Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA

Summary

This study is for adults aged 18-65 with myelodysplastic syndrome (MDS) or severe aplastic anemia (AA) who are scheduled to receive a donor stem cell transplant (allogeneic hematopoietic stem cell transplant). After the transplant, it is critical for the body to start making its own blood cells again. A common and serious problem is a delay in the recovery of platelets (the cells that help stop bleeding), which increases the risk of bleeding, infection, and death. This study aims to see if a new treatment can help platelets recover faster and more safely after transplant. We are comparing two drugs: Romiplostim: A long-acting injection given just once a week. rhTPO (Recombinant Human Thrombopoietin): A standard injection given every day. Both drugs are designed to help the body make more platelets. The main question is whether the once-weekly romiplostim works as well or better than the daily rhTPO, and if it is safe. About 66 patients will participate. By random chance (like flipping a coin), each participant will be assigned to receive either romiplostim or rhTPO. The treatment will start a few days after the transplant and continue until platelets recover to a safe level or for up to 8 weeks. Doctors will closely monitor all participants for 100 days to track platelet recovery, need for transfusions, side effects, and overall health.

Timeline

Start

2026-01-31

Primary completion

2028-01-31

Completion

2028-12-31

Publications

• Background Scordo M, Gilbert LJ, Hanley DM, Flynn JR, Devlin SM, Nguyen LK, Ruiz JD, Shah GL, Sauter CS, Chung DJ, Landau HJ, Lahoud OB, Lin RJ, Dahi PB, Perales MA, Giralt SA, Soff GA. Open-label pilot study of romiplostim for thrombocytopenia after autologous hematopoietic cell transplantation. Blood Adv. 2023 Apr 25;7(8):1536-1544. doi: 10.1182/bloodadvances.2022007838.
• Background Xie C, Zhao H, Bao X, Fu H, Lou L. Pharmacological characterization of hetrombopag, a novel orally active human thrombopoietin receptor agonist. J Cell Mol Med. 2018 Nov;22(11):5367-5377. doi: 10.1111/jcmm.13809. Epub 2018 Aug 29.
• Background Wang R, Cai J, Chen Z, Tian H, Cong D, Bai Y, Zhang W. Recombinant human thrombopoietin does not promote platelet engraftment in newly diagnosed multiple myeloma patients following autologous stem cell transplantation. Sci Rep. 2025 Feb 13;15(1):5393. doi: 10.1038/s41598-025-89535-7.
• Background Wang H, Huang M, Zhao Y, Qi JQ, Chen C, Tang YQ, Qiu HY, Fu CC, Tang XW, Wu DP, Ruan CG, Han Y. Recombinant Human Thrombopoietin Promotes Platelet Engraftment and Improves Prognosis of Patients with Myelodysplastic Syndromes and Aplastic Anemia after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2017 Oct;23(10):1678-1684. doi: 10.1016/j.bbmt.2017.06.010. Epub 2017 Jun 19.
• Background Han TT, Xu LP, Liu DH, Liu KY, Wang FR, Wang Y, Yan CH, Chen YH, Sun YQ, Ji Y, Wang JZ, Zhang XH, Huang XJ. Recombinant human thrombopoietin promotes platelet engraftment after haploidentical hematopoietic stem cell transplantation: a prospective randomized controlled trial. Ann Hematol. 2015 Jan;94(1):117-28. doi: 10.1007/s00277-014-2158-1. Epub 2014 Jul 30.
• Background Blumberg N, Heal JM, Phillips GL, Phipps RP. Platelets--to transfuse or not to transfuse. Lancet. 2012 Oct 13;380(9850):1287-9. doi: 10.1016/S0140-6736(12)60983-0. Epub 2012 Aug 8. No abstract available.
• Background Bolwell B, Pohlman B, Sobecks R, Andresen S, Brown S, Rybicki L, Wentling V, Kalaycio M. Prognostic importance of the platelet count 100 days post allogeneic bone marrow transplant. Bone Marrow Transplant. 2004 Feb;33(4):419-23. doi: 10.1038/sj.bmt.1704330.
• Background Kuzmina Z, Eder S, Bohm A, Pernicka E, Vormittag L, Kalhs P, Petkov V, Stary G, Nepp J, Knobler R, Just U, Krenn K, Worel N, Greinix HT. Significantly worse survival of patients with NIH-defined chronic graft-versus-host disease and thrombocytopenia or progressive onset type: results of a prospective study. Leukemia. 2012 Apr;26(4):746-56. doi: 10.1038/leu.2011.257. Epub 2011 Sep 16.
• Background Kanda Y, Chiba S, Hirai H, Sakamaki H, Iseki T, Kodera Y, Karasuno T, Okamoto S, Hirabayashi N, Iwato K, Maruta A, Fujimori Y, Furukawa T, Mineishi S, Matsuo K, Hamajima N, Imamura M. Allogeneic hematopoietic stem cell transplantation from family members other than HLA-identical siblings over the last decade (1991-2000). Blood. 2003 Aug 15;102(4):1541-7. doi: 10.1182/blood-2003-02-0430. Epub 2003 Apr 24.
• Background Yamazaki R, Kuwana M, Mori T, Okazaki Y, Kawakami Y, Ikeda Y, Okamoto S. Prolonged thrombocytopenia after allogeneic hematopoietic stem cell transplantation: associations with impaired platelet production and increased platelet turnover. Bone Marrow Transplant. 2006 Sep;38(5):377-84. doi: 10.1038/sj.bmt.1705444.
• Background Mohty M, Kuentz M, Michallet M, Bourhis JH, Milpied N, Sutton L, Jouet JP, Attal M, Bordigoni P, Cahn JY, Boiron JM, Blaise D; Societe Francaise de Greffe de Moelle et de Therapie Cellulaire (SFGM-TC). Chronic graft-versus-host disease after allogeneic blood stem cell transplantation: long-term results of a randomized study. Blood. 2002 Nov 1;100(9):3128-34. doi: 10.1182/blood.V100.9.3128.
• Results Huang XJ. Current status of haploidentical stem cell transplantation for leukemia. J Hematol Oncol. 2008 Dec 31;1:27. doi: 10.1186/1756-8722-1-27.

Drugs

Indications