학술논문
Real-world data suggest effectiveness of the allogeneic mesenchymal stromal cells preparation MSC-FFM in ruxolitinib-refractory acute graft-versus-host disease.
Document Type
Article
Author
Bonig, Halvard; Verbeek, Mareike; Herhaus, Peter; Braitssch, Krischan; Beutel, Gernot; Schmid, Christoph; Müller, Nadine; Bug, Gesine; Döring, Michaela; von Stackelberg, Arend; Tischer, Johanna; Ayuk, Francis; Wulf, Gerald; Holtick, Udo; Pfeffermann, Lisa-Marie; Jahrsdörfer, Bernd; Schrezenmeier, Hubert; Kuci, Selim; Kuci, Zyrafete; Zens, Anke
Source
Subject
*ACUTE diseases
*GRAFT versus host disease
*STROMAL cells
*HEMATOPOIETIC stem cell transplantation
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Language
ISSN
1479-5876
Abstract
Background: Patients with steroid-refractory acute graft-versus-host disease (aGvHD) not tolerating/responding to ruxolitinib (RR-aGvHD) have a dismal prognosis. Methods: 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. Results: 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). Conclusion: 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. [ABSTRACT FROM AUTHOR]