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.
Phase:
Phase 2/Phase 3
Details
Lead Sponsor:
The First Affiliated Hospital of Soochow University