Children and adults with refractory acute Graft-versus-Host disease respond to treatment with the mesenchymal stromal cell preparation “MSC-FFM”

• Verfasst von: Bönig, Halvard-Björn [VerfasserIn]
• Verfasst von: Greil, Johann [VerfasserIn]
• Titel: Children and adults with refractory acute Graft-versus-Host disease respond to treatment with the mesenchymal stromal cell preparation “MSC-FFM”
• Titelzusatz: outcome report of 92 patients
• Verf.angabe: Halvard Bonig, Zyrafete Kuçi, Selim Kuçi, Shahrzad Bakhtiar, Oliver Basu, Gesine Bug, Mike Dennis, Johann Greil, Aniko Barta, Krisztián M. Kállay, Peter Lang, Giovanna Lucchini, Raj Pol, Ansgar Schulz, Karl-Walter Sykora, Irene Teichert von Luettichau, Grit Herter-Sprie, Mohammad Ashab Uddin, Phil Jenkin, Abdulrahman Alsultan, Jochen Buechner, Jerry Stein, Agnes Kelemen, Andrea Jarisch, Jan Soerensen, Emilia Salzmann-Manrique, Martin Hutter, Richard Schäfer, Erhard Seifried, Shankara Paneesha, Igor Novitzky-Basso, Aharon Gefen, Neta Nevo, Gernot Beutel, Paul-Gerhardt Schlegel, Thomas Klingebiel and Peter Bader
• E-Jahr: 2019
• Jahr: 5 December 2019
• Umfang: 13 S.
• Fussnoten: Gesehen am 21.02.2020
• Titel Quelle: Enthalten in: Cells
• Ort Quelle: Basel : MDPI, 2012
• Jahr Quelle: 2019
• Band/Heft Quelle: 8(2019,12) Artikel-Nummer 1577, 13 Seiten
• ISSN Quelle: 2073-4409
• DOI: doi:10.3390/cells8121577
• Datenträger: Online-Ressource
• Sprache: eng
• Sach-SW: cell therapy
• Sach-SW: graft-versus host
• Sach-SW: hospital exemption
• Sach-SW: mesenchymal stromal cell
• Sach-SW: refractory aGvHD
• Sach-SW: steroid-resistant aGvHD
• Sach-SW: transplantation
• K10plus-PPN: 1690628251

Abstract

(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.