Platelet-lysate-Expanded Mesenchymal Stromal Cells as a Salvage Therapy for Severe Resistant Graft-versus-Host Disease in a Pediatric Population

被引：144

作者：

Lucchini, Giovanna ^{[1
]}

Introna, Martino ^{[2
]}

Dander, Erica ^{[1
]}

Rovelli, Attilio ^{[1
]}

Balduzzi, Adriana ^{[1
]}

Bonanomi, Sonia ^{[1
]}

Salvade, Agnese ^{[1
]}

Capelli, Chiara ^{[2
]}

Belotti, Daniela ^{[1
]}

Gaipa, Giuseppe ^{[1
]}

Perseghin, Paolo ^{[3
]}

Vinci, Paola ^{[1
]}

Lanino, Edoardo ^{[4
]}

Chiusolo, Patrizia ^{[5
]}

Orofino, Maria Grazia ^{[6
]}

Marktel, Sarah ^{[7
]}

Golay, Jose ^{[2
]}

Rambaldi, Alessandro ^{[2
]}

Biondi, Andrea ^{[1
]}

D'Amico, Giovanna ^{[1
]}

Biagi, Ettore ^{[1
]}

机构：

[1] Univ Milano Bicocca, Osped San Gerardo, Pediat Clin, Monza, Italy

[2] Osped Riuniti Bergamo, USC Ematol, I-24100 Bergamo, Italy

[3] Osped San Gerardo, Ctr Trasfus UOS Aferesi, Monza, Italy

[4] IRCCS Giannina Gaslini, Dipartimento Ematol Oncol Pediat, Genoa, Italy

[5] Univ Cattolica S Cuore, Ist Ematol, Pediat Clin, Rome, Italy

[6] Univ Cagliari, Pediat Clin, Cagliari, Italy

[7] Univ Milan, Osped San Raffaele, Ist Sci, Unita Immunoematol Pediat & Trapianto Midollo Oss, I-20127 Milan, Italy

来源：

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION | 2010年 / 16卷 / 09期

关键词：

Bone marrow transplantation; Mesenchymal stromal cells; Cell therapy; GVHD; REFRACTORY ACUTE GVHD; STEM-CELLS; IFN-GAMMA; MARROW; TRANSPLANTATION; IMMUNOSUPPRESSION; WASHOUTS;

D O I：

10.1016/j.bbmt.2010.03.017

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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 II 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. Biol Blood Marrow Transplant 16: 1293-1307 (2010) (C) 2010 American Society for Blood and Marrow Transplantation

引用

页码：1293 / 1301

页数：9

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