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

Pediatric Blood and Cancer

Volumn 63, Issue 2, 2016, Pages 355-357

  Chellapandian, Deepak ^a   Krueger, Joerg ^a   Schechter, Tal ^a   Gassas, Adam ^a   Weitzman, Sheila ^a   Naqvi, Ahmed ^a   Ali, Muhammad ^a  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

Author keywords

donor recipient chimerism; hematopoietic stem cell transplantation; reduced intensity conditioning; XIAP

Indexed keywords

ALEMTUZUMAB; CYCLOSPORIN A; DEXAMETHASONE; ETOPOSIDE; FERRITIN; FIBRINOGEN; FLUDARABINE; MELPHALAN; MYCOPHENOLIC ACID; SOLUBLE INTERLEUKIN 2 RECEPTOR; TRIACYLGLYCEROL; X LINKED INHIBITOR OF APOPTOSIS;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; BONE MARROW TRANSPLANTATION; CASE REPORT; CD3+ T LYMPHOCYTE; CHILD; CHIMERA; DONOR LYMPHOCYTE INFUSION; EPSTEIN BARR VIRUS INFECTION; FEVER; GRAFT VERSUS HOST REACTION; HEMOPHAGOCYTIC SYNDROME; HERPES VIRUS INFECTION; HUMAN; HUMAN HERPESVIRUS 6; HYPERTENSION; MALE; NEUTROPHIL; NEUTROPHIL COUNT; NONSENSE MUTATION; PANCYTOPENIA; POLYMERASE CHAIN REACTION; PRESCHOOL CHILD; PRIORITY JOURNAL; PROTEIN DEFICIENCY; REDUCED INTENSITY CONDITIONING; REMISSION; SPLENOMEGALY; STEM CELL; STEM CELL BOOST; UNSPECIFIED SIDE EFFECT; VIRUS REACTIVATION; X LINKED INHIBITOR OF APOPTOSIS DEFICIENCY;

EID: 84945293238     PISSN: 15455009     EISSN: 15455017     Source Type: Journal    
DOI: 10.1002/pbc.25756     Document Type: Article

References (12)

1. Seemayer TA, Gross TG, Egeler RM, Pirruccello SJ, Davis JR, Kelly CM, Okano M, Lanyi A, Sumegi J. X-linked lymphoproliferative disease: Twenty-five years after the discovery. Pediatr Res 1995; 38:471–478.
2. Rigaud S, Fondanèche MC, Lambert N, Pasquier B, Mateo V, Soulas P, Galicier L, Le Deist F, Rieux-Laucat F, Revy P, Fischer A, de Saint Basile G, Latour S. XIAP deficiency in humans causes an X-linked lymphoproliferative syndrome. Nature 2006; 444:110–114.
3. Marsh RA, Madden L, Kitchen BJ, Mody R, McClimon B, Jordan MB, Bleesing JJ, Zhang K, Filipovich AH. XIAP deficiency: A unique primary immunodeficiency best classified as X-linked familial hemophagocytic lymphohistiocytosis and not as X-linked lymphoproliferative disease. Blood 2010; 116:1079–1082.
4. Pachlopnik Schmid J, Canioni D, Moshous D, Touzot F, Mahlaoui N, Hauck F, Kanegane H, Lopez-Granados E, Mejstrikova E, Pellier I, Galicier L, Galambrun C, Barlogis V, Bordigoni P, Fourmaintraux A, Hamidou M, Dabadie A, Le Deist F, Haerynck F, Ouachée-Chardin M, Rohrlich P, Stephan JL, Lenoir C, Rigaud S, Lambert N, Milili M, Schiff C, Chapel H, Picard C, de Saint Basile G, Blanche S, Fischer A, Latour S. Clinical similarities and differences of patients with X-linked lymphoproliferative syndrome type 1 (XLP-1/SAP deficiency) versus type 2 (XLP-2/XIAP deficiency). Blood 2011; 117:1522–1529.
5. Marsh RA, Rao K, Satwani P, Lehmberg K, Müller I, Li D, Kim MO, Fischer A, Latour S, Sedlacek P, Barlogis V, Hamamoto K, Kanegane H, Milanovich S, Margolis DA, Dimmock D, Casper J, Douglas DN, Amrolia PJ, Veys P, Kumar AR, Jordan MB, Bleesing JJ, Filipovich AH. Allogeneic hematopoietic cell transplantation for XIAP deficiency: An international survey reveals poor outcomes. Blood 2013; 121:877–883.
6. Jost PJ, Grabow S, Gray D, McKenzie MD, Nachbur U, Huang DC, Bouillet P, Thomas HE, Borner C, Silke J, Strasser A, Kaufmann T. XIAP discriminates between type I and type II FAS-induced apoptosis. Nature 2009; 460:1035–1039.
7. Cooper N, Rao K, Goulden N, Webb D, Amrolia P, Veys P. The use of reduced-intensity stem cell transplantation in haemophagocytic lymphohistiocytosis and Langerhans cell histiocytosis. Bone Marrow Transplant 2008; 42:S47–S50.
8. Marsh RA, Vaughn G, Kim MO, Li D, Jodele S, Joshi S, Mehta PA, Davies SM, Jordan MB, Bleesing JJ, Filipovich AH. Reduced-intensity conditioning significantly improves survival of patients with hemophagocytic lymphohistiocytosis undergoing allogeneic hematopoietic cell transplantation. Blood 2010; 116:5824–5831.
9. Henter JI, Horne A, Aricó M, Egeler RM, Filipovich AH, Imashuku S, Ladisch S, McClain K, Webb D, Winiarski J, Janka G. HLH- 2004: Diagnostic and therapeutic guidelines for hemophagocytic lymphohistiocytosis. Pediatr Blood Cancer 2007; 48:124–131.
10. Terrell CE, Jordan MB. Perforin deficiency impairs a critical immunoregulatory loop involving murine CD8(+) T cells and dendritic cells. Blood 2013; 121:5184–5191.
11. Yabal M, Müller N, Adler H, Knies N, Groß CJ, Damgaard RB, Kanegane H, Ringelhan M, Kaufmann T, Heikenwälder M, Strasser A, Groß O, Ruland J, Peschel C, Gyrd-Hansen M, Jost PJ. XIAP restricts TNF- and RIP3-dependent cell death and inflammasome activation. Cell Rep 2014; 7:1796–1808.
12. Marsh RA, Kim MO, Liu C, Bellman D, Hart L, Grimley M, Kumar A, Jodele S, Myers KC, Chandra S, Leemhuis T, Mehta PA, Bleesing JJ, Davies SM, Jordan MB, Filipovich AH. An intermediate alemtuzumab schedule reduces the incidence of mixed chimerism following reduced-intensity conditioning hematopoietic cell transplantation for hemophagocytic lymphohistiocytosis. Biol Blood Marrow Transplant 2013; 19:1625–1631.