Effective treatment of steroid and therapy-refractory acute graft-versus-host disease with a novel mesenchymal stromal cell product (MSC-FFM)
| Autor: | Jan Soerensen, Grit Herter-Sprie, Agnes Kelemen, Raj Pol, Aniko Barta, Erhard Seifried, Emilia Salzmann-Manrique, Thomas Klingebiel, Jerry Stein, Halvard Bonig, Andrea Jarisch, Mike Dennis, Abdulrahman Alsultan, Peter Bader, A. Schulz, Karl-Walter Sykora, Irene von Luettichau, Zyrafete Kuçi, Jochen Buechner, Martin Hutter, Giovanna Lucchini, Gesine Bug, Mohammad Ashab Uddin, Shahrzad Bakhtiar, Peter Lang, Phil Jenkin, Richard Schäfer, Krisztián M. Kállay, Oliver Basu, Johann Greil, Selim Kuçi |
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| Jazyk: | angličtina |
| Rok vydání: | 2018 |
| Předmět: |
Male
0301 basic medicine medicine.medical_specialty Stromal cell Adolescent medicine.medical_treatment Medizin Graft vs Host Disease Mesenchymal Stem Cell Transplantation Gastroenterology Article Steroid 03 medical and health sciences 0302 clinical medicine Text mining Refractory Internal medicine Humans Medicine Potency Cumulative incidence Child Survival rate Transplantation business.industry Mesenchymal stem cell Infant Newborn Infant Hematology ddc Survival Rate Treatment Outcome 030104 developmental biology Child Preschool 030220 oncology & carcinogenesis Acute Disease Female Steroids business |
| Zdroj: | Bone Marrow Transplantation |
| Popis: | 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. |
| Databáze: | OpenAIRE |
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