MHC class II deficiency cured by unrelated mismatched umbilical cord blood transplantation: Case report and review of 68 cases in the literature

Pediatric Transplantation

Volumn 15, Issue 4, 2011, Pages

  Siepermann, M ^a   Gudowius, S ^a,d   Beltz, K ^b   Strier, U ^c   Feyen, O ^a   Troeger, A ^a   Gobel U ^a   Laws, H J ^a   Kogler G ^a   Meisel, R ^a   Dilloo, D ^a   Niehues, T ^b  

^a UNIVERSITY HOSPITAL DÜSSELDORF   (Germany)

^b HEINRICH HEINE UNIVERSITY   (Germany)

^c Department of General Pediatrics   (Germany)

^d Altonaer Kinderkrankenhaus   (Germany)

Author keywords

BLS; cord blood transplantation; hematopoietic stem cell transplantation; immune reconstitution; MHC class II deficiency; severe combined immunodeficiency

Indexed keywords

ANTIBIOTIC AGENT; BUSULFAN; CD3 ANTIGEN; CORTICOSTEROID; CORTISONE; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; FLUDARABINE; GANCICLOVIR; HLA DR ANTIGEN; IMMUNOGLOBULIN; IMMUNOGLOBULIN A; IMMUNOGLOBULIN G; IMMUNOSUPPRESSIVE AGENT; THYMOCYTE ANTIBODY;

ADENOVIRUS INFECTION; ANTIBIOTIC THERAPY; ARTICLE; BACTERIAL GROWTH; BARE LYMPHOCYTE SYNDROME; CASE REPORT; CD4+ T LYMPHOCYTE; CD8+ T LYMPHOCYTE; CHRONIC DIARRHEA; CONSANGUINEOUS MARRIAGE; CORD BLOOD STEM CELL TRANSPLANTATION; CORTICOSTEROID THERAPY; CYTOTOXIC T LYMPHOCYTE; DRUG DOSE REDUCTION; FAILURE TO THRIVE; FAMILY HISTORY; FLOW CYTOMETRY; GENE MUTATION; GRAFT VERSUS HOST REACTION; HELPER CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA TYPING; HOMOZYGOSITY; HUMAN; HUMAN HERPESVIRUS 6; IMMUNOGLOBULIN BLOOD LEVEL; INFANT; INTENSIVE CARE UNIT; LEUKOCYTE COUNT; LYMPHOCYTE COUNT; LYMPHOCYTOPENIA; MALE; MEDICAL HISTORY; MEMORY T LYMPHOCYTE; MYELOABLATIVE CONDITIONING; NATURAL KILLER CELL; NEUTROPHIL COUNT; OUTCOME ASSESSMENT; PNEUMOCYSTIS JIROVECI; PNEUMONIA; POLYMERASE CHAIN REACTION; PSEUDOMONAS AERUGINOSA; RESPIRATORY DISTRESS; RESPIRATORY SYNCYTIAL PNEUMOVIRUS; SEPTICEMIA; STREPTOCOCCUS PNEUMONIAE; THROAT CULTURE; TREATMENT OUTCOME; UMBILICAL CORD BLOOD; UPPER RESPIRATORY TRACT INFECTION; VIRUS DETECTION; VIRUS LOAD;

FETAL BLOOD; FOLLOW-UP STUDIES; GRAFT SURVIVAL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY ANTIGENS CLASS II; HISTOCOMPATIBILITY TESTING; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; INFANT; MALE; RISK ASSESSMENT; SEVERITY OF ILLNESS INDEX; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME;

EID: 79957452283     PISSN: 13973142     EISSN: 13993046     Source Type: Journal    
DOI: 10.1111/j.1399-3046.2010.01292.x     Document Type: Article

References (27)

1. Elhasid R, Etzioni A,. Major histocompatibility complex class II deficiency: A clinical review. Blood Rev 1996: 10: 242-248. (Pubitemid 27042827)
2. Reith W, Mach B,. The bare lymphocyte syndrome and the regulation of MHC expression. Annu Rev Immunol 2001: 19: 331-373. (Pubitemid 32368039)
3. Klein C, Cavazzana-Calvo M, Le Deist F, et al., Major histocompatibility complex class II deficiency: Clinical manifestations, immunologic features, and outcome. J Pediatr 1993: 123: 921-928. (Pubitemid 23353255)
4. Canioni D, Patey N, Cuenod B, Benkerrou M, Brousse N., Major histocompatibility complex class II deficiency needs an early diagnosis: report of a case. Pediatr Pathol Lab Med 1997: 17: 645-651. (Pubitemid 27263941)
5. Myers LA, Patel DD, Puck JM, Buckley RH., Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 2002: 99: 872-878. (Pubitemid 34525548)
6. Saleem MA, Arkwright PD, Davies EG, Cant AJ, Veys PA., Clinical course of patients with major histocompatibility complex class II deficiency. Arch Dis Child 2000: 83: 356-359.
7. Bhattacharya A, Slatter MA, Chapman CE, et al., Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplant 2005: 36: 295-299. (Pubitemid 41195161)
8. Parmar S, Del Lima M, Zou Y, et al., Donor-recipient mismatches in MHC class I chain-related gene A in unrelated donor transplantation lead to increased incidence of acute graft-versus-host disease. Blood 2009: 114: 2884-2887.
9. Godthelp BC, Van Eggermond MC, Peijnenburg A, et al., Incomplete T-cell immune reconstitution in two major histocompatibility complex class II-deficiency/bare lymphocyte syndrome patients after HLA-identical sibling bone marrow transplantation. Blood 1999: 94: 348-358. (Pubitemid 29300168)
10. Renella R, Picard C, Neven B, et al., Human leucocyte antigen-identical haematopoietic stem cell transplantation in major histocompatiblity complex class II immunodeficiency: Reduced survival correlates with an increased incidence of acute graft-versus-host disease and pre-existing viral infections. Br J Haematol 2006: 134: 510-516. (Pubitemid 44141717)
11. Gluckman E, Rocha V,. Cord blood transplantation: state of the art. Haematologica 2009: 94: 451-454.
12. Kurtzberg J, Laughlin M, Graham ML, et al., Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med 1996: 335: 157-166. (Pubitemid 26249452)
13. Knutsen AP, Wall DA,. Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: Two-year experience. J Clin Immunol 2000: 20: 466-476. (Pubitemid 32099712)
14. Niehues T, Rocha V, Filipovich AH, et al., Factors affecting lymphocyte subset reconstitution after either related or unrelated cord blood transplantation in children - a Eurocord analysis. Br J Haematol 2001: 114: 42-48. (Pubitemid 32702631)
15. Kern S, Eichler H, Stoeve J, Klüter H, Bieback K., Comparative analysis of mesenchymal stem cells from bone marrow, umbilical cord blood, or adipose tissue. Stem Cells 2006: 24: 1294-1301. (Pubitemid 44464698)
16. Kleen TO, Kadereit S, Fanning LR, et al., Recipient-specific tolerance after HLA-mismatched umbilical cord blood stem cell transplantation. Transplantation 2005: 80: 1316-1322. (Pubitemid 41746811)
17. Encabo A, Solves P, Carbonell-Uberos F, Miñana MD., The functional immaturity of dendritic cells can be relevant to increased tolerance associated with cord blood transplantation. Transfusion 2007: 47: 272-279. (Pubitemid 46146562)
18. Moretta A, Maccario R, Fagioli F, et al., Analysis of immune reconstitution in children undergoing cord blood transplantation. Exp Hematol 2001: 29: 371-379. (Pubitemid 32220129)
19. Mayani H, Lansdorp PM,. Biology of human umbilical cord blood-derived hematopoietic stem/progenitor cells. Stem Cells 1998: 16: 153-165. (Pubitemid 28235160)
20. Knobloch C, Ballas M, Wölpl A, Friedrich W., Allorecognition and T cell repertoire selection in severe combined immunodeficiency lacking HLA class II antigens. Transplantation 1992: 53: 1295-1301.
21. Van Eggermond MC, Rijkers GT, Kuis W, Zegers BJ, Van Den Elsen PJ. T cell development in a major histocompatibility complex class II-deficient patient. Eur J Immunol 1993: 23: 2585-2591. (Pubitemid 23295143)
22. Gennery AR, Cant AJ,. Cord blood stem cell transplantation in primary immune deficiencies. Curr Opin Allergy Clin Immunol 2007: 7: 528-534. (Pubitemid 350076449)
23. Hanley PJ, Cruz CR, Savoldo B, et al., Functionally active virus-specific T cells that target CMV, adenovirus, and EBV can be expanded from naive T-cell populations in cord blood and will target a range of viral epitopes. Blood 2009: 114: 1958-1967.
24. Fischer A, Landais P, Friedrich W, et al., Bone marrow transplantation (BMT) in Europe for primary immunodeficiencies other than severe combined immunodeficiency: A report from the European Group for BMT and the European Group for Immunodeficiency. Blood 1994: 83: 1149-1154. (Pubitemid 24058568)
25. Klein C, Cavazzana-Calvo M, Le Deist F, et al., Bone marrow transplantation in major histocompatibility complex class II deficiency: a single-center study of 19 patients. Blood 1995: 85: 580-587.
26. Bonduel M, Pozo A, Zelazko M, et al., Successful related umbilical cord blood transplantation for graft failure following T cell-depleted non-identical bone marrow transplantation in a child with major histocompatibility complex class II deficiency. Bone Marrow Transplant 1999: 24: 437-440. (Pubitemid 29403454)
27. Godthelp BC, Van Eggermond MC, Peijnenburg A, et al., T cell immune reconstitution after allogeneic bone marrow transplantation in bare lymphocyte syndrome. Hum Immunol 2000: 61: 898-907.