Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia

Few therapeutic options are available for patients with aplastic anemia who are ineligible for transplantation or refractory to immunosuppressive therapy. Eltrombopag was recently shown to produce trilineage responses in refractory patients. However, the effects of real-life use of this drug remain...

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Autores principales: Lengline, Etienne, Drenou, Bernard, Peterlin, Pierre, Tournilhac, Olivier, Abraham, Julie, Berceanu, Ana, Dupriez, Brigitte, Guillerm, Gaelle, Raffoux, Emmanuel, de Fontbrune, Flore Sicre, Ades, Lionel, Balsat, Marie, Chaoui, Driss, Coppo, Paul, Corm, Selim, Leblanc, Thierry, Maillard, Natacha, Terriou, Louis, Socié, Gerard, de Latour, Regis Peffault
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Ferrata Storti Foundation 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5792265/
https://www.ncbi.nlm.nih.gov/pubmed/29170252
http://dx.doi.org/10.3324/haematol.2017.176339
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author Lengline, Etienne
Drenou, Bernard
Peterlin, Pierre
Tournilhac, Olivier
Abraham, Julie
Berceanu, Ana
Dupriez, Brigitte
Guillerm, Gaelle
Raffoux, Emmanuel
de Fontbrune, Flore Sicre
Ades, Lionel
Balsat, Marie
Chaoui, Driss
Coppo, Paul
Corm, Selim
Leblanc, Thierry
Maillard, Natacha
Terriou, Louis
Socié, Gerard
de Latour, Regis Peffault
author_facet Lengline, Etienne
Drenou, Bernard
Peterlin, Pierre
Tournilhac, Olivier
Abraham, Julie
Berceanu, Ana
Dupriez, Brigitte
Guillerm, Gaelle
Raffoux, Emmanuel
de Fontbrune, Flore Sicre
Ades, Lionel
Balsat, Marie
Chaoui, Driss
Coppo, Paul
Corm, Selim
Leblanc, Thierry
Maillard, Natacha
Terriou, Louis
Socié, Gerard
de Latour, Regis Peffault
author_sort Lengline, Etienne
collection PubMed
description Few therapeutic options are available for patients with aplastic anemia who are ineligible for transplantation or refractory to immunosuppressive therapy. Eltrombopag was recently shown to produce trilineage responses in refractory patients. However, the effects of real-life use of this drug remain unknown. This retrospective study (2012–2016) was conducted by the French Reference Center for Aplastic Anemia on patients with relapsed/refractory aplastic anemia, and patients ineligible for antithymocyte globulin or transplantation, who received eltrombopag for at least 2 months. Forty-six patients with aplastic anemia were given eltrombopag without prior antithymocyte globulin treatment (n=11) or after antithymocyte globulin administration (n=35) in a relapsed/refractory setting. Eltrombopag (median daily dose 150 mg) was introduced 17 months (range, 8–50) after the diagnosis of aplastic anemia. At last followup, 49% were still receiving treatment, 9% had stopped due to a robust response, 2% due to toxicity and 40% due to eltrombopag failure. Before eltrombopag treatment, all patients received regular transfusions. The overall rates of red blood cell and platelet transfusion independence were 7%, 33%, 46% and 46% at 1, 3, 6 months and last follow-up. Responses were slower to develop in antithymocyte treatment-naïve patients. In patients achieving transfusion independence, hemoglobin concentration and platelet counts improved by 3 g/dL (interquartile range, 1.4–4.5) and 42×10(9)/L (interquartile range, 11–100), respectively. Response in at least one lineage (according to National Institutes of Health criteria) was observed in 64% of antithymocyte treatment-naïve and 74% of relapsed/refractory patients, while trilineage improvement was observed in 27% and 34%, respectively. We found high rates of hematologic improvement and transfusion independence in refractory aplastic anemia patients but also in patients ineligible for antithymocyte globulin receiving first-line treatment. In conclusion, elderly patients unfit for antithymocyte globulin therapy may benefit from eltrombopag.
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spelling pubmed-57922652018-02-13 Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia Lengline, Etienne Drenou, Bernard Peterlin, Pierre Tournilhac, Olivier Abraham, Julie Berceanu, Ana Dupriez, Brigitte Guillerm, Gaelle Raffoux, Emmanuel de Fontbrune, Flore Sicre Ades, Lionel Balsat, Marie Chaoui, Driss Coppo, Paul Corm, Selim Leblanc, Thierry Maillard, Natacha Terriou, Louis Socié, Gerard de Latour, Regis Peffault Haematologica Article Few therapeutic options are available for patients with aplastic anemia who are ineligible for transplantation or refractory to immunosuppressive therapy. Eltrombopag was recently shown to produce trilineage responses in refractory patients. However, the effects of real-life use of this drug remain unknown. This retrospective study (2012–2016) was conducted by the French Reference Center for Aplastic Anemia on patients with relapsed/refractory aplastic anemia, and patients ineligible for antithymocyte globulin or transplantation, who received eltrombopag for at least 2 months. Forty-six patients with aplastic anemia were given eltrombopag without prior antithymocyte globulin treatment (n=11) or after antithymocyte globulin administration (n=35) in a relapsed/refractory setting. Eltrombopag (median daily dose 150 mg) was introduced 17 months (range, 8–50) after the diagnosis of aplastic anemia. At last followup, 49% were still receiving treatment, 9% had stopped due to a robust response, 2% due to toxicity and 40% due to eltrombopag failure. Before eltrombopag treatment, all patients received regular transfusions. The overall rates of red blood cell and platelet transfusion independence were 7%, 33%, 46% and 46% at 1, 3, 6 months and last follow-up. Responses were slower to develop in antithymocyte treatment-naïve patients. In patients achieving transfusion independence, hemoglobin concentration and platelet counts improved by 3 g/dL (interquartile range, 1.4–4.5) and 42×10(9)/L (interquartile range, 11–100), respectively. Response in at least one lineage (according to National Institutes of Health criteria) was observed in 64% of antithymocyte treatment-naïve and 74% of relapsed/refractory patients, while trilineage improvement was observed in 27% and 34%, respectively. We found high rates of hematologic improvement and transfusion independence in refractory aplastic anemia patients but also in patients ineligible for antithymocyte globulin receiving first-line treatment. In conclusion, elderly patients unfit for antithymocyte globulin therapy may benefit from eltrombopag. Ferrata Storti Foundation 2018-02 /pmc/articles/PMC5792265/ /pubmed/29170252 http://dx.doi.org/10.3324/haematol.2017.176339 Text en Copyright© 2018 Ferrata Storti Foundation Material published in Haematologica is covered by copyright. All rights are reserved to the Ferrata Storti Foundation. Use of published material is allowed under the following terms and conditions: https://creativecommons.org/licenses/by-nc/4.0/legalcode. Copies of published material are allowed for personal or internal use. Sharing published material for non-commercial purposes is subject to the following conditions: https://creativecommons.org/licenses/by-nc/4.0/legalcode, sect. 3. Reproducing and sharing published material for commercial purposes is not allowed without permission in writing from the publisher.
spellingShingle Article
Lengline, Etienne
Drenou, Bernard
Peterlin, Pierre
Tournilhac, Olivier
Abraham, Julie
Berceanu, Ana
Dupriez, Brigitte
Guillerm, Gaelle
Raffoux, Emmanuel
de Fontbrune, Flore Sicre
Ades, Lionel
Balsat, Marie
Chaoui, Driss
Coppo, Paul
Corm, Selim
Leblanc, Thierry
Maillard, Natacha
Terriou, Louis
Socié, Gerard
de Latour, Regis Peffault
Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
title Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
title_full Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
title_fullStr Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
title_full_unstemmed Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
title_short Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia
title_sort nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the french reference center for aplastic anemia
topic Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5792265/
https://www.ncbi.nlm.nih.gov/pubmed/29170252
http://dx.doi.org/10.3324/haematol.2017.176339
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