Outcome of unrelated umbilical cord blood transplantation in 88 patients with primary immunodeficiency in Japan

British Journal of Haematology

Volumn 154, Issue 3, 2011, Pages 363-372

  Morio, Tomohiro ^a   Atsuta, Yoshiko ^b   Tomizawa, Daisuke ^a   Nagamura Inoue, Tokiko ^c   Kato, Koji ^d   Ariga, Tadashi ^e   Kawa, Keisei ^f   Koike, Kazutoshi ^g   Tauchi, Hisamichi ^h   Kajiwara, Michiko ⁱ   Hara, Toshiro ^j   Kato, Shunichi ^k  

^a TOKYO MEDICAL AND DENTAL UNIVERSITY   (Japan)

^b NAGOYA UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^c UNIVERSITY OF TOKYO   (Japan)

^d JAPANESE RED CROSS NAGOYA FIRST HOSPITAL   (Japan)

^e HOKKAIDO UNIVERSITY GRADUATE SCHOOL OF MEDICINE   (Japan)

^f RESEARCH INSTITUTE FOR MATERNAL AND CHILD HEALTH   (Japan)

^g Ibaraki Children's Hospital   (Japan)

^h EHIME UNIVERSITY SCHOOL OF MEDICINE   (Japan)

ⁱ TOKYO MEDICAL AND DENTAL UNIVERSITY   (Japan)

^j KYUSHU UNIVERSITY   (Japan)

^k TOKAI UNIVERSITY SCHOOL OF MEDICINE   (Japan)

more..

Author keywords

Cord blood transplantation; Primary immunodeficiency; Reduced intensity conditioning; Severe combined immunodeficiency; Wiskott Aldrich syndrome

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; CYTARABINE; ETOPOSIDE; FLUDARABINE; LEUKOCYTE ANTIGEN; MAJOR HISTOCOMPATIBILITY ANTIGEN CLASS 2; MELPHALAN; TACROLIMUS; THYMOCYTE ANTIBODY;

ADOLESCENT; ADULT; ARTICLE; BACTERIAL INFECTION; CAUSE OF DEATH; CHILD; CHRONIC GRANULOMATOUS DISEASE; CORD BLOOD STEM CELL TRANSPLANTATION; DRUG MEGADOSE; ENGRAFTMENT; GRAFT FAILURE; GRAFT VERSUS HOST REACTION; HUMAN; IMMUNE DEFICIENCY; JAPAN; MAJOR CLINICAL STUDY; MORTALITY; MYCOSIS; MYELOABLATIVE CONDITIONING; NEUTROPHIL; OVERALL SURVIVAL; PRESCHOOL CHILD; PRIORITY JOURNAL; PROGNOSIS; REDUCED INTENSITY CONDITIONING; RETICULOCYTE COUNT; RISK FACTOR; SCHOOL CHILD; SEVERE COMBINED IMMUNODEFICIENCY; SEVERE CONGENITAL NEUTROPENIA; THROMBOCYTE COUNT; UMBILICAL CORD BLOOD; VIRUS INFECTION; WHOLE BODY RADIATION; WISKOTT ALDRICH SYNDROME;

ADOLESCENT; CHILD; CHILD, PRESCHOOL; CORD BLOOD STEM CELL TRANSPLANTATION; EPIDEMIOLOGIC METHODS; GRAFT SURVIVAL; GRAFT VS HOST DISEASE; HISTOCOMPATIBILITY; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; INFANT; INFANT, NEWBORN; OPPORTUNISTIC INFECTIONS; SEVERE COMBINED IMMUNODEFICIENCY; TRANSPLANTATION CONDITIONING; TREATMENT OUTCOME; WISKOTT-ALDRICH SYNDROME; YOUNG ADULT;

EID: 79960204921     PISSN: 00071048     EISSN: 13652141     Source Type: Journal    
DOI: 10.1111/j.1365-2141.2011.08735.x     Document Type: Article

References (39)

1. Antoine, C., Muller, S., Cant, A., Cavazzana-Calvo, M., Veys, P., Vossen, J., Fasth, A., Heilmann, C., Wulffraat, N., Seger, R., Blanche, S., Friedrich, W., Abinun, M., Davies, G., Bredius, R., Schulz, A., Landais, P. & Fischer, A.; European Group for Blood and Marrow Transplanation; European Society for Immunodeficiency (2003) Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. [see comment]. Lancet, 361, 553-560.
2. Atsuta, Y., Suzuki, R., Nagamura-Inoue, T., Taniguchi, S., Takahashi, S., Kai, S., Sakamaki, H., Kouzai, Y., Kasai, M., Fukuda, T., Azuma, H., Takanashi, M., Okamoto, S., Tsuchida, M., Kawa, K., Morishima, Y., Kodera, Y. & Kato, S.; Japan Cord Blood Bank Network (2009) Disease-specific analyses of unrelated cord blood transplantation compared with unrelated bone marrow transplantation in adult patients with acute leukemia. Blood, 113, 1631-1638.
3. Bhattacharya, A., Slatter, M., Curtis, A., Chapman, C.E., Barge, D., Jackson, A., Flood, T.J., Abinun, M., Cant, A.J. & Gennery, A.R. (2003) Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplantation, 31, 403-405.
4. Bhattacharya, A., Slatter, M.A., Chapman, C.E., Barge, D., Jackson, A., Flood, T.J., Abinun, M., Cant, A.J. & Gennery, A.R. (2005) Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplantation, 36, 295-299.
5. Brown, J.A. & Boussiotis, V.A. (2008) Umbilical cord blood transplantation: basic biology and clinical challenges to immune reconstitution. Clinical Immunology, 127, 286-297.
6. Cuvelier, G.D.E., Schultz, K.R., Davis, J., Hirschfeld, A.F., Junker, A.K., Tan, R. & Turvey, S.E. (2009) Optimizing outcomes of hematopoietic stem cell transplantation for severe combined immunodeficiency. Clinical Immunology, 131, 179-188.
7. Dvorak, C.C. & Cowan, M.J. (2008) Hematopoietic stem cell transplantation for primary immunodeficiency disease. Bone Marrow Transplantation, 41, 119-126.
8. Fagioli, F., Biasin, E., Berger, M., Nesi, F., Saroglia, E.H., Miniero, R., Martino, S. & Tovo, P.A. (2003) Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome. Bone Marrow Transplantation, 31, 133-136.
9. Friedrich, W., Schutz, C., Schulz, A., Benninghoff, U. & Honig, M. (2009) Results and long-term outcome in 39 patients with Wiskott-Aldrich syndrome transplanted from HLA-matched and -mismatched donors. Immunologic Research, 44, 18-24.
10. Gluckman, E., Rocha, V., Boyer-Chammard, A., Locatelli, F., Arcese, W., Pasquini, R., Ortega, J., Souillet, G., Ferreira, E., Laporte, J.P., Fernandez, M. & Chastang, C. (1997) Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. [see comment]. New England Journal of Medicine, 337, 373-381.
11. Gooley, T.A., Leisenring, W., Crowley, J. & Storer, B.E. (1999) Estimation of failure probabilities in the presence of competing risks: new representations of old estimators. Statistics in Medicine, 18, 695-706.
12. Gray, R. (1988) A class of k-sample tests for comparing the cumulative incidence of a competing risk. Annals of Statistics, 16, 1141-1154.
13. Griffith, L.M., Cowan, M.J., Kohn, D.B., Notarangelo, L.D., Puck, J.M., Schultz, K.R., Buckley, R.H., Eapen, M., Kamani, N.R., O'Reilly, R.J., Parkman, R., Roifman, C.M., Sullivan, K.E., Filipovich, A.H., Fleisher, T.A. & Shearer, W.T. (2008) Allogeneic hematopoietic cell transplantation for primary immune deficiency diseases: current status and critical needs. Journal of Allergy & Clinical Immunology, 122, 1087-1096.
14. Knutsen, A.P. & Wall, D.A. (2000) Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: two-year experience. Journal of Clinical Immunology, 20, 466-476.
15. Knutsen, A.P., Steffen, M., Wassmer, K. & Wall, D.A. (2003) Umbilical cord blood transplantation in Wiskott Aldrich syndrome. Journal of Pediatrics, 142, 519-523.
16. Kobayashi, R., Ariga, T., Nonoyama, S., Kanegane, H., Tsuchiya, S., Morio, T., Yabe, H., Nagatoshi, Y., Kawa, K., Tabuchi, K., Tsuchida, M., Miyawaki, T. & Kato, S. (2006) Outcome in patients with Wiskott-Aldrich syndrome following stem cell transplantation: an analysis of 57 patients in Japan. British Journal of Haematology, 135, 362-366.
17. Kurtzberg, J., Laughlin, M., Graham, M.L., Smith, C., Olson, J.F., Halperin, E.C., Ciocci, G., Carrier, C., Stevens, C.E. & Rubinstein, P. (1996) Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. [see comment]. New England Journal of Medicine, 335, 157-166.
18. Kurtzberg, J., Prasad, V.K., Carter, S.L., Wagner, J.E., Baxter-Lowe, L.A., Wall, D., Kapoor, N., Guinan, E.C., Feig, S.A., Wagner, E.L. & Kernan, N.A. (2008) Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies. Blood, 112, 4318-4327.
19. Laughlin, M.J., Eapen, M., Rubinstein, P., Wagner, J.E., Zhang, M.-J., Champlin, R.E., Stevens, C., Barker, J.N., Gale, R.P., Lazarus, H.M., Marks, D.I., van Rood, J.J., Scaradavou, A. & Horowitz, M.M. (2004) Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia. [see comment]. New England Journal of Medicine, 351, 2265-2275.
20. Mazzolari, E., Forino, C., Guerci, S., Imberti, L., Lanfranchi, A., Porta, F. & Notarangelo, L.D. (2007) Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency. Journal of Allergy & Clinical Immunology, 120, 892-899.
21. McGhee, S.A., Stiehm, E.R., Cowan, M., Krogstad, P. & McCabe, E.R.B. (2005) Two-tiered universal newborn screening strategy for severe combined immunodeficiency. Molecular Genetics & Metabolism, 86, 427-430.
22. Michel, G., Rocha, V., Chevret, S., Arcese, W., Chan, K.W., Filipovich, A., Takahashi, T.A., Vowels, M., Ortega, J., Bordigoni, P., Shaw, P.J., Yaniv, I., Machado, A., Pimentel, P., Fagioli, F., Verdeguer, A., Jouet, J.P., Diez, B., Ferreira, E., Pasquini, R., Rosenthal, J., Sievers, E., Messina, C., Iori, A.P., Garnier, F., Ionescu, I., Locatelli, F., Gluckman, E. & Eurocord Group (2003) Unrelated cord blood transplantation for childhood acute myeloid leukemia: a Eurocord Group analysis. Blood, 102, 4290-4297.
23. Morinishi, Y., Imai, K., Nakagawa, N., Sato, H., Horiuchi, K., Ohtsuka, Y., Kaneda, Y., Taga, T., Hisakawa, H., Miyaji, R., Endo, M., Oh-Ishi, T., Kamachi, Y., Akahane, K., Kobayashi, C., Tsuchida, M., Morio, T., Sasahara, Y., Kumaki, S., Ishigaki, K., Yoshida, M., Urabe, T., Kobayashi, N., Okimoto, Y., Reichenbach, J., Hashii, Y., Tsuji, Y., Kogawa, K., Yamaguchi, S., Kanegane, H., Miyawaki, T., Yamada, M., Ariga, T. & Nonoyama, S. (2009) Identification of severe combined immunodeficiency by T-cell receptor excision circles quantification using neonatal guthrie cards. Journal of Pediatrics, 155, 829-833.
24. Neven, B., Leroy, S., Decaluwe, H., Le Deist, F., Picard, C., Moshous, D., Mahlaoui, N., Debre, M., Casanova, J.-L., Dal Cortivo, L., Madec, Y., Hacein-Bey-Abina, S., de Saint Basile, G., de Villartay, J.-P., Blanche, S., Cavazzana-Calvo, M. & Fischer, A. (2009) Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency. Blood, 113, 4114-4124.
25. Ozsahin, H., Cavazzana-Calvo, M., Notarangelo, L.D., Schulz, A., Thrasher, A.J., Mazzolari, E., Slatter, M.A., Le Deist, F., Blanche, S., Veys, P., Fasth, A., Bredius, R., Sedlacek, P., Wulffraat, N., Ortega, J., Heilmann, C., O'Meara, A., Wachowiak, J., Kalwak, K., Matthes-Martin, S., Gungor, T., Ikinciogullari, A., Landais, P., Cant, A.J., Friedrich, W. & Fischer, A. (2008) 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, 111, 439-445.
26. Przepiorka, D., Weisdorf, D., Martin, P., Klingemann, H.G., Beatty, P., Hows, J. & Thomas, E.D. (1995) 1994 consensus conference on acute GVHD grading. Bone Marrow Transplantation, 15, 825-828.
27. Rao, K., Amrolia, P.J., Jones, A., Cale, C.M., Naik, P., King, D., Davies, G.E., Gaspar, H.B. & Veys, P.A. (2005) Improved survival after unrelated donor bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen. Blood, 105, 879-885.
28. Rocha, V. & Gluckman, E.; Eurocord and European Blood and Marrow Transplant Group. (2006) Clinical use of umbilical cord blood hematopoietic stem cells. Biology of Blood and Marrow Transplantation, 12, 34-41.
29. Rocha, V., Wagner, Jr, J.E., Sobocinski, K.A., Klein, J.P., Zhang, M.J., Horowitz, M.M. & Gluckman, E. (2000) Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources. New England Journal of Medicine, 342, 1846-1854.
30. Rocha, V., Labopin, M., Sanz, G., Arcese, W., Schwerdtfeger, R., Bosi, A., Jacobsen, N., Ruutu, T., de Lima, M., Finke, J., Frassoni, F. & Gluckman, E.; Acute Leukemia Working Party of European Blood Marrow Transplant, Group; Eurocord-Netcord Registry (2004) Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia. [see comment]. New England Journal of Medicine, 351, 2276-2285.
31. Rubinstein, P., Carrier, C., Scaradavou, A., Kurtzberg, J., Adamson, J., Migliaccio, A.R., Berkowitz, R.L., Cabbad, M., Dobrila, N.L., Taylor, P.E., Rosenfield, R.E. & Stevens, C.E. (1998) Outcomes among 562 recipients of placental-blood transplants from unrelated donors. [see comment]. New England Journal of Medicine, 339, 1565-1577.
32. Sakata, N., Kawa, K., Kato, K., Yabe, H., Yabe, M., Nagasawa, M., Mugishima, H., Kigasawa, H., Tsuchida, M., Akiyama, Y., Morisima, Y., Kodera, Y. & Kato, S. (2004) Unrelated donor marrow transplantation for congenital immunodeficiency and metabolic disease: an update of the experience of the Japan Marrow Donor Program. International Journal of Hematology, 80, 174-182.
33. Sawczyn, K.K., Quinones, R., Malcolm, J., Foreman, N., Garrington, T., Gore, L., Gao, D. & Giller, R. (2005) Cord blood transplant in childhood ALL. [see comment]. Pediatric Blood & Cancer, 45, 964-970.
34. Szabolcs, P. & Niedzwiecki, D. (2008) Immune reconstitution in children after unrelated cord blood transplantation. Biology of Blood and Marrow Transplantation, 14, 66-72.
35. Thomson, B.G., Robertson, K.A., Gowan, D., Heilman, D., Broxmeyer, H.E., Emanuel, D., Kotylo, P., Brahmi, Z. & Smith, F.O. (2000) Analysis of engraftment, graft-versus-host disease, and immune recovery following unrelated donor cord blood transplantation. Blood, 96, 2703-2711.
36. Tomizawa, D., Yuki, A., Nagasawa, M., Morio, T., Kajiwara, M., Sekine, T., Shimizu, N., Kato, M., Yachie, A. & Mizutani, S. (2005) Novel adopted immunotherapy for mixed chimerism after unrelated cord blood transplantation in Omenn syndrome. European Journal of Haematology, 75, 441-444.
37. Tono, C., Takahashi, Y., Terui, K., Sasaki, S., Kamio, T., Tandai, S., Sato, T., Kudo, K., Toki, T., Tachibana, N., Yoshioka, T., Nakahata, T., Morio, T., Nishikomori, R. & Ito, E. (2007) Correction of immunodeficiency associated with NEMO mutation by umbilical cord blood transplantation using a reduced-intensity conditioning regimen. Bone Marrow Transplantation, 39, 801-804.
38. Wagner, J.E., Rosenthal, J., Sweetman, R., Shu, X.O., Davies, S.M., Ramsay, N.K., McGlave, P.B., Sender, L. & Cairo, M.S. (1996) Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease. Blood, 88, 795-802.
39. Zeidler, C., Welte, K., Barak, Y., Barriga, F., Bolyard, A.A., Boxer, L., Cornu, G., Cowan, M.J., Dale, D.C., Flood, T., Freedman, M., Gadner, H., Mandel, H., O'Reilly, R.J., Ramenghi, U., Reiter, A., Skinner, R., Vermylen, C. & Levine, J.E. (2000) Stem cell transplantation in patients with severe congenital neutropenia without evidence of leukemic transformation. Blood, 95, 1195-1198.