Mesenchymal stromal cell (MSC) infusions have been reported to be effective in patients with steroid-refractory, acute graft-versus-host disease (aGvHD) but comprehensive data on paediatric patients are limited. We retrospectively analysed a cohort of 37 children (aged 3months-17years) treated with MSCs for steroid-refractory grade III-IV aGvHD. All patients but three received multiple MSC infusions. Complete response (CR) was observed in 24 children (65%), while 13 children had either partial (n=8) or no response (n=5). Cumulative incidence of transplantation-related mortality (TRM) in patients who did or did not achieve CR was 17% and 69%, respectively (P=0001). After a median follow-up of 29years, overall survival (OS) was 37%; it was 65% vs. 0% in patients who did or did not achieve CR, respectively (P=0001). The median time from starting steroids for GvHD treatment to first MSC infusion was 13d (range 5-85). Children treated between 5 and 12d after steroid initiation showed a trend for better OS (56%) and lower TRM (17%) as compared with patients receiving MSCs 13-85d after steroids (25% and 53%, respectively; P=022 and 006, respectively). Multiple MSC infusions are safe and effective for children with steroid-refractory aGvHD, especially when employed early in the disease course.

Ball, L. M., Bernardo, M. E., Roelofs, H., Van Tol, M. J. D., Contoli, B., Zwaginga, J. J., Avanzini, M. A., Conforti, A., Bertaina, A., Giorgiani, G., Jol-Van Der Zijde, C. M., Zecca, M., Le Blanc, K., Frassoni, F., Egeler, R. M., Fibbe, W. E., Lankester, A. C., Locatelli, F., Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease, <<BRITISH JOURNAL OF HAEMATOLOGY>>, 2013; 163 (4): 501-509. [doi:10.1111/bjh.12545] [https://hdl.handle.net/10807/243856]

Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease

Locatelli, Franco
2013

Abstract

Mesenchymal stromal cell (MSC) infusions have been reported to be effective in patients with steroid-refractory, acute graft-versus-host disease (aGvHD) but comprehensive data on paediatric patients are limited. We retrospectively analysed a cohort of 37 children (aged 3months-17years) treated with MSCs for steroid-refractory grade III-IV aGvHD. All patients but three received multiple MSC infusions. Complete response (CR) was observed in 24 children (65%), while 13 children had either partial (n=8) or no response (n=5). Cumulative incidence of transplantation-related mortality (TRM) in patients who did or did not achieve CR was 17% and 69%, respectively (P=0001). After a median follow-up of 29years, overall survival (OS) was 37%; it was 65% vs. 0% in patients who did or did not achieve CR, respectively (P=0001). The median time from starting steroids for GvHD treatment to first MSC infusion was 13d (range 5-85). Children treated between 5 and 12d after steroid initiation showed a trend for better OS (56%) and lower TRM (17%) as compared with patients receiving MSCs 13-85d after steroids (25% and 53%, respectively; P=022 and 006, respectively). Multiple MSC infusions are safe and effective for children with steroid-refractory aGvHD, especially when employed early in the disease course.
2013
Inglese
Ball, L. M., Bernardo, M. E., Roelofs, H., Van Tol, M. J. D., Contoli, B., Zwaginga, J. J., Avanzini, M. A., Conforti, A., Bertaina, A., Giorgiani, G., Jol-Van Der Zijde, C. M., Zecca, M., Le Blanc, K., Frassoni, F., Egeler, R. M., Fibbe, W. E., Lankester, A. C., Locatelli, F., Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease, <<BRITISH JOURNAL OF HAEMATOLOGY>>, 2013; 163 (4): 501-509. [doi:10.1111/bjh.12545] [https://hdl.handle.net/10807/243856]
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/10807/243856
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