Thrombopoietin Receptor Agonists in Post-Hematopoietic Cell Transplantation Complicated by Prolonged Thrombocytopenia: A Comprehensive Review

Abstract

Abdelrahman Elsayed,1 Basant Elsayed,1 Mohamed Elmarasi,1 Ahmed Adel Elsabagh,1 Engy Elsayed,2 Ibrahim Elmakaty,1 Mohamed Yassin2,3 1Department of Medical Education, Hamad Medical Corporation, Doha, Qatar; 2College of Medicine, Qatar University, Doha, Qatar; 3Hematology Section, National Center for Cancer Care and Research (NCCCR), Doha, QatarCorrespondence: Ibrahim Elmakaty; Mohamed Yassin, Email [email protected]; [email protected]: Hematopoietic cell transplantation (HCT) is a well-established procedure that has become a therapeutic mainstay for various hematological conditions. Prolonged thrombocytopenia following HCT is associated with a significant risk of morbidity and mortality, yet no universally recognized treatment protocol exists for such a complication. First-generation thrombopoietin receptor (TpoR) agonists as well as second-generation agents are known for their role in enhancing platelet production, and their use is expanding across various thrombocytopenic conditions. Therefore, we conducted this comprehensive review of the literature to provide an updated evaluation of the use of TpoR agonists and explore their efficacy and safety in the treatment of extended post-HCT thrombocytopenia. The literature search was conducted using PubMed database from 1996 through December 2023, using a predefined strategy with medical subject headings terms. We identified 64 reports on the utility of TpoR agonists, five of them were randomized controlled trials and the rest were retrospective observational studies and case series, with a total number of 1730 patients. Second-generation TpoR agonists appear more convenient than subcutaneous recombinant human thrombopoietin (rhTpo) as they can be orally administered and exhibit similar efficacy in platelet recovery, as indicated by recent trial results. Among these agents, avatrombopag, unlike eltrombopag, does not require any dietary restrictions, which could be more favorable for patients. However, eltrombopag remains the most extensively studied agent. TpoR agonists had promising effects in the treatment of post-HCT thrombocytopenia with a good safety profile so far, highlighting the potential benefit of their use.Keywords: hematopoietic cell transplantation, thrombocytopenia, thrombopoietin receptor agonists, recombinant human thrombopoietin, romiplostim, eltrombopag

Keywords

• hematopoietic cell transplantation
• thrombocytopenia
• thrombopoietin receptor agonists
• recombinant human thrombopoietin
• romiplostim
• eltrombopag.