Immune (idiopathic) aplastic anemia (IAA) represents the prototypical manifestation of acquired bone marrow failure disorders, featuring pancytopenia and an empty bone marrow. The immune-mediated pathophysiology of IAA provided the basis for developing therapeutic strategies. First-line treatment options encompass hematopoietic stem cell transplant (HSCT) and immunosuppressive therapy (IST), with treatment selection guided by age and donor availability. IST, involving antithymocyte globulin (ATG) and cyclosporine, serves as the cornerstone therapy for patients ineligible for HSCT. Incorporating eltrombopag, a thrombopoietin receptor agonist, has enhanced response rates, particularly in severe cases. For comorbid or frail patients unfit for ATG-based therapy, combinations of ciclosporin with eltrombopag offer a viable option. In refractory or relapsed cases unfit for HSCT, second-line options, including eltrombopag monotherapy or alternative IST regimens, should be considered. However, alternative immunosuppression strategies have shown variable efficacy and safety profiles, emphasizing the need for reasonable use within well-designed studies. While substantial progress has been made in managing IAA, challenges persist, notably in addressing treatment failure, relapse, and clonal evolution. Continued research is imperative to optimizing outcomes and enhancing patients’ quality of life.

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Non-transplant Treatments for Acquired Bone Marrow Failure Disorders

  • Pedro H. Prata,
  • Camilla Frieri,
  • Antonio M. Risitano,
  • Régis Peffault de Latour

摘要

Immune (idiopathic) aplastic anemia (IAA) represents the prototypical manifestation of acquired bone marrow failure disorders, featuring pancytopenia and an empty bone marrow. The immune-mediated pathophysiology of IAA provided the basis for developing therapeutic strategies. First-line treatment options encompass hematopoietic stem cell transplant (HSCT) and immunosuppressive therapy (IST), with treatment selection guided by age and donor availability. IST, involving antithymocyte globulin (ATG) and cyclosporine, serves as the cornerstone therapy for patients ineligible for HSCT. Incorporating eltrombopag, a thrombopoietin receptor agonist, has enhanced response rates, particularly in severe cases. For comorbid or frail patients unfit for ATG-based therapy, combinations of ciclosporin with eltrombopag offer a viable option. In refractory or relapsed cases unfit for HSCT, second-line options, including eltrombopag monotherapy or alternative IST regimens, should be considered. However, alternative immunosuppression strategies have shown variable efficacy and safety profiles, emphasizing the need for reasonable use within well-designed studies. While substantial progress has been made in managing IAA, challenges persist, notably in addressing treatment failure, relapse, and clonal evolution. Continued research is imperative to optimizing outcomes and enhancing patients’ quality of life.