Calcineurin inhibitor and eltrombopag combination for acquired aplastic anemia: results from a large national database

Ther Adv Hematol. 2025 Nov 30;16:20406207251394775. doi: 10.1177/20406207251394775. eCollection 2025.

ABSTRACT

BACKGROUND: Acquired aplastic anemia (AA) is rare bone marrow failure syndrome characterized by pancytopenia due to immune-mediated destruction of hematopoietic stem and progenitor cells, and leads to an increased risk of bleeding and infectious complications. The first-line treatment option for individuals with severe AA who are ineligible for allogeneic hematopoietic stem cell transplantation is triple therapy, comprised equine anti-thymocyte globulin, calcineurin inhibitor (CNI), and eltrombopag (EPAG). However, this approach is associated with considerable treatment-related complications, requires close inpatient monitoring as well as specialized care and expertise, limiting its feasibility in less-experienced centers. Emerging evidence suggests that double therapy with CNI and EPAG combination reduces the transfusion burden in severe AA.

OBJECTIVE: Evaluate the real-world effectiveness of CNI and EPAG combination therapy in reducing transfusion burden among adults with AA.

METHODS: We conducted a retrospective cohort study using Blue Cross Blue Shield Axis Database to examine the change in transfusion requirements in adult patients with newly diagnosed AA who have received combination therapy with CNI and EPAG. Individuals were stratified into baseline transfusion-independent, low, and high transfusion subgroups. Transfusion requirements were reassessed after 6 months from initiation of double therapy.

RESULTS: The majority of the 153 identified patients with AA started therapy shortly after diagnosis and were adherent to treatment and monitoring during the initial 6 months. Among patients with baseline transfusion requirements (97; 63.4%), 66% achieved TI status. Additionally, 75.5% of patients with a high baseline transfusion burden demonstrated a ⩾50% reduction in transfusion requirements.

CONCLUSION: These findings underscore the efficacy and feasibility of double therapy with CNI and EPAG for adults with AA in the real-world setting, offering an alternative for patients when triple therapy is not possible.

PMID:41333634 | PMC:PMC12665816 | DOI:10.1177/20406207251394775