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ABSTRACT
Introduction: For many decades, immunosuppressive therapy (IST) for severe aplastic anemia (SAA) has not advanced much beyond standard horse antithymocyte globulin (h-ATG) plus cyclosporine. Many attempts to enhance lymphocyte depletion or modulate autoreactive effector cells did not yield better outcomes. Furthermore, efforts to enhance hematopoiesis with growth factors were of limited efficacy.
Areas covered: The development of eltrombopag in SAA will be reviewed in patients who remained unresponsive after initial IST. Overall hematologic response rate was about 40–50% as single agent with multilineage recoveries observed in this setting. When combined to standard IST in front line, the addition of eltrombopag produced the highest hematologic response rate seen in SAA especially when all drugs are initiated concomitantly. Possible mechanisms for this result will be discussed.
Expert opinion: The demonstration of activity of eltrombopag in SAA represents an important option when IST therapy fails. The possible risk of clonal evolution has been anticipated and closely monitored in all eltrombopag protocols in SAA. However, to date, an increase in cumulative incidence has not been observed. Longer follow-up will better define this long-term risk. The incorporation of this agent in treatment algorithms is rapidly evolving in the salvage and also in the upfront settings.
Declaration of interest
P Scheinberg has acted in an advisory role and speaker for Novartis and also as speaker for Pfizer and Amgen. The author has no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.