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Eltrombopag for post-transplant cytopenias due to poor graft function


Persistent cytopenia due to poor graft function (PoGF) is a relatively common complication which may affect up to 20% of patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Treatment options for PoGF remain limited, and reinfusion of additional HSC is often the only way to rescue hematopoiesis. Here we describe a retrospective single-center experience with the thrombopoietin-mimetic agent eltrombopag for the treatment of PoGF. Thirteen patients have received eltrombopag for either PoGF (n = 12) or primary graft failure (n = 1). In the 12 PoGF patients eltrombopag was started at the median time of 79 days after HSCT, due to persistent thrombocytopenia, with concomitant anemia and neutropenia in 7 and 3 patients, respectively. The treatment was started at the dose of 50 mg per day, and eventually increased up to 150 mg in case of lack of response. Hematological response was seen in 7 patients, with 6 complete responses. Hematological responses were seen both in patients with evidence of immune-mediated pathophysiology, and with possible infectious/iatrogenic causes. In responding patients, eltrombopag was discontinued in 6/7 patients without further relapse. These results suggest that eltrombopag is safe and possibly effective in the setting of the treatment of PoGF, and pave the way for future prospective studies.

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Author information

SM, LM, and AMR conceived the study; PR performed hematopoietic progenitor cell in vitro cultures. Al the remaining authors contributed with SM, LM and AMR to the clinical management of the patients, and collected the clinical data from their clinical records. SM, LM, and AMR wrote the manuscript, which was critically revised by all the other authors.

Conflict of interest

AMR is involved in the clinical development of eltrombopag for the treatment of aplastic anemia, serving as Principal Investigator of a phase III trial (NCT02099747), as a member of an advisory board and as a consultant for Novartis; AMR has also received lecture fees from Novartis. The paper describes an off-label use of eltrombopag. The remaining authors declare that they have no conflict of interest.

Correspondence to Antonio M Risitano.

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