Nontransplant therapy for bone marrow failure

Danielle M. Townsley, Thomas Winkler
Hematology. American Society of Hematology. Education Program · 2016 · vol. 2016(1) , pp. 83–89 · doi:10.1182/asheducation-2016.1.83 · PMID:27913466 · PMC6142431 · W2558806998
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Nontransplant therapies, including eltrombopag and danazol, are expanding options for bone marrow failure, with eltrombopag showing promise in refractory aplastic anemia and danazol preventing telomere attrition in constitutional bone marrow failure.

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Abstract

Nontransplant therapeutic options for acquired and constitutional aplastic anemia have significantly expanded during the last 5 years. In the future, transplant may be required less frequently. That trilineage hematologic responses could be achieved with the single agent eltrombopag in refractory aplastic anemia promotes new interest in growth factors after years of failed trials using other growth factor agents. Preliminary results adding eltrombopag to immunosuppressive therapy are promising, but long-term follow-up data evaluating clonal evolution rates are required before promoting its standard use in treatment-naive disease. Danazol, which is traditionally less preferred for treating cytopenias, is capable of preventing telomere attrition associated with hematologic responses in constitutional bone marrow failure resulting from telomere disease.

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MeSH descriptors

Anemia, Aplastic Anemia, Refractory Benzoates Danazol Hydrazines Immunosuppression Therapy Pyrazoles Anemia, Aplastic Anemia, Refractory Benzoates Danazol Humans Hydrazines Immunosuppression Therapy Pyrazoles

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