TY  - JOUR
A1  - Bönig, Halvard-Björn
A1  - Kuçi, Zyrafete
A1  - Kuçi, Selim
A1  - Bakhtiar, Shahrzad
A1  - Basu, Oliver
A1  - Bug, Gesine
A1  - Dennis, Mike
A1  - Greil, Johann
A1  - Barta, Aniko
A1  - Kállay, Krisztián
A1  - Lang, Peter
A1  - Lucchini, Giovanna
A1  - Pol, Raj
A1  - Schulz, Ansgar
A1  - Sykora, Karl-Walter
A1  - Teichert- von Lüttichau, Irene
A1  - Herter-Sprie, Grit
A1  - Uddin, Mohammad Ashab
A1  - Jenkin, Phil
A1  - Alsultan, Abdulrahman
A1  - Büchner, Jochen
A1  - Stein, Jerry
A1  - Kelemen, Agnes
A1  - Jarisch, Andrea
A1  - Sörensen, Jan
A1  - Salzmann-Manrique, Emilia
A1  - Hutter, Martin
A1  - Schäfer, Richard
A1  - Seifried, Erhard
A1  - Paneesha, Shankara
A1  - Novitzky-Basso, Igor
A1  - Gefen, Aharon
A1  - Nevo, Neta
A1  - Beutel, Gernot
A1  - Schlegel, Paul-Gerhardt
A1  - Klingebiel, Thomas
A1  - Bader, Peter
T1  - Children and adults with refractory acute graft-versus-host disease respond to treatment with the mesenchymal stromal cell preparation "MSC-FFM"—outcome report of 92 patients
T2  - Cells
N2  - (1) Background: Refractory acute graft-versus-host disease (R-aGvHD) remains a leading cause of death after allogeneic stem cell transplantation. Survival rates of 15% after four years are currently achieved; deaths are only in part due to aGvHD itself, but mostly due to adverse effects of R-aGvHD treatment with immunosuppressive agents as these predispose patients to opportunistic infections and loss of graft-versus-leukemia surveillance resulting in relapse. Mesenchymal stromal cells (MSC) from different tissues and those generated by various protocols have been proposed as a remedy for R-aGvHD but the enthusiasm raised by initial reports has not been ubiquitously reproduced.
(2) Methods: We previously reported on a unique MSC product, which was generated from pooled bone marrow mononuclear cells of multiple third-party donors. The products showed dose-to-dose equipotency and greater immunosuppressive capacity than individually expanded MSCs from the same donors. This product, MSC-FFM, has entered clinical routine in Germany where it is licensed with a national hospital exemption authorization. We previously reported satisfying initial clinical outcomes, which we are now updating. The data were collected in our post-approval pharmacovigilance program, i.e., this is not a clinical study and the data is high-level and non-monitored.
(3) Results: Follow-up for 92 recipients of MSC-FFM was reported, 88 with GvHD ≥°III, one-third only steroid-refractory and two-thirds therapy resistant (refractory to steroids plus ≥2 additional lines of treatment). A median of three doses of MSC-FFM was administered without apparent toxicity. Overall response rates were 82% and 81% at the first and last evaluation, respectively. At six months, the estimated overall survival was 64%, while the cumulative incidence of death from underlying disease was 3%.
(4) Conclusions: MSC-FFM promises to be a safe and efficient treatment for severe R-aGvHD.
KW  - graft-versus host
KW  - transplantation
KW  - mesenchymal stromal cell
KW  - cell therapy
KW  - hospital exemption
KW  - steroid-resistant aGvHD
KW  - refractory aGvHD
Y1  - 2019
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/52753
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-527539
SN  - 2073-4409
N1  - This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited
VL  - 8
IS  - 12, Art. 1577
SP  - 1
EP  - 13
PB  - MDPI
CY  - Basel
ER  -