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Effective treatment of steroid and therapy-refractory acute graft-versus-host disease with a novel mesenchymal stromal cell product (MSC-FFM)

The inability to generate mesenchymal stromal cells (MSCs) of consistent potency likely is responsible for inconsistent clinical outcomes of patients with aGvHD receiving MSC products. We developed a novel MSC manufacturing protocol characterized by high in vitro potency and near-identity of individ...

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Bibliographische Detailangaben
Hauptverfasser: Bader, Peter (VerfasserIn) , Greil, Johann (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 29 January 2018
In: Bone marrow transplantation
Year: 2018, Jahrgang: 53, Heft: 7, Pages: 852-862
ISSN:1476-5365
DOI:10.1038/s41409-018-0102-z
Online-Zugang:Verlag, Volltext: https://doi.org/10.1038/s41409-018-0102-z
Verlag: https://www.nature.com/articles/s41409-018-0102-z
Volltext
Verfasserangaben:Peter Bader, Zyrafete Kuçi, Shahrzad Bakhtiar, Oliver Basu, Gesine Bug, Michael Dennis, Johann Greil, Aniko Barta, Krisztián M. Kállay, Peter Lang, Giovanna Lucchini, Raj Pol, Ansgar Schulz, Karl-Walter Sykora, Irene 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, Thomas Klingebiel, Halvard Bonig, Selim Kuçi
Beschreibung
Zusammenfassung:The inability to generate mesenchymal stromal cells (MSCs) of consistent potency likely is responsible for inconsistent clinical outcomes of patients with aGvHD receiving MSC products. We developed a novel MSC manufacturing protocol characterized by high in vitro potency and near-identity of individual doses, referred to as “MSC-Frankfurt am Main (MSC-FFM)”. Herein, we report outcomes of the 69 patients who have received MSC-FFM. These were 51 children and 18 adults with refractory aGvHD grade II (4%), III (36%) or IV (59%). Patients were refractory either to frontline therapy (steroids) (29%) or to steroids and 1-5 additional lines of immunosuppressants (71%) were given infusions in four weekly intervals. The day 28 overall response rate was 83%; at the last follow-up, 61% and 25% of patients were in complete or partial remission. The median follow-up was 8.1 months. Six-month estimate for cumulative incidence of non-relapse mortality was 27% (range, 16-38); leukemia relapse mortality was 2% (range, 0-5). This was associated with a superior six-month overall survival (OS) probability rate of 71% (range, 61-83), compared to the outcome of patients not treated with MSC-FFM. This novel product was effective in children and adults, suggesting that MSC-FFM represents a promising therapy for steroid refractory aGvHD.
Beschreibung:Gesehen am 02.03.2020
Beschreibung:Online Resource
ISSN:1476-5365
DOI:10.1038/s41409-018-0102-z

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