Multicenter Validation of the Scoring System of Pre-Transplant Serum Ferritin and Disease Risk in Patients with Acute Myeloid Leukemia and Myelodysplastic Syndrome After Allogeneic Hematopoietic Stem Cell Transplantation

Abstract

Abstract 2032 Background:Recent studies have suggested that iron overload is one of important predictors for outcome in allogeneic hematopoietic stem cell transplantation (allo-HSCT). We previously reported that a new scoring system by combination with pre-transplant serum ferritin (SF) and disease risk is useful for predicting outcome in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) who underwent allo-HSCT at Kanagawa Cancer Center (KCC) (Bone Marrow Transplantation 46: 150–151, 2011). To clarify the availability of this scoring system in another cohort, we performed a validation in multicenter study. Patients and methods:We retrospectively collected clinical data of adult patients with AML and MDS who received allo-HSCT between 2000 and 2010 at three institutes. The patients analyzed in the previous study were excluded. Pre-transplant SF value was categorized as low-SF (<1000 ng/ml) and high-SF (≥1000 ng/ml) according to the previous report. First/second complete remission in AML and refractory anemia in MDS were defined as standard disease risk and others were defined as high risk. Pre-transplant SF and disease risk were each assigned a score, and the sum total was tested as prognostic score based on three risk groups: low (score=0), intermediate (score=1) and high (score=2). The log-rank test was used for comparisons of Kaplan-Meier curves. Cox regression model was used for multivariate analyses. Results:A total of 153 patients were analyzed. Their median age was 48 years old (range: 17–65), with 57 males and 96 females. Related or unrelated bone marrow (n=106), related peripheral blood stem cell (n=14) or unrelated cord blood (n=33) were transplanted as stem cell sources. Myeloablative conditioning regimens were used in 112 patients while reduced-intensity regimens in 41. The median level of pre-transplant SF was 1,139 ng/ml (range:22–6,262) in all patients. Standard and high disease risks were 82 and 71 patients, respectively. There were no significant differences of patient characteristics among the three institutes except pre-transplant SF level. The median follow-up period among survivors was 25.9 months (range: 7.1–124). Univariate analysis for 5-year overall survival (OS) demonstrated an inferior outcome in patient with high SF (41% vs. 48%, p=0.025) or high disease risk (21% vs. 61%, p<0.001), compared with low SF or standard disease risk, respectively. Multivariate analysis for 5-year OS demonstrated high SF (HR: 1.794, 95% CI: 1.11–2.91, p=0.018) and high disease risk (HR: 2.08, 95% CI: 1.26–3.41, p=0.004) at transplantation as inferior prognostic predictors. According to the scoring system with pre-transplant SF and disease risk, the 5-year OS of the low (n=36), intermediate (n=82) and high (n=35) risk group was 68%, 42% and 20%, respectively (p<0.001). Conclusions:The usefulness of the simple scoring system with pre-transplant SF and disease risk was validated by the multicenter analysis. This prognostic scoring system might help to stratify patients in clinical studies and choose patients for iron chelating therapy. [Display omitted] Disclosures:No relevant conflicts of interest to declare.