Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease

Chronic Granulomatous Disease (CGD) is an inherited disorder of neutrophil function. Patients are profoundly immunocompromised and are plagued early in life with recurrent and life threatening infections. The prognosis for CGD patients whose infection persists despite appropriate medical and surgical intervention is extremely poor. Allogeneic stem cell transplantation can cure CGD however the mortality of this procedure is high for patients who are actively infected. Ongoing clinical trials at the NIH and elsewhere suggest that the use of non-myeloablative conditioning for allogeneic stem cell transplantation is safer and less toxic for patients who are free of infection at the time of transplant. The goal of this phase II study is to investigate the safety and efficacy of this novel approach to allogeneic stem cell transplantation for CGD patients who are actively infected. Following a preparative regimen designed to provide intense immunosuppression without myeloablation, patients will receive a peripheral blood stem cell graft from an HLA identical parent or sibling. Donor T-cells will be infused post-transplant if donor stem engraftment is unsatisfactory. The end points of this study are engraftment, degree of donor-host chimerism, incidence of acute and chronic GvHD, infection status, immune reconstitution, and transplant related morbidity and mortality.