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

被引：180

作者：

Ball, Lynne M. ^{[1
]}

Bernardo, Maria E. ^{[2
,3
]}

Roelofs, Helene ^{[4
]}

van Tol, Maarten J. D. ^{[1
]}

Contoli, Benedetta ^{[3
]}

Zwaginga, Jaap Jan ^{[4
,5
]}

Avanzini, Maria Antonia ^{[2
]}

Conforti, Antonella ^{[3
]}

Bertaina, Alice ^{[2
,3
]}

Giorgiani, Giovanna ^{[2
]}

Jol-van der Zijde, Cornelia M. ^{[1
]}

Zecca, Marco ^{[2
]}

Le Blanc, Katarina ^{[6
]}

Frassoni, Francesco ^{[7
]}

Egeler, Rudolph Maarten ^{[1
,8
]}

Fibbe, Willem E. ^{[4
]}

Lankester, Arjan C. ^{[1
]}

Locatelli, Franco ^{[3
]}

机构：

[1] Leiden Univ, Med Ctr, Dept Paediat, Stem Cell Transplantat Unit, Leiden, Netherlands

[2] Fdn IRCCS Policlin S Matteo, Dept Paediat Haematol Oncol, Pavia, Italy

[3] Univ Pavia, IRCCS Osped Pediat Bambino Gesu, Dept Paediat Haematol Oncol, Rome, Italy

[4] Leiden Univ, Med Ctr, Dept Immunohaematol & Blood Transfus, Leiden, Netherlands

[5] Sanquin Blood supply, Ctr Clin Transfus Res, Leiden, Netherlands

[6] Karolinska Inst, Karolinska Univ Hosp, Dept Lab Med, Haematol Ctr, Stockholm, Sweden

[7] IRCCS, Ist G Gaslini, Ctr Cellule Staminali, Genoa, Italy

[8] Univ Toronto, Hosp Sick Children, Dept Stem Cell Transplantat, Toronto, ON M5G 1X8, Canada

来源：

BRITISH JOURNAL OF HAEMATOLOGY | 2013年 / 163卷 / 04期

关键词：

steroid-refractory acute graft-versus-host disease; mesenchymal stromal cells; transplantation-related mortality; haematopoietic stem cell transplantation in children; STEM-CELLS; PEDIATRIC-PATIENTS; EXTRACORPOREAL PHOTOCHEMOTHERAPY; LYMPHOCYTE-PROLIFERATION; TRANSPLANT RECIPIENTS; MARROW; INHIBIT; RESISTANT; RISK; DACLIZUMAB;

D O I：

10.1111/bjh.12545

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

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 3months-17years) 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=0001). After a median follow-up of 29years, overall survival (OS) was 37%; it was 65% vs. 0% in patients who did or did not achieve CR, respectively (P=0001). The median time from starting steroids for GvHD treatment to first MSC infusion was 13d (range 5-85). Children treated between 5 and 12d after steroid initiation showed a trend for better OS (56%) and lower TRM (17%) as compared with patients receiving MSCs 13-85d after steroids (25% and 53%, respectively; P=022 and 006, respectively). Multiple MSC infusions are safe and effective for children with steroid-refractory aGvHD, especially when employed early in the disease course.

引用

页码：501 / 509

页数：9

共 51 条

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