Study of Reduced-Intensity Stem Cell Transplant for Children and Young Adults with Non-Malignant Diseases Using Campath, Fludarabine, and Melphalan Conditioning to Minimize Toxicity and Support Donor Cell Engraftment

What this Trial Is About

Researchers are evaluating a reduced-intensity preparative regimen for hematopoietic stem cell transplantation in children and young adults with non-malignant disorders. This approach aims to maximize immune suppression without using radiation or high-dose chemotherapy, intending to reduce early and late toxicities. The study also focuses on preventing complications like graft-versus-host disease GVHD by using specific prophylaxis methods during matched and mismatched donor transplants from bone marrow and cord blood sources. The trial includes different groups based on the type of non-malignant disorder and the donor match. Treatments involve drug regimens such as Campath, Fludarabine, Melphalan, and sometimes Thiotepa, given before transplantation to prepare the body. GVHD prevention drugs like Tacrolimus, Cyclosporine, Mycophenolate mofetil MMF, Abatacept, and Methotrexate are administered on specific days before and after the stem cell infusion, which occurs on day 0. Participants will be closely monitored for donor cell engraftment and major toxicities within the first 100 days after transplant. Assessments include measuring blood counts for neutrophil and platelet recovery, tracking acute and chronic GVHD occurrences up to two years post-transplant, and evaluating immune recovery one year after transplant. Overall and disease-free survival will also be followed for two years. The study spans from preparative treatment through long-term follow-up, with safety and effectiveness monitored at regular intervals.

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases