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Efficacy of eltrombopag for treatment of thrombocytopenia in the setting of allogeneic hematopoietic cell transplantation: a systematic review and meta-analysis

Bone Marrow Transplantation volume 61, pages 211–218 (2026)Cite this article

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Abstract

Thrombocytopenia following an allogeneic hematopoietic cell transplant (allo-HCT) is a potentially serious complication, and the efficacy of eltrombopag, a thrombopoietin receptor agonist, in this context is unclear due to inconsistent findings. Additionally, other post-allograft outcomes in eltrombopag treated patients such as transfusion independence, overall survival (OS), and non-relapse mortality have not been systematically reviewed. The aim of this systematic review/meta-analysis (SR/MA) is to evaluate the efficacy of eltrombopag in allo-HCT-induced thrombocytopenia by analyzing data from 16 eligible studies. Pooled rate of response for platelets counts achieving >30 × 109/L and >50 × 109/L were 72% and 56%, respectively. When evaluating the composite endpoint of platelets response and transfusion independence, pooled rates for >30 × 109/L and >50 × 109/L plus transfusion independence were 47% and 56%, respectively. Pooled OS, bleeding-related mortality and mortality from GVHD/infection were 68%, 6%, and 19%, respectively. These findings show that eltrombopag is an effective treatment of allo-HCT-induced thrombocytopenia. Optimal dose and duration of treatment remain to be determined in a large prospective study. Results of this SR/MA suggest a beneficial effect of eltrombopag for thrombocytopenia after allo-HCT. These results could represent the benchmark to be used for future prospective and comparative studies to better understand the benefit of this intervention.

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Data availability

This work represents the results of a systematic review and meta-analysis. Data were extracted from published information in the public domain. The methodology section explains the criteria used to select studies for inclusion in the meta-analysis. All data generated or analyzed during this study are included in this published article.

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Author information

Authors and Affiliations

  1. Judy Genshaft Honors College, University of South Florida, Tampa, FL, USA

    Arni Kumar

  2. College of Arts and Sciences, University of South Florida, Tampa, FL, USA

    Arin Singh

  3. Morsani College of Medicine, University of South Florida, Tampa, FL, USA

    Tea Reljic

  4. Division of Hematology-Oncology and Blood and Marrow Transplantation and Cellular Therapy Programs, Mayo Clinic, Jacksonville, FL, USA

    Madiha Iqbal, Hemant Murthy, Ricardo Parrondo, Ernesto Ayala, Vivek Roy & Mohamed A. Kharfan-Dabaja

  5. Division of Hematology-Oncology and O’Neal Comprehensive Cancer Center, University of Alabama at Birmingham, Birmingham, AL, USA

    Razan Mohty

  6. Department of Blood and Marrow Transplantation and Cellular Immunotherapy Program, Moffitt Cancer Center, Tampa, FL, USA

    Taiga Nishihori

  7. Blood and Marrow Transplantation Program, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

    Mahmoud Aljurf

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Contributions

AK, AS, TR, MI, RM, HM, and MAK-D were responsible for designing the study protocol, screening potentially eligible studies, extracting and analyzing the data, interpreting results, and writing the manuscript. TN, RP, EA, VR, and MA were responsible for analyzing the data, interpreting the results and reviewing and provided feedback on the final manuscript.

Corresponding author

Correspondence to Mohamed A. Kharfan-Dabaja.

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Competing interests

AK, AS, TR, RM, EA, VR, MA declare no financial conflicts of interest. MI declares consultancy for US Sanofi, Bristol Myers Squibb and ADC therapeutics; HM declares consultancy or advisory board member: Bristol Myers Squibb, CRISPR Therapeutics, Incyte, Jazz Pharmaceuticals, Senti Bioscience, Autolus, Kite/Gilead, Sobi; medical monitor for Bone Marrow Transplant Clinical Trial Network; TN declares clinical trial research support to the institution from Novartis and Karyopharm; RP declares serving on the advisory board for Sanofi Aventis and Astra Zeneca, has received honoraria from Sanofi Aventis, Astra Zeneca and Beigene, and has received research funding from Bristol Myers Squibb Foundation, Beigene, Cullinan Therapeutics, and GlaxoSmithKline; MAK-D declares research/grant from Bristol Myers Squibb, Novartis, and Pharmacyclics and lecture/honoraria from Kite Pharma.

Ethics approval

All methods were performed in accordance with the relevant guidelines and regulations. The study selection process was summarized using the PRISMA flow diagram and methodology for conduct of systematic reviews followed the Cochrane Collaboration Handbook.

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Kumar, A., Singh, A., Reljic, T. et al. Efficacy of eltrombopag for treatment of thrombocytopenia in the setting of allogeneic hematopoietic cell transplantation: a systematic review and meta-analysis. Bone Marrow Transplant 61, 211–218 (2026). https://doi.org/10.1038/s41409-025-02760-6

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