1. Platelet-lysate-expanded mesenchymal stromal cells as a salvage therapy for severe resistant graft-versus-host disease in a pediatric population
- Author
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Lucchini, G, Introna, M, Dander, E, Rovelli, A, Balduzzi, A, Bonanomi, S, Salvadè, A, Capelli, C, Belotti, D, Gaipa, G, Perseghin, P, Vinci, P, Lanino, E, Chiusolo, P, Orofino, M, Marktel, S, Golay, J, Rambaldi, A, Biondi, A, D'Amico, G, Biagi, E, DANDER, ERICA, BELOTTI, DANIELA, GAIPA, GIUSEPPE, Orofino, MG, BIONDI, ANDREA, BIAGI, ETTORE, Lucchini, G, Introna, M, Dander, E, Rovelli, A, Balduzzi, A, Bonanomi, S, Salvadè, A, Capelli, C, Belotti, D, Gaipa, G, Perseghin, P, Vinci, P, Lanino, E, Chiusolo, P, Orofino, M, Marktel, S, Golay, J, Rambaldi, A, Biondi, A, D'Amico, G, Biagi, E, DANDER, ERICA, BELOTTI, DANIELA, GAIPA, GIUSEPPE, Orofino, MG, BIONDI, ANDREA, and BIAGI, ETTORE
- Abstract
Despite advances in graft-versus-host-disease (GVHD) treatment, it is estimated that overall survival (OS) at 2 years for hematopoietic cell transplantation (HCT) recipients who experience steroid-resistant GVHD is 10%. Among recent therapeutic approaches for GVHD treatment, mesenchymal stromal cells (MSCs) hold a key position. We describe a multicenter experience of 11 pediatric patients diagnosed with acute or chronic GVHD (aGVHD, cGVHD) treated for compassionate use with GMP-grade unrelated HLA-disparate donors' bone marrow-derived MSCs, expanded in platelet-lysate (PL)-containing medium. Eleven patients (aged 4-15 years) received intravenous (i.v.) MSCs for aGVHD or cGVHD, which was resistant to multiple lines of immunosuppression. The median dose was 1.2 x 10(6)/kg (range: 0.7-3.7 x 10(6)/kg). No acute side effects were observed, and no late side effects were reported at a median follow-up of 8 months (range: 4-18 months). Overall response was obtained in 71.4% of patients, with complete response in 23.8% of cases. None of our patients presented GVHD progression upon MSC administration, but 4 patients presented GVHD recurrence 2 to 5 months after infusion. Two patients developed chronic limited GVHD. This study underlines the safety of PL-expanded MSC use in children. MSC efficacy seems to be greater in aGVHD than in cGVHD, even after failure of multiple lines of immunosuppression.
- Published
- 2010