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.

Bonig, Halvard, Kuçi, Zyrafete, Kuçi, Selim, Bakhtiar, Shahrzad, Basu, Oliver, Bug, Gesine, Dennis, Mike, Greil, Johann, Barta, Aniko, Kállay, Krisztián M, Lang, Peter, Lucchini, Giovanna, Pol, Raj, Schulz, Ansgar, Sykora, Karl-Walter, Teichert von Luettichau, Irene, Herter-Sprie, Grit, Ashab Uddin, Mohammad, Jenkin, Phil, Alsultan, Abdulrahman, Buechner, Jochen, Stein, Jerry, Kelemen, Agnes, Jarisch, Andrea, Soerensen, Jan, Salzmann-Manrique, Emilia, Hutter, Martin, Schäfer, Richard, Seifried, Erhard, Paneesha, Shankara, Novitzky-Basso, Igor, Gefen, Aharon, Nevo, Neta, Beutel, Gernot, Schlegel, Paul-Gerhardt, Klingebiel, Thomas and Bader, Peter (2019) 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. Cells, 8 (12). ISSN 2073-4409. This article is available to all UHB staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their UHB Athens login IDs

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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.

Item Type:	Article
Additional Information:	This article is available to all UHB staff and students via ASK Discovery tool http://tinyurl.com/z795c8c by using their UHB Athens login IDs
Subjects:	QU Biochemistry QW Microbiology. Immunology WH Haemic and lymphatic systems. Haematology
Divisions:	Planned IP Care > Oncology and Clinical Haematology
Related URLs:	https://www.ncbi.nlm.nih.gov/pubmed/?ter...
Depositing User:	Jamie Edgar
Date Deposited:	13 Dec 2019 13:55
Last Modified:	13 Dec 2019 13:55
URI:	http://www.repository.uhblibrary.co.uk/id/eprint/2684

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