Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid‐refractory,grade III–IV acute graft‐versus‐host disease |
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| Authors: | 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 |
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| Affiliation: | 1. Department of Paediatrics, Stem Cell Transplantation Unit, Leiden University Medical Centre, , Leiden, The Netherlands;2. Department of Paediatric Haematology/Oncology, Fondazione IRCCS Policlinico S. Matteo, , Pavia, Italy;3. Department of Paediatric Haematology/Oncology, University of Pavia, IRCCS Ospedale Pediatrico Bambino Gesù, , Rome, Italy;4. Department of Immunohaematology and Blood Transfusion, Leiden University Medical Centre, , Leiden, The Netherlands;5. The Centre for Clinical Transfusion Research, Sanquin Blood supply, , Leiden, The Netherlands;6. Department of Laboratory Medicine, Karolinska Institutet, Haematology Centre, Karolinska University Hospital, , Stockholm, Sweden;7. Centro Cellule Staminali, Istituto G. Gaslini, IRCCS, , Genova, Italy;8. Department of Stem Cell Transplantation, Hospital for Sick Children/University of Toronto, , Toronto, ON, Canada |
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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. |
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| Keywords: | steroid‐refractory acute graft‐versus‐host disease mesenchymal stromal cells transplantation‐related mortality haematopoietic stem cell transplantation in children |
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