Abstract

The prognosis of acute myeloid leukemia (AML) in elderly (≥65 years) patients is poor and treatment remains non-consensual especially for those who are not eligible for intensive therapies. Our group has shown that in vitro the iron chelator deferasirox (DFX) synergizes with vitamin D (VD) to promote monocyte differentiation in primary AML cells. Herein, we present results from a retrospective case-control study in which the association of DFX (1–2 g/d) and 25-hydroxycholecalciferol (100,000 IU/week) (DFX/VD) was proposed to patients following demethylating agents failure. Median survival of patients treated with DFX/VD combination (n = 17) was significantly increased in comparison with matched patients receiving best supportive care (BSC) alone (n = 13) (10.4 versus 4 months respectively). In addition, the only factor associated to an increased overall survival in DFX/VD-treated patients was serum VD levels. We conclude that DFX/VD treatment correlated with increased overall survival of AML patients in this retrospective cohort of elderly patients.

Highlights

  • Acute myeloid leukemia (AML) is a group of heterogeneous malignant diseases characterized by uncontrolled cell growth and differentiation arrest [1]

  • Because of cumulated evidences from experimental data and that both iron chelators and vitamin D (VD) analogues are safe drugs and currently used in AML patients, we proposed deferasirox and 25-hydroxycholecalciferol combination therapy to a 69-yr-old AML patient, who was refractory to high dose chemotherapy

  • We show that patients who received this combination therapy presented an increased overall survival and that VD serum level was the stronger factor that positively correlated with overall survival

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Summary

Introduction

Acute myeloid leukemia (AML) is a group of heterogeneous malignant diseases characterized by uncontrolled cell growth and differentiation arrest [1]. It has been shown that the use of demethylating agents (such as 5-azacytidine or decitabine) may induce hematological responses and increase life expectancy in elderly patients [7]. These observations provided a strong rationale for the use of iron chelators/VD therapy in the setting of high risk or relapsing AML elderly patients after failure or not fit enough for chemotherapy.

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