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Hematopoietic cell transplantation in myelodysplastic syndromes

Author
Robert S Negrin, MD
Section Editor
Nelson J Chao, MD
Deputy Editor
Alan G Rosmarin, MD

INTRODUCTION

The myelodysplastic syndromes (MDS) encompass a series of hematologic conditions characterized by chronic cytopenias with abnormal cellular maturation. Concomitant risks are infection, bleeding, anemia, and an increased risk of progression to acute leukemia, which is often refractory to standard treatments. (See "Clinical manifestations and diagnosis of the myelodysplastic syndromes".)

There is no agreed upon standard treatment approach for patients with symptomatic MDS, and patients should be encouraged to enroll in clinical trials whenever available. Our treatment approach is similar to that proposed by the MDS Panel for Practice Guidelines of the National Comprehensive Cancer Network (NCCN) and recommendations from the European LeukemiaNet [1,2]. This approach incorporates knowledge of the patient's performance status, the International Prognostic Scoring System (IPSS) (table 1) (calculator 1) and revised IPSS (IPSS-R) (table 2) (calculator 2) MDS risk categories, and other disease characteristics (ie, cytopenias present, serum erythropoietin level) to help guide management decisions. (See "Overview of the treatment of myelodysplastic syndromes", section on 'Selection of initial treatment for patients with symptomatic MDS'.)

Allogeneic hematopoietic cell transplantation (HCT) is a treatment option for patients with intermediate-2, high, or very high risk MDS. In contrast, autologous HCT is not typically employed for patients with MDS. A choice among treatment options in these populations is discussed in more detail separately. (See "Treatment of high or very high risk myelodysplastic syndromes", section on 'Choice of therapy'.)

The treatment of MDS with HCT will be reviewed here. The clinical manifestations, diagnosis, prognosis, and treatment of this disorder with other therapies and the role of HCT in children with MDS are discussed separately. (See "Hematopoietic cell transplantation for Diamond-Blackfan anemia and the myelodysplastic syndromes in children and adolescents" and "Overview of the treatment of myelodysplastic syndromes".)

The term "hematopoietic cell transplantation" (HCT) will be used throughout this review as a general term to cover transplantation of progenitor cells from any source (eg, bone marrow, peripheral blood, cord blood). Otherwise, the source of such cells will be specified (eg, autologous peripheral blood progenitor cell transplantation). (See "Sources of hematopoietic stem cells".)

                      

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Literature review current through: Nov 2016. | This topic last updated: Mon Jul 18 00:00:00 GMT+00:00 2016.
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References
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