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Eltrombopag as frontline treatment of aplastic anaemia in routine practice: implications on cost and efficacy

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Abstract

The thrombopoietin mimetic eltrombopag (EPAG) is efficacious in clinical trials of newly diagnosed moderate (M), severe (S) and very severe (vS) aplastic anaemia (AA). Its use in routine practice and resource-constrained settings is not well described. Twenty-five men and 38 women at a median age of 54 (18–86) years with newly diagnosed AA treated consecutively in a 7-year period with EPAG (N = 6), EPAG/cyclosporine (CsA) (N = 33) and EPAG/CsA/anti-thymocyte globulin (ATG) (N = 24) were analyzed. Because EPAG was not reimbursed, peak doses ranged from 25 to 200 mg/day depending on affordability. EPAG/CsA-treated patients were older (median age: 61 years) with less severe AA (MAA, N = 15; SAA, N = 14; vSAA, N = 4), whereas EPAG/CsA/ATG-treated patients were younger (median age: 44 years) with more severe AA (MAA, N = 2; SAA, N = 12, vSAA, N = 10). The overall/trilineage response rates were 83%/50% for EPAG-treated patients; 79%/42% for EPAG/CsA-treated patients and 75%/63% for EPAG/CsA/ATG-treated patients. Adverse events included grade 1 liver derangement (N = 7) and grade 1 dyspepsia (N = 3). The 5-year overall survivals/failure-free survivals were 62%/80% for the entire cohort; 55%/75% for EPAG/CsA-treated patients and 82%/78% for EPAG/CsA/ATG-treated patients. EPAG showed robust efficacy in AA in routine practice. However, EPAG dosage and combinations remain to be optimized for AA of different severities.

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Authors and Affiliations

  1. Department of Medicine, Queen Mary Hospital, Pokfulam Road, Hong Kong, China

    Yu-Yan Hwang, Thomas S. Y. Chan, Joycelyn P. Y. Sim & Yok-Lam Kwong

  2. Department of Medicine, Pamela Youde Eastern Hospital, Hong Kong, China

    Florence H. Y. Chan & Bonnie Kho

  3. Department of Medicine, Queen Elizabeth Hospital, Hong Kong, China

    Chris W. P. Lau & June S. M. Lau

  4. Department of Medicine, Tseung Kwan O Hospital, Hong Kong, China

    Yan-Yan Luk & Chi-Kuen Lau

  5. Department of Medicine, Princess Margaret Hospital, Hong Kong, China

    Grace W. N. Lau & Vivien Mak

  6. Department of Medicine, Tuen Mun Hospital, Hong Kong, China

    Ka-Po Chan & Sze-Fai Yip

  7. Department of Medicine, United Christian Hospital, Hong Kong, China

    Kwan-Hung Leung & Shek-Ying Lin

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  1. Yu-Yan Hwang
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  15. Joycelyn P. Y. Sim
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Contributions

Y.Y. Hwang: treated the patients, collected the data, wrote and approved the manuscript

T.S.Y. Chan: treated the patients, wrote and approved the manuscript

F.H.Y. Chan: treated the patients, collected the data and approved the manuscript

C.W.P. Lau: treated the patients, collected the data and approved the manuscript

Y.Y. Luk: treated the patients, collected the data and approved the manuscript

G.W.N. Lau: treated the patients, collected the data and approved the manuscript

K.P. Chan: treated the patients, collected the data and approved the manuscript

K.H. Leung: treated the patients, collected the data and approved the manuscript

Bonnie Kho: treated the patients and approved the manuscript

J.S.M. Lau: treated the patients and approved the manuscript

C.K. Lau: treated the patients and approved the manuscript

V. Mak: treated the patients and approved the manuscript

S.F. Yip: treated the patients and approved the manuscript

S.Y. Lin: treated the patients and approved the manuscript

J.P.Y. Sim: treated the patients and approved the manuscript

Y.L. Kwong: conceived the study, treated the patients, wrote and approved the manuscript

Corresponding author

Correspondence to Yok-Lam Kwong.

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The patient gave informed consent to treatment.

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The authors declare no competing interests.

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Hwang, YY., Chan, T.S.Y., Chan, F.H.Y. et al. Eltrombopag as frontline treatment of aplastic anaemia in routine practice: implications on cost and efficacy. Ann Hematol 101, 1163–1172 (2022). https://doi.org/10.1007/s00277-022-04780-4

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  • DOI: https://doi.org/10.1007/s00277-022-04780-4

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