Efficacy of bone marrow-derived mesenchymal stem cells in the treatment of sclerodermatous chronic graft-versus-host disease: clinical report

Biol Blood Marrow Transplant. 2010 Mar;16(3):403-12. doi: 10.1016/j.bbmt.2009.11.006. Epub 2009 Nov 17.

Abstract

The success of treatment for sclerodermatous chronic graft-versus-host disease (ScGVHD) remains disappointing. The immunomodulatory ability of bone marrow (BM)-derived mesenchymal stem cells (MSCs) shows promise in treating GVHD, especially given its previous success in treating patients with acute GVHD (aGVHD). The potential efficacy and safety issues for treating cGVHD, particularly ScGVHD, remain to be clarified, however. Here, we report 4 patients with ScGVHD who received MSCs expanded ex vivo from unrelated donors by intra-BM injection. After MSC infusion, the ratio of helper T lymphocyte (Th) 1 cells to Th2 cells was dramatically reversed, with an increase in Th1 and a decrease in Th2 achieving a new balance. Correspondingly, symptoms gradually improved in all 4 patients. During the course of MSC treatment, the patients' vital signs and laboratory results remained normal. At the time of this report, none of the 4 patients had experienced recurrence of leukemia. Although this study alone cannot guarantee the application of MSCs in ScGVHD, our findings strongly suggest that this treatment is therapeutically practicable, with no detectable side effects. This approach may provide new insight into the clinical treatment of ScGVHD, with the aim of greatly increasing the survival rate in patients with leukemia who undergo allogeneic BM transplantation (BMT).

Publication types

  • Clinical Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adipocytes / cytology
  • Adult
  • Bone Marrow Cells / cytology*
  • Cell Differentiation / drug effects
  • Cell Proliferation
  • Chondrocytes / cytology
  • Female
  • Graft vs Host Disease / complications
  • Graft vs Host Disease / pathology
  • Graft vs Host Disease / therapy*
  • Hematologic Neoplasms / therapy
  • Humans
  • Immunophenotyping
  • Interferon-gamma / metabolism
  • Interleukins / metabolism
  • Lymphocyte Count
  • Male
  • Mesenchymal Stem Cell Transplantation / adverse effects
  • Mesenchymal Stem Cell Transplantation / methods*
  • Mesenchymal Stem Cells / cytology
  • Mesenchymal Stem Cells / drug effects
  • Mesenchymal Stem Cells / immunology
  • Osteoblasts / cytology
  • Scleroderma, Localized / etiology
  • Scleroderma, Localized / pathology
  • Scleroderma, Localized / therapy*
  • T-Lymphocyte Subsets / cytology
  • T-Lymphocyte Subsets / immunology
  • T-Lymphocyte Subsets / metabolism
  • T-Lymphocytes / cytology
  • T-Lymphocytes / immunology
  • T-Lymphocytes / metabolism
  • Th1 Cells / cytology
  • Th1 Cells / immunology
  • Th1 Cells / metabolism
  • Th2 Cells / cytology
  • Th2 Cells / immunology
  • Th2 Cells / metabolism
  • Treatment Outcome

Substances

  • Interleukins
  • Interferon-gamma