Selective expansion of donor-derived regulatory T cells after allogeneic bone marrow transplantation in a patient with IPEX syndrome

Pediatric Transplantation

Volumn 18, Issue 1, 2014, Pages

  Horino, Satoshi ^a,b   Sasahara, Yoji ^a   Sato, Miki ^a   Niizuma, Hidetaka ^a   Kumaki, Satoru ^a   Abukawa, Daiki ^b   Sato, Atsushi ^b   Imaizumi, Masue ^b   Kanegane, Hirokazu ^c   Kamachi, Yoshiro ^d   Sasaki, Shinya ^e   Terui, Kiminori ^e   Ito, Etsuro ^e   Kobayashi, Ichiro ^f   Ariga, Tadashi ^f   Tsuchiya, Shigeru ^a   Kure, Shigeo ^a  

^a TOHOKU UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^b MIYAGI CHILDREN S HOSPITAL   (Japan)

^c UNIVERSITY OF TOYAMA   (Japan)

^d NAGOYA UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^e HIROSAKI UNIVERSITY   (Japan)

^f HOKKAIDO UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

Author keywords

allogeneic hematopoietic stem cell transplantation; enteropathy; Forkhead box protein 3; immune dysregulation; polyendocrinopathy; reduced intensity conditioning; regulatory T cells; X linked syndrome

Indexed keywords

AUTOANTIBODY; BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; IMMUNOSUPPRESSIVE AGENT; MELPHALAN; METHOTREXATE; PREDNISOLONE; TRANSCRIPTION FACTOR FOXP3;

ACUTE LYMPHOBLASTIC LEUKEMIA; ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; BLOODY DIARRHEA; CASE REPORT; CD4+ CD25+ T LYMPHOCYTE; CELL EXPANSION; CHILD; CHIMERA; COLITIS; DIARRHEA; DONOR; DRUG DOSE INCREASE; GASTROINTESTINAL SYMPTOM; GRAFT VERSUS HOST REACTION; HLA MATCHING; HUMAN; HUMAN CELL; IMMUNOSUPPRESSIVE TREATMENT; INFANT; IPEX SYNDROME; LOW DRUG DOSE; MALE; MISSENSE MUTATION; MYELOABLATIVE CONDITIONING; PRESCHOOL CHILD; REDUCED INTENSITY CONDITIONING; REGULATORY T LYMPHOCYTE; REMISSION; SIBLING; THROMBOCYTE TRANSFUSION; WHOLE BODY RADIATION;

ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ENTEROPATHY; FORKHEAD BOX PROTEIN 3; IMMUNE DYSREGULATION; POLYENDOCRINOPATHY; REDUCED INTENSITY CONDITIONING; REGULATORY T CELLS; X-LINKED SYNDROME;

BONE MARROW TRANSPLANTATION; CHILD; GASTROINTESTINAL DISEASES; GENETIC DISEASES, X-LINKED; HUMANS; MALE; T-LYMPHOCYTES, REGULATORY; TRANSPLANTATION, HOMOLOGOUS;

EID: 84891830230     PISSN: 13973142     EISSN: 13993046     Source Type: Journal    
DOI: 10.1111/petr.12184     Document Type: Article

References (19)

1. Powell BR, Bruist NR, Stenzel P,. An X-linked syndrome of diarrhea, polyendocrinopathy, and fatal infection in infancy. J Pediatr 1982: 100: 731-737.
2. Wildin RS, Smyk-Pearson S, Filipovich AH,. Clinical and molecular features of the immunodysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome. J Med Genet 2002: 39: 537-545.
3. Taddio A, Faleschini E, Valencic E, et al. Medium-term survival without hematopoietic stem cell transplantation in a case of IPEX: Insights into nutritional and immunosuppressive therapy. Eur J Pediatr 2007: 166: 1195-1197.
4. Gambineri E, Perroni L, Passerini L, et al. Clinical and molecular profile of a new series of patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome: Inconsistent correlation between forkhead box protein 3 expression and disease severity. J Allergy Clin Immunol 2008: 122: 1105-1112.
5. Baud O, Goulet O, Canioni D, et al. Treatment of the immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome (IPEX) by allogeneic bone marrow transplantation. N Engl J Med 2001: 344: 1758-1762.
6. Mazzolari E, Forino C, Fontana M, et al. A new case of IPEX receiving bone marrow transplantation. Bone Marrow Transplant 2005: 35: 1033-1034.
7. Rao A, Kamani N, Filopovich A, et al. Successful bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Blood 2007: 109: 383-385.
8. Lucas KG, Ungar D, Comito M, et al. Submyeloablative cord blood transplantation corrects clinical defects seen in IPEX syndrome. Bone Marrow Transplant 2007: 39: 55-56.
9. Dorsey MJ, Petrovic A, Morrow MR, et al. FOXP3 expression following bone marrow transplantation for IPEX syndrome after reduced-intensity conditioning. Immunol Res 2009: 44: 179-184.
10. Zhan H, Sinclair J, Adams S, et al. Immune reconstitution and recovery of FOXP3 (forkhead box protein 3)-expressing T cells after transplantation for IPEX (immune dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome. Pediatrics 2008: 121: 998-1002.
11. high FOXP3-positive T cells in IPEX syndrome after nonmyeloablative hematopoietic stem cell transplantation. Blood 2009: 113: 5689-5691.
12. Burroughs LM, Torgerson TR, Storb R, et al. Stable hematopoietic cell engraftment after low-intensity nonmyeloablative conditioning in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome. J Allergy Clin Immunol 2010: 126: 1000-1005.
13. Tanaka H, Suzuki K, Nakahata T, et al. Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome. Acta Paediatr 2004: 93: 142-143.
14. Tanaka H, Tsugawa K, Kudo M, et al. Low-dose cyclosporine A in a patient with X-linked immune dysregulation, polyendocrinopathy and enteropathy. Eur J Pediatr 2005: 164: 779-780.
15. Thiede C, Florek M, Bornhäuser M, et al. Rapid quantification of mixed chimerism using multiplex amplification of short tandem repeat markers and fluorescence detection. Bone Marrow Transplant 1999: 23: 1055-1060.
16. low) cells in patients with immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome. Clin Immunol 2011: 141: 111-120.
17. Kobayashi I, Kubota M, Yamada M, et al. Autoantibodies to villin occur frequently in IPEX, a severe immune dysregulation, syndrome caused by mutation of FOXP3. Clin Immunol 2011: 141: 83-89.
18. Smyk-Pearson SK, Bakke AC, Held PK, et al. Rescue of the autoimmune scurfy mouse by partial bone marrow transplantation or injection with T-enriched splenocytes. Clin Exp Immunol 2003: 133: 193-199.
19. Ozsahin H, Cavazzana-Calvo M, Notarangelo LD, et al. Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: Collaborative study of the European Society for Immunodeficiencies and European Group for Blood and Marrow Transplantation. Blood 2008: 111: 439-445. Technology