In this single-center retrospective analysis, the response of 76 patients with high-risk myelodysplastic syndrome (MDS) treated with azacitidine (AZA) was reviewed, especially the cytogenetic response (CGR) to determine the response to treatment and the possible impact of karyotype evolution (KE) on the course of the disease.
So far, the only treatment option with curative potential for RA MDS patients is allogeneic stem cell transplantation. Although transplantation procedures have improved and an increasing number of patients can be transplanted, most patients with MDS are ineligible due to their age or comorbidities. For patients with MDS-AR, demethylating agents (DMARDs) such as azacitidine (AZA) and decitabine lead to significantly longer overall survival (OS) and AML-free survival and better quality of life compared to conventional treatment regimens.
With the present data they were able to demonstrate that in MDS-AR patients treated with AZA there is a close relationship between cytogenetic and hematologic response: patients with cytogenetic response showed significantly more hematologic improvement than non-responders and patients with hematologic response at 3 months significantly more often achieved ICER. KE was associated with significantly worse OS and PFS. Peripheral blood cytogenetic monitoring may serve as an additional less invasive, simple but useful tool to monitor clonal response to therapy.
