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<p>Effective management of aplastic anemia (AA) requires early recognition and timely initiation of appropriate therapy. The therapeutic approach includes supportive care, immunosuppressive therapy (IST), and hematopoietic stem cell transplantation (HSCT), with the choice guided by disease severity, patient age, comorbidities, and donor availability. Supportive care remains fundamental and involves red blood cell transfusions when hemoglobin falls to ≤ 7&#xa0;g/dL, platelet transfusion in the presence of clinically significant bleeding or when platelet counts fall below 10,000/µL (or &lt; 20,000/µL in febrile patients), consistent with common practices in Indian settings. Individuals with profound neutropenia should receive antibacterial and antifungal prophylaxis according to institutional protocols, while patients undergoing IST require antiviral prophylaxis. Iron chelation therapy should be individualized based on transfusion burden and iron parameters. Current evidence does not support the routine use of granulocyte colony-stimulating factor (G-CSF), erythropoiesis-stimulating agents, or granulocyte infusions. IST typically consists of anti-thymocyte globulin (ATG) combined with cyclosporine (CSA), with or without eltrombopag (EPAG). ATG plus CSA is recommended as first-line therapy for transfusion-dependent non-severe AA. For young and medically fit patients (generally ≤ 40–50 years) with severe or very severe AA (SAA/VSAA) and an available matched sibling donor (MSD), HSCT remains the treatment of choice. In patients over 40 years, the decision between IST and HSCT should be individualized based on performance status and institutional expertise. Matched unrelated donor or haploidentical HSCT is considered in patients who fail initial IST or do not have an MSD.</p>

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Treatment Overview of Aplastic Anemia

  • 期刊论文

  • 发表日期：

    2026年5月13日

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  • Tuphan Kanti Dolai,
  • Kaustav Ghosh,
  • Shipla Roy

摘要

Effective management of aplastic anemia (AA) requires early recognition and timely initiation of appropriate therapy. The therapeutic approach includes supportive care, immunosuppressive therapy (IST), and hematopoietic stem cell transplantation (HSCT), with the choice guided by disease severity, patient age, comorbidities, and donor availability. Supportive care remains fundamental and involves red blood cell transfusions when hemoglobin falls to ≤ 7 g/dL, platelet transfusion in the presence of clinically significant bleeding or when platelet counts fall below 10,000/µL (or < 20,000/µL in febrile patients), consistent with common practices in Indian settings. Individuals with profound neutropenia should receive antibacterial and antifungal prophylaxis according to institutional protocols, while patients undergoing IST require antiviral prophylaxis. Iron chelation therapy should be individualized based on transfusion burden and iron parameters. Current evidence does not support the routine use of granulocyte colony-stimulating factor (G-CSF), erythropoiesis-stimulating agents, or granulocyte infusions. IST typically consists of anti-thymocyte globulin (ATG) combined with cyclosporine (CSA), with or without eltrombopag (EPAG). ATG plus CSA is recommended as first-line therapy for transfusion-dependent non-severe AA. For young and medically fit patients (generally ≤ 40–50 years) with severe or very severe AA (SAA/VSAA) and an available matched sibling donor (MSD), HSCT remains the treatment of choice. In patients over 40 years, the decision between IST and HSCT should be individualized based on performance status and institutional expertise. Matched unrelated donor or haploidentical HSCT is considered in patients who fail initial IST or do not have an MSD.