The DOI matches its Crossref record; 28 of the 28 references it cites were found in a registry or an index.
Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia
Open accessAbstract
BACKGROUND: A single-group, phase 1-2 study indicated that eltrombopag improved the efficacy of standard immunosuppressive therapy that entailed horse antithymocyte globulin (ATG) plus cyclosporine in patients with severe aplastic anemia. METHODS: In this prospective, investigator-led, open-label, multicenter, randomized, phase 3 trial, we compared the efficacy and safety of horse ATG plus cyclosporine with or without eltrombopag as front-line therapy in previously untreated patients with severe aplastic anemia. The primary end point was a hematologic complete response at 3 months. RESULTS: Patients were assigned to receive immunosuppressive therapy (Group A, 101 patients) or immunosuppressive therapy plus eltrombopag (Group B, 96 patients). The percentage of patients who had a complete response at 3 months was 10% in Group A and 22% in Group B (odds ratio, 3.2; 95% confidence interval [CI], 1.3 to 7.8; P = 0.01). At 6 months, the overall response rate (the percentage of patients who had a complete or partial response) was 41% in Group A and 68% in Group B. The median times to the first response were 8.8 months (Group A) and 3.0 months (Group B). The incidence of severe adverse events was similar in the two groups. With a median follow-up of 24 months, a karyotypic abnormality that was classified as myelodysplastic syndrome developed in 1 patient (Group A) and 2 patients (Group B); event-free survival was 34% and 46%, respectively. Somatic mutations were detected in 29% (Group A) and 31% (Group Β) of the patients at baseline; these percentages increased to 66% and 55%, respectively, at 6 months, without affecting the hematologic response and 2-year outcome. CONCLUSIONS: The addition of eltrombopag to standard immunosuppressive therapy improved the rate, rapidity, and strength of hematologic response among previously untreated patients with severe aplastic anemia, without additional toxic effects. (Funded by Novartis and others; RACE ClinicalTrials.gov number, NCT02099747; EudraCT number, 2014-000363-40.).
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Cites
Young, N. S. (2018) · New England Journal of Medicine · doi:10.1056/nejmra1413485 · Cited by 722
Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia
Yoshizato, T., Dumitriu, B., Hosokawa, K., Makishima, H., Yoshida, K., Townsley, D., Sato-Otsubo, A., Sato, Y., Liu, D., Suzuki, H., et al. (2015) · New England Journal of Medicine · doi:10.1056/nejmoa1414799 · Cited by 646
Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia
Townsley, D. M., Scheinberg, P., Winkler, T., Desmond, R., Dumitriu, B., Rios, O., Weinstein, B., Valdez, J., Lotter, J., Feng, X., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1613878 · Cited by 562
Eltrombopag and Improved Hematopoiesis in Refractory Aplastic Anemia
Olnes, M. J., Scheinberg, P., Calvo, K. R., Desmond, R., Tang, Y., Dumitriu, B., Parikh, A. R., Soto, S., Biancotto, A., Feng, X., et al. (2012) · New England Journal of Medicine · doi:10.1056/nejmoa1200931 · Cited by 539
Horse versus Rabbit Antithymocyte Globulin in Acquired Aplastic Anemia
Scheinberg, P., Nunez, O., Weinstein, B., Scheinberg, P., Biancotto, A., Wu, C. O., & Young, N. S. (2011) · New England Journal of Medicine · doi:10.1056/nejmoa1103975 · Cited by 504
Thrombopoietin receptor agonists: ten years later
Ghanima, W., Cooper, N., Rodeghiero, F., Godeau, B., & Bussel, J. B. (2019) · Haematologica · doi:10.3324/haematol.2018.212845 · Cited by 416
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Desmond, R., Townsley, D. M., Dumitriu, B., Olnes, M. J., Scheinberg, P., Bevans, M., Parikh, A. R., Broder, K., Calvo, K. R., Wu, C. O., et al. (2014) · Blood · doi:10.1182/blood-2013-10-534743 · Cited by 402
Rosenfeld, S., Follmann, D., Nunez, O., & Young, N. S. (2003) · JAMA · doi:10.1001/jama.289.9.1130 · Cited by 396
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Metadata
- Authors
- Peffault de Latour, R., Kulasekararaj, A., Iacobelli, S., Terwel, S. R., Cook, R., Griffin, M., Halkes, C. J. M., Recher, C., Barraco, F., Forcade, E., Vallejo, J.-C., Drexler, B., Mear, J.-B., Smith, A. E., Angelucci, E., Raymakers, R. A. P., de Groot, M. R., Daguindau, E., Nur, E., Barcellini, W., Russell, N. H., Terriou, L., Iori, A.-P., La Rocca, U., Sureda, A., Sánchez-Ortega, I., Xicoy, B., Jarque, I., Cavenagh, J., Sicre de Fontbrune, F., Marotta, S., Munir, T., Tjon, J. M. L., Tavitian, S., Praire, A., Clement, L., Rabian, F., Marano, L., Hill, A., Palmisani, E., Muus, P., Cacace, F., Frieri, C., van Lint, M.-T., Passweg, J. R., Marsh, J. C. W., Socié, G., Mufti, G. J., Dufour, C., & Risitano, A. M.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2022-01-06
- Volume
- 386
- Issue
- 1
- Pages
- 11-23
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W4206559161
- Cited by
- 304
- References
- 28
- FWCI
- 31.50
- Type
- journal article
- Language
- en
- Open access
- bronze
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- Peffault de Latour, R., Kulasekararaj, A., Iacobelli, S., Terwel, S. R., Cook, R., Griffin, M., Halkes, C. J. M., Recher, C., Barraco, F., Forcade, E., Vallejo, J.-C., Drexler, B., Mear, J.-B., Smith, A. E., Angelucci, E., Raymakers, R. A. P., de Groot, M. R., Daguindau, E., Nur, E., . . . Risitano, A. M. (2022). Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. New England Journal of Medicine. https://doi.org/10.1056/nejmoa2109965
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
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