Management of Adult Patients with Myelodysplastic Syndromes

Abstract

The myelodysplastic syndromes (MDS) form a heterogeneous group of clonal disorders with an increasing incidence in the elderly population and an emerging impact on healthcare resources. MDS are caused by gene mutations affecting the haematopoietic stem cells, leading to ineffective haematopoiesis, characterised by dysplasia and cytopenia, and a propensity to evolve towards secondary acute myeloid leukaemia (AML). Accurate diagnosis and risk assessment are essential for the correct treatment allocation. In lower-risk MDS patients, median survival reaches 3–8 years and mortality is mainly caused by cytopenia (cardiovascular events, infections, and bleeding). Therefore, the treatment for these patients should be focussed on reduction of disease-related complications, disease progression, and improvement of quality of life. In contrast, in higher-risk MDS patients, median survival ranges from 1–3 years and death from transformation to AML exceeds non-leukaemic mortality. Treatment should be aimed to delay progression to AML and improve overall survival. Allogeneic haematopoietic stem cell transplant remains the only curative option for higher-risk MDS patients. However, only a minority of patients are eligible for such intensive treatment. Consequently, most patients are managed with supportive care and palliative treatment, including growth factors, immune-modulators, and hypomethylating agents. Since elderly patients with chronic cytopenia are frequently seen in general practice, awareness of the wide spectrum of presentations of MDS and potential courses of lower and higher-risk diseases are important for primary healthcare physicians.