Treatment of immunodeficiency: Long-term outcome and quality of life

Journal of Allergy and Clinical Immunology

Volumn 122, Issue 6, 2008, Pages 1065-1068

  Shearer, William T ^a   Notarangelo, Luigi D ^b   Griffith, Linda M ^c  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b BOSTON CHILDREN S HOSPITAL   (United States)

^c NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES   (United States)

Author keywords

Hematoietic stem cell transplantation; long term follow up; quality of life; severe combined immunodeficiency

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; FLUDARABINE; GAMMA INTERFERON RECEPTOR 1; I KAPPA B KINASE GAMMA; IMMUNOGLOBULIN; INTERLEUKIN 12 RECEPTOR BETA1; INTERLEUKIN 12P40; LECTIN; METHYLPREDNISOLONE; STAT3 PROTEIN; TRANSCRIPTION FACTOR FOXP3;

AUTOSOMAL RECESSIVE INHERITANCE; CD34 SELECTION; CONSANGUINEOUS MARRIAGE; EDITORIAL; GENE MUTATION; GENETIC HETEROGENEITY; GENETIC SUSCEPTIBILITY; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA MATCHING; HUMAN; HYPERIMMUNOGLOBULINEMIA D AND PERIODIC FEVER SYNDROME; IMMUNE DEFICIENCY; LONG TERM CARE; MYCOBACTERIOSIS; OMENN SYNDROME; OUTCOME ASSESSMENT; PRIORITY JOURNAL; QUALITY OF LIFE; SEVERE COMBINED IMMUNODEFICIENCY; TREATMENT OUTCOME; WISKOTT ALDRICH SYNDROME;

DISEASE-FREE SURVIVAL; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; QUALITY OF LIFE; SURVIVAL RATE; TIME FACTORS;

EID: 57249087374     PISSN: 00916749     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jaci.2008.10.033     Document Type: Editorial

References (38)

1. Buckley R.H. Molecular defects in human severe combined immunodeficiency and approaches to immune reconstitution. Annu Rev Immunol 22 (2004) 625-655
2. Dvorak C.C., and Cowan M.J. Hematopoietic stem cell transplantation for primary immunodeficiency disease. Bone Marrow Transplant 41 (2008) 119-126
3. Filipovich A.H. Hematopoietic cell transplantation for correction of primary immunodeficiencies. Bone Marrow Transplant 42 (2008) S49-S52
4. Titman P., Pink E., Skucek E., O'Hanion K., Cole T.L., Gaspara J., et al. Cognitive and behavioral abnormalities in children following haematopoietic stem cell transplantation for severe congenital immunodeficiencies. Blood 112 (2008) 3907-3913
5. Gatti R.A., Meuwissen H.J., Allen H.D., Hong R., and Good R.A. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 2 (1968) 1366-1369
6. Bach F.H., Albertini R.J., Joo P., Anderson J.L., and Bortin M.M. Bone marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet 2 (1968) 1364-1366
7. Reisner T.M., Kapoor N., Kirkpatrick D., Pollack M.S., Cunningham-Rundles S., Dupont B., et al. Transplantation for severe combined immunodeficiency with HLA-A, B, D, DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells. Blood 61 (1983) 341-348
8. Buckley R.H., Schiff S.E., Schiff R.I., Markert L., Williams L.W., Roberts J.L., et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. New Engl J Med 340 (1999) 508-516
9. Roifman C.M., Somech R., and Grunebaum E. Matched unrelated bone marrow transplant for T + combined immunodeficiency. Bone Marrow Transplant 41 (2008) 947-952
10. Knutsen A.P., and Wall D.A. Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: two-year experience. J Clin Immunol 20 (2000) 466-476
11. Barge R.M.Y., Starrenburg C.W.J., Falkenburg J.H.F., Fibbe W.E., Marijt E.W., and Willemze R. Long-term follow-up of myeloablative allogeneic stem cell transplantation using Campath "in the bag" as T-cell depletion: the Leiden experience. Bone Marrow Transplant 37 (2006) 1129-1134
12. Read E.J., and Carter C.S. T-cell depletion of hematopoietic progenitor cell products for allogeneic transplantation. In: Sacher R.A. (Ed). Cellular therapy: new frontiers in transfusion medicine (2002), American Association of Blood Banks, Bethesda (MD) 29-44
13. Antoine C., Müller S., Cant A., Cavazzana-Calvo M., Veys P., Vossen J., et al. European Group for Blood and Marrow Transplantation, European Society for Immunodeficiency. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet 361 (2003) 553-560
14. Mazzolari E., Forino C., Guerci S., Imperti L., Lanfranchi A., Porta F., et al. Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency. J Allergy Clin Immunol 120 (2007) 892-899
15. Grunebaum E., Mazzolari E., Porta F., Dallera D., Atkinson A., Reid B., et al. Bone marrow transplantation for severe combined immune deficiency. JAMA 295 (2006) 508-518
16. Griffith L.M., Cowan M.J., Kohn D.B., Notarangelo L.D., Puck J.K., Schultz K.R., et al. Allogeneic hematopoietic cell transplantation for primary immune deficiency disease current status and critical needs. J Allergy Clin Immunol 122 (2008) 1087-1096
17. Lin M., Epport K., Azen C., Parkman R., Kohn D.B., and Shah A.J. Long-term neurocognitive function of pediatric patients with severe combined immunodeficiency (SCID): pre- and post-hematopoietic stem cell transplant (HSCT). J Clin Immunol (2008) Sep 20. [Epub ahead of print]
18. Patel N.C., Chinen J., Rosenblatt H.M., Hanson I.C., Brown B.S., Paul M.E., et al. Long-term outcomes of nonconditioned patients with severe combined immunodeficiency transplanted with HLA-identical or haploidentical bone marrow depleted of T cells with anti-CD6 mAb. J Allergy Clin Immunol 122 (2008) 1185-1193
19. Pai S.Y., Levy O., Jabara H.H., Glickman J.N., Sachs J., Nurko S., et al. Allogeneic transplantation successfully corrects immune defects, but not susceptibility to colitis, in a patient with nuclear factor-κB essential modulator deficiency. J Allergy Clin Immunol 122 (2008) 1113-1118
20. Somech R., Kavadas F., Atkinson A., Grunebaum E., and Roifman C.M. High-dose methylprednisolone is effective in the management of acute graft-versus-host disease in severe combined immune deficiency. J Allergy Clin Immunol 122 (2008) 1215-1216
21. Al-Muhsen S., and Casanova J.L. The genetic heterogeneity of mendelian susceptibility to mycobacterial diseases. J Allergy Clin Immunol 122 (2008) 1043-1051
22. Ozcan E., Notarangelo L.D., and Geha R.S. Primary immune deficiencies with aberrant IgE production. J Allergy Clin Immunol 122 (2008) 1054-1062
23. De Ravin S.S., Naumann N., Cowen E.W., Friend J., Hilligoss D., Marquesen M., et al. Chronic granulomatous disease as a risk factor for autoimmune disease. J Allergy Clin Immunol 122 (2008) 1097-1103
24. Villa A., Notarangelo L.D., and Roifman C.M. Omenn syndrome: inflammation in leaky severe combined immunodeficiency. J Allergy Clin Immunol 122 (2008) 1082-1086
25. Grunebaum E., Bates A., and Roifman C.M. Omenn syndrome is associated with mutations in DNA ligase IV. J Allergy Clin Immunol 122 (2008) 1219-1220
26. Notarangelo L.D., and Sorensen R. Is it necessary to identify molecular defects in primary immunodeficiency disease?. J Allergy Clin Immunol 122 (2008) 1069-1073
27. Davis C., Kancherla V.S., Reddy A., Yeh H.W., Noroski L.M., Rosenblatt H., et al. Development of specific T-cell responses to Candida and tetanus antigens in partial DiGeorge syndrome. J Allergy Clin Immunol 122 (2008) 1194-1199
28. Gambineri E., Perroni L., Passerini L., Bianchi L., Doglioni C., Meschi F., 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 122 (2008) 1105-1112
29. Hanson E.P., Monaco-Shawver L., Solt L.A., Madge L.A., Banerjee P.P., May M.J., et al. Hypomorphic nuclear factor-κB essential modulator mutation database and reconstitution system identifies phenotypic and immunologic diversity. J Allergy Clin Immunol 122 (2008) 1169-1177
30. Kavadas F.D., Giliani S., Gu Y., Mazzolari E., Bates A., Pegoiani E., et al. Variability of clinical and laboratory features among patients with ribonuclease mitochondrial RNA processing endoribonuclease gene mutations. J Allergy Clin Immunol 122 (2008) 1178-1184
31. Nahum A., Bates A., Sharfe N., and Roifman C.M. Association of the lymphoid tyrosine phosphatase, R620W variant, with chronic mucocutaneous candidiasis. J Allergy Clin Immunol 122 (2008) 1220-1222
32. Ehlayel M., de Beaucoudrey L., Fike F., Nahas S.A., Feinberg J., Casanova J.L., et al. Simultaneous presentation of 2 rare hereditary immunodeficiencies: IL-12 receptor β1 deficiency and ataxia-telangiectasia. J Allergy Clin Immunol 122 (2008) 1217-1219
33. Sheehan W.J., Delmonte O.M., Miller D.T., Roberts A.E., Bonilla F.A., Morra M., et al. Novel presentation of Omenn syndrome in association with aniridia. J Allergy Clin Immunol (2008) In press
34. Syllabe L., and Henner K. Contribution a l'independance de l'athetose double idiopathique et congenitale: atteinte familiale, syndrome dystrophique, signe du reseau vasculaire conjonctival, integrite psychique. Rev Neurol (Paris) 1 (1926) 541-562
35. Shearer W.T., Cunningham-Rundles C., and Ochs H.D. Editorial: primary immunodeficiency: looking backwards, looking forwards. J Allergy Clin Immunol 113 (2004) 607-609
36. Shearer W.T. Editorial. Importance of technology for the future of allergy and immunology. J Allergy Clin Immunol 114 (2004) 406-408
37. Shearer W.T. Editorial: the lst 80 years in primary immunodeficiency: how far have we come, have far need we go?. J Allergy Clin Immunol 117 (2006) 748-752
38. Shearer W.T., Malech H., and Puck J.M. Editorial: primary immunodeficiencies: meeting the challenges. J Allergy Clin Immunol 120 (2007) 753-755