Abstract

Hypomethylating drugs are useful and have been approved for the treatment of myelodysplastic syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML). However, phase 2 and 3 studies that assessed these agents in MDS, have included only a small number of patients with CMML, and there are just a few specific reports on CMML patients. The Azacitidine is actually authorised for the treatment of CMML patients with 10–29% marrow blasts without myeloproliferative disorder, who are not eligible for haematopoietic stem cell transplantation. This hypomethylating agent in MDS is known for causing transient cytopenias, most often occurring during the first 2 cycles. Here we report a case of an atypical delayed and prolonged hematologic toxicity during Azacitidine treatment in a CMML patient; furthermore we also reviewed the literature regarding the efficacy of the drug and the management of hematologic adverse effects, in term of dose adjustments or alternative schedule of administration, in specific CMML setting.

Highlights

  • Chronic Myelomonocytic Leukemia (CMML) is a clonal disorder of haematopoietic stem cell, often occurring in elderly patients, sharing heterogeneous clinical and morphological features of both myelodysplastic syndromes (MDS) and chronic myeloproliferative disorders (MPD)

  • We report a case of an atypical delayed and prolonged hematologic toxicity during Azacitidine treatment in a patient affected by CMML; we reviewed the literature regarding the efficacy of the drug and the management of hematologic adverse effects, in term of dose adjustments or alternative schedule of administration, in specific CMML setting

  • The efficacy of Azacitidine and Decitabine for the treatment of MDS has been confirmed by several national and international clinical trials.[7,8,9,12]. These studies proved efficacy of hypomethylating agents compared to best supportive care

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Summary

Introduction

CMML is a clonal disorder of haematopoietic stem cell, often occurring in elderly patients, sharing heterogeneous clinical and morphological features of both MDS and chronic myeloproliferative disorders (MPD). We report a case of an atypical delayed and prolonged hematologic toxicity during Azacitidine treatment in a CMML patient; we reviewed the literature regarding the efficacy of the drug and the management of hematologic adverse effects, in term of dose adjustments or alternative schedule of administration, in specific CMML setting.

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