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

Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 65
Healthy Volunteers: f
View:

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

Locations
Other Locations
China
The First Affiliated Hospital of Soochow University
RECRUITING
Suzhou
Contact Information
Primary
Meng Zhou, MD
zhoumeng@suda.edu.cn
+86
Backup
Yue Han, PhD,MD
hanyue@suda.edu.cn
+8615606133001
Time Frame
Start Date: 2022-10-22
Estimated Completion Date: 2027-07-31
Participants
Target number of participants: 120
Treatments
Other: supportive care
patients in this group will receive supportive care, including blood products transfusion, anti-infective therapy rather than rhTPO or TPO-RAs.
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.
Sponsors
Leads: The First Affiliated Hospital of Soochow University

This content was sourced from clinicaltrials.gov