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Nationwide survey in France on the use of romiplostim in patients with refractory severe aplastic anemia

Bone Marrow Transplantation volume 54, pages 1161–1163 (2019)Cite this article

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Immunosuppressive therapy with horse anti-thymoglobulin (ATG) and cyclosporine (CsA) is the standard of care for patients with severe aplastic anemia (SAA) who are ineligible for hematopoietic stem cell transplantation (HSCT) [1]. While hematological responses correlate strongly with long-term survival [2], approximately 30% of the patients will fail to respond, and therapeutic options for those with refractory/relapsed SAA are limited [3].

Thrombopoietin (TPO) is the main regulator of platelet production, through interaction with its receptor c-mpl on hematopoietic stem cells, megakaryocytes, and platelets [4]. While recent reports have shown that eltrombopag (ELT) can induce tri-lineage responses in refractory AA patients [5,6,7], little data have been published about romiplostim (ROM) [8], a second TPO receptor agonist (TPO-RA), whose real-life effects remain largely unknown. We therefore conducted a retrospective study in 8 centers in France, on behalf of the French Reference Center for Aplastic Anemia (FRCAA), to evaluate the risks and benefits of ROM in patients with SAA.

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Acknowledgements

Author contributions

FSdF, L-PZ, GS, and RPdL analyzed the data and helped write the manuscript. FSdF, JA, AC, LT, CC, EL, AC, GS, and RPdL took care of the patients and provided clinical information. L-PZ and RPL wrote the manuscript. RPL coordinated the research. FSdF, RPL, and L-PZ helped design the study and revised the manuscript for intellectual content.

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Authors and Affiliations

  1. Centre de Référence Aplasie Médullaire, Service d’Hématologie Greffe, Assistance Publique des Hôpitaux de Paris, Hôpital Saint-Louis, Paris, France

    Lin-Pierre Zhao, Flore Sicre De Fontbrune, Etienne Lengline, Gérard Socié & Régis Peffault de Latour

  2. Service d’Hématologie, Assistance Publique des Hôpitaux de Paris, Hôpital Cochin, Paris, France

    Adrien Contejean

  3. Service d’Hématologie et de Thérapie Cellulaire, CHU de Limoges, Limoges, France

    Julie Abraham

  4. Service des Maladies du Sang, Hôpital Claude Huriez, Lille, France

    Louis Terriou

  5. Service d’Hématologie, CHU Clermont-Ferrand, Clermont-Ferrand, France

    Cécile Chabrot

  6. Service d’Hématologie, Institut Paoli-Calmettes, Marseille, France

    Amandine Charbonnier

  7. Université Paris Diderot, Paris, France

    Gérard Socié & Régis Peffault de Latour

  8. INSERM, U1160, Paris, France

    Gérard Socié

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  1. Lin-Pierre Zhao
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  2. Flore Sicre De Fontbrune
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  8. Etienne Lengline
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  9. Gérard Socié
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Correspondence to Régis Peffault de Latour.

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Zhao, LP., Sicre De Fontbrune, F., Contejean, A. et al. Nationwide survey in France on the use of romiplostim in patients with refractory severe aplastic anemia. Bone Marrow Transplant 54, 1161–1163 (2019). https://doi.org/10.1038/s41409-019-0452-1

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