Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease

Lynne M. Ball, Maria E. Bernardo, Helene Roelofs, Maarten J D van Tol, Benedetta Contoli, Jaap Jan Zwaginga, Maria Antonia Avanzini, Antonella Conforti, Alice Bertaina, Giovanna Giorgiani, Cornelia M. Jol-van der Zijde, Marco Zecca, Katarina Le Blanc, Francesco Frassoni, Rudolph Maarten Egeler, Willem E. Fibbe, Arjan C. Lankester, Franco Locatelli

Research output: Contribution to journalArticle

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Abstract

Mesenchymal stromal cell (MSC) infusions have been reported to be effective in patients with steroid-refractory, acute graft-versus-host disease (aGvHD) but comprehensive data on paediatric patients are limited. We retrospectively analysed a cohort of 37 children (aged 3 months-17 years) treated with MSCs for steroid-refractory grade III-IV aGvHD. All patients but three received multiple MSC infusions. Complete response (CR) was observed in 24 children (65%), while 13 children had either partial (n = 8) or no response (n = 5). Cumulative incidence of transplantation-related mortality (TRM) in patients who did or did not achieve CR was 17% and 69%, respectively (P = 0·001). After a median follow-up of 2·9 years, overall survival (OS) was 37%; it was 65% vs. 0% in patients who did or did not achieve CR, respectively (P = 0·001). The median time from starting steroids for GvHD treatment to first MSC infusion was 13 d (range 5-85). Children treated between 5 and 12 d after steroid initiation showed a trend for better OS (56%) and lower TRM (17%) as compared with patients receiving MSCs 13-85 d after steroids (25% and 53%, respectively; P = 0·22 and 0·06, respectively). Multiple MSC infusions are safe and effective for children with steroid-refractory aGvHD, especially when employed early in the disease course.

Original languageEnglish
Pages (from-to)501-509
Number of pages9
JournalBritish Journal of Haematology
Volume163
Issue number4
DOIs
Publication statusPublished - Nov 2013

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Graft vs Host Disease
Mesenchymal Stromal Cells
Steroids
Transplantation
Survival
Mortality
Pediatrics
Incidence

Keywords

  • Haematopoietic stem cell transplantation in children
  • Mesenchymal stromal cells
  • Steroid-refractory acute graft-versus-host disease
  • Transplantation-related mortality

ASJC Scopus subject areas

  • Hematology

Cite this

Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease. / Ball, Lynne M.; Bernardo, Maria E.; Roelofs, Helene; van Tol, Maarten J D; Contoli, Benedetta; Zwaginga, Jaap Jan; Avanzini, Maria Antonia; Conforti, Antonella; Bertaina, Alice; Giorgiani, Giovanna; Jol-van der Zijde, Cornelia M.; Zecca, Marco; Le Blanc, Katarina; Frassoni, Francesco; Egeler, Rudolph Maarten; Fibbe, Willem E.; Lankester, Arjan C.; Locatelli, Franco.

In: British Journal of Haematology, Vol. 163, No. 4, 11.2013, p. 501-509.

Research output: Contribution to journalArticle

Ball, Lynne M. ; Bernardo, Maria E. ; Roelofs, Helene ; van Tol, Maarten J D ; Contoli, Benedetta ; Zwaginga, Jaap Jan ; Avanzini, Maria Antonia ; Conforti, Antonella ; Bertaina, Alice ; Giorgiani, Giovanna ; Jol-van der Zijde, Cornelia M. ; Zecca, Marco ; Le Blanc, Katarina ; Frassoni, Francesco ; Egeler, Rudolph Maarten ; Fibbe, Willem E. ; Lankester, Arjan C. ; Locatelli, Franco. / Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid-refractory, grade III-IV acute graft-versus-host disease. In: British Journal of Haematology. 2013 ; Vol. 163, No. 4. pp. 501-509.
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