Abstract
Background:Pediatric rheumatic disease treatment has seen a surge in innovative therapies including numerous biologic medications that show great potential. Yet, many children with advanced disease are not able to reach remission on available treatment. Mesenchymal stem cells have immunomodulatory properties, and transplantation of these stem cells have been used to successfully treat immunologic conditions like graft-versus-host disease. Mesenchymal stem cell research in adult lupus has been encouraging, but there is a lack of studies in pediatric rheumatology. This retrospective case series will highlight three children with refractory autoimmune disease who received mesenchymal stem cell therapy and review the potential benefits and risks of this emerging treatment modality.Case Presentation:Three children with established diagnoses of systemic lupus erythematosus, mixed connective tissue disease and juvenile idiopathic arthritis were under the care of pediatric rheumatology at a large, tertiary-care, teaching institution for many years. Multiple non-biologic and biologic disease-modifying anti-rheumatic drugs failed to significantly decrease disease activity, and as a result, the families chose to undergo mesenchymal stem cell therapy. After transplantation, all children improved and were able to taper off conventional immunosuppressive drugs. No serious adverse events occurred. Conclusion:The three cases presented in this study reflect comparable beneficial outcomes and minimal risks published in adult studies. Mesenchymal stem cell transplantation is a promising adjunctive treatment option that necessitates further research and development of standardized infusion therapy protocols.
Mesenchymal stem cell treatment for refractory pediatric rheumatic diseases: A single center case series
