The Use of Eltrombopag Post HSCT in BMFD

November 25, 2025 updated by: HAN Yue, The First Affiliated Hospital of Soochow University

A Multicenter Clinical Study of a TPO Receptor Agonist (Eltrombopag) in the Acceleration of Engraftment Post Hematopoietic Stem Cell Transplantation of Bone Marrow Failure Diseases

Bone marrow failure disease(BMFD) is a kind of bone marrow due to congenital or acquired hematopoietic stem cells (hemopoietic stem cell, HSC) function damage. Allogenic hemopoietic stem cell transplantation (Allo-HSCT) might be the most possible treatment to cure the disease.However, 5-26% of patients have been reported to have delayed platelet engraftment (DPE), which is defined as persistent severe thrombocytopenia (<20 × 109/L) for >35 days after transplantation . To date, no standard treatment and prevention has been recommended for DPE. In patients with DPE, the amount of transfusion, the increased risk of infection, and the prolonged average hospital stay were independent risk factors affecting the prognosis of allo-HSCT patients. Due to continuous and progressive failure in the bone marrow hematopoiesis, thrombocytopenia post HSCT is more common in BMFD patients and often achieves low response to conventional therapy, such as platelet transfusion. Therefore, it is of great significance to effectively promote hematopoietic reconstruction to improve the prognosis of transplant patients.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Bone marrow failure disease(BMFD) is a kind of bone marrow due to congenital or acquired hematopoietic stem cells (hemopoietic stem cell, HSC) function damage. Generally, BMFD can be divided into two categories based on pathogenesis, which is primary and secondary BMFD. The latter is commonly seen secondary to infection, cancer, drugs, while aplastic anemia(AA), hypoplastic myelodysplastic syndromes(hMDS), paroxysmal nocturnal haemoglobinuria(PNH) and Fanconi anaemia(FA) are included in primary BMFD. Although immunotherapy or allogenic hemopoietic stem cell transplantation (Allo-HSCT) can be selected based on different individuals, allo-HSCT is still the most effective treatment for diseases that pose a greater threat to life or have a higher degree of malignancy, such as sereve aplastic anemia(SAA), MDS and PNH. Patients undergoing allo-HSCT typically achieve neutrophil and megakaryocyte reconstruction within 2 weeks and 3 weeks after transplantation respectively. However, 5-26% of patients have been reported to have delayed platelet engraftment (DPE), which is defined as persistent severe thrombocytopenia (<20 × 109/L) for >35 days after transplantation . To date, no standard treatment and prevention has been recommended for DPE. In patients with DPE, the amount of transfusion, the increased risk of infection, and the prolonged average hospital stay were independent risk factors affecting the prognosis of allo-HSCT patients. Due to continuous and progressive failure in the bone marrow hematopoiesis, thrombocytopenia post HSCT is more common in BMFD patients and often achieves low response to conventional therapy, such as platelet transfusion. Therefore, it is of great significance to effectively promote hematopoietic reconstruction to improve the prognosis of transplant patients.

Study Type

Interventional

Enrollment (Actual)

118

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
    • Jiangsu
      • Suzhou, Jiangsu, China, 215006
        • the First Affiliated Hospital of Soochow University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

8 years to 61 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

Patients diagnosed as bone marrow failure disease who received allo-HSCT; Physical strength score 0-3 according to WHO standard

Exclusion Criteria:

  1. single or double umbilical cord blood transplantation;
  2. allergic to any of the research drugs involved in the protocol;
  3. simultaneously suffering from another malignant tumor;
  4. pregnant or lactating women;
  5. participating in other clinical researchers at the same time;
  6. patients with at least one following high risk factors of thrombosis: past medical history of thromboembolism, concurrent grade 2 to 3 hypertension (systolic BP>=160mmHg or diastolic BP>=100mmHg) , diabetes, obesity(BMI>30), family history of stroke, smoke for more than 10 years , or history of catheter thrombosis;
  7. severe cataract;
  8. Severe infectious diseases (uncured tuberculosis, pulmonary aspergillosis, viral infection, active hepatitis B/C; for positive HBsAg and HBcAg, patient is excluded if hepatitis B DNA nucleic acid test is positive, DNA negative patients can enter this clinical trial; patients with hepatitis C who have a positive hepatitis C RNA nucleic acid test are excluded).;
  9. Abnormal liver and kidney function: creatinine level ≥177 μmol/l (1.5mg/dl), transaminase and bilirubin levels increased significantly (3 times or more than the upper limit of normal), and who cannot be enrolled at the discretion of clinician.
  10. In moribund condition or concurrent severe liver, kidney, heart, nerve, lung, infectious or metabolic diseases, the severity of which will cause the patient to be unable to tolerate the treatment regimen, or may die within 7-10 days

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: supportive care
patients in this group will receive supportive care, including blood products transfusion, anti-infective therapy rather than rhTPO or TPO-RAs.
Drug: Supportive care
Patients receive transfusion of blood products, including platelets and red blood cells.
Other Names:
  • support
Experimental: Eltrombopag group
Eltrombopag treatment will be started at the dose of 50mg/d from the 1st day post hematopoietic stem cell transplantation, and the dose will be titrated by 25mg each every 7 days up to 100mg/d according to the tolerability. If not tolerable, reduce the dose to the previous tolerable level (if not tolerable at 50mg/d, reduce to 25mg/d) and maintain this dose for the following 7 days, with the attempt to restart dose escalation after this 7-day period.
Drug: Eltrombopag 25 MG Oral Tablet
Since thrombopoietin receptor agonists (TPO-RAs) have never been regularly used for promotion of cell engraftment post hematopoietic stem cell transplantation HSCT), we design this eltrombopag intervention to evaluate the safety and efficacy of TPO-RAs post HSCT.
Other Names:
  • TPO-RA

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number and proportion of platelet engraftment on day 35 post HSCT.
Time Frame: From day 28 to 35 post HSCT
Platelet engraftment is defined as platelet count >20×109/L in consecutive 7 days (namely day 28 to day 35 after HSCT) without platelet transfusion in the prior 7 days (in other words, no platelet transfusion after day 21).
From day 28 to 35 post HSCT

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number and proportion of subjects achieving neutrophil engraftment at day 35 post HSCT.
Time Frame: From day 28 to 35 post HSCT
Neutrophil engraftment is defined as ANC>0.5×109/L for consecutive 3 days without administration of G-csf
From day 28 to 35 post HSCT
Hematopoietic reconstruction time.
Time Frame: From day 1 to day 180 post HSCT
Reconstruction time is defined as the time from day 1 after allo-HSCT to platelet or neutrophil engraftment
From day 1 to day 180 post HSCT
Overall survival (OS)
Time Frame: From day 1 to day 720 post HSCT or the time of the patients enrolled dead or quitting
OS is defined as the time from the date of day 1 post HSCT to the date of death due to any cause.
From day 1 to day 720 post HSCT or the time of the patients enrolled dead or quitting

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The First Affiliated Hospital of Soochow University

Investigators

  • Study Chair: Depei Wu, PhD,MD, the First Affiliated Hospital of Soochow University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 22, 2022

Primary Completion (Actual)

July 31, 2025

Study Completion (Actual)

October 31, 2025

Study Registration Dates

First Submitted

July 12, 2022

First Submitted That Met QC Criteria

July 17, 2022

First Posted (Actual)

July 20, 2022

Study Record Updates

Last Update Posted (Estimated)

December 3, 2025

Last Update Submitted That Met QC Criteria

November 25, 2025

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SOOCHOW-HY-2022-08

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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