, 0.1-0.79]).

Conclusion

Patients with MDS and IOL receiving ICT had improved survival compared with patients not receiving ICT, suggesting a possible beneficial effect on clinical outcome. Prospective studies of ICT in MDS are warranted.