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

NCT07400341 | Recruiting Phase 2 DMC

• 1 other identifier: SOOCHOW-WXJ-2025-1294
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

MDS (Myelodysplastic Syndrome); Aplastic Anemia (AA)

Keywords

Romiplostim; Recombinant Human Thrombopoietin (rhTPO); Platelet Engraftment; Delayed Platelet Recovery

Outcome Measures

Primary Outcomes (2)

• Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days)

  To preliminarily evaluate and compare the efficacy of romiplostim versus rhTPO in promoting platelet recovery after allo-HSCT. Engraftment is defined as achieving a platelet count ≥50×10\^9/L without transfusion support for 7 consecutive days.

  From the start of transplantation until Day 60 post-transplant (or until the engraftment criterion is met).

• Incidence of ≥ Grade 3 Adverse Events within 100 Days Post-Transplant (including GVHD and thrombotic events)

  To assess and compare the safety profile between romiplostim and rhTPO, focusing on severe adverse events. Events of special interest include acute/chronic GVHD (graded per NIH criteria) and thrombotic events (e.g., TA-TMA, deep vein thrombosis).

  From the start of transplantation until Day 100 post-transplant.

Secondary Outcomes (5)

• 60-Day Platelet Engraftment Rate

  At Day 60 post-transplant.

• Platelet Transfusion Requirements (Days 0-60)

  From Day 0 (day of transplant) to Day 60 post-transplant.

• Overall Survival (OS)

  From date of transplant until death from any cause, assessed up to 2 years.

• Progression-Free Survival (PFS)

  From date of transplant until disease progression/relapse or death from any cause, assessed up to 2 years.

• Non-Relapse Mortality (NRM)

  From date of transplant until death from non-relapse causes, assessed up to 2 years.

Other Outcomes (5)

• Immune reconstitution

  At post-transplant day +30 and day +100 (±7 days)

• Megakaryocyte Count and Maturity in Bone Marrow

  At post-transplant day +14 (±3 days) and day +28 (±3 days)

• Serum Thrombopoietin (TPO) Level

  At post-transplant day +14 (±3 days) and day +28 (±3 days)

Study Arms (2)

rhTPO (Recombinant Human Thrombopoietin)

ACTIVE COMPARATOR

Participants will receive subcutaneous rhTPO injections at a fixed dose of 15000 U once daily, beginning on transplant day +4. Treatment continues until the platelet count is ≥50×10⁹/L without transfusion for 7 days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first.

Drug: Recombinant Human Thrombopoietin

Romiplostim

EXPERIMENTAL

Participants will receive subcutaneous Romiplostim injections at a starting dose of 5.0 µg/kg once weekly, beginning on transplant day +4. The dose will be adjusted weekly based on platelet counts. Treatment continues until the platelet count is ≥50×10⁹/L without transfusion for 7 days, until day +60 post-transplant, or for a maximum of 8 weeks, whichever occurs first.

Drug: Romiplostim

Interventions

Recombinant Human Thrombopoietin DRUG

Recombinant human thrombopoietin is a cytokine that stimulates platelet production by binding to the TPO receptor on megakaryocytes. In this study, it is administered as a subcutaneous injection once daily.

Also known as: rhTPO

rhTPO (Recombinant Human Thrombopoietin)

Romiplostim DRUG

Romiplostim is a thrombopoietin receptor agonist (TPO-RA) that stimulates platelet production. It is a fusion protein (peptibody) that binds to and activates the TPO receptor, promoting megakaryocyte proliferation and differentiation. In this study, it is administered as a subcutaneous injection once weekly.

Also known as: TPO-RA

Romiplostim

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 18-65 years (inclusive).
• Diagnosis of Myelodysplastic Syndrome (MDS) per WHO criteria, or Severe/Very Severe Aplastic Anemia (SAA/VSAA) per Camitta criteria, and deemed eligible for allogeneic hematopoietic stem cell transplantation (allo-HSCT).
• Planned to receive allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).
• Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
• Persistent platelet count \<20×10⁹/L with platelet transfusion dependence between post-transplant days +4 and +10. Transfusion dependence is defined as platelet count not doubling within 24-48 hours after transfusion or ongoing need for prophylactic transfusion.
• Adequate cardiac, hepatic, and renal function as required for transplantation, per investigator assessment.
• Voluntary participation with written informed consent obtained prior to any study-specific procedures.

You may not qualify if:

• Active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment.
• History of arterial thrombosis, or venous thromboembolism within the past 6 months (unless cured or stable for over 6 months).
• Active transplant-associated thrombotic microangiopathy (TA-TMA).
• Pre-transplant bone marrow biopsy showing fibrosis grade ≥ MF-2 (according to WHO criteria).
• Known hypersensitivity to Romiplostim, recombinant human thrombopoietin (rhTPO), or any of their excipients.
• Pregnant or lactating women.
• Women of childbearing potential or men with partners of childbearing potential who are unwilling to use highly effective contraception during the study period and for at least 3 months after the last dose of study drug.
• Any other condition that, in the opinion of the investigator, would make the patient unsuitable for participation in the study.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First Affiliated Hospital of Soochow University, Department of Hematology

Suzhou, Jiangsu, 215006, China

RECRUITING

Related Publications (12)

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  PMID: 36409612 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.

  PMID: 30156363 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.

  PMID: 39948233 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.

  PMID: 28642072 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.

  PMID: 25069650 BACKGROUND

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  PMID: 22877505 BACKGROUND

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  PMID: 14688814 BACKGROUND

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  PMID: 21926960 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.

  PMID: 12714500 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.

  PMID: 16915226 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.

  PMID: 12384409 BACKGROUND

• 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.

  PMID: 19117511 RESULT

MeSH Terms

Conditions

Anemia, Refractory, with Excess of Blasts; Anemia, Aplastic

Interventions

romiplostim; Thrombopoietin

Condition Hierarchy (Ancestors)

Anemia, Refractory; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Bone Marrow Failure Disorders

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Central Study Contacts

Depei Wu Professor, Director of Hematology Department, MD

CONTACT

+86-512-67972861; depeiwu@suda.edu.cn

Xiaojin Wu Associate Professor, MD

CONTACT

+86-512-67972861; wuxiaojin@suda.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2026
• First Posted: February 10, 2026
• Study Start: January 31, 2026
• Primary Completion (Estimated): January 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: April 15, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)