Study of Post-transplant Cyclophosphamide with Abatacept, Vedolizumab, and Ruxolitinib to Prevent Graft-versus-host Disease in Children and Young Adults with Hematoloblastosis After Stem Cell Transplantation

What this Trial Is About

Researchers are evaluating a combination treatment to prevent graft-versus-host disease (GVHD) in children and young adults under 21 years old with various blood cancers and disorders after stem cell transplantation. The study focuses on patients with diseases such as acute lymphoblastic leukemia, myeloblastic leukemia, biphenotypic leukemia, bilinear leukemia, non-Hodgkin malignant lymphoma, and myelodysplastic syndrome. This is a phase 2 and 3 clinical trial aiming to improve outcomes after hematopoietic stem cell transplantation (HSCT) from unrelated or haploidentical donors, using a myeloablative conditioning regimen including treosulfan, total body irradiation, etoposide, and fludarabine. The treatment involves a conditioning regimen with treosulfan or total body irradiation combined with etoposide and fludarabine before transplantation. To prevent GVHD, patients receive post-transplant cyclophosphamide on days +3 and +4; abatacept on days +5, +14, +28, +60, and +90; vedolizumab on days 0, +14, +28, and +60; and oral ruxolitinib twice daily from day -3 to day +90 after transplantation. Donor selection considers cytomegalovirus status, donor-recipient sex, medical and psychological suitability, and blood type compatibility. Therapy duration is 120 days for high-risk patients and 180 days for others, with follow-up extending up to 3 years after HSCT. Participants undergo extensive monitoring including disease remission status, donor chimerism, minimal residual disease, and cytogenetics at multiple time points up to one year. Blood samples are collected for immune cell studies and pathogen-specific immune function, along with weekly viral monitoring by PCR for up to 100 days. Researchers assess acute and chronic GVHD using clinical criteria and biopsies when necessary, adjusting immunosuppressive therapy accordingly. Safety is tracked closely, with plans for interim and final data analyses based on GVHD incidence and transplant-related mortality. Overall, participant involvement includes regular clinical exams, laboratory tests, and long-term follow-up for up to three years.

Preventing of GVHD with Post-transplantation Cyclophosphamide, Abatacept, Vedolizumab and Ruxolitinib At Children and Young Adults with Hemoblastosis