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<p>Immunosuppressive therapy (IST) has been transformative in the management of aplastic anemia (AA) that is immune-mediated, especially for patients who are ineligible for hematopoietic stem cell transplantation. This manuscript offers a detailed account of IST, covering its historical development, immunological underpinnings, clinical administration and outcomes from both Indian and global cohorts. Evidence indicates that the combination of cyclosporine A (CsA) and anti-thymocyte globulin (ATG), with the addition of thrombopoietin receptor agonist (TPO RA)- eltrombopag (EPAG), yields overall response rates between 60 and 90%. Indian data reveal comparable efficacy to international experience, albeit with unique challenges related to access and long-term monitoring. Ongoing research is focused on novel immunosuppressants, risk stratification using molecular markers, and preventing relapses or clonal evolution.</p>

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Immunosuppressive Therapy in Aplastic Anemia

  • 期刊论文

  • 发表日期：

    2026年7月23日

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  • Mukul Aggarwal,
  • Manoranjan Mahapatra

摘要

Immunosuppressive therapy (IST) has been transformative in the management of aplastic anemia (AA) that is immune-mediated, especially for patients who are ineligible for hematopoietic stem cell transplantation. This manuscript offers a detailed account of IST, covering its historical development, immunological underpinnings, clinical administration and outcomes from both Indian and global cohorts. Evidence indicates that the combination of cyclosporine A (CsA) and anti-thymocyte globulin (ATG), with the addition of thrombopoietin receptor agonist (TPO RA)- eltrombopag (EPAG), yields overall response rates between 60 and 90%. Indian data reveal comparable efficacy to international experience, albeit with unique challenges related to access and long-term monitoring. Ongoing research is focused on novel immunosuppressants, risk stratification using molecular markers, and preventing relapses or clonal evolution.