Eltrombopag in Treating Post‐Allogeneic Hematopoietic Stem Cell Transplantation Cytopenias: Efficacy, Response Durability, and Cost Benefit of Early Drug Tapering

The utilization of eltrombopag (EPAG) in the management of post allogenic hematopoietic cell transplant (HCT) cytopenia has exhibited promising outcomes. Isolated thrombocytopenia and poor graft function (PGF) are factors that adversely influence transplant outcomes and patient well-being. This retrospective study aims to assess EPAG efficacy in this context as a primary outcome and evaluate early EPAG tapering post complete response (CR) for cost efficiency and response durability as a secondary outcome. We analyzed 39 patients who underwent allogeneic HCT; EPAG was administered to 89.7% of patients for PGF and 10.3% for isolated thrombocytopenia. The overall response rate (ORR) after 4 weeks of EPAG treatment was 71.8%, with 48.7% achieving a complete response (CR). Early tapering of EPAG post-CR was explored for cost benefit among responders. Early tapering was defined as the start of tapering after 4 weeks of achieving CR and was found to be associated with significant response durability with a trend towards cost effectiveness. Eltrombopag response was independent of megakaryocyte number or density prior to drug initiation. The study suggests EPAG as a safe treatment for PGF and isolated thrombocytopenia post-allogeneic HCT and proposes early drug tapering as a potentially cost-beneficial approach.