@article{ScheerVokuhlBlanketal.2019, author = {Scheer, Monika and Vokuhl, Christian and Blank, Bernd and Hallmen, Erika and von Kalle, Thekla and M{\"u}nter, Marc and Wessalowski, R{\"u}diger and Hartwig, Maite and Sparber-Sauer, Monika and Schlegel, Paul-Gerhardt and Kramm, Christof M. and Kontny, Udo and Spriewald, Bernd and Kegel, Thomas and Bauer, Sebastian and Kazanowska, Bernarda and Niggli, Felix and Ladenstein, Ruth and Ljungman, Gustaf and Jahnukainen, Kirsi and Fuchs, J{\"o}rg and Bielack, Stefan S. and Klingebiel, Thomas and Koscielniak, Ewa}, title = {Desmoplastic small round cell tumors: Multimodality treatment and new risk factors}, series = {Cancer Medicine}, volume = {8}, journal = {Cancer Medicine}, number = {2}, doi = {10.1002/cam4.1940}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-228444}, pages = {527-545}, year = {2019}, abstract = {Background To evaluate optimal therapy and potential risk factors. Methods Data of DSRCT patients <40 years treated in prospective CWS trials 1997-2015 were analyzed. Results Median age of 60 patients was 14.5 years. Male:female ratio was 4:1. Tumors were abdominal/retroperitoneal in 56/60 (93\%). 6/60 (10\%) presented with a localized mass, 16/60 (27\%) regionally disseminated nodes, and 38/60 (63\%) with extraperitoneal metastases. At diagnosis, 23/60 (38\%) patients had effusions, 4/60 (7\%) a thrombosis, and 37/54 (69\%) elevated CRP. 40/60 (67\%) patients underwent tumor resection, 21/60 (35\%) macroscopically complete. 37/60 (62\%) received chemotherapy according to CEVAIE (ifosfamide, vincristine, actinomycin D, carboplatin, epirubicin, etoposide), 15/60 (25\%) VAIA (ifosfamide, vincristine, adriamycin, actinomycin D) and, 5/60 (8\%) P6 (cyclophosphamide, doxorubicin, vincristine, ifosfamide, etoposide). Nine received high-dose chemotherapy, 6 received regional hyperthermia, and 20 received radiotherapy. Among 25 patients achieving complete remission, 18 (72\%) received metronomic therapies. Three-year event-free (EFS) and overall survival (OS) were 11\% (±8 confidence interval [CI] 95\%) and 30\% (±12 CI 95\%), respectively, for all patients and 26.7\% (±18.0 CI 95\%) and 56.9\% (±20.4 CI 95\%) for 25 patients achieving remission. Extra-abdominal site, localized disease, no effusion or ascites only, absence of thrombosis, normal CRP, complete tumor resection, and chemotherapy with VAIA correlated with EFS in univariate analysis. In multivariate analysis, significant factors were no thrombosis and chemotherapy with VAIA. In patients achieving complete remission, metronomic therapy with cyclophosphamide/vinblastine correlated with prolonged time to relapse. Conclusion Pleural effusions, venous thrombosis, and CRP elevation were identified as potential risk factors. The VAIA scheme showed best outcome. Maintenance therapy should be investigated further.}, language = {en} } @article{BonigKuciKucietal.2019, author = {Bonig, Halvard and Ku{\c{c}}i, Zyrafete and Ku{\c{c}}i, Selim and Bakhtiar, Shahrzad and Basu, Oliver and Bug, Gesine and Dennis, Mike and Greil, Johann and Barta, Aniko and K{\´a}llay, Kriszti{\´a}n M. and Lang, Peter and Lucchini, Giovanna and Pol, Raj and Schulz, Ansgar and Sykora, Karl-Walter and Teichert von Luettichau, Irene and Herter-Sprie, Grit and Ashab Uddin, Mohammad and Jenkin, Phil and Alsultan, Abdulrahman and Buechner, Jochen and Stein, Jerry and Kelemen, Agnes and Jarisch, Andrea and Soerensen, Jan and Salzmann-Manrique, Emilia and Hutter, Martin and Sch{\"a}fer, Richard and Seifried, Erhard and Paneesha, Shankara and Novitzky-Basso, Igor and Gefen, Aharon and Nevo, Neta and Beutel, Gernot and Schlegel, Paul-Gerhardt and Klingebiel, Thomas and Bader, Peter}, title = {Children and adults with Refractory acute Graft-versus-Host Disease respond to treatment with the Mesenchymal Stromal cell preparation "MSC-FFM"—Outcome report of 92 patients}, series = {Cells}, volume = {8}, journal = {Cells}, number = {12}, issn = {2073-4409}, doi = {10.3390/cells8121577}, url = {http://nbn-resolving.de/urn:nbn:de:bvb:20-opus-193878}, pages = {1577}, year = {2019}, abstract = {(1) Background: Refractory acute graft-versus-host disease (R-aGvHD) remains a leading cause of death after allogeneic stem cell transplantation. Survival rates of 15\% after four years are currently achieved; deaths are only in part due to aGvHD itself, but mostly due to adverse effects of R-aGvHD treatment with immunosuppressive agents as these predispose patients to opportunistic infections and loss of graft-versus-leukemia surveillance resulting in relapse. Mesenchymal stromal cells (MSC) from different tissues and those generated by various protocols have been proposed as a remedy for R-aGvHD but the enthusiasm raised by initial reports has not been ubiquitously reproduced. (2) Methods: We previously reported on a unique MSC product, which was generated from pooled bone marrow mononuclear cells of multiple third-party donors. The products showed dose-to-dose equipotency and greater immunosuppressive capacity than individually expanded MSCs from the same donors. This product, MSC-FFM, has entered clinical routine in Germany where it is licensed with a national hospital exemption authorization. We previously reported satisfying initial clinical outcomes, which we are now updating. The data were collected in our post-approval pharmacovigilance program, i.e., this is not a clinical study and the data is high-level and non-monitored. (3) Results: Follow-up for 92 recipients of MSC-FFM was reported, 88 with GvHD ≥°III, one-third only steroid-refractory and two-thirds therapy resistant (refractory to steroids plus ≥2 additional lines of treatment). A median of three doses of MSC-FFM was administered without apparent toxicity. Overall response rates were 82\% and 81\% at the first and last evaluation, respectively. At six months, the estimated overall survival was 64\%, while the cumulative incidence of death from underlying disease was 3\%. (4) Conclusions: MSC-FFM promises to be a safe and efficient treatment for severe R-aGvHD.}, language = {en} }