title: Low-dose total-body irradiation and alemtuzumab-based reduced-intensity conditioning regimen results in durable engraftment and correction of clinical disease among children with chronic granulomatous disease.
abstract: HSCT with MAC is associated with durable donor engraftment for patients with CGD; however, MAC is limited by high rates of RRT. We used a novel RIC regimen with LD-TBI (200 cGy × two doses), fludarabine (30 mg/m(2) × three doses), and proximal alemtuzumab (0.5 mg/kg/dose × one dose) and unrelated donor grafts for consecutive patients with high-risk CGD who were not candidates for MAC at our institution. Among four children with CGD transplanted at our institution, three PBSC recipients are alive with sustained donor engraftment (median follow-up: two yr) and resolution of pre-HSCT active infections while one patient with bone marrow graft is alive after graft failure and autologous recovery. RIC may be a curative option for children with high-risk CGD.
authors.affiliation: Division of Hematology, Oncology, Blood and Marrow Transplantation, Children's Hospital Los Angeles, Los Angeles, CA, USA.
authors.forename: Bhakti
authors.initials: B
authors.lastname: Mehta
authors.forename: Kris
authors.initials: K
authors.lastname: Mahadeo
authors.forename: Neena
authors.initials: N
authors.lastname: Kapoor
authors.forename: Hisham
authors.initials: H
authors.lastname: Abdel-Azim
mesh_terms: D000293:Adolescent; D000074323:Alemtuzumab; D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage / Q000627:therapeutic use*; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D046528:Chimerism; D006085:Graft Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006105:Granulomatous Disease, Chronic / Q000628:therapy*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D019172:Transplantation Conditioning / Q000379:methods*; D061349:Unrelated Donors; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives / Q000627:therapeutic use; D014916:Whole-Body Irradiation
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents; D000074323:Alemtuzumab; D014740:Vidarabine; C024352:fludarabine
keywords: chronic granulomatous disease; hematopoietic stem cell transplantation; pediatric; reduced-intensity conditioning; unrelated donor