Novel dynamic outcome indicators and clinical endpoints in myelodysplastic syndrome; the European LeukemiaNet MDS Registry and MDS-RIGHT project perspective

摘要

Myelodysplastic syndromes (MDS) are chronic bone marrow (BM) disorders characterized by peripheral blood cytopenias, predominantly in older persons with an average age at diagnosis of 75 years.1,2 The natural history of MDS is heterogeneous, ranging from indolent conditions to forms similar to acute myeloid leukemia (AML). In 1997, an International Prognostic Scoring System (IPSS) was established based on the percentage of BM blasts, number of cytopenias and cytogenetic characteristics.3 In 2012, this prognostic scoring system was refined (IPSS-R) to include better categorization of cytopenias, blast cell percentage and an improved risk stratification of the cytogenetic risk groups.4 Generally, MDS are divided into two prognostic groups: lower-risk MDS (LR-MDS) with patients from the (very) low risk or intermediate risk groups, and higher-risk MDS (HR-MDS) with patients from the (very) poor risk groups, as defined within the IPSS-R.2