Eltrombopag for Treatment of Thrombocytopenia after Allogeneic Hematopoietic Cell Transplantation

Abstract

Persistent thrombocytopenia is a common complication after allogeneic hematopoietic cell transplantation (HCT). Eltrombopag is an oral thrombopoietin receptor agonist whose efficacy against persistent thrombocytopenia after allogeneic HCT has not been well characterized. This retrospective study evaluated the safety and efficacy of eltrombopag in 12 consecutive patients with persistent thrombocytopenia after allogeneic HCT. Eltrombopag was started at 12.5 mg once daily and the dose was increased by 12.5 mg daily every week until platelet counts exceeded 50,000/μL. Five patients had prolonged isolated thrombocytopenia (PIT) and 7 patients had secondary failure of platelet recovery (SFPR). The cumulative incidence rate of successful platelet recovery to ≥50,000/μL without transfusion support was 60% in PIT patients and 71% in SFPR patients. No patients discontinued the drug because of adverse events or intolerability. Notably, the rate of platelet recovery was higher (100% versus 58%; P = .0017) and recovery was faster (median, 33 days versus 137 days; P = .0078) in patients with normal numbers of bone marrow megakaryocytes before starting eltrombopag than in those with decreased numbers of megakaryocytes. Eltrombopag is a promising treatment for both PIT and SFPR after allogeneic HCT. The number of megakaryocytes in bone marrow before eltrombopag treatment may predict the response to eltrombopag.

Usefulness of thrombopoietin receptor agonists for persistent clinically relevant thrombocytopenia after allogeneic stem cell transplantation

“…Although these publications may suffer from publication bias of positive outcomes, overall they show promising results. [8][9][10][11][12][13][14][15] In this preliminary study in which we recruited 20 alloSCT recipients with posttransplant severe thrombocytopenia, we have tried to perform a careful and thoughtful descriptive analysis to emphasize the importance to keep working on improving the treatment in common and relevant post-SCT complications.…”

“…7 However, the reported experience on their use after alloSCT is currently limited. [8][9][10][11][12][13][14][15] In this study, we retrospectively evaluated TPOa efficacy and safety in 20 consecutive adult alloSCT recipients who received either romiplostim or eltrombopag as a compassionate use for treating severe thrombocytopenia postengraftment in alloSCT recipients, exploring the potential predictive factors of response in this cohort.…”

Fresh or Cryopreserved CD34 + -Selected Mobilized Peripheral Blood Stem and Progenitor Cells for the Treatment of Poor Graft Function after Allogeneic Hematopoietic Cell Transplantation

“…Second allo-HSCT and infusion of additional unmanipulated stem cells are associated with high rate of GVHD and treatment-related mortality (TRM) [4–8]. The efficacy and safety of long term growth factor after allo-HSCT is unclear [9, 10]; Therefore SCB without conditioning have recently been utilized for PGF with promising results [9–19]. …”

Complex Transfusion Issues in Pediatric Hematopoietic Stem Cell Transplantation

“…While one agent, eltrombopag, is currently approved for pediatric use in chronic ITP [77], it has possible adverse effect of reticulin deposition in the marrow space and risk of malignant transformation of hematopoietic progenitors. The most appropriate place to investigate this in pediatric patients may be those who fail to engraft platelets despite otherwise intact donor hematopoiesis and those with ongoing platelet transfusion need in the context of graft-versus-host disease, similar to early successes reported in adults [78].…”