The Use of Eltrombopag Post HSCT in BMFD

NCT05466201 | Completed Phase 2 DMC

• 1 other identifier: SOOCHOW-HY-2022-08
• Study Type: interventional
• Target: 118
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Stem Cell Transplant Complications

Keywords

Hematopoietic stem cell transplantation; Bone marrow failure syndrome; Eltrombopag; Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• Number and proportion of platelet engraftment on day 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 Outcomes (3)

• Number and proportion of subjects achieving neutrophil engraftment at day 35 post HSCT.

  From day 28 to 35 post HSCT

• Hematopoietic reconstruction time.

  From day 1 to day 180 post HSCT

• Overall survival (OS)

  From day 1 to day 720 post HSCT or the time of the patients enrolled dead or quitting

Study Arms (2)

supportive care

OTHER

patients in this group will receive supportive care, including blood products transfusion, anti-infective therapy rather than rhTPO or TPO-RAs.

Drug: Supportive care

Eltrombopag group

EXPERIMENTAL

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

Interventions

Eltrombopag 25 MG Oral Tablet DRUG

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.

Also known as: TPO-RA

Eltrombopag group

Supportive care DRUG

Patients receive transfusion of blood products, including platelets and red blood cells.

Also known as: support

supportive care

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• single or double umbilical cord blood transplantation;
• allergic to any of the research drugs involved in the protocol;
• simultaneously suffering from another malignant tumor;
• pregnant or lactating women;
• participating in other clinical researchers at the same time;
• 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;
• severe cataract;
• 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).;
• 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.
• 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

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (1)

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, 215006, China

Location

Related Publications (5)

• Nash RA, Kurzrock R, DiPersio J, Vose J, Linker C, Maharaj D, Nademanee AP, Negrin R, Nimer S, Shulman H, Ashby M, Jones D, Appelbaum FR, Champlin R. A phase I trial of recombinant human thrombopoietin in patients with delayed platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2000;6(1):25-34. doi: 10.1016/s1083-8791(00)70049-8.

  PMID: 10707996 BACKGROUND

• Marotta S, Marano L, Ricci P, Cacace F, Frieri C, Simeone L, Trastulli F, Vitiello S, Cardano F, Pane F, Risitano AM. Eltrombopag for post-transplant cytopenias due to poor graft function. Bone Marrow Transplant. 2019 Aug;54(8):1346-1353. doi: 10.1038/s41409-019-0442-3. Epub 2019 Jan 24.

  PMID: 30679824 BACKGROUND

• Mahat U, Rotz SJ, Hanna R. Use of Thrombopoietin Receptor Agonists in Prolonged Thrombocytopenia after Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2020 Mar;26(3):e65-e73. doi: 10.1016/j.bbmt.2019.12.003. Epub 2019 Dec 9.

  PMID: 31830528 BACKGROUND

• Zeidan AM, Battiwalla M, Berlyne D, Winkler T. Aplastic Anemia and MDS International Foundation (AAMDSIF): Bone marrow failure disease scientific symposium 2016. Leuk Res. 2017 Feb;53:8-12. doi: 10.1016/j.leukres.2016.11.011. Epub 2016 Nov 24.

  PMID: 27923195 BACKGROUND

• Dominietto A, Raiola AM, van Lint MT, Lamparelli T, Gualandi F, Berisso G, Bregante S, Frassoni F, Casarino L, Verdiani S, Bacigalupo A. Factors influencing haematological recovery after allogeneic haemopoietic stem cell transplants: graft-versus-host disease, donor type, cytomegalovirus infections and cell dose. Br J Haematol. 2001 Jan;112(1):219-27. doi: 10.1046/j.1365-2141.2001.02468.x.

  PMID: 11167808 BACKGROUND

MeSH Terms

Conditions

Bone Marrow Failure Disorders; Thrombocytopenia

Interventions

eltrombopag; Tablets; Palliative Care

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Blood Platelet Disorders; Cytopenia

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations; Patient Care; Therapeutics; Health Services; Health Care Facilities Workforce and Services

Study Officials

• Depei Wu, PhD,MD

  The First Affiliated Hospital of Soochow University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: July 12, 2022
• First Posted: July 20, 2022
• Study Start: October 22, 2022
• Primary Completion: July 31, 2025
• Study Completion: October 31, 2025
• Last Updated: December 3, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)