TY - JOUR
T1 - Platelet-lysate-expanded mesenchymal stromal cells as a salvage therapy for severe resistant graft-versus-host disease in a pediatric population
AU - Lucchini, Giovanna
AU - Introna, Martino
AU - Dander, Erica
AU - Rovelli, Attilio
AU - Balduzzi, Adriana
AU - Bonanomi, Sonia
AU - Salvadè, Agnese
AU - Capelli, Chiara
AU - Belotti, Daniela
AU - Gaipa, Giuseppe
AU - Perseghin, Paolo
AU - Vinci, Paola
AU - Lanino, Edoardo
AU - Chiusolo, Patrizia
AU - Orofino, Maria Grazia
AU - Marktel, Sarah
AU - Golay, Jose
AU - Rambaldi, Alessandro
AU - Biondi, Andrea
AU - D'Amico, Giovanna
AU - Biagi, Ettore
PY - 2010/9
Y1 - 2010/9
N2 - 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 × 106/kg (range: 0.7-3.7 × 106/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.
AB - 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 × 106/kg (range: 0.7-3.7 × 106/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.
KW - Bone marrow transplantation
KW - Cell therapy
KW - GVHD
KW - Mesenchymal stromal cells
UR - http://www.scopus.com/inward/record.url?scp=77955509074&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=77955509074&partnerID=8YFLogxK
U2 - 10.1016/j.bbmt.2010.03.017
DO - 10.1016/j.bbmt.2010.03.017
M3 - Article
C2 - 20350611
AN - SCOPUS:77955509074
VL - 16
SP - 1293
EP - 1301
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 9
ER -