Clonal Dynamics of Hematopoiesis in Aplastic Anemia after Immunosuppression and Eltrombopag.

doi:10.1056/EVIDoa2500174

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Article

Clonal Dynamics of Hematopoiesis in Aplastic Anemia after Immunosuppression and Eltrombopag.

NEJM evidence - 1 May 2026

Kaya Deniz Ece, Cook Riley, Iacobelli Simona, Napolitani Giorgio, Gerlevik Sila, Seymen Nogayhan, Sicre de Fontbrune Flore, Griffin Morag, Frieri Camilla, Halkes Constantijn J M, Recher Christian, Barraco Fiorenza, Forcade Edouard, Méar Jean-Baptiste, Laurino Marica, Drexler Beatrice, Daguindau Etienne, van Os Marleen, Terwel Sofie, Dufour Carlo, Karimi Mohammad M, Kulasekararaj Austin G, Peffault De Latour Regis, Risitano Antonio M, Mufti Ghulam

Abstract excerpt

BACKGROUND: A Phase 3 randomized trial compared immunosuppressive therapy with or without eltrombopag in untreated patients with aplastic anemia (AA) and showed that addition of eltrombopag increased the rate, rapidity, and durability of hematological response, without increasing transformation to myeloid malignancies. We sought to systematically investigate clonal hematopoiesis (CH) dynamics from patient samples...

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PMID: 42047558
DOI: 10.1056/EVIDoa2500174

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