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Multicenter evaluation of the addition of eltrombopag to immunosuppressive therapy for adults with severe aplastic anemia

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Abstract

Eltrombopag has been shown to improve response rates when added to standard therapy in adults with severe aplastic anemia in controlled trial settings. However, outcomes in real-world populations have mostly been examined in small retrospective studies. This robust, multicenter, retrospective cohort study across six academic health systems compared outcomes in patients who received immunosuppressive therapy with or without eltrombopag. The study included 82 patients who received front-line therapy from January 2014 to August 2021. Overall response rates at 6 months did not differ significantly for patients receiving eltrombopag versus immunosuppressive therapy alone (58% v. 65%, p = 0.56). However, complete response rates at 6 and 12 months were over two times higher in the eltrombopag arm (29% v. 12%, p = 0.06 and 48% v. 18%, p = 0.005). Rates of hepatotoxicity were similar across both arms. Eltrombopag addition did not impact overall survival (median not reached in either arm at 2 years, p = 0.86) or disease-free survival (median not reached v. 13.3 months at 2 years, p = 0.20). Eltrombopag may not produce as large of a benefit in real-world settings compared to controlled trial settings but may offer patients deeper responses with similar rates of toxicity to immunosuppressive therapy alone.

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Data availability

The data that support the findings of this study are available from the corresponding author upon reasonable request.

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Acknowledgements

We thank Taylor Coe-Eisenberg, PharmD for her contribution to statistical analysis and review of the manuscript. We also thank Hilary Bond, JD, MSc for her coordination of data use agreements across all participating sites.

Funding

No funds, grants, or other support was received.

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Authors and Affiliations

  1. Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA

    Lauren T. Shinn, Lydia L. Benitez, Anthony J. Perissinotti, Dale Bixby, Patrick Burke, Kristen Pettit & Bernard L. Marini

  2. Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, NC, USA

    Justin H. Reid, Kaitlyn M. Buhlinger & Hendrik van Deventer

  3. Department of Pharmacy, University of Utah Huntsman Cancer Institute, Salt Lake City, UT, USA

    Dylan Barth & Charlotte B. Wagner

  4. Department of Pharmacy, Virginia Commonwealth University Medical Center, Richmond, VA, USA

    Kyle Zacholski & Ruchi Desai

  5. Department of Pharmacy, University of Kentucky HealthCare, Lexington, KY, USA

    Ashley Soule

  6. Department of Pharmacy, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Sarah E. Stump & Taylor M. Weis

Authors
  1. Lauren T. Shinn
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  2. Lydia L. Benitez
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  11. Ashley Soule
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  13. Taylor M. Weis
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  14. Dale Bixby
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  15. Patrick Burke
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  17. Bernard L. Marini
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Corresponding author

Correspondence to Bernard L. Marini.

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Conflicts of interest

Dylan Barth accepted a position with AbbVie, Inc. and Taylor Weis with Blueprint Medicines after data collection and analysis. All other authors report no relevant competing interests.

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Shinn, L.T., Benitez, L.L., Perissinotti, A.J. et al. Multicenter evaluation of the addition of eltrombopag to immunosuppressive therapy for adults with severe aplastic anemia. Int J Hematol 118, 682–689 (2023). https://doi.org/10.1007/s12185-023-03670-3

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  • DOI: https://doi.org/10.1007/s12185-023-03670-3

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