Romiplostim versus daily rhTPO for speeding platelet recovery after donor stem-cell transplant

A Randomized Phase II Study of Romiplostim vs. rhTPO for Platelet Engraftment After Allo-HSCT in Patients With MDS and Aplastic Anemia (PROMPT)

This trial will test whether a once-weekly romiplostim injection helps platelets recover faster and safely than daily rhTPO in adults 18–65 with MDS or severe aplastic anemia after a donor stem-cell transplant.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 66 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: The First Affiliated Hospital of Soochow University (Academic / other)
• Locations: 1 site (Suzhou, Jiangsu)
• Trial ID: NCT07400341 on ClinicalTrials.gov

What this trial studies

This is a randomized, open-label, parallel-controlled Phase II trial enrolling about 74 adults to yield 66 evaluable patients with MDS or severe/very severe aplastic anemia undergoing allogeneic hematopoietic stem-cell transplant. Participants who remain platelet transfusion–dependent (platelets <20×10^9/L) between post-transplant days +4 and +10 are randomized 1:1, stratified by underlying disease, to receive once-weekly romiplostim or daily recombinant human thrombopoietin (rhTPO). Treatment starts from day +4 with dose adjustments per protocol and safety monitoring for bleeding, thrombosis, and transplant-related complications. The primary goal is to provide a preliminary estimate of efficacy for platelet engraftment and to compare safety between the two regimens.

Who should consider this trial

Good fit: Adults aged 18–65 with MDS or severe/very severe aplastic anemia undergoing allo-HSCT who remain platelet transfusion–dependent (platelets <20×10^9/L) between post-transplant days +4 and +10 are ideal candidates.

Not a fit: Patients with uncontrolled active infection, significant prior thrombosis, active transplant-associated thrombotic microangiopathy, pre-transplant marrow fibrosis ≥ MF-2, or those outside the age range are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If successful, once-weekly romiplostim could shorten time to platelet recovery, reduce transfusion needs, and lower bleeding risk after transplant.

How similar studies have performed: Thrombopoietin agonists have shown promise in small post-transplant studies for improving platelet recovery, but direct head-to-head comparisons of romiplostim versus daily rhTPO are limited.

Eligibility criteria

Inclusion Criteria:

1. Age 18-65 years (inclusive).
2. 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).
3. Planned to receive allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).
4. Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
5. 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.
6. Adequate cardiac, hepatic, and renal function as required for transplantation, per investigator assessment.
7. Voluntary participation with written informed consent obtained prior to any study-specific procedures.

Exclusion Criteria:

1. Active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment.
2. History of arterial thrombosis, or venous thromboembolism within the past 6 months (unless cured or stable for over 6 months).
3. Active transplant-associated thrombotic microangiopathy (TA-TMA).
4. Pre-transplant bone marrow biopsy showing fibrosis grade ≥ MF-2 (according to WHO criteria).
5. Known hypersensitivity to Romiplostim, recombinant human thrombopoietin (rhTPO), or any of their excipients.
6. Pregnant or lactating women.
7. 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.
8. Any other condition that, in the opinion of the investigator, would make the patient unsuitable for participation in the study.

Where this trial is running

Suzhou, Jiangsu

• The First Affiliated Hospital of Soochow University, Department of Hematology — Suzhou, Jiangsu, China (Recruiting)

Study contacts

• Study coordinator: Depei Wu Professor, Director of Hematology Department, MD

Conditions MDS; Aplastic Anemia; Romiplostim; Recombinant Human Thrombopoietin; Platelet Engraftment; Delayed Platelet Recovery