Platelet-lysate-expanded mesenchymal stromal cells as a salvage therapy for severe resistant graft-versus-host disease in a pediatric population

Giovanna Lucchini, Martino Introna, Erica Dander, Attilio Rovelli, Adriana Balduzzi, Sonia Bonanomi, Agnese Salvadè, Chiara Capelli, Daniela Belotti, Giuseppe Gaipa, Paolo Perseghin, Paola Vinci, Edoardo Lanino, Patrizia Chiusolo, Maria Grazia Orofino, Sarah Marktel, Jose Golay, Alessandro Rambaldi, Andrea Biondi, Giovanna D'AmicoEttore Biagi

Research output: Contribution to journalArticlepeer-review


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.

Original languageEnglish
Pages (from-to)1293-1301
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Issue number9
Publication statusPublished - Sep 2010


  • Bone marrow transplantation
  • Cell therapy
  • GVHD
  • Mesenchymal stromal cells

ASJC Scopus subject areas

  • Transplantation
  • Hematology


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