Factors affecting response to 5-azacytidine and prognosis of myelodysplastic syndrome. Is long-term survival a realistic goal?

Abstract

The introduction of hypomethylating agents (HMAs) 5-azacytidine and decitabine has altered the prognosis of patients with myelodysplastic syndrome (MDS). Over the past few years, the International Prognostic Scoring System (IPSS) and the revised IPSS (IPSS-R) have been used both to define the prognosis of patients with MDS and to select patients to be treated with HMAs. Nevertheless, the prognosis of individual patients with MDS can differ considerably from the one calculated with the use of the above-mentioned prognostic systems. Thus, some patients may achieve long-term survival irrespective of their initial prognostic score. Several factors besides those used to define the IPSS/IPSS-R are analyzed in this review article; these include age and gender, the baseline hematologic characteristics, the comorbidities, the cytogenetic and molecular profile of the patients, as well as their response to treatment with 5-azacytidine. Thus, insight into a more personalized way of managing patients with MDS is given and long-term survival is set as a more realistic goal of treatment with 5-azacytidine.

Keywords: 5-azacytidine; Hypomethylating agents; Myelodysplastic syndrome; Prognosis; Response.