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

What Was Tested

This registered phase 2 trial tested Romiplostim for anemia.

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.

It had two arms (groups): rhTPO (Recombinant Human Thrombopoietin) (active comparator) and Romiplostim (experimental).

The registry classes the interventions as drug.

More on the Plan

This is a prospective, single-center, randomized, open-label, parallel-controlled Phase II clinical trial.

Study Design and Population: The study will enroll approximately 74 patients to achieve 66 evaluable subjects, randomized in a 1:1 ratio to the romiplostim group or the rhTPO group.

Randomization will be stratified by the underlying disease (MDS vs.

Eligible patients are aged 18-65 years, diagnosed with MDS or severe/very severe AA (SAA/VSAA), and are candidates for allo-HSCT from a matched sibling, haploidentical, or unrelated donor (including cord blood).

Who Took Part

The registry lists 66 planned participants were the target.

It was open to people of any sex aged from 18 years up to 65 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: 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. Adequate cardiac, hepatic, and renal function as required for transplantation, per investigator assessment.

The full record lists 7 inclusion and 8 exclusion criteria; among the exclusions: active, uncontrolled bacterial, fungal, or viral infection at the time of enrollment; active transplant-associated thrombotic microangiopathy (ta-tma); pre-transplant bone marrow biopsy showing fibrosis grade ≥ mf-2 (according to who criteria).

How the Study Was Run

The set-up was randomized, parallel-group and open-label.

Random assignment meant chance, not the researchers, decided who got Romiplostim and who got the comparison, which limits bias.

Groups ran in parallel: each of the anemia participants stayed in one arm throughout.

Nothing was hidden in this open-label Romiplostim study: the anemia participants and staff both knew the treatment.

It started in 2026-01-31 with completion listed for 2028-01-31.

It ran at 1 site in China.

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Rate of Platelet Engraftment by Day +60 (Platelets ≥50×10^9/L without transfusion for 7 consecutive days) (time frame: 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) (time frame: From the start of transplantation until Day 100 post-transplant).

It also tracked 5 secondary outcomes, for example 60-Day Platelet Engraftment Rate, Platelet Transfusion Requirements (Days 0-60), Overall Survival (OS), Progression-Free Survival (PFS) and Non-Relapse Mortality (NRM).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of Romiplostim in 66 people with anemia.

It is still recruiting; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Romiplostim compound page, Anemia research.