PS1120 IRON MOBILIZATION IN A REAL LIFE COHORT OF APLASTIC ANEMIA PATIENTS TREATED WITH ELTROMBOPAG
Authors: F. Cavallaro, B. Fattizzo, G. Levati, G. Milesi, W. Barcellini
Journal: HemaSphere (2019)

Background: Most aplastic anemia (AA) patients are heavily transfusion dependent of both platelets and red blood cells (RBC), and iron overload may become an issue. Excess iron not only hampers the function of several systems (liver, heart, kidney, endocrine system), but has also a negative effect on normal hematopoiesis, which is already particularly poor in AA patients. TPO mimetic eltrombopag (EPAG) improves hematopoiesis in about 40% of patients with both relapsed/refractory and newly‐diagnosed AA, and recent reports demonstrated iron‐mobilizing activity in vitro; furthermore, harmonic oscillation of serum ferritin concomitant to EPAG initiation and discontinuation have been also observed in vivo, indicating an iron mobilization effect Aims: To evaluate iron mobilization mediated by EPAG in a real life cohort of AA patients Methods: Clinical and hematologic parameters, including iron panel, of 10 AA patients treated with EPAG at our Institution in the last 18 months were collected. Changes in iron status and their relationship with EPAG treatment were systematically recorded Results: Table 1 shows hematologic parameters and iron status at baseline and after EPAG treatment. Median age was 43 years (23–91), and male to female ratio 1:1. At the time of EPAG initiation 6 cases presented with severe AA and 60% were RBC transfusion dependent. Three patients were therapy naïve and received EPAG together with frontline IST, starting at 150 mg/day since day+14; seven cases were relapsed/refractory ones, previously treated with ATG and cyclosporine (2), cyclosporine alone (2), or steroids only (2). As regards iron status, ferritin levels and plasma iron increased from EPAG start to month +1 and +3 (p = 0.04), and transferrin saturation was persistently augmented versus baseline at month +1,+3, and +6 (p = 0.01, p = 0.03, P = 0.04, respectively). Considering non transfused patients, we observed a similar increase of these parameters after EPAG start, although baseline values were lower than in transfusion‐dependent cases. At month +12 an amelioration of iron status was observed, both in transfusion dependent and not, particularly iron and transferring saturation that did not further increase. Notably, iron chelation was performed in 2 transfused cases only, possibly accounting for this improvement. Considering hematologic parameters, Hb, ANC, and PLT values significantly ameliorated from baseline to month +3, and along the follow up. Mean Hb increase from baseline was 0.7 ± 1.6 g/dL, mean ANC increase 0.3 ± 0.48x10 9 /L, and mean PLT 27 ± 38x10 9 /L. Transfusion requirement was progressively lost, and ORR increased from 56% at month+3 to 100% at month +6 and thereafter (although CR was observed in 1 patient only) Summary/Conclusion: In our real life cohort of AA patients we observed an increase of iron and transferrin saturation levels soon after EPAG initiation, irrespective of the transfusion status. This data support the iron‐mobilizing effect of EPAG image