Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning

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 decreas...

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Veröffentlicht in:Pediatric blood & cancer 2016-02, Vol.63 (2), p.355-357
Hauptverfasser: Chellapandian, Deepak, Krueger, Joerg, Schechter, Tal, Gassas, Adam, Weitzman, Sheila, Naqvi, Ahmed, Ali, Muhammad
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container_end_page 357
container_issue 2
container_start_page 355
container_title Pediatric blood & cancer
container_volume 63
creator Chellapandian, Deepak
Krueger, Joerg
Schechter, Tal
Gassas, Adam
Weitzman, Sheila
Naqvi, Ahmed
Ali, Muhammad
description 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. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.
doi_str_mv 10.1002/pbc.25756
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subjects Alemtuzumab
Antibodies, Monoclonal, Humanized - administration & dosage
Child, Preschool
donor-recipient chimerism
Genetic Diseases, X-Linked - surgery
Hematology
hematopoietic stem cell transplantation
Hematopoietic Stem Cell Transplantation - methods
Humans
Lymphoproliferative Disorders - surgery
Male
Melphalan - administration & dosage
Myeloablative Agonists - therapeutic use
Oncology
Pediatrics
reduced intensity conditioning
Transplantation Conditioning - methods
Transplantation, Homologous - methods
Vidarabine - administration & dosage
Vidarabine - analogs & derivatives
XIAP
title Successful Allogeneic Hematopoietic Stem Cell Transplantation in XIAP Deficiency Using Reduced-Intensity Conditioning
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