Multiple infusions of mesenchymal stromal cells induce sustained remission in children with steroid‐refractory, grade III–IV acute graft‐versus‐host disease. (31st August 2013)

• Record Type: Journal Article
• Authors: 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
• Is Part Of: British journal of haematology. Volume 163:Number 4(2013:Nov.)
• Page Start: 501
• Page End: 509
• Publication Date: 2013-08-31
• Subjects: steroid‐refractory acute graft‐versus‐host disease -- mesenchymal stromal cells -- transplantation‐related mortality -- haematopoietic stem cell transplantation in children; Hematology -- Periodicals; Blood -- Diseases -- Periodicals; 616.15
• DOI: 10.1111/bjh.12545
• Languages: English
• ISSNs: 0007-1048

Abstract

Summary: 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.