The Journal of the Association of Physicians of India
September 1, 2026 · via Europe PMC
Safety and Effectiveness of Cyclosporine and Eltrombopag in Acquired Aplastic Anemia: Experience from a Tertiary Care Center in Eastern India.
Baul S, Sharma A, Ghosh K, Saha SK, Dolai TK.
AI summary — for doctors, generated from the abstract only
Cyclosporine plus eltrombopag without ATG produced a 62.5% response rate at 6 months in adults with severe aplastic anemia who could not receive ATG.
What was studied
Single-center retrospective study of 40 adults with severe or very severe acquired aplastic anemia treated with cyclosporine and eltrombopag from 2020 to 2025 in Eastern India.
Key findings
- Overall response 62.5% (17.5% complete, 45% partial) at 6 months
- Median age 56.5 years; 72.5% severe and 27.5% very severe
- Hemoglobin, neutrophil and platelet counts improved significantly
- Authors describe toxicity as acceptable
Clinical relevance
Hematologists treating older or comorbid aplastic anemia patients have a practical ATG-free option, especially where resources are limited.
Who should care
Limitations
Small, retrospective, uncontrolled series with short follow-up at 3-6 months and details of safety cut from the abstract.
Consider cyclosporine plus eltrombopag when ATG is contraindicated, with close monitoring.
This is an AI-generated summary of the published abstract, not a clinical recommendation. It may not reflect the full text or subsequent updates — verify against the original source before acting on it.
Abstract
Introduction
Acquired aplastic anemia (AAA) is an immune-mediated bone marrow failure syndrome traditionally treated with antithymocyte globulin (ATG)-based immunosuppressive therapy. However, many patients-particularly older adults and those with significant comorbidities-are unable to tolerate ATG. The combination of cyclosporine (CSA) and eltrombopag (EPAG) without ATG has emerged as a potential alternative, though real-world data from India remain limited.
Materials and methods
This single-center, retrospective observational study included consecutive adult patients (n = 40; ≥18 years) with severe or very severe AAA treated with CSA and EPAG without ATG between January 2020 and December 2025, study ends December 2025. Patients had contraindications to ATG due to age and/or comorbidities. Hematologic responses were assessed at 3-6 months. Overall response rate (ORR) was defined as complete plus partial response. Safety was evaluated using CTCAE v5.0 criteria.
Results
The median age was 56.5 years, with 72.5% having severe and 27.5% very severe AAA. At 6 months, complete response was observed in 17.5% and partial response in 45%, yielding an ORR of 62.5%. Notable improvements were observed in hemoglobin, absolute neutrophil count, and platelet count (all p < 0.001). Younger age (≤60 years), fewer comorbidities (≤1), higher baseline blood counts, and severe (vs very severe) disease were significantly associated with response. Treatment was well tolerated; hepatic adverse events were mild (grade ≤2), transient, and did not necessitate treatment discontinuation.
Conclusion
Cyclosporine combined with eltrombopag is a relatively safe and effective ATG-free therapeutic option for selected patients with acquired aplastic anemia, particularly older individuals and those with comorbidities. This regimen offers meaningful hematologic recovery with acceptable toxicity and represents a pragmatic alternative in real-world and resource-limited settings.
DOI
10.59556/japi.74.1689
PMID
42902644
Research metadata (titles, abstracts, authors, publication dates) is sourced from Europe PMC, which aggregates content from PubMed/MEDLINE, PubMed Central, and other providers under the U.S. National Library of Medicine (NLM/NCBI). NLM does not claim copyright on abstracts; the original journal publishers or authors may hold rights to the full articles. AI-generated summaries are derived only from the abstract, never the full text, and are always labeled as AI-generated.
Want more like this?
Browse more from the feed. A free account unlocks the full feed, saving and communities.
Back to the research feed