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Eltrombopag for the treatment of poor graft function following allogeneic stem cell transplant: a retrospective multicenter study

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Abstract

This retrospective study assessed the effectiveness of eltrombopag (EPAG), a thrombopoietin receptor agonist, in the treatment of poor graft function (PGF) following an allogeneic haemopoietic stem cell transplantation (HSCT). Complete response was defined as normalization of blood counts, whereas partial response was defined as transfusion independence. A total of 48 patients with full donor chimerism after HSCT, received EPAG for a median of 120 days (range 10–591). Patients with uni- bi- or tri-lineage cytopenia started treatment at a median of 95 days (range 17–877) after HSCT. The overall response rate was 75%: 24 patients had a complete response and 12 had a partial response. Positive predictors of response were an HLA-matched donor, a CD34+ dose at transplant > 4 × 106/kg, and starting EPAG treatment at least 90 days after HSCT. Patients with more than one positive predictor had a response rate of 92% for the overall patient cohort and 94% for patients with tri-lineage cytopenia. One-year survival was 89% for complete responders, 60% for partial responders and 20% for non-responders (p = 0.0004). EPAG improves peripheral blood counts in patients with poor graft function following HSCT. Response to EPAG can be predicted and has a significant impact on survival.

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Data are available from the corresponding author on reasonable request.

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Authors and Affiliations

  1. Dipartimento di Diagnostica per Immagini, Radioterapia Oncologica ed Ematologia, Fondazione Policlinico Universitario A. Gemelli IRCCS, Rome, Italy

    Sabrina Giammarco, Simona Sica, Patrizia Chiusolo, Luca Laurenti, Federica Sorá, Elisabetta Metafuni & Andrea Bacigalupo

  2. Sezione di Ematologia, Dipartimento di Scienze Radiologiche ed Ematologiche, Università Cattolica del Sacro Cuore, Rome, Italy

    Simona Sica, Patrizia Chiusolo, Luca Laurenti, Federica Sorá & Andrea Bacigalupo

  3. Azienda Ospedaliera BMM, Centro Trapianti di Midollo Osseo, Reggio Calabria, Italy

    Massimo Martino

  4. A.O.U. Cittá della Salute e della Scienza di Torino, Presidio MolinetteHaematology, Turin, Italy

    Alessandro Busca

  5. Bone Marrow Transplant Unit, Department of Clinical Medicine and Surgery, Federico II University of Naples, Naples, Italy

    Antonio Risitano

  6. Hematology and BMT Unit AORN S. G. Moscati, Avellino, Italy

    Antonio Risitano

  7. Hospital Universitario de Donostia, San Sebastian, Spain

    Carlos Vallejo

Authors
  1. Sabrina Giammarco
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  2. Simona Sica
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  3. Patrizia Chiusolo
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  4. Luca Laurenti
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  5. Federica Sorá
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  6. Massimo Martino
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  7. Elisabetta Metafuni
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  8. Alessandro Busca
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  9. Antonio Risitano
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  10. Carlos Vallejo
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  11. Andrea Bacigalupo
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Contributions

SG and AB designed the study and read, revised, and approved drafts of the manuscript. SS, PC, LL, FS, MM, AB, AR and CV provided data on patients and reviewed the manuscript. All authors read and approved the final manuscript.

Corresponding author

Correspondence to Sabrina Giammarco.

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Conflict of interest

The authors state that they have no conflicts of interest to declare.

Ethics approval

This research study was conducted retrospectively from data obtained for clinical purposes. This retrospective study was approved by the Ethics Committee of Fondazione Policlinico A. Gemelli IRCCS (prot.0030921/20; 23/07/2020).

Informed consent

Every patient gave written informed consent for the use of EPAG as an off-label drug.

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Giammarco, S., Sica, S., Chiusolo, P. et al. Eltrombopag for the treatment of poor graft function following allogeneic stem cell transplant: a retrospective multicenter study. Int J Hematol 114, 228–234 (2021). https://doi.org/10.1007/s12185-021-03153-3

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  • DOI: https://doi.org/10.1007/s12185-021-03153-3

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