Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia
Abstract
What is this summary about?
Severe aplastic anemia (SAA) and very severe aplastic anemia (vSAA) are blood diseases of the bone marrow. If a suitable donor for bone marrow transplant as initial treatment is unavailable, standard immunosuppression is used. Standard immunosuppression treatment includes horse antithymocyte globulin (hATG) and cyclosporin A (CsA). This summary investigated the results of standard immunosuppression treatment (Group A) versus standard immunosuppression treatment with a medication called eltrombopag (Group B) in participants with SAA and vSAA. Eltrombopag is a medicine that improves the blood platelet level and is taken by mouth (orally).
What were the results of the study?
Compared to Group A, more participants in Group B showed increased blood cell level to a normal range without SAA or vSAA and faster treatment response. Side effects were similar in both groups even with the addition of eltrombopag for Group B. Participants in both groups reported feeling well after 6, 12 and 24 months. Differences in the participant-reported scores (overall health, physical, emotional, and social) between Group A and Group B were minimal.
What do the results of the study mean?
Immunosuppression treatment (hATG plus CsA) with eltrombopag benefited participants with SAA and vSAA and could be the new standard for SAA in persons who cannot undergo bone marrow transplant. At this time, eltrombopag is only approved in specific countries to treat the condition under study that is discussed in this summary.
Clinical Trial Registration:NCT02099747 (RACE study)
Patients with severe and very severe aplastic anemia benefited from and had faster response when treated with horse antithymocyte globulin and cyclosporin A plus eltrombopag. This could be the new standard when bone marrow transplant cannot be the initial treatment.
This is an abstract of the Plain Language Summary of Publication article.
To read the full Plain Language Summary of this article, click here to view the PDF.
Link to original article here
Acknowledgments
The authors thank all the participants in this trial, the staff members at the trial locations, the members of the data and safety monitoring board, and all the investigators at the clinical sites. The authors on this Plain Language Summary of Publication would also like to thank the authors of the primary manuscript: Sofie R Terwel, Riley Cook, Christian Recher, Fiorenza Barraco, Edouard Forcade, Juan Carlos Vallejo, Beatrice Drexler, Jean-Baptiste Mear, Alexander E Smith, Emanuele Angelucci, Reinier AP Raymakers, Marco R De Groot, Etienne Daguindau, Nur Erfan, Wilma Barcellini, Nigel H Russell, Louis Terriou, Anna Paola Iori, Ursula La Rocca, Anna Sureda, Isabel Sánchez-Ortega, Blanca Xicoy, Isidro Jarque, James Cavenagh, Flore Sicre de Fontbrune, Serena Marotta, Talha Munir, Jennifer ML Tjon, Suzanne Tavitian, Aline Praire, Laurence Clement, Florence Rabian, Luana Marano, Anita Hill, Elena Palmisani, Petra Muus, Fabiana Cacace, Camilla Frieri, Maria Teresa van Lint, Jakob R Passweg, Judith CW Marsh, Gérard Socié and Ghulam J Mufti.
Financial disclosure
The RACE study was sponsored by the European Society for Blood and Marrow Transplantation (EBMT). The study was funded by Novartis, Pfizer, a grant from Alexion Pharma, a grant (A22324) from Cancer Research UK and grants (10024 and 14017) from Bloodwise UK (previously called Leukaemia and Lymphoma Research). Pfizer funded the development of this plain language summary. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.
Competing interests disclosure
R Peffault de Latour has received honoraria and been a consultant for Alexion Pharmaceuticals, Amgen Inc., Apellis Pharmaceuticals, Novartis, Pfizer, and Sobi Inc.; and received research funding and grants from Alexion Pharmaceuticals, Amgen Inc., Novartis and Pfizer. A Kulasekararaj has been on the advisory board and a consultant for Alexion Pharmaceuticals, Amgen Inc., BioCryst Pharmaceuticals, Celgene Corporation, F Hoffman-La Roche and Novartis; and received funding for travel from Alexion Pharmaceuticals and Novartis. M Griffin is on the advisory board of Alexion, Amgen, BioCryst Pharmaceuticals and Novartis; consultant for BioCryst Pharmaceuticals and Regeneron; and received lecture fees/honoraria from Alexion, Novartis and Sobi Inc. C Dufour has been a consultant for BioCryst Pharmaceuticals, Inc. and Novartis. AM Risitano received grants from Achillion, Alexion Pharmaceuticals, Alnylam Pharmaceuticals, BioCryst Pharmaceuticals, Novartis, Pfizer, RA Pharmaceuticals, Roche and Samsung; and is on the advisory board and received lecture fees from Apellis. The authors have no other competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript apart from those disclosed.
Writing disclosure
Medical writing support was provided by Priyanka Nair at Pfizer. Editorial support was provided by Melissa Furtado and Kanchan Bhati at Pfizer.
Open access
This work is licensed under the Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 Unported License. To view a copy of this license, visit http://creativecommons.org/licenses/by-nc-nd/4.0/
