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Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS

Overall Goal:  

To build a trans-NIH Intramural Research Program (IRP)-based collaborative translational science network of myeloid dysplastic syndromes (MDS) clinical trial data, accompanying biospecimens and correlative laboratory data.

Specific Aim: 

Characterize the disease course and biology of MDS and use biological samples to lead research aimed at developing novel treatment strategies to significantly improve patient outcomes.

About the Study

The Comprehensive Molecular and Clinical Evaluation of Pediatric and Adult MDS protocol is an NCI CCR intramural research program study characterizing the biology and natural history of disease in adults as well as pediatric, adolescents, and young adults (AYA). In addition, comprehensive research analyses based on the hypothesis that chronic inflammation in MDS arising from a variety of contributors (i.e., germline and somatic gene mutations, altered transcriptional and translational regulation, metabolic reprogramming, and microenvironmental factors) results in hematopoietic differentiation and maturation blocks leading to dysplasia, cytopenias, and disease progression are underway in and effort to develop novel therapeutic targeting strategies. To learn more, see NCT05350748 or contact our clinical team.

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Core biopsy H&E of dysplastic megakaryocites.
Core biopsy H&E of dysplastic megakaryocytes. Image courtesy of Kathy Calvo, M.D., Ph.D.