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

• DOI: 10.1038/s41409-018-0102-z
• Publication Date: 2018-01-23T15:19:34Z
• Authors: 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-M...; Martin Hutter; Richard Schäfer; Erhard Seifried; Thomas Klingebiel; Halvard Bonig; Selim Kuçi
• Keywords: Refractory (planetary science); Cumulative incidence

The inability to generate mesenchymal stromal cells (MSCs) of consistent potency likely is responsible for inconsistent clinical outcomes patients with aGvHD receiving MSC products. We developed a novel manufacturing protocol characterized by high in vitro and near-identity individual doses, referred as "MSC-Frankfurt am Main (MSC-FFM)". Herein, we report the 69 who have received MSC-FFM. These were 51 children 18 adults refractory grade II (4%), III (36%) or IV (59%). Patients either frontline therapy (steroids) (29%) steroids 1-5 additional lines immunosuppressants (71%) given infusions four weekly intervals. day 28 overall response rate was 83%; at last follow-up, 61% 25% complete partial remission. median follow-up 8.1 months. Six-month estimate cumulative incidence non-relapse mortality 27% (range, 16-38); leukemia relapse 2% 0-5). This associated superior six-month survival (OS) probability 71% 61-83), compared outcome not treated product effective adults, suggesting that MSC-FFM represents promising steroid aGvHD.