Long-term follow-up after the application of mesenchymal stromal cells in children and adolescents with steroid-refractory graft-versus-host disease
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Long-term follow-up after the application of mesenchymal stromal cells in children and adolescents with steroid-refractory graft-versus-host disease. / Döring, Michaela; Cabanillas Stanchi, Karin Melanie; Lenglinger, Katrin; Treuner, Claudia; Gieseke, Friederike; Erbacher, Annika; Mezger, Markus; Vaegler, Martin; Schlegel, Paul-Gerhardt; Greil, Johann; Bettoni da Cunha Riehm, Claudia; Faul, Christoph; Schumm, Michael; Lang, Peter; Handgretinger, Rupert; Müller, Ingo.
In: STEM CELLS DEV, Vol. 30, No. 5, 03.2021, p. 234-246.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Long-term follow-up after the application of mesenchymal stromal cells in children and adolescents with steroid-refractory graft-versus-host disease
AU - Döring, Michaela
AU - Cabanillas Stanchi, Karin Melanie
AU - Lenglinger, Katrin
AU - Treuner, Claudia
AU - Gieseke, Friederike
AU - Erbacher, Annika
AU - Mezger, Markus
AU - Vaegler, Martin
AU - Schlegel, Paul-Gerhardt
AU - Greil, Johann
AU - Bettoni da Cunha Riehm, Claudia
AU - Faul, Christoph
AU - Schumm, Michael
AU - Lang, Peter
AU - Handgretinger, Rupert
AU - Müller, Ingo
PY - 2021/3
Y1 - 2021/3
N2 - Steroid-refractory graft-versus-host disease (GvHD) is a life-threatening complication after allogeneic hematopoietic stem cell transplantation (alloHSCT). Alternative treatment options are often insufficient. Several studies have proven the efficacy of mesenchymal stromal cells (MSCs) in the treatment of therapy-refractory acute GvHD in adult and pediatric patients. Long-term data in pediatric patients are scarce. In this retrospective analysis, a total of 25 patients with a median age of 10.6 years (range 0.6-22.1 years) who received bone marrow-derived MSCs after alloHSCT for the treatment of steroid-refractory III and IV GvHD were analyzed. The median observation period of the surviving patients was 9.3 years (1.3-12.7 years) after HSCT. Among the 25 patients, 10 (40.0%) died [relapse (n = 3), multiorgan failure (n = 6), cardiorespiratory failure (n = 1)] at median 0.5 years (0.2-2.3 years) after HSCT. Partial response and complete remission (PR, CR) of the GvHD were achieved in 76.0% and 24.0% of the patients, respectively. Transplant-related mortality was 0% in the patients who achieved CR after MSC treatment and 26.3% for those with PR. A median improvement by one intestinal or liver GvHD stage (range 1-4) could be achieved after MSC application. No potentially MSC-related long-term adverse effects, for example, secondary malignancy, were identified. In conclusion, the intravenous application of allogeneic MSCs was safe and proved effective for the treatment of steroid-refractory GvHD. However, larger, prospective, and randomized trials are needed to evaluate these findings.
AB - Steroid-refractory graft-versus-host disease (GvHD) is a life-threatening complication after allogeneic hematopoietic stem cell transplantation (alloHSCT). Alternative treatment options are often insufficient. Several studies have proven the efficacy of mesenchymal stromal cells (MSCs) in the treatment of therapy-refractory acute GvHD in adult and pediatric patients. Long-term data in pediatric patients are scarce. In this retrospective analysis, a total of 25 patients with a median age of 10.6 years (range 0.6-22.1 years) who received bone marrow-derived MSCs after alloHSCT for the treatment of steroid-refractory III and IV GvHD were analyzed. The median observation period of the surviving patients was 9.3 years (1.3-12.7 years) after HSCT. Among the 25 patients, 10 (40.0%) died [relapse (n = 3), multiorgan failure (n = 6), cardiorespiratory failure (n = 1)] at median 0.5 years (0.2-2.3 years) after HSCT. Partial response and complete remission (PR, CR) of the GvHD were achieved in 76.0% and 24.0% of the patients, respectively. Transplant-related mortality was 0% in the patients who achieved CR after MSC treatment and 26.3% for those with PR. A median improvement by one intestinal or liver GvHD stage (range 1-4) could be achieved after MSC application. No potentially MSC-related long-term adverse effects, for example, secondary malignancy, were identified. In conclusion, the intravenous application of allogeneic MSCs was safe and proved effective for the treatment of steroid-refractory GvHD. However, larger, prospective, and randomized trials are needed to evaluate these findings.
U2 - 10.1089/scd.2020.0191
DO - 10.1089/scd.2020.0191
M3 - SCORING: Journal article
C2 - 33446053
VL - 30
SP - 234
EP - 246
JO - STEM CELLS DEV
JF - STEM CELLS DEV
SN - 1547-3287
IS - 5
ER -
