Treatment of graft versus host disease with mesenchymal stromal cells: A phase I study on 40 adult and pediatric patients

Martino Introna, Giovanna Lucchini, Erica Dander, Stefania Galimberti, Attilio Rovelli, Adriana Balduzzi, Daniela Longoni, Fabio Pavan, Francesca Masciocchi, Alessandra Algarotti, Caterina Micò, Anna Grassi, Sara Deola, Irene Cavattoni, Giuseppe Gaipa, Daniela Belotti, Paolo Perseghin, Matteo Parma, Enrico Pogliani, Josee GolayOlga Pedrini, Chiara Capelli, Sergio Cortelazzo, Giovanna D'Amico, Andrea Biondi, Alessandro Rambaldi, Ettore Biagi

Research output: Contribution to journalArticle

103 Citations (Scopus)

Abstract

This phase I multicenter study was aimed at assessing the feasibility and safety of intravenous administration of third party bone marrow-derived mesenchymal stromal cells (MSC) expanded in platelet lysate in 40 patients (15 children and 25 adults), experiencing steroid-resistant grade II to IV graft-versus-host disease (GVHD). Patients received a median of 3 MSC infusions after having failed conventional immunosuppressive therapy. A median cell dose of 1.5×106/kg per infusion was administered. No acute toxicity was reported. Overall, 86 adverse events and serious adverse events were reported in the study, most of which (72.1%) were of infectious nature. Overall response rate, measured at 28days after the last MSC injection, was 67.5%, with 27.5% complete response. The latter was significantly more frequent in patients exhibiting grade II GVHD as compared with higher grades (61.5% versus 11.1%, P= .002) and was borderline significant in children as compared with adults (46.7 versus 16.0%, P= .065). Overall survival at 1 and 2years from the first MSC administration was 50.0% and 38.6%, with a median survival time of 1.1years. In conclusion, MSC can be safely administered on top of conventional immunosuppression for steroid resistant GVHD treatment. Eudract Number 2008-007869-23, NCT01764100.

Original languageEnglish
Pages (from-to)375-381
Number of pages7
JournalBiology of Blood and Marrow Transplantation
Volume20
Issue number3
DOIs
Publication statusPublished - 1 Mar 2014
Externally publishedYes

Fingerprint

Graft vs Host Disease
Mesenchymal Stromal Cells
Pediatrics
Steroids
Therapeutics
Survival
Immunosuppressive Agents
Intravenous Administration
Immunosuppression
Multicenter Studies
Blood Platelets
Bone Marrow
Safety
Injections

Keywords

  • Cell therapy
  • Hematopoietic stem cell transplantation (HSCT)
  • Immunosuppressive treatment
  • Mesenchymal stromal cells
  • Steroid refractory graft-versus-host disease (GVHD)

ASJC Scopus subject areas

  • Hematology
  • Transplantation

Cite this

Treatment of graft versus host disease with mesenchymal stromal cells : A phase I study on 40 adult and pediatric patients. / Introna, Martino; Lucchini, Giovanna; Dander, Erica; Galimberti, Stefania; Rovelli, Attilio; Balduzzi, Adriana; Longoni, Daniela; Pavan, Fabio; Masciocchi, Francesca; Algarotti, Alessandra; Micò, Caterina; Grassi, Anna; Deola, Sara; Cavattoni, Irene; Gaipa, Giuseppe; Belotti, Daniela; Perseghin, Paolo; Parma, Matteo; Pogliani, Enrico; Golay, Josee; Pedrini, Olga; Capelli, Chiara; Cortelazzo, Sergio; D'Amico, Giovanna; Biondi, Andrea; Rambaldi, Alessandro; Biagi, Ettore.

In: Biology of Blood and Marrow Transplantation, Vol. 20, No. 3, 01.03.2014, p. 375-381.

Research output: Contribution to journalArticle

Introna, M, Lucchini, G, Dander, E, Galimberti, S, Rovelli, A, Balduzzi, A, Longoni, D, Pavan, F, Masciocchi, F, Algarotti, A, Micò, C, Grassi, A, Deola, S, Cavattoni, I, Gaipa, G, Belotti, D, Perseghin, P, Parma, M, Pogliani, E, Golay, J, Pedrini, O, Capelli, C, Cortelazzo, S, D'Amico, G, Biondi, A, Rambaldi, A & Biagi, E 2014, 'Treatment of graft versus host disease with mesenchymal stromal cells: A phase I study on 40 adult and pediatric patients', Biology of Blood and Marrow Transplantation, vol. 20, no. 3, pp. 375-381. https://doi.org/10.1016/j.bbmt.2013.11.033
Introna, Martino ; Lucchini, Giovanna ; Dander, Erica ; Galimberti, Stefania ; Rovelli, Attilio ; Balduzzi, Adriana ; Longoni, Daniela ; Pavan, Fabio ; Masciocchi, Francesca ; Algarotti, Alessandra ; Micò, Caterina ; Grassi, Anna ; Deola, Sara ; Cavattoni, Irene ; Gaipa, Giuseppe ; Belotti, Daniela ; Perseghin, Paolo ; Parma, Matteo ; Pogliani, Enrico ; Golay, Josee ; Pedrini, Olga ; Capelli, Chiara ; Cortelazzo, Sergio ; D'Amico, Giovanna ; Biondi, Andrea ; Rambaldi, Alessandro ; Biagi, Ettore. / Treatment of graft versus host disease with mesenchymal stromal cells : A phase I study on 40 adult and pediatric patients. In: Biology of Blood and Marrow Transplantation. 2014 ; Vol. 20, No. 3. pp. 375-381.
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AU - Introna, Martino

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AU - Dander, Erica

AU - Galimberti, Stefania

AU - Rovelli, Attilio

AU - Balduzzi, Adriana

AU - Longoni, Daniela

AU - Pavan, Fabio

AU - Masciocchi, Francesca

AU - Algarotti, Alessandra

AU - Micò, Caterina

AU - Grassi, Anna

AU - Deola, Sara

AU - Cavattoni, Irene

AU - Gaipa, Giuseppe

AU - Belotti, Daniela

AU - Perseghin, Paolo

AU - Parma, Matteo

AU - Pogliani, Enrico

AU - Golay, Josee

AU - Pedrini, Olga

AU - Capelli, Chiara

AU - Cortelazzo, Sergio

AU - D'Amico, Giovanna

AU - Biondi, Andrea

AU - Rambaldi, Alessandro

AU - Biagi, Ettore

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