Therapy of high risk myelodysplastic syndromes (MDS) remains unsatisfactory. The only treatment that alters the natural history of the disease is allogeneic stem-cell transplantation, which can be offered to a limited number of young patients.
Modern therapy predominantly focuses on prolonging survival, postponing potential acute myeloid leukaemia (AML) transformation and improving quality of life (QoL). New epigenetic therapy, especially methyltransferase inhibitors, has achieved these goals.
Here, we present the experience of the Czech MDS Cooperative Group with 5-azaci- tidine treatment of patients with intermediate II and high risk MDS patients (IPSS), chronic myelomonocytic leukaemia II (CMML II) and AML with less than 30% myeloblasts in the bone marrow. Conclusion.
Our results correlate very well with other published data (AZA 001, CALGB 9221). OS data are comparable with other “real life” studies.
It is also important to take into consideration that our group also included very high risk patients (relapsed, refractory to previous treatment, post-transplant patients and AML with more than 30% myeloblasts). Nevertheless, our results are very promising and support the positive effect of azacitidine compared to standard therapy in high risk MDS patients.
The analysis performed so far shows that from the aspect of OS, best response correlates with: cytogenetic aberration, peripheral blast count, neutrophil count and ECOG PS.