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Updated: May 1, 2026

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
[Identification of myelodysplastic syndromes patients]
Abstract:
Myelodysplastic syndromes (MDS) are an heterogeneous group of hematological malignancies characterized by ineffective hematopoiesis and peripheral cytopenias, with a variable risk of developing acute myeloid leukemia. An accurate diagnosis is the basis of the possible selection of the most appropriate therapy. The most important parameters for an accurate prognosis and therapy are the biological characteristics of the disease, the clinical presentation, the fragility of the patient and the presence of comorbidities. MDS are mainly geriatric diseases and the presence of comorbidities and patient's poor performance status adversely affect the prognosis. Our group has proposed a system to identify the patients who have low probabilities to achieve benefit from treatment with azacitidine. A number of validated prognostic scores exist in MDS. The most widely used, although dated, is the International Prognostic Scoring System (IPSS). On IPSS scores we still base therapeutic choices. Recently a revised IPSS has been built on a larger cohort of patients still to be further validated, also in MDS treated patients. We at present can predict response to azacitidine and survival in patients treated with this drug. The goals of MDS therapy are for low-risk patients: improving quality of life through the improvement of hematopoiesis and for high risk patients: prolong survival and delay progression to acute myeloid leukemia. Currently, according to the calculated IPSS risk of the patient, age, biological parameters and comorbidities we may propose individualized therapies, which may vary from watch-and-wait to hypomethylating agent therapy, to an allogeneic transplant. Moreover, the new concept of sequential lines of therapy has gained consideration.

