Cord Blood Transplant for Children and Young Adults With Blood Cancers and Non-malignant Blood Disorders Phase 2 Study Evaluating 1-Year Treatment Outcomes

What this Trial Is About

Researchers are studying children and young adults with life-threatening blood cancers and non-malignant blood disorders who do not have a matched related donor for allogeneic transplantation. This phase 2 trial aims to investigate the treatment-related mortality one year after receiving a myeloablative cord blood transplant. The study focuses on both malignant and non-malignant hematologic conditions to better understand outcomes in this vulnerable population. Participants with malignant disorders receive chemotherapy drugs including clofarabine, fludarabine phosphate, and busulfan before the transplant. They also receive immune suppression drugs tacrolimus and mycophenolate mofetil starting several days before the transplant to help prevent graft versus host disease GVHD. Patients with non-malignant disorders receive additional treatments such as rituximab and rabbit anti-thymocyte globulin before chemotherapy and transplant. All patients undergo cord blood transplantation on day 0, followed by supportive care including filgrastim injections to aid blood cell recovery and additional rituximab for non-malignant cases. Throughout the study, participants have regular blood tests, and imaging scans including computed tomography CT and positron emission tomography PET to monitor their condition. Researchers track treatment-related mortality and recovery over one year after the transplant. The study involves careful monitoring of organ function, immune response, and overall health status. Participants may have medication adjustments based on their response, with the goal of understanding safety and outcomes in this treatment approach.

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders