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Life-threatening complications after stem cell transplantation can be treated much better than before with a new therapy / Guidelines expected to be adapted / Study led by Freiburg researchers published in the New England Journal of Medicine

For many people with acute leukemia, the transplantation of blood stem cells from healthy donors is the last chance of treatment. However, in around one in two patients, the immune cells used attack the recipient's tissue a few months after the transplant, sometimes causing life-threatening damage. Experts refer to this as a chronic graft-versus-host reaction or graft-versus-host disease (GVHD). If GVHD cannot be controlled by subsequent cortisone treatment, physicians will be able to fall back on a new therapy in future. Scientists at the Medical Center - University of Freiburg have now shown that treatment with the active substance ruxolitinib works significantly better for these patients than the previous standard therapy. The results of the global phase 3 study were published in the New England Journal of Medicine on July 15, 2021.

"The results of our study will improve the treatment of patients with chronic GVHD worldwide. These patients now have a much more effective treatment option than before. I expect that our results will soon lead to a change in treatment guidelines," says study leader Prof. Dr. Robert Zeiser, Head of the Department of Tumor Immunology and Immune Regulation at the Department of Medicine I at the Medical Center - University of Freiburg.

Research from Freiburg makes stem cell transplants safer overall

In the REACH-3 study, 329 patients with cortisone-resistant chronic GVHD were treated. Treatment with ruxolitinib brought improvement in 50 percent of patients who did not respond to cortisone. In contrast, with the previous standard therapy, this figure was just half (26 percent). The effectiveness of the therapy also lasted longer.

In a previous study, Zeiser has already shown that ruxolitinib is also very effective in acute graft-versus-host reactions. The diseases sound very similar, but have different causes. In acute GVHD, the antibody-producing B cells are directed against the recipient's tissue. In chronic GVHD, on the other hand, it is primarily T cells that control the overall immune regulation. It usually progresses more slowly than acute GVHD and is usually accompanied by a severe reduction in quality of life. "With our research, we have succeeded in making stem cell transplants much safer overall," says Zeiser happily.

Original title of the study: Ruxolitinib (RUX) vs Best Available Therapy (BAT) in Patients (Pts) with Glucocorticoid-Refractory Chronic Graft-vs-Host Disease (cGVHD): Primary Findings from the Phase 3, Randomized REACH3 Study
DOI: 10.1056/NEJMoa2033122
Link to the study: www.nejm.org/doi/full/10.1056/NEJMoa2033122

Contact:
Prof. Dr. Robert Zeiser
Head of the Department of Tumor Immunology
Department of Medicine I (Specialties: Hematology, Oncology, Stem Cell Transplantation)
Department of Medicine I (Specialties: Hematology, Oncology and Stem Cell Transplantation)
Phone: 0761 270-34580
robert.zeiser@uniklinik-freiburg.de