Treatment of transfusional iron overload (TIO) in patients with myelodysplastic syndrome (MDS)
Authors: A. Raptis, M. S. Duh, J. R. Weiner, S. Wang, B. Fortner, N. Mody-Patel, J. Scott
Journal: Journal of Clinical Oncology (2008)

7098 Background: More than 90% of patients (pts) with MDS are anemic (Greenberg, Blood, 1997) and may become dependent on repeated transfusions of packed red blood cells (PRBC), placing them at risk for TIO. TIO can result in organ damage if not treated with iron chelation therapies (ICT) such as oral deferasirox or IV deferoxamine. Although recent data suggest treatment of TIO improves survival in lower- risk MDS pts (Rose, ASH 2007 Meeting), little is known about the rates of TIO treatment and potential differences between treated and untreated pts. Methods: Retrospective medical records review of pts diagnosed with MDS between 1998 and 2007 (1 academic center, 3 community practices) was conducted. Inclusion criteria were diagnosis of MDS or severe anemia ≥6 months before data extraction, aged ≥ 21 years at diagnosis and received ≥1 PRBC transfusion. ICT eligibility was defined as ≥20 units of PRBC or ≥2 serum ferritin (SF) tests of ≥1,000 mcg/L. Study endpoint was proportion of ICT-eligible pts receiving ≥1 ICT prescription or infusion. Key characteristics and outcomes of treated and untreated groups were described. Results: Data for 116 pts were extracted, with 55 (47%) found to be ICT-eligible and 29 (53%) of those receiving ICT. Among the 24 ICT-eligible pts with lower-risk MDS [IPSS (Low & Intermediate-1) or WHO/FAB (RA & RARS)] at baseline, 14 (58%) received ICT. Treated pts tended to be those with lower-risk MDS and higher SF level at baseline than untreated pts. Additional findings are described in Table. Updated results will be presented for a target sample of 300 pts. Conclusions: Preliminary findings indicate that <60% of ICT-eligible MDS pts, including those with lower-risk MDS, actually received ICT in practice, suggesting that more proactive management of TIO may be warranted. Further investigation of differences between treated and untreated ICT-eligible pts may elucidate the benefits and limitations of ICT, especially among lower-risk MDS pts. All ICT-Eligible Treated Untreated Number of pts 116 29 26 Observation period (yrs), mean ± SD 2.5 ± 2.0 2.7 ± 1.9 2.2 ± 1.6 Practice setting, % Academic 13.8 24.1 26.9 Community 86.2 75.9 73.1 Female, % 53.4 41.4 46.2 Age at first MDS diagnosis (yrs), mean ± SD 70.6 ± 12.3 68.2 ± 11.8 69.3 ± 12.6 Risk classification, % Early-stage MDS 45.7 48.3 38.5 Other 24.1 31.0 42.3 Not reported 30.2 20.7 19.2 Selected lab values at 1st test post-MDS diagnosis, mean ± SD Hematocrit (%) 28.8 ± 4.9 28.0 ± 4.0 27.9 ± 6.0 Hemoglobin (g/dl) 9.7 ± 1.6 9.1 ± 1.6 9.7 ± 1.6 SF (mcg/L) 881 ± 1,082 1,490 ± 1,288 939 ± 1,084 Annual no. PRBC transfusions, mean ± SD 6.9 ± 8.4 13.3 ± 10.7 11.4 ± 7.0 Annual units of PRBC transfused, mean ± SD 13.5 ± 15.7 25.0 ± 19.4 23.0 ± 13.2 Transformed to acute myeloid leukemia (%) 8.6 13.8 15.4 Died during study period (%) 15.5 20.7 38.5 Author Disclosure Employment or Leadership Consultant or Advisory Role Stock Ownership Honoraria Research Expert Testimony Other Remuneration Novartis Novartis Novartis