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Updated: May 11, 2026

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Myelodysplastic syndromes: what do hospitalists need to know?
Amer M Zeidan1, Bishoy Faltas, B Douglas Smith
1Department of Oncology, The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University, Baltimore, Maryland, USA. azeidan1@jhmi.edu
Abstract:
Myelodysplastic syndromes (MDS) comprise a heterogeneous group of clonal hematopoietic stem cell disorders characterized by dysplasia, ineffective hematopoiesis leading to peripheral blood cytopenias, and a variable risk of leukemic progression. MDS primarily affects the elderly, and although its prevalence is increasing, MDS is frequently overlooked and underdiagnosed. MDS should be suspected in any patient with unexplained cytopenias, and a bone marrow evaluation is ultimately needed to make the diagnosis and exclude other causes of bone marrow failure. The last 15 years have witnessed significant advances in our understanding of the complex pathogenesis, classification and prognostication, and therapeutic approaches to MDS. Several prognostic models facilitate outcome prediction and risk-adapted therapy. The addition of azacitidine, decitabine, and lenalidomide to erythropoiesis-stimulating agents in our armamentarium offered new effective therapeutic options for many patients who are not candidates for intensive interventions. Improved understanding of the genetic, epigenetic, and immunologic mechanisms that operate in MDS will help develop better prognostication tools and rationally design more effective therapies. Hospitalists are likely to encounter both patients with MDS and patients in whom MDS should be suspected. In this review of MDS, we focus on the epidemiology, diagnosis, pathogenesis, classification and prognostic tools, and treatment options.
Insights
Myelodysplastic syndromes (MDS) are clonal stem cell disorders often overlooked in the elderly. Advances in understanding pathogenesis and new therapies offer improved prognostication and treatment options.
Area of Science:
- Hematology
- Oncology
- Stem Cell Biology
Background:
- Myelodysplastic syndromes (MDS) are heterogeneous clonal hematopoietic stem cell disorders.
- Characterized by ineffective hematopoiesis, peripheral cytopenias, and risk of leukemic transformation.
- MDS is increasingly prevalent, particularly in the elderly, yet often underdiagnosed.
Purpose of the Study:
- To review the epidemiology, diagnosis, pathogenesis, classification, prognostication, and treatment of MDS.
- To highlight the importance of suspecting MDS in patients with unexplained cytopenias.
- To discuss recent advances in understanding and managing MDS.
Main Methods:
- Review of recent literature on MDS.
- Focus on diagnostic criteria and prognostic models.
- Summary of current and emerging therapeutic strategies.
Main Results:
- Significant advances in understanding MDS pathogenesis, classification, and prognostication over the last 15 years.
- New therapeutic options including azacitidine, decitabine, and lenalidomide have improved patient outcomes.
- Prognostic models aid in risk-adapted therapy selection.
Conclusions:
- MDS requires a high index of suspicion, especially in elderly patients with cytopenias.
- Bone marrow evaluation is crucial for diagnosis and excluding other conditions.
- Continued research into genetic, epigenetic, and immunologic mechanisms will drive development of better therapies and prognostication tools.
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