MSCs With or Without Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment

Nanfang Hospital, Southern Medical University

Status and phase: Unknown; Phase 3; Phase 2

Conditions: Poor Graft Function; Delayed Platelet Engraftment; Hematological Diseases; Stem Cell Transplantation, Hematopoietic

Treatments: Biological: PBSC; Biological: MSCs

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02240992

NFH-PBSC-MSC-PGF-2014

Details and patient eligibility

About

The purpose of this study is to compare the efficacy of mesenchymal stem cells (MSCs) with or without granulocyte colony-stimulating factor (G-CSF) mobilized peripheral stem cells (PBSC) in treating patients experiencing poor graft function or delayed platelet engraftment after allogeneic hematopoietic stem cell transplantation.

Full description

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) is the only cure for many hematologic diseases. However, about 5-27% of patients would suffer from poor graft function (PGF) and more recipients might develop delayed platelet engraftment (DPE) after allo-HSCT. These complications are associated with considerable mortality related to infections or hemorrhagic complications. Treatment of PGF and DPE usually involves hematopoietic growth factors such as granulocyte colony-stimulating factor (G-CSF) and thrombopoietin (TPO), or second transplantation, but these methods have dismal effect or even a significant risk of graft-versus-host disease (GVHD).

Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs include improving hematopoietic engraftment, preventing and treating graft-versus-host disease after allo-HSCT and so on. Some studies have shown that MSCs combined with PBSC or cord blood could be useful to improve engraftment after HSCT. Several reports suggested MSCs might be effective in the treatment of PGF.

However, the efficacy of MSCs as single-drug treatment for PGF or DPE is unsatisfactory in our previous study. Therefore, in the present study, G-CSF mobilized PBSC will be used combined with MSCs in the patients with PGF or DPE after allo-HSCT.

Enrollment: 120 estimated patients

Sex: All

Ages: 14 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age：14-65 years
• PGF or DPE after allo-HSCT
• Subjects (or their legally acceptable representatives) must have signed an informed consent document

Exclusion criteria

• Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
• Patients with any conditions not suitable for the trial (investigators' decision)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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120 participants in 2 patient groups

MSCs&PBSC

Experimental group

Description:

PBSC will be intravenously infused at a dose of 2×10\^8/kg. MSCs will be intravenously infused at a dose of 1×10\^6 cells/kg once per week or until complete response(CR) . If the patients do not achieve CR or partial remission (PR) within 4 weeks, they will swithed to other therapy. If the patients achieve PR within 4 weeks, a second course of the same treatment will be given.

Treatment:

Biological: MSCs

Biological: PBSC

MSCs

Experimental group

Description:

MSCs will be intravenously infused at a dose of 1×10\^6 cells/kg once per week or until CR. If the patients have no response (NR) within 4 weeks, they will switch to other therapy. If the patients achieve PR within 4 weeks, a second course of the same treatment will be given.

Treatment:

Biological: MSCs