Using Eltrombopag to Treat Delayed Platelet Engraftment After Stem Cell Transplant

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

Phase2; Phase3 Interventional The First Affiliated Hospital of Soochow University · NCT05466201

This study is testing if a medication called Eltrombopag can help patients who have had a stem cell transplant and are struggling with low platelet counts recover better and have fewer complications.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment120 (estimated)
Ages12 Years to 65 Years
SexAll
SponsorThe First Affiliated Hospital of Soochow University Academic / other
Drugs / interventionsimmunotherapy
Locations1 site (Suzhou, Jiangsu)
Trial IDNCT05466201 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the effectiveness of Eltrombopag in patients with bone marrow failure disease who have undergone allogenic hematopoietic stem cell transplantation (allo-HSCT). The study aims to address the issue of delayed platelet engraftment, which affects a significant percentage of these patients, leading to severe thrombocytopenia and increased complications. Participants will receive Eltrombopag alongside supportive care to evaluate its impact on platelet recovery and overall patient outcomes. The trial is designed to gather data on the safety and efficacy of this treatment approach in a Phase 2 and Phase 3 setting.

Who should consider this trial

Good fit: Ideal candidates are patients diagnosed with bone marrow failure disease who have received allo-HSCT and have a physical strength score of 0-3 according to WHO standards.

Not a fit: Patients who have undergone umbilical cord blood transplantation, have severe infectious diseases, or have high-risk factors for thrombosis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve platelet recovery and reduce complications for patients undergoing stem cell transplantation.

How similar studies have performed: While there have been studies on thrombocytopenia treatments, the specific use of Eltrombopag for delayed platelet engraftment post-HSCT is a novel approach.

Eligibility criteria

Show full inclusion / exclusion criteria
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

Where this trial is running

Suzhou, Jiangsu

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Stem Cell Transplant ComplicationsHematopoietic stem cell transplantationBone marrow failure syndromeEltrombopagThrombocytopenia
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.