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Real-world data suggest effectiveness of the allogeneic mesenchymal stromal cells preparation MSC-FFM in ruxolitinib-refractory acute graft-versus-host disease

Patients with steroid-refractory acute graft-versus-host disease (aGvHD) not tolerating/responding to ruxolitinib (RR-aGvHD) have a dismal prognosis. We retrospectively assessed real-world outcomes of RR-aGvHD treated with the random-donor allogeneic MSC preparation MSC-FFM, available via Hospital E...

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Main Authors: Bönig, Halvard (Author) , Verbeek, Mareike (Author) , Herhaus, Peter (Author) , Braitsch, Krischan (Author) , Beutel, Gernot (Author) , Schmid, Christoph (Author) , Müller, Nadine Zoé (Author) , Bug, Gesine (Author) , Döring, Michaela (Author) , Stackelberg, Arend von (Author) , Tischer, Johanna (Author) , Ayuk, Francis (Author) , Wulf, Gerald (Author) , Holtick, Udo (Author) , Pfeffermann, Lisa-Marie (Author) , Jahrsdörfer, Bernd (Author) , Schrezenmeier, Hubert (Author) , Kuci, Selim (Author) , Kuci, Zyrafete (Author) , Zens, Anke (Author) , Tribanek, Michael (Author) , Zeiser, Robert (Author) , Huenecke, Sabine (Author) , Bader, Peter (Author)
Format: Article (Journal)
Language:English
Published: 21 November 2023
In: Journal of translational medicine
Year: 2023, Volume: 21, Pages: 1-11
ISSN:1479-5876
DOI:10.1186/s12967-023-04731-1
Online Access:Verlag, kostenfrei, Volltext: https://doi.org/10.1186/s12967-023-04731-1
Verlag, kostenfrei, Volltext: http://translational.medicine.biomedcentral.com/articles/10.1186/s12967-023-04731-1
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Author Notes:Halvard Bonig, Mareike Verbeek, Peter Herhaus, Krischan Braitsch, Gernot Beutel, Christoph Schmid, Nadine Müller, Gesine Bug, Michaela Döring, Arend von Stackelberg, Johanna Tischer, Francis Ayuk, Gerald Wulf, Udo Holtick, Lisa-Marie Pfeffermann, Bernd Jahrsdörfer, Hubert Schrezenmeier, Selim Kuci, Zyrafete Kuci, Anke Zens, Michael Tribanek, Robert Zeiser, Sabine Huenecke and Peter Bader
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Summary:Patients with steroid-refractory acute graft-versus-host disease (aGvHD) not tolerating/responding to ruxolitinib (RR-aGvHD) have a dismal prognosis. We retrospectively assessed real-world outcomes of RR-aGvHD treated with the random-donor allogeneic MSC preparation MSC-FFM, available via Hospital Exemption in Germany. MSC-FFM is provided as frozen cell dispersion for administration as i.v. infusion immediately after thawing, at a recommended dose of 1-2 million MSCs/kg body weight in 4 once-weekly doses. 156 patients, 33 thereof children, received MSC-FFM; 5% had Grade II, 40% had Grade III, and 54% had Grade IV aGvHD. Median (range) number of prior therapies was 4 (1-10) in adults and 7 (2-11) in children. The safety profile of MSC-FFM was consistent with previous reports for MSC therapies in general and MSC-FFM specifically. The overall response rate at Day 28 was 46% (95% confidence interval [CI] 36-55%) in adults and 64% (45-80%) in children; most responses were durable. Probability of overall survival at 6, 12 and 24 months was 47% (38-56%), 35% (27-44%) and 30% (22-39%) for adults, and 59% (40-74%), 42% (24-58%) and 35% (19-53%) for children, respectively (whole cohort: median OS 5.8 months). A recent real-world analysis of outcomes for 64 adult RR-aGvHD patients not treated with MSCs reports survival of 20%, 16% and 10% beyond 6, 12 and 24 months, respectively (median 28 days). Our data thus suggest effectiveness of MSC-FFM in RR-aGvHD.
Item Description:Gesehen am 01.10.2024
Physical Description:Online Resource
ISSN:1479-5876
DOI:10.1186/s12967-023-04731-1

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