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Novel Approaches in MDS

Program: Education Program
Hematology Disease Topics & Pathways:
MDS, Clinical Practice (Health Services and Quality), Diversity, Equity, and Inclusion (DEI) , Chronic Myeloid Malignancies, Diseases, Myeloid Malignancies
Saturday, December 10, 2022: 4:00 PM-5:15 PM
288-290 (Ernest N. Morial Convention Center)

Description:
Myelodysplastic syndromes (MDS) are characterized clinically by a hyperproliferative bone marrow, reflective of ineffective hematopoiesis, and usually accompanied by one or more peripheral cytopenias. This session will focus on the rapid advances in both diagnosis and treatment in the entire spectrum of the clinical presentation of MDS. The identification of various molecular mutations that impact the pathogenesis of the disease has resulted in new molecularly defined subtypes of MDS and a new prognostic classification, the IPSS-M. This has also led to advances in our understanding of the evolution of cytopenias to low risk MDS and the role of clonal hematopoiesis (CH) and inflammation in this process. This is an emerging area in the field of MDS where identification, close observation, and possibly early treatment of these entities may form part of the future strategy of the care of the MDS patient.Lastly, this session will detail the innovative therapies in combination with hypomethylating gents that are moving forward in the treatment of high risk MDS and their role in the treatment of MDS with various molecular mutations.

Dr. Mario Cazzola will discuss the increasing importance of gene sequencing  and the role of molecular mutational data in the diagnosis and  risk stratification of MDS. New molecularly-defined MDS entities in the updated pathology classifications will be discussed. The Molecular International Prognostic Scoring System for MDS (IPSS-M) which incorporates molecular mutational data into MDS risk classification, will also be illustrated, using a case-based approach.

Dr. Amit Verma will discuss the role of inflammation in the pathogenesis of ineffective hematopoiesis and cytopenias in MDS. He will outline the various inflammatory mediators that are elevated in MDS, including the TGF-beta pathways as well as inflammatory cascades triggered by spliceosome mutations in MDS. He will also summarize the translational/clinical efforts targeting these pathways in proposed and ongoing clinical trials.

Dr. Borate will discuss the various advances in clinical trials looking at different combinations with hypomethylating agents that are moving forward in high risk MDS. She will focus on agents that target  various dysregulated pathways in MDS including the BCL-2 pathway, various immunotherapy targeting agents including CD47 and TIM-3 as well as other biomarker and mutation specific agents being explored in high risk MDS. Lastly she will discuss the role of traditional AML chemotherapy agents in this high risk population.

Chair:
Uma Borate, MD, The Ohio State University
Disclosures:
Borate: Incyte: Consultancy, Research Funding; BMS: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sumitomo Pharma: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Pfizer: Research Funding; Novartis: Membership on an entity's Board of Directors or advisory committees, Research Funding; Genentech: Membership on an entity's Board of Directors or advisory committees, Research Funding; Daiichi Sankyo: Membership on an entity's Board of Directors or advisory committees; Takeda: Consultancy, Membership on an entity's Board of Directors or advisory committees; AbbVie/Genentech: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Jazz: Consultancy, Research Funding.
Myelodysplastic syndromes (MDS) are characterized clinically by a hyperproliferative bone marrow, reflective of ineffective hematopoiesis, and usually accompanied by one or more peripheral cytopenias. This session will focus on the rapid advances in both diagnosis and treatment in the entire spectrum of the clinical presentation of MDS. The identification of various molecular mutations that impact the pathogenesis of the disease has resulted in new molecularly defined subtypes of MDS and a new prognostic classification, the IPSS-M. This has also led to advances in our understanding of the evolution of cytopenias to low risk MDS and the role of clonal hematopoiesis (CH) and inflammation in this process. This is an emerging area in the field of MDS where identification, close observation, and possibly early treatment of these entities may form part of the future strategy of the care of the MDS patient.Lastly, this session will detail the innovative therapies in combination with hypomethylating gents that are moving forward in the treatment of high risk MDS and their role in the treatment of MDS with various molecular mutations.

Dr. Mario Cazzola will discuss the increasing importance of gene sequencing  and the role of molecular mutational data in the diagnosis and  risk stratification of MDS. New molecularly-defined MDS entities in the updated pathology classifications will be discussed. The Molecular International Prognostic Scoring System for MDS (IPSS-M) which incorporates molecular mutational data into MDS risk classification, will also be illustrated, using a case-based approach.

Dr. Amit Verma will discuss the role of inflammation in the pathogenesis of ineffective hematopoiesis and cytopenias in MDS. He will outline the various inflammatory mediators that are elevated in MDS, including the TGF-beta pathways as well as inflammatory cascades triggered by spliceosome mutations in MDS. He will also summarize the translational/clinical efforts targeting these pathways in proposed and ongoing clinical trials.

Dr. Borate will discuss the various advances in clinical trials looking at different combinations with hypomethylating agents that are moving forward in high risk MDS. She will focus on agents that target  various dysregulated pathways in MDS including the BCL-2 pathway, various immunotherapy targeting agents including CD47 and TIM-3 as well as other biomarker and mutation specific agents being explored in high risk MDS. Lastly she will discuss the role of traditional AML chemotherapy agents in this high risk population.

Mario Cazzola, MD

University of Pavia, Pavia, Italy

Amit Verma, MD

Albert Einstein College of Medicine, Bronx, NY

Uma Borate, MD

Division of Hematology, The Ohio State University, Columbus, OH

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