Eltrombopag for Delayed Platelet Recovery and Secondary Thrombocytopenia Following Allogeneic Stem Cell Transplantation in Children

Abstract

The delay in platelet recovery after hematopoietic stem cell transplantation (HSCT) is closely related to the overall survival rate of transplanted children. The use of platelet-producing agents such as eltrombopag and romiplostim has made great progress in treating diseases such as immune thrombocytopenia and aplastic anemia. However, the use of such drugs in patients with thrombocytopenia after transplantation, especially in children, is rare. This study aimed to report eltrombopag treatment for 3 children with primary platelet engraftment failure and secondary thrombocytopenia after allogeneic HSCT. Of these patients, 2 had platelets stabilized at ≥50×10/L after eltrombopag treatment and subsequent withdrawal of eltrombopag. All 3 patients showed no clear adverse reactions. The results indicated a wide application prospect of eltrombopag treatment in children with thrombocytopenia after allogeneic HSCT.

Eltrombopag for post-transplant cytopenias due to poor graft function

“…Thus, eltrombopag seems a good candidate agent to be investigated in the setting of post-HSCT thrombocytopenia or PoGF, which are obviously associated with a similar quantitative impairment of the HSC compartment [19]. Some few anecdotic cases have been described in the last 2 years; [24][25][26][27][28][29][30][31][32][33] however, data from clinical trials are not available, with the exception of a preliminary report from a phase II placebo-controlled randomized trial [34]. In this study, which enrolled 60 transplanted (53 allogeneic and 7 autologous) patients with persistent thrombocytopenia or neutropenia, the response rate in the experimental arm was 36%, but results were statistically inconclusive in terms of superiority in comparison with the control arm (28% responses) [34].…”

Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children

“…[16][17][18][19][20] In children in the same context, data are limited to scarce case reports. [21][22][23] To our knowledge, there have been no studies on the use of eltrombopag to treat PGF post-HSCT in children. Our retrospective series, though small, shows that eltrombopag was both effective and safe in this setting, with results similar to those reported in adults.…”

Eltrombopag for thrombocytopenia following allogeneic hematopoietic stem cell transplantation in children

“…In the pediatric post‐HSCT setting, only few previously published case reports 14‐16 described the successful use of ELT as treatment for thrombocytopenia. Li et al 14 reported three children transplanted for nonmalignant disease treated for both primary and secondary failure of platelet engraftment. Treatment was effective in two patients, but was not able to produce stable increase in the platelet count in a patient transplanted for Gaucher disease with primary platelet engraftment failure.…”