Mesenchymal Stem Cells for Treatment of Refractory Chronic Graft-versus-host Disease

Nanfang Hospital, Southern Medical University

Status and phase

Unknown

Phase 2

Conditions

Chronic Graft-versus-host Disease

Treatments

Biological: Mesenchymal stem cells

Biological: Non-mesenchymal stem cells

Study type

Interventional

Funder types

Other

Identifiers

NCT01765660

NFH-MSCs-cGVHD-2013

Details and patient eligibility

About

The purpose of this study is to evaluate the utility of treating patients experiencing refractory chronic graft-versus-host disease with ex-vivo-expanded BM-drived mesenchymal stem cells from third-party donors. The objective was to evaluate the effect and safety of such treatment on refractory chronic graft-versus-host disease.

Full description

Allogeneic hematopoietic stem cell transplantation(allo-HSCT) can cure many hematologic diseases. Although great progress has been made in the prevention and treatment of side effects associated with transplantation,chronic graft-versus-host disease(cGVHD) remains an important complication that occurs in about 50% patients. The mortality of cGVHD and its complication could reach up to 50%,and cGVHD seriously influence the quality of life. At present, glucocorticoids and cyclosporine (CsA) are the first line treatment of cGVHD, but their effective rates are only 50%. If first line treatment is ineffective, second line drugs would be taken, such as mycophenolate mofetil(MMF)and rituximab. The effective rates of second line drugs are 30%-61%. The effective rates and prognosis of refractory cGVHD are even worse.

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 GVHD after allo-HSCT and so on. However, the efficacy of treatment of refractory cGVHD using expanded BM-derived MSCs from a third-party donor is rarely reported. 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 in treating patients with refractory cGVHD.

Enrollment

60 estimated patients

Sex

All

Ages

12 to 65 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

A patient age of 12-65 years
Recipients of allogeneic hematopoietic stem cell transplantation
Patients with refractory cGVHD
On a voluntary basis, patients are divided into MSCs and Non-MSCs group
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)