Premium
Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid‐refractory, grade III – IV acute graft‐versus‐host disease
Author(s) -
Ball Lynne M.,
Bernardo Maria E.,
Roelofs Helene,
Tol Maarten J. D.,
Contoli Benedetta,
Zwaginga Jaap Jan,
Avanzini Maria Antonia,
Conforti Antonella,
Bertaina Alice,
Giorgiani Giovanna,
Jolvan der Zijde Cornelia M.,
Zecca Marco,
Le Blanc Katarina,
Frassoni Francesco,
Egeler Rudolph Maarten,
Fibbe Willem E.,
Lankester Arjan C.,
Locatelli Franco
Publication year - 2013
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/bjh.12545
Subject(s) - medicine , refractory (planetary science) , steroid , graft versus host disease , mesenchymal stem cell , gastroenterology , transplantation , incidence (geometry) , cumulative incidence , surgery , pathology , biology , physics , astrobiology , hormone , optics
Summary Mesenchymal stromal cell ( MSC ) infusions have been reported to be effective in patients with steroid‐refractory, acute graft‐versus‐host disease (a G v HD ) but comprehensive data on paediatric patients are limited. We retrospectively analysed a cohort of 37 children (aged 3 months‐17 years) treated with MSC s for steroid‐refractory grade III – IV a G v HD . 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 G v HD 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 MSC s 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 a G v HD , especially when employed early in the disease course.