title: Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning.
abstract: Hematopoietic stem cell transplantation (HSCT) is currently the only available curative therapy for X-linked inhibitor of apoptosis (XIAP) deficiency. Myeloablative conditioning regimens are associated with high mortality rates. Reduced-intensity conditioning (RIC) is recommended in order to decrease treatment-related toxicities, but RIC regimens increase the risk for mixed donor-recipient chimerism that may progress to graft loss. We report our experience with a patient with XIAP deficiency who was successfully treated with allogeneic HSCT using a RIC protocol. Post-transplant chimerism was vigilantly monitored and maintained with donor lymphocyte infusions and a stem cell boost to a level that prevented hemophagocytic lymphohistiocytosis recurrence.
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Deepak
authors.initials: D
authors.lastname: Chellapandian
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Joerg
authors.initials: J
authors.lastname: Krueger
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Tal
authors.initials: T
authors.lastname: Schechter
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Adam
authors.initials: A
authors.lastname: Gassas
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Sheila
authors.initials: S
authors.lastname: Weitzman
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Ahmed
authors.initials: A
authors.lastname: Naqvi
authors.affiliation: Division of Hematology/Oncology/BMT, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada, M5G 1X8.
authors.forename: Muhammad
authors.initials: M
authors.lastname: Ali
mesh_terms: D000074323:Alemtuzumab; D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage; D002675:Child, Preschool; D040181:Genetic Diseases, X-Linked / Q000601:surgery*; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D008232:Lymphoproliferative Disorders / Q000601:surgery*; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D019653:Myeloablative Agonists / Q000627:therapeutic use*; D019172:Transplantation Conditioning / Q000379:methods*; D014184:Transplantation, Homologous / Q000379:methods; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D019653:Myeloablative Agonists; D000074323:Alemtuzumab; D014740:Vidarabine; C024352:fludarabine; D008558:Melphalan
keywords: XIAP; donor-recipient chimerism; hematopoietic stem cell transplantation; reduced intensity conditioning