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Thrombopoietin receptor agonist discontinuation rates and reasons among patients with immune thrombocytopenia: a study of administrative claims linked with medical chart review

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Abstract

Administrative claims provide a rich data source for retrospective studies of real-world clinical practice, yet some important data may be inconsistent or unavailable. This study explored factors influencing discontinuation of thrombopoietin receptor agonists (TPO-RAs) among patients with immune thrombocytopenia (ITP), by adding medical chart abstraction for additional details. Adult (≥ 18 years) patients with continuous commercial or Medicare Advantage with Part D health insurance coverage were included. Inclusion criteria were ≥ 1 claim for eltrombopag or romiplostim and ≥ 2 diagnoses of ITP between December 31, 2017, and January 1, 2020. Providers were asked to provide access to medical charts for abstraction. The analyses included only patients who discontinued TPO-RA and described patient characteristics, treatment patterns, platelet values, and reasons for discontinuation. Among 207 ITP patients treated with a TPO-RA, 137 (66%) discontinued treatment during the observation period. The mean TPO-RA treatment duration was 185 days. Mean platelet count at the time of discontinuation was 197 × 109/L. The most common reason for discontinuation was improvement of the patient’s condition (42%). Other reasons included worsening of ITP/lack of response (12%), adverse events (12%), and cost-related or social reasons (23%). No reason was reported for 10%. Notably 26% of patients who discontinued remained off all ITP therapy for the remainder of the study, with a mean treatment-free period of 262 days. These results emphasize that some patients with ITP are able to discontinue TPO-RA therapy and achieve durable treatment-free periods.

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Acknowledgements

Medical writing assistance was provided by Caroline Jennermann of Optum, which was contracted by Novartis to perform this study. Victoria Barghout provided critical appraisal for data interpretation.

Funding

This study was funded by Novartis Pharmaceutical Corporation.

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Authors and Affiliations

  1. Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA

    Adam Cuker

  2. Optum HEOR, Eden Prairie, MN, USA

    Lincy Lal, Caitlin Elliott, Maureen Carlyle & Carolyn Martin

  3. Novartis Pharmaceuticals Corporation, East Hanover, NJ, USA

    Anuja Roy

  4. Novartis Pharma AG, Basel, Switzerland

    Jens Haenig & Ricardo Viana

Authors
  1. Adam Cuker
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Contributions

All authors contributed to the study conception and design. Data collection and analysis were performed by Lincy Lal, Carolyn Martin, Maureen Carlyle, and Caitlin Elliott. All authors contributed to the writing and editing of the manuscript and approved the final manuscript.

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Correspondence to Carolyn Martin.

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Conflict of interest

Adam Cuker served as a consultant for Synergy, has received authorship royalties from UpToDate, and his institution has received research support on his behalf from Alexion, Bayer, Novartis, Novo Nordisk, Pfizer, Sanofi, Spark, and Takeda. Lincy Lal was an employee of Optum, which was paid by Novartis to conduct this study, at the time of the study. Anuja Roy was an employee of Novartis at the time of the study. Caitlin Elliott was an employee of Optum, which was paid by Novartis to conduct this study. Maureen Carlyle is an employee of Optum, which was paid by Novartis to conduct this study. Carolyn Martin is an employee of Optum, which was paid by Novartis to conduct this study. Jens Haenig is a Novartis AG employee and holds shares. Ricardo Viana is a Novartis AG employee and holds shares.

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Cuker, A., Lal, L., Roy, A. et al. Thrombopoietin receptor agonist discontinuation rates and reasons among patients with immune thrombocytopenia: a study of administrative claims linked with medical chart review. Ann Hematol 101, 1915–1924 (2022). https://doi.org/10.1007/s00277-022-04888-7

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