Efficacy and safety of human umbilical cord derived mesenchymal stem cell therapy in children with severe aplastic anemia following allogeneic hematopoietic stem cell transplantation: a retrospective case series of 37 patients

Pediatr Hematol Oncol. 2014 Feb;31(1):39-49. doi: 10.3109/08880018.2013.867556. Epub 2014 Jan 2.

Abstract

The treatment of pediatric severe aplastic anemia (SAA) with allogeneic hematopoietic stem cell transplantation (allo-HSCT), presents major challenges including the risks of graft failure, septic complications, and graft-versus-host disease (GVHD). Additive infusions of human umbilical cord derived mesenchymal stem cell (hUC-MSC) may be administered to improve patient survival. We retrospectively examined 37 pediatric patients with SAA who received allo-HSCT and subsequent infusions of hUC-MSC suspension at a dose of 1.0 × 10(6 )/kg. The times and doses of hUC-MSC infusions were increased in patients with severe GVHD. All patients received hUC-MSC infusions. The median time to post-transplantation neutrophil count of greater than 0.5 × 10(9 )/L was 14 days (range, 11-20 days) and time to post-transplantation platelet count of greater than 20 × 10(9 )/L was 19 days (14-29 days). The overall frequency of acute GVHD (aGVHD) was 45.9% (17/37). These aGVHD episodes occurred at a median time of post-transplantation 47 days (15-83 days). The frequency of chronic GVHD (cGVHD) was 18.9% (7/37); cGVHD developed from aGVHD in 10.8% (4/37) of patients. The GVHD-associated mortality rate was 18.9% (7/37) and aGVHD-specific mortality rate was 8.1% (3/37). The median overall survival time was 35 months (9-67 months) and the three-year overall survival rate was 74.2% (28/37). Seven patients died of GVHD, one patient died of a severe invasive fungal infection, and one patient died of renal failure. In conclusion, post-transplantation hUC-MSC infusions seemed to be safely infused in children with SAA who have previously received allo-HSCT.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Allografts
  • Anemia, Aplastic / surgery*
  • Blood Cell Count
  • Bone Marrow Transplantation / statistics & numerical data
  • Child
  • Child, Preschool
  • Cord Blood Stem Cell Transplantation* / statistics & numerical data
  • Female
  • Graft Survival
  • Graft vs Host Disease / diagnosis
  • Graft vs Host Disease / epidemiology
  • Hematopoietic Stem Cell Mobilization
  • Hematopoietic Stem Cell Transplantation* / statistics & numerical data
  • Humans
  • Infant
  • Male
  • Mesenchymal Stem Cell Transplantation* / statistics & numerical data
  • Mycoses / etiology
  • Mycoses / mortality
  • Peripheral Blood Stem Cell Transplantation / statistics & numerical data
  • Postoperative Complications / epidemiology
  • Renal Insufficiency / etiology
  • Renal Insufficiency / mortality
  • Retrospective Studies
  • Sepsis / etiology
  • Sepsis / mortality
  • Survival Rate
  • Transplantation Conditioning
  • Treatment Outcome