Myeloproliferative Neoplasm (MPN) Driver Mutations Are Enriched during Hematopoietic Stem Cell Differentiation in Patterns That Correlate with Clinical Phenotype and Treatment Response

Conclusion. The pattern of clonal enrichment of an MPN stem cell during hematopoiesis is unique to individual pts and varies significantly among the 3 major WHO subtypes of PV, ET, and PMF. This pattern also differs in relation to the disease stage, and is informative of treatment effects. WB VAF does not predict the proportion of immature HSPC harboring driver mutation. For these reasons, evaluating the clonal heterogeneity of MPN driver mutations, particularly those in HSPCs, may provide a useful surrogate measure to qualify response to novel agents in pre-clinical and clinical studies in MPNs. This is currently being tested.DisclosuresRitchie: NS Pharma: Research Funding; Incyte: Consultancy, Speakers Bureau; Bristol-Myers Squibb: Research Funding; ARIAD Pharmaceuticals: Speakers Bureau; Astellas Pharma: Research Funding; Pfizer: Consultancy, Research Funding; Novartis: Consultancy, Other: Travel, Accommodations, Expenses, Research Funding, Speakers Bureau; Celgene: Consultancy, Other: Travel, Accommodations, Expenses, Speakers Bureau.
Source: Blood - Category: Hematology Authors: Tags: 635. Myeloproliferative Syndromes: Basic Science: Poster III Source Type: research