ABSTRACT
While the pathogenesis of MDS is not yet fully defined, hematopoietic failure resulting in peripheral blood cytopenias is the central characteristic of the disease. Several preclinical and clinical observations point to the body’s intrinsic cellular defense mechanism as an important contributor to MDS-associated ineffective hematopoiesis. These observations have clinical relevance because in some patients it is possible to use therapies that alter the number and activity of specific immune cell populations, thereby modifying a disordered immune response and restoring normal hematopoiesis. Indeed, the current (2008 v.2) iteration of the National Comprehensive Cancer Network (NCCN) guideline for MDS therapy (www.nccn.org) recommends an early assessment of whether a patient is a potential immunotherapy candidate as part of the suggested management algorithm, attesting to the considerable promise of this treatment modality.
