Eltrombopag treatment promotes platelet recovery and reduces platelet transfusion for patients with post-transplantation thrombocytopenia

NCCN Guidelines® Insights: Hematopoietic Growth Factors, Version 1.2022

“…14 In a retrospective analysis of 32 patients with prolonged post-HSCT thrombocytopenia, 66% achieved overall recovery and 44% achieved complete recovery when treated with eltrombopag. 15 Patients who responded to eltrombopag also received fewer platelet transfusions than nonresponders (median, 11 vs 95 units; P,.001). After a median follow-up of 364 days, the OS was 100% for responders and 37% for nonresponders (P,.001).…”

“…14 Several studies have examined the efficacy of TPO-RAs in patients with prolonged thrombocytopenia following HCT, including those with secondary failure of platelet recovery. [14][15][16] An extensive systematic review of 37 case reports suggests that patients with prolonged post-HSCT thrombocytopenia may respond to either eltrombopag (overall response rate [ORR], 70%) or romiplostim (ORR, 82%), with no evidence of serious adverse effects. However, the strength of the evidence is weak due to the retrospective nature of the studies, lack of control groups, and heterogeneity of the data.…”

Novel Perspectives on Thrombopoietin Receptor Agonists Applications

“…71 Platelet transfusions remain the backbone of therapy, while boost doses of CD34 þ cells have been used in PGF. 68 Some retrospective studies, as seen in ►Table 2, examined TPO-RA's role in this context, with reported response rates varying from 39.3 to 83.3%, [72][73][74][75][76][77][78][79][80][81][82][83] whereas results from few prospective studies are mostly conflicting. 84,85 Both ROMI and EP, and recently avatrombopag, proved to be as safe and effective and possibly cost-effective in comparison to transfusions in thrombocytopenia after allo-HSCT and PGF.…”

“…84,85 Both ROMI and EP, and recently avatrombopag, proved to be as safe and effective and possibly cost-effective in comparison to transfusions in thrombocytopenia after allo-HSCT and PGF. 69,[72][73][74][75][76][77][78][79][80][81][82][83][84] Notably, EP can elicit multilineage responses and along with its immunomodulatory and anti-inflammatory effects can induce enhancement of impaired hematopoiesis in allo-HSCT patients with PGF. 69,74,75,[78][79][80][81] EPinduced iron chelation is also beneficial in these patients, which are characterized by high ferritin levels and iron overload, since iron decrease could result in BM microenvironment reconstitution.…”

“…69,[72][73][74][75][76][77][78][79][80][81][82][83][84] Notably, EP can elicit multilineage responses and along with its immunomodulatory and anti-inflammatory effects can induce enhancement of impaired hematopoiesis in allo-HSCT patients with PGF. 69,74,75,[78][79][80][81] EPinduced iron chelation is also beneficial in these patients, which are characterized by high ferritin levels and iron overload, since iron decrease could result in BM microenvironment reconstitution. 78 Moreover, newly discovered antiviral properties of EP could contribute to impediment of cytomegalovirus (CMV) reactivation, which is a major cause of PGF.…”