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

Autor: Shinn LT; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA., Benitez LL; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA., Perissinotti AJ; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA., Reid JH; Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, NC, USA., Buhlinger KM; Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, NC, USA., van Deventer H; Department of Pharmacy, University of North Carolina Medical Center, Chapel Hill, NC, USA., Barth D; Department of Pharmacy, University of Utah Huntsman Cancer Institute, Salt Lake City, UT, USA., Wagner CB; Department of Pharmacy, University of Utah Huntsman Cancer Institute, Salt Lake City, UT, USA., Zacholski K; Department of Pharmacy, Virginia Commonwealth University Medical Center, Richmond, VA, USA., Desai R; Department of Pharmacy, Virginia Commonwealth University Medical Center, Richmond, VA, USA., Soule A; Department of Pharmacy, University of Kentucky HealthCare, Lexington, KY, USA., Stump SE; Department of Pharmacy, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Weis TM; Department of Pharmacy, Memorial Sloan Kettering Cancer Center, New York, NY, USA., Bixby D; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA., Burke P; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA., Pettit K; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA., Marini BL; Department of Clinical Pharmacy, Michigan Medicine and University of Michigan College of Pharmacy, 1540 E. Hospital Dr., Room 251-B, Ann Arbor, MI, USA. bernmari@med.umich.edu.
Jazyk: angličtina
Zdroj: International journal of hematology [Int J Hematol] 2023 Dec; Vol. 118 (6), pp. 682-689. Date of Electronic Publication: 2023 Oct 26.
DOI: 10.1007/s12185-023-03670-3
Abstrakt: 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.
(© 2023. Japanese Society of Hematology.)
Databáze: MEDLINE
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