Researchers at Vall d'Hebron University Hospital in Barcelona, Spain have published a study investigating the use of eltrombopag, a synthetic thrombopoietin receptor agonist, in children with poor graft function (PGF) after allogenic hematopoietic stem cell transplantation (HSCT). These findings, published in Pediatric Transplantation, demonstrate eltrombopag may be a safe and effective treatment.
For certain types of blood cancers, HSCT is a critical life-saving treatment. In approximately 5–27% of these patients, PGF, characterized by pancytopenia or low red blood cells, will occur. PGF may be caused by several factors and can have life-threatening consequences. While few treatments for PGF exist, eltrombopag has not currently been tested for use in children.
The goal of this retrospective study was to examine the use of eltrombopag in children. Among 198 patients who developed PGF, ten presented PGF and five were treated with eltrombopag. The overall response rate was 80% with three patients achieving complete response, one partial response, and one showing no response to eltrombopag. The median time to response was eight weeks with eltrombopag showing a favorable safety profile. Although limited by its design and small size, this study provides among the first data of eltrombopag safety and efficacy for PGF in children.
Uria-Oficialdegui ML, Alonso L, Benitez-Carabante MI, Renedo B, Oliveras M, Diaz-de-Heredia C. Use of eltrombopag for the treatment of poor graft function after hematopoietic stem cell transplantation in children. Pediatr Transplant. 2021.