Mesenchymal Stem Cells Combined With Cord Blood for Treatment of Graft Failure

Nanfang Hospital, Southern Medical University

Status and phase: Unknown; Phase 2

Conditions: Graft Failure; Umbilical Cord Blood; Hematopoietic Stem Cell Transplantation; Hematological Diseases; Mesenchymal Stem Cells

Treatments: Biological: Mesenchymal stem cells and cord blood; Biological: Mesenchymal stem cells

Study type: Interventional

Funder types: Other

Identifiers

NCT01763099

NFH-MSC-auto-HSCT-2013

Details and patient eligibility

About

The purpose of this study is to evaluate the utility of treating patients experiencing graft failure after autologous hematopoietic stem cell transplantation with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors or mesenchymal stem cells combined with cord blood. The first objective of this study was to evaluate the effect of such treatment on graft failure, and second object was to investigate the safety of such treatment.

Full description

Autologous hematopoietic stem cell transplantation (auto-HSCT) are considered the standard of care for many malignancies, such as lymphoma, myeloma and some leukemias, and so on. Graft failure after auto-HSCT is a formidable complication. It occurs in 2-9.5% of patients and is associated with considerable morbidity and mortality related to infections and hemorrhagic complications. There are various options for the management of graft failure. The most common treatment of graft failure is growth factors such as granulocyte colony-stimulating factor (G-CSF)and recombinant erythropoietin,but it usually effective in the short term and no effect on platelet counts.

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 are evolving rapidly with goals of improving hematopoietic engraftment, preventing and treating graft-versus-host disease (GVHD) after allogeneic hematopoietic stem cell transplantation and so on.As an important source of hematopoietic stem cell, cord blood has been widely used in clinical practice. It is reported that cord blood combined with MSCs can increase engraftment after allogeneic hematopoietic stem cell transplantation. However, to our knowledge, the report about efficacy of treatment of graft that develops after auto-HSCT using expanded BM-derived MSCs from a third-party donor combined with cord blood is absent.If such treatment could be shown to be effective and safe, BM-derived MSCs could potentially be used as an universal donor material. This would have a major impact because the generation of donor-specific MSCs is time-consuming, costly, and often impractical if the clinical status of a patient is urgent.

In the present study, the investigators will prospectively evaluate the efficacy and safety of ex-vivo-expanded BM-derived MSCs from third-party donors or MSCs combined with cord blood in treating patients with graft failure after auto-HSCT.

Enrollment: 60 estimated patients

Sex: All

Ages: 14 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• A patient age of 14-65 years
• Graft failure developing after auto-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: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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60 participants in 2 patient groups

Mesenchymal stem cells

Experimental group

Description:

Mesenchymal stem cells group refers to treatment with mesenchymal stem cells (1×10\^6 cells/kg, intravenously)

Treatment:

Biological: Mesenchymal stem cells

Mesenchymal stem cells and cord blood

Experimental group

Description:

Mesenchymal stem cells and cord blood group refers to treatment with mesenchymal stem cells (at a dose of 1×10\^6 cells/kg) and cord blood

Treatment:

Biological: Mesenchymal stem cells and cord blood