Treatment patterns, resource utilization and clinical outcomes in patients with higher risk myelodysplastic syndromes (MDS) in United States community practices

Abstract

Management of higher-risk myelodysplastic syndromes (HR-MDS) is challenging in the real world. We studied 200 patients with HR-MDS within a large US community hospital network. We describe the clinical presentation, patient-related factors, prognostic characteristics, treatment patterns, clinical outcomes and resource utilization. Patients with HR-MDS, treated in our community setting, were elderly (median age 76 years) with a high comorbidity burden. First-line therapy was hypomethylating agent (HMA) monotherapy (20%), lenalidomide (2%), and venetoclax (2%), while the rest were treated with supportive care. Sixty-one percent of the 200, were subsequently hospitalized within 6 months of initial diagnosis. Overall survival was 11.8 months. Curative transplantation was infrequent, HMA-based therapy was underutilized, responses were not durable, most patients became transfusion-dependent or transformed to AML, and resource utilization was substantial and was highly correlated with total in-hospital days. There is a clear unmet need for tolerable treatments that can produce durable remissions in this population.

Keywords:

• Myelodysplastic syndrome
• MDS
• real-world evidence
• community-based

Acknowledgements

We would like to acknowledge former members of the Sarah Cannon Health Economics and Outcomes Research (HEOR) team for their contribution to project management, study feasibility, design, data documentation and development of the statistical analysis plan including Angela Junglen, Emily Pless, Molly Altman, Lindsay Sears, Ryan Patrick, and Jared Holder. We would also like to thank Michael Frost and Joseph Short for managing the HCA Healthcare Research Institute Operations team and securing resources for manual screening and chart abstraction. Finally, we would like to acknowledge Chandra Corn for managing the project to close and Laura Debusk for manuscript technical review and formatting.

Authors’ contributions

MB, SAAE, and XC conceptualized the study design, secured funding, and contributed to all aspects of the study including the development of the statistical analysis plan, interpretation of results, manuscript writing and review, and leading the study team. RL performed the data cleaning and analysis, contributed to the manuscript preparation, and reviewed all results and manuscript drafts. CM oversaw the data management, created the manual screening tool, critically reviewed results, aggregated the cancer registry data, curated the manual abstraction results, and critically reviewed the results and manuscript drafts. MM served as both data coordinator and, later, data analyst where she performed the manual screening for higher-risk MDS, maintained the screening and abstraction datasets, and critically reviewed the results and manuscript drafts. EF and DR performed the chart review and abstraction of reasons for HMA discontinuation and AML progression, along with critically reviewing the manuscript drafts. DS oversaw the data science, reviewed results, and critically reviewed the final manuscript. DK contributed to study design, securing funding, and critical review of the manuscript drafts. SI provided thoughtful review and editing of the manuscript.

Disclosure statement

The views expressed in this publication represent those of the author(s) and do not necessarily represent the official views of Novartis, HCA Healthcare, or any affiliated entities. None of the authors declare any conflict of interest related to the current study beyond employment with said entities. MB, DS, MC, RL, and SAAE reported stock ownership in HCA Healthcare.

Data availability statement

The data that support the findings of this study are available upon request from the corresponding author. The data are not publicly available due to privacy restrictions.

Additional information

Funding

This study was funded by Novartis and analysis was performed by teams at Sarah Cannon and HCA Healthcare. Employees of both Novartis and Sarah Cannon/HCA Healthcare contributed to drafting, reviewing, and finalizing the manuscript.