Eltrombopag After Allogeneic Haematopoietic Cell Transplantation in a Case of Poor Graft Function and Systematic Review of the Literature

Background: Late graft failure after allogeneic haematopoietic cell transplantation (HCT) can result from the failed engraftment of long-term engrafting cells. The use of thrombopoietin (TPO) receptor agonists (TRA) has been extensively studied and remains an important component of experimental ex vivo stem cell expansion protocols, but its use in allogeneic transplantation is still evolving.

Methods: We describe the use of eltrombopag, a TRA, to stimulate the rescue of late graft failure in a patient following allogeneic HCT, and we performed a systematic review of published studies describing the use of TRAs following allogeneic transplantation.

Results: A total of eight publications were identified from our systematic search and included observational case studies (five studies, total of seven patients) that primarily addressed ITP or isolated thrombocytopenia at various time points after allogeneic HCT and prospective clinical trials (three studies, total of 177 patients with 95 patients receiving TRAs). No studies reported specifically on the use of TRAs for the treatment of trilineage graft failure as a means of in vivo stem cell expansion. The use of TRAs following allogeneic HCT appears safe and promising.

Conclusion: The use of eltrombopag or other TRAs to treat poor graft function after allogeneic HCT is intriguing and warrants further study.