Efficacy and Safety of Avatrombopag in Aplastic Anemia - A Comprehensive Review of Clinical Evidence

Sep 28, 2026

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Aplastic anemia is a bone marrow failure disorder that causes cytopenias affecting oxygen delivery, immune defense, and hemostasis. Standard care combines immunosuppression with or without hematopoietic stem cell transplantation, and thrombopoietin receptor agonists support residual hematopoiesis. This review evaluates ten 2023–October 2025 clinical studies of avatrombopag in acquired aplastic anemia, showing overall response rates of 55%–85%, complete responses up to one-third, and onset within 1–2 months. Avatrombopag reduces transfusion needs, improves hematologic outcomes in severe and non-severe disease, including patients previously intolerant to eltrombopag, with a favorable safety profile.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages9

Reading Time23 min

AccessFree to download

Abstract

Key clinical outcomes

Safety profile

Plain language summary

Benefits in liver-compromised settings

Evidence across patient groups

Efficacy and safety of avatrombopagin aplastic anemia: a comprehensive review of clinical evidence Mostafa F. Mohammed Saleh, Abdulrahman Nasiri, Ahmed KotbAbdrabou, Alfadil Haroon, Hadeel Samarkandi, Hazza Alzahrani, Mahmoud Aljurf and Ali Alahmari Abstract: Aplastic anemia is a bone marrow failure disorder marked by cytopenias that impair oxygen delivery, immune defense, and hemostasis. Standard therapy traditionally combines immunosuppression with or without hematopoietic stem cell transplantation, and more recently incorporates thrombopoietin receptor agonists to stimulate residual hematopoiesis. Eltrombopag improved outcomes when added to immunosuppressive therapy, but its association with hepatotoxicity limits its suitability for patients with underlying liver disease or elevated baseline liver enzymes. Avatrombopag is a newer oral thrombopoietin receptor agonist that does not require dietary restrictions and does not undergo significant hepatic metabolism, which offers a potential therapeutic advantage in settings where liver function is compromised. This review evaluated ten clinical studies published from 2023 to October 2025 that investigated avatrombopag in acquired aplastic anemia across varied patient populations, including treatment-naive, relapsed or refractory cases, older adults, and patients with secondary aplastic anemia related to chemoradiation. Across these studies, overall response rates ranged from 55% to 85%, and complete response rates reached up to one-third of treated patients. Response onset typically occurred within 1–2 months, which aligns with clinical decision timelines for assessing therapeutic benefit. Avatrombopag supported reductions in transfusion requirements and sustained hematologic improvement in both severe and non-severe disease. Patients previously intolerant or non-responsive to eltrombopag also demonstrated clinical improvement, which suggests pharmacologic differences translate into meaningful therapeutic effects. Importantly, avatrombopag demonstrated a favorable safety profile in all reviewed settings. Reports did not identify clinically relevant hepatotoxicity, clonal evolution, or treatmentlimiting adverse effects. Its tolerability in patients with liver dysfunction distinguishes it from earlier agents in this drug class. Ongoing trials will clarify optimal dosing strategies and define its future role within first-line therapy and salvage treatment pathways for aplastic anemia. Plain language summary A new treatment option for aplastic anemia: how avatrombopag may help patients with fewer side effects Aplastic anemia is a rare but serious disease where the bone marrow stops making enough blood cells. People with this condition often feel tired, bruise easily, or get frequent infections. The standard treatment for those who can’t get a bone marrow transplant is a combination of medications that suppress the immune system. Another TherAdv Hematol 2026, Vol. 17: 1–9 DOI: 10. 1177/ 20406207251409305 © The Author(s), 2026. Article reuse guidelines: [sagepub.com/journals](sagepub.com/journals)permissions Correspondence to: Abdulrahman Nasiri Department of Internal Medicine, College of Medicine, Imam Mohammad Ibn Saud Islamic University (IMSIU), PO Box 5701, Riyadh 11432, Saudi Arabia [AMNasiri@imamu.edu.sa](AMNasiri@imamu.edu.sa)[ ](AMNasiri@imamu.edu.sa)Mostafa F. Mohammed Saleh Department of Hematology, Stem Cell Transplant & Cellular Therapy, Cancer Centre of Excellence, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia Clinical Hematology Unit, Department of Internal Medicine, Faculty of Medicine, Assiut University, Assiut, Egypt Ahmed Kotb Abdrabou Department of Hematology, Stem Cell Transplant & Cellular Therapy, Cancer Centre of Excellence, King Faisal Specialist Hospital and Research Center, Riyadh, Saudi Arabia Clinical Hematology Unit, Internal Medicine Department, Faculty of Medicine, Zagazig University, Zag

FAQs

How does avatrombopag differ from earlier thrombopoietin receptor agonists in aplastic anemia?

Avatrombopag is an oral thrombopoietin receptor agonist designed to stimulate residual hematopoiesis. Unlike eltrombopag, it does not require dietary restrictions and shows no clinically relevant hepatotoxicity in the reviewed studies.

What treatment benefits were observed across the included clinical studies?

Overall response rates ranged from 55% to 85%, with complete responses up to one-third of treated patients. Response typically began within 1–2 months, and patients often saw reduced transfusion requirements and sustained hematologic improvement.

What safety concerns were reported for avatrombopag?

Across the reviewed settings, reports did not identify clinically relevant hepatotoxicity, clonal evolution, or treatment-limiting adverse effects. Tolerability in patients with liver dysfunction was highlighted as a distinguishing advantage.

Sep 28, 2026

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