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Updated: Jul 11, 2025

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
[Recent findings in myelodysplastic syndrome]
Abstract:
Myelodysplastic syndromes (MDS) represent a heterogeneous group of myeloid disorders characterized by peripheral blood cytopenias and increased risk of transformation to acute myeloid leukemia (AML).Recent developments include the classification and the estimation of prognosis. In 2022 the former 2016 WHO classification was replaced by the ICC and WHO 2022 classification. Both classifications have included precursor lesions (CHIP and ICUS), both distinguish between three molecularly cytogenetically defined subgroups - del(5q), TP53, SF3B1 - and morphologically defined subgroups with differences in blast threshold (WHO: 20%; ICC: 10%) for the differentiation from AML. However, although prognostic factors influenced the classification-subgroups, it is important to distinguish the prognosis, which is crucial for optimal therapeutic decision making. Since 2022, the IPSS-M has been available for this purpose, which represents an expansion of the well-established IPSS-R. It could improve prognosis estimation by adding molecular data, recently this could have been confirmed in real world cohorts. The IPSS-M also represents an important extension with regard to prognosis estimation for patients with therapy-related MDS.In 2020 Luspatercept has been approved for transfusion-dependent lower risk MDS patients harboring ring sideroblasts ± an SF3B1 mutation after failure of an erythropoiesis stimulating agent. The COMMANDS trial has just reported an interim analysis, where the superiority of luspatercept in the 1st line compared to erythropoietin could be demonstrated. In addition, data from the phase III trial with Imeltelstat give reason to hope that we will be able to offer a new second-line therapy to LR-MDS patients. For higher risk MDS patients azacitidine therapy remains the standard of care, results of phase III trials of combination therapies must be awaited.

