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March 14, 2026Current Topics in Medicinal Chemistry0 citations

Treatment Strategies for Refractory and Relapsed Aplastic Anemia – Challenges and Emerging Hope: A Review

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GLGuizhen LyuDLDongbing Li

Key Points

  • To review recent treatment advancements for refractory and relapsed aplastic anemia, focusing on emerging therapies.
  • Reviewed existing literature on severe aplastic anemia treatments.
  • Analyzed effectiveness and safety of various treatment modalities.
  • Discussed alternative donor hematopoietic stem cell transplantation options.
  • Emerging therapies like thrombopoietin receptor agonists show promise.
  • Salvage immunosuppressive therapy demonstrates effectiveness in certain cases.
  • Alternative donor transplantations, including haploidentical and cord blood, provide new options.

Abstract

Aplastic Anemia (AA) is a serious hematological disease characterized by bone marrow failure and pancytopenia, which is difficult to treat, particularly in refractory and relapsed cases. This review will consider the recent developments on the treatment of Severe Aplastic Anemia (SAA), with particular emphasis on new therapeutic modalities and their possible role in enhancing patient outcomes. We discuss the effectiveness and safety of Salvage Immunosuppressive Therapy (IST), Hematopoietic Stem Cell Transplantation (HSCT), and newer agents like Thrombopoietin Receptor Agonists (TPO-RAs) such as eltrombopag. Moreover, we also address the contribution of alternative donor HSCT, including haploidentical and umbilical cord blood transplantations, and the prospects of emerging immunomodulatory drugs. The present review is intended to give a detailed account of existing treatment options, identify active research areas, and provide practical recommendations to clinicians dealing with refractory and relapsed SAA, and it underlines the necessity of individualized, mechanism-driven strategies to maximize patient care.

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Lyu et al. (2026) studied this question.

synapsesocial.com/papers/69b4fc0eb39f7826a300c9f4https://doi.org/10.2174/0115680266435593260113191100
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