Long-term follow-up after the application of mesenchymal stromal cells in children and adolescents with steroid-refractory graft-versus-host disease

Item Type:	Article
Title:	Long-term follow-up after the application of mesenchymal stromal cells in children and adolescents with steroid-refractory graft-versus-host disease
Creators Name:	Döring, M. and Cabanillas Stanchi, K.M. and Lenglinger, K. and Treuner, C. and Gieseke, F. and Erbacher, A. and Mezger, M. and Vaegler, M. and Schlegel, P.G. and Greil, J. and Bettoni da Cunha Riehm, C. and Faul, C. and Schumm, M. and Lang, P. and Handgretinger, R. and Müller, I.
Abstract:	Steroid-refractory graft-versus-host disease (GvHD) is a life-threatening complication after allogeneic hematopoietic stem cell transplantation (alloHSCT). Alternative treatment options are often insufficient. Several studies have proven the efficacy of mesenchymal stromal cells (MSC) in the treatment of therapy-refractory acute GvHD in adult and pediatric patients. Long-term data in pediatric patients is scarce. In this retrospective analysis, a total of 25 patients with a median age of 10.6 years (range 0.6-22.1 years) who received bone marrow-derived MSC after alloHSCT for the treatment of steroid-refractory III and IV GvHD, were analyzed. The median observation period of the surviving patients was 9.3 years (1.3-12.7 years) after HSCT. Among the 25 patients, 10 (40.0%) died (relapse (n=3), multi-organ failure (n=6), cardiorespiratory failure (n=1)) at median 0.5 years (0.2-2.3 years) after HSCT. Partial response and complete remission (PR, CR) of the GvHD was achieved in 76.0% and 24.0% of the patients, respectively. Transplant-related mortality was 0% in the patients who achieved CR after MSC treatment, and 26.3% for those with PR. A median improvement by one intestinal or liver GvHD stage (range 1-4) could be achieved after MSC application. No potentially MSC-related long-term adverse effects, e. g. secondary malignancy, were identified. In conclusion, the intravenous application of allogeneic MSC was safe and proved effective for the treatment of steroid-refractory GvHD. However, larger, prospective and randomized trials are needed to evaluate these findings.
Keywords:	Mesenchymal Stromal Cells, Mesenchymal Stem Cells, Pediatric Patients, Graft-Versus-Host Disease, Intestinal Graft-Versus-Host Disease, Hematopoietic Stem Cell Transplantation
Source:	Stem Cells and Development
ISSN:	1547-3287
Publisher:	Mary Ann Liebert
Volume:	30
Number:	5
Page Range:	234-246
Date:	19 February 2021
Official Publication:	https://doi.org/10.1089/scd.2020.0191
PubMed:	View item in PubMed

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