hATG+CsA vs hATG+CsA+Eltrombopag for SAA

Summary

The null hypothesis of no difference in CR% at 3 months between the arms will be tested against the alternative of a difference in CR% at an alpha level of .05 by assessing the odds ratio for arm yielded by this model.

Timeline

Start

2015-07

Primary completion

2020-12

Completion

2020-12

Outcome

Met primary endpoint

paper complete response at 3 months was 10% in Group A and 22% in Group B PMID 34986284 ↗

paper (odds ratio, 3.2; 95% confidence interval [CI], 1.3 to 7.8; P = 0.01). PMID 34986284 ↗

Publications

• Kaya DE, Cook R, Iacobelli S, Napolitani G, Gerlevik S, Seymen N, Sicre de Fontbrune F, Griffin M, Frieri C, Halkes CJM, Recher C, Barraco F, Forcade E, Mear JB, Laurino M, Drexler B, Daguindau E, van Os M, Terwel S, Dufour C, Karimi MM, Kulasekararaj AG, Peffault De Latour R, Risitano AM, Mufti G. Clonal Dynamics of Hematopoiesis in Aplastic Anemia after Immunosuppression and Eltrombopag. NEJM Evid. 2026 May;5(5):EVIDoa2500174. doi: 10.1056/EVIDoa2500174. Epub 2026 Apr 28.
• de Latour RP, Kulasekararaj A, Iacobelli S, Griffin M, Halkes CJ, Dufour C, Risitano AM. Plain language summary of RACE study results: addition of eltrombopag to standard treatment of severe aplastic anemia. Immunotherapy. 2024 Feb;16(3):135-142. doi: 10.2217/imt-2023-0200. Epub 2023 Dec 13.
• Peffault de Latour R, Kulasekararaj A, Iacobelli S, Terwel SR, Cook R, Griffin M, Halkes CJM, Recher C, Barraco F, Forcade E, Vallejo JC, Drexler B, Mear JB, Smith AE, Angelucci E, Raymakers RAP, de Groot MR, Daguindau E, Nur E, Barcellini W, Russell NH, Terriou L, Iori AP, La Rocca U, Sureda A, Sanchez-Ortega I, Xicoy B, Jarque I, Cavenagh J, Sicre de Fontbrune F, Marotta S, Munir T, Tjon JML, Tavitian S, Praire A, Clement L, Rabian F, Marano L, Hill A, Palmisani E, Muus P, Cacace F, Frieri C, van Lint MT, Passweg JR, Marsh JCW, Socie G, Mufti GJ, Dufour C, Risitano AM; Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation. Eltrombopag Added to Immunosuppression in Severe Aplastic Anemia. N Engl J Med. 2022 Jan 6;386(1):11-23. doi: 10.1056/NEJMoa2109965.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Eltrombopag	Small molecule	—	Oral
Background	ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE)	Protein / enzyme biologic	—	—
Background	cyclosporin A	Unknown	—	—

Indications