The Journal of the Association of Physicians of India · 2026
Safety and Effectiveness of Cyclosporine and Eltrombopag in Acquired Aplastic Anemia: Experience from a Tertiary Care Center in Eastern India.
СтатьяНаблюдательное исследованиеНазвание и аннотация: Перевод пока не подготовлен
Обработка статьи · этапы, ошибки и управление
Загрузка состояния…
Ошибки и ограничения
История · было → стало
Состояние обновляется каждые 5 секунд, пока блок открыт. Процент показывается только там, где известен объём работы. Пауза останавливает продолжение этапа; текущий запрос к источнику может завершиться.
Baul Shuvraneel, Sharma Abhishek, Ghosh Kaustav, Saha Subhra Kamal, Dolai Tuphan Kanti
Аннотация
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.
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.