Peripheral Blood Stem Cell Combined With Mesenchymal Stem Cells for Treatment of Poor Graft Function

Nanfang Hospital, Southern Medical University

Status and phase: Unknown; Phase 2

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

Treatments: Biological: MSCs; Biological: PBSC

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02083718

NFH-PBSC-MSC-2014

Details and patient eligibility

About

The purpose of this study is to evaluate the utility of treating patients experiencing poor graft function after allogeneic hematopoietic stem cell transplantation with Granulocyte Colony-Stimulating Factor (G-CSF) mobilized peripheral stem cell combined (PBSC) with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors. Our first objective was to evaluate the effect of such treatment on poor graft function, and second object was to investigate the safety of such treatment.

Full description

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) is the only cure for many hematologic diseases. However, poor graft function (PGF) is an important complication after allo-HSCT that occurs in 5-27% of patients, and is associated with considerable mortality related to infections or hemorrhagic complications. Treatment of PGF usually involves the prescription of hematopoietic growth factors such as granulocyte colony-stimulating factor (G-CSF), or second transplantation, but these methods are associated with 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 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 after allo-HSCT.

Enrollment: 120 estimated patients

Sex: All

Ages: 14 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• A patient age of 14-65 years
• Poor graft function developing after allo-HSCT
• Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study

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: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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120 participants in 1 patient group

PBSC & MSCs

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. The vital signs of all patients will be closely monitored during and for 24h after administration.If the NEU and PLT levels do not attain the completely response(CR)standards within 28d, a second course of the same treatment will be given.

Treatment:

Biological: PBSC

Biological: MSCs