ASH 2023: Validation of Composite Complete Response Definitions in Higher-Risk Myelodysplastic Syndromes Treated with Hypomethylating Agents: Real-World Analysis

Introduction 

Dr. Jan Bewersdorf stopped by to talk about his study in Higher-Risk Myelodysplastic Syndromes. The study aims to validate the Composite Complete Response (cCR) definitions introduced in the International Working Group (IWG) 2023 criteria for patients with HR-MDS undergoing treatment with Hypomethylating Agents. The IWG 2023 criteria redefined complete response (CR) and introduced provisional endpoints like CR with partial hematologic recovery (CRh), CR with limited count recovery (CR-L), and CR equivalent (CRequ) to enhance the assessment of treatment efficacy. Watch the video below or continue reading to hear more about this exciting research. 

Methods

This was a retrospective analysis, it utilized data from the Validate Database, comprising high-risk MDS patients treated with hypomethylating agents. Patients meeting eligibility criteria underwent response assessments based on both IWG 2006 and 2023 criteria. 

Results

Of 629 eligible patients, achieving IWG 2023-defined composite complete response was associated with significantly improved overall survival (26.5 months vs. 14.3 months for non-composite complete response). Notably, complete response and complete response with limited count recovery, including Complete Response with bi-lineage (CRbi) and uni-lineage (CRuni) count recovery, showed improved overall survival. However, the small number of patients achieving complete response with partial hematologic recovery and complete response is equivalent to limited specific assessments. Multivariable analysis confirmed that achieving a composite complete response per IWG 2023 criteria was independently associated with improved overall survival.

Discussion 

In this real-world analysis, a composite complete response based on IWG 2023 criteria demonstrated a significant association with improved overall survival in high-risk-MDS patients treated with hypomethylating agents-based therapy. Notably, the different complete responses proved valuable in assessing treatment responses. Ongoing additions to the database will allow for further detailed analyses and insights.

Overall, the study supports the clinical utility of IWG 2023 criteria in evaluating treatment outcomes in HR-MDS patients and emphasizes the importance of incorporating these criteria in future clinical trials assessing response rates.

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