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M Ouachee-Chardin, C Elie, G. De Saint Basile, F Le Deist et al. Hematopoietic stem cell transplantation in hemophagocytic lymphohistiocytosis (HLH) : a single center report of 48 patients. Pediatrics 2006 Apr; 117(4): e743-750.

Schmid JM, Junge SA, Hossle JP, Schneider EM, Roosnek E, Seger RA, Gungor T. Transient hemophagocytosis with deficient cellular cytotoxicity, monoclonal immunoglobulin M gammopathy, increased T-cell numbers, and hypomorphic NEMO mutation. Pediatrics May 2006;117;1049-1056

Heiko von Goessel, Johann Peter Hossle, Reinhard Seger and Tayfun Gungor. Characterization of 17 new cases of X-linked chronic granulomatous disease with seven novel mutations in the CYBB gene. Experimental Hematology 2006;528-535

Y. Chalandon, J-M Tiercy, U. Schanz, T. Gungor, R. Seger, J. Halter, C. Helg, B. Chapuis, A. Gratwohl, A. Tichelli, G. Nicoloso de Faveri, E. Roosnek and JR Passweg, on behalf of the Swiss Transplant Working Group for Blood and Marrow Transplantation (STABMT) and the Swiss National Donor Registry. Impact of high-resolution matching in allogeneic-unrelated donor stem cell transplantation in Switzerland. Bone Marrow Transplantation 2006;37(10):909-16.

Formankova R, Sedlacek P, Keslova P, Sramkova L, Vavra V, Sediva A, Bartunkova J, Hrusak O, Spisek R, Dlask K, Litzman J, Parizkova E, Stary J Haematopoietic stem cell transplantation for primary immunodeficiencies -a 10-year single-centre experience. Suppl. BMT; 2005; 00:35:22

Wulffraat NM, Vastert B, Tyndall A. Treatment of refractory autoimmune diseases with autologous stem cell transplantation: focus on juvenile idiopathic arthritis. Bone Marrow Transplant. 2005 Mar;35 Suppl 1:S27-9.

Almousa H, Ouachee-Chardin M, Picard C, Radford-Weiss I, Caillat-Zucman S, Cavazzana-Calvo M, Blanche S, de Saint Basile G, Le Deist F, Fischer A. Transient familial haemophagocytic lymphohistiocytosis reactivation post-CD34 haematopoietic stem cell transplantation. Br J Haematol. 2005 Aug;130(3):404-8.

Ferry C, Ouachee M, Leblanc T, Michel G, Notz-Carrere A, Tabrizi R, Flood T, Lutz P, Fischer A, Gluckman E, Donadieu J. Hematopoietic stem cell transplantation in severe congenital neutropenia: experience of the French SCN register. Bone Marrow Transplant. 2005 Jan;35(1):45-50.

Dawson C, Slatter MA, Gennery AR. In utero transplantation: baby steps towards an effective therapy. Bone Marrow Transplant. 2005 Sep;36(6):563-4

Gallimore CI, Taylor C, Gennery AR, Cant AJ, Galloway A, Lewis D, Gray JJ. Use of a heminested reverse transcriptase PCR assay for detection of astrovirus in environmental swabs from an outbreak of gastroenteritis in a pediatric primary immunodeficiency unit. J Clin Microbiol. 2005 Aug;43(8):3890-4.

Gennery AR, Hodges E, Williams AP, Harris S, Villa A, Angus B, Cant AJ, Smith JL. Omenn's syndrome occurring in patients without mutations in recombination activating genes. Clin Immunol. 2005 Sep;116(3):246-56.

Bhattacharya A, Slatter MA, Chapman CE, Barge D, Jackson A, Flood TJ, Abinun M, Cant AJ, Gennery AR. Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplant. 2005 Aug;36(4):295-9.

Slatter MA, Bhattacharya A, Flood TJ, Abinun M, Cant AJ, Gennery AR. Use of two unrelated umbilical cord stem cell units in stem cell transplantation for Wiskott-Aldrich syndrome. Pediatr Blood Cancer. 2005 Jul 8 Epub

Ege M, Ma Y, Manfras B, Kalwak K, Lu H, Lieber MR, Schwarz K, Pannicke U. Omenn syndrome due to ARTEMIS mutations. Blood. 2005 Jun 1;105(11):4179-86.

Gratwohl A, Passweg J, Bocelli-Tyndall C, Fassas A, van Laar JM, Farge D, Andolina M, Arnold R, Carreras E, Finke J, Kotter I, Kozak T, Lisukov I, Lowenberg B, Marmont A, Moore J, Saccardi R, Snowden JA, van den Hoogen F, Wulffraat NM, Zhao XW, Tyndall A; Autologous hematopoietic stem cell transplantation for autoimmune diseases. Bone Marrow Transplant. 2005 May;35(9):869-79.

Slatter MA, Bhattacharya A, Abinun M, Flood TJ, Cant AJ, Gennery AR. Outcome of boost haemopoietic stem cell transplant for decreased donor chimerism or graft dysfunction in primary immunodeficiency. Bone Marrow Transplant. 2005 Apr;35(7):683-9.

Gennery AR, Slatter MA, Bhattacharya A, Jeggo PA, Abinun M, Flood TJ, Cant AJ. Bone marrow transplantation for Nijmegan breakage syndrome. J Pediatr Hematol Oncol. 2005 Apr;27(4):239.

Slatter MA, Read S, Taylor CE, Crooks BN, Abinun M, Flood TJ, Cant AJ, Wright C, Gennery AR. Adenovirus type F subtype 41 causing disseminated disease following bone marrow transplantation for immunodeficiency. J Clin Microbiol. 2005 Mar;43(3):1462-4.

Grewal SS, Wynn R, Abdenur JE, Burton BK, Gharib M, Haase C, Hayashi RJ, Shenoy S, Sillence D, Tiller GE, Dudek ME, van Royen-Kerkhof A, Wraith JE, Woodard P, Young GA, Wulffraat N, Whitley CB, Peters C. Safety and efficacy of enzyme replacement therapy in combination with hematopoietic stem cell transplantation in Hurler syndrome. Genet Med. 2005 Feb;7(2):143-6.

Gungor T, Halter J, Klink A, Junge S, Stumpe KD, Seger R, Schanz U. Successful low toxicity hematopoietic stem cell transplantation for high-risk adult chronic granulomatous disease patients. Transplantation. 2005 Jun 15;79(11):1596-606

Cesaro S, Oneto R, Messina C, Gibson BE, Buzyn A, Steward C, Gluckman E, Bredius R, Boogaerts M, Vermylen C, Veys P, Marsh J, Badell I, Michel G, Gungor T, Niethammer D, Bordigoni P, Oswald C, Favre C, Passweg J, Dini G; EBMT Severe Aplastic Anaemia and Paediatric Diseases Working Party. Haematopoietic stem cell transplantation for Shwachman-Diamond disease: a study from the European Group for blood and marrow transplantation. Br J Haematol. 2005 Oct;131(2):231-6

Kanchan Rao, Persis J Amrolia, Alison Jones, Catherine M Cale, Paru Naik, Doug King, Graham E. Davies, H B Gaspar, and Paul A. Veys. Improved survival after unrelated donor bone marrow transplant in children with primary immunodeficiency using a redused intensity conditioning regimen. Blood. 2005 Jan 15;105(2):879-85. Epub 2004 Sep 14.

L. Dal-Cortivo, A. Fischer, M. Cavazzana-Calvo, S. Caillat-Zucman. Does haploidentical transplantation in children with primary immunodeficiencies have the potential to exploit donor NK cell alloreactivity? Bone Marrow Transplantation 2004, doi:10.1038/sj.bmt.1704663, 1-3

Bredius R.G.M., K. Templeton, S. Scheltinga, E. Claas, A. Kroes, J.M. Vossen. Prospective study of respiratory viral infections in pediatric hemopoietic stem cell transplantation patients. Pediatric Infectious Dis J. 2004 Jun;23(6):518-22

S. Caillat-Zucman, Elie Haddad, F. Le Deist, M. Gannagé, N. Jabado, S. Hacein-Bey, S. Blanche, J-L Casanova, A. Fischer and M. Cavazzana-Calvo. Impact of HLA matching on outcome after hematopoietic stem cell transplantation study in children with inherited diseases : a single center comparative analysis of genoidentical, haploidentical or unrelated donors. Bone Marrow Transplant. 2004 Jun;33(11):1089-95

De Kleer IM, Brinkman DMC, Ferster A, Abinun M, Quartier P, van der Net JJ, ten Cate R, Wedderburn L, Horneff G, Oppermann J, Zintl F, Foster HE, Prieur AM, Fasth A, van Rossum MAJ, Kuis W, Wulffraat NM. Autologous stem cell transplantation for refractory juvenile idiopathic arthritis: analysis of clinical effects, mortality and transplant related morbidity. Annals of the Rheumatic Diseases Oct. 2004;63:1318-1326

Gennery AR, Khawaja K, Veys P, Bredius RGM, Notarangelo LD, Mazzolari E, Fischer A, Landais P, Cavazzana-Calvo M, Friedrich W, Fasth A, Wulffraat NM, Matthes-Martin S, Bensoussan D, Bordigoni P, Lange A, Pagliuca A, Andolina M, Cant AJ, Davies EG. Treatment of CD40 ligand deficiency by haematopoietic stem cell transplantation : a survey of the European experience, 1993-2002. Blood 2004 Feb. 1;103(3)1152-7

C.G. Steward, I. Pellier, A. Mahajan, M. T. Ashworth, A. G. Stuart, A. Fasth, D. Lang, A. Fischer, W. Friedrich and A. S. Schulz on behalf of the Working Party on Inborn Errors of the European Blood and Marrow Transplantation Group. Severe pulmonary hypertension: a frequent complication of stem cell transplantation for malignant infantile osteopetrosis. British Journal of Haematology 2004 Jan;124(1)63-71

Wulffraat NM, Brinkman D, Ferster A, Opperman J, ten Cate R, Wedderburn L, Foster H, Abinun M, Prieur AM, Horneff G, Zintl F, de Kleer I, Kuis W. Long-term follow-up of autoloous stem cell transplantation for refractory juvenil idiopathic arthritis. Bone Marrow Transplant. 2003; 32:S61-S64

C.G. Steward, I. Pellier, A. Mahajan, M. T. Ashworth, A. G. Stuart, A. Fasth, D. Lang, A. Fischer, W. Friedrich and A. S. Schulz on behalf of the Working Party on Inborn Errors of the European Blood and Marrow Transplantation Group. Severe pulmonary hypertension: a frequent complication of stem cell transplantation for malignant infantile osteopetrosis. British Journal of Haematology. 2003; 124,1-9

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, Abinum M, Davies G, Bredius R, Schulz A, Landais P, Fischer A. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. The Lancet 2003;361:553-559

Peters C, Steward CG on behalf of the National Marrow Donor Program, International Bone Marrow Transplant Registry and Working Party on Inborn Errors. Hematopoietic cell transplantation for inherited metabolic diseases : an overview of outcomes and practice guidelines. European Bone Marrow Transplant Group. Bone Marrow Transplantation 17 Feb. 2003;31:229-239

Wulffraat NM, Brinkman D, Ferster A, Opperman J, ten Cate R, Wedderburn L, Foster H, Abinun M, Prieur AM, Horneff G, Zintl F, de Kleer I, Kuis W. Long-term follow-up of autologous stem cell transplantation for refractory juvenile idiopathic arthritis. Bone Marrow Transplantation 01 Aug. 2003;32:S61-S64

Ball LM, Lankester AC, Giordano PC, van Weel MH, Harteveld CL, Bredius RGM, Smiers FJ, Egeler ME, Vossen JMJJ. Paediatric allogeneic bone marrow transplantation for b-thalassemia:the Dutch experience. Bone Marrow Transplantation 2003 Jun;31(12):1081-7

Driessen GJ, Gerritsen EJ, Fischer A, Fasth A, Hop WC, Veys P, Porta F, Cant A, Steward CG, Vossen JM, Uckan D, Friedrich W. Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: an EBMT report. Bone Marrow Transplantation 2003 Oct;32(7):657-63

Feldmann J, Le Deist F, Ouachee-Chardin M, Certain S, Alexander S, Quartier P, Haddad E, Wulffraat NM, Casanova JL, Blanche S, Fischer A, de Saint Basile G. Functional consequences of perforin gene mutations in 22 patients with familial haemophagocytic lymphohistiocytosis. British Journal of Haematology 2002 Jun;117(4)965-72

Seger RA, Gungor T, Belohradsky BH, Blanche S, Bordigoni P, Bartolomeo P, Flood T, Landais P, Muller S, Ozsahin H, Passwell J, Porta F, Slavin S, Wulffraat NM, Zintl F, Nagler A, Cant A and Fischer A. Treatment of chronic granulomtous disease with myeloablative conditioning and an unmodified hematopoietic allograft: A survey of the European experience 1985-2000. Blood 2002 Dec. 15;100(13):4344-50

André-Schmutz I, Le Deist F, Hacein-Bey S, Vitetta E, Schindler J, Chedeville G, Vilmer E, Fischer A and Cavazzana-Calvo M. Immune reconstitution without graft-versus-host disease after haemopoietic stem-cell transplantation: a phase 1/2 study. Lancet 2002 Jul 13;360(9327):98-9

Hamel Y., Blake N., Gabrielsson S., Haigh T. Jooss K., Martinache C., Caillat-Zucman S., Rickinson A.B., Hacein-Bey S., Fischer A and Cavazzana-Calvo M. Adenovirally transduced dendritic cells induce bispecific CTL responses against both adenovirus and CMV pp65 or EBNA3C protein : a novel approach for immunotherapy. Human Gene Therapy May 1, 2002;13:855-866

Dziembowska M, Fondaneche MC, Vedrenne J, Barbieri G, Wiszniewski W, Picard C, Cant AJ, Siemle V, et al. Three novel mutations of the CIITA gene in MHC class II-deficient patients with a severe immunodeficiency. Immunogenetics 2002 Feb;53(10-11):821-9

Haddad E., Le Deist F., Acouturier P., Cavazzana-Calvo M., Banche S., de Saint Basile G., Fischer A. Long-term chimerism and B-cell function after Bone Marrow Transplanation in patients with Severe Combined Immunodeficiency with B cells : a single – center study of 22 patients. Blood 1999 Oct.15;94(8):2923-30

Bertrand Y., Landais P., Friedrich W., Guerritsen B., Morgan G., Fasth A., Cavazzana-Calvo M., Porta F., Cant A., Espanol T., Muller S., Veys P., Vossen J., Haddad, Fischer A. Influence of SCID phenotype on the outcome of HLA non identical, T-cell depleted bone marrow transplantation. A retrospective European survey from the European group for BMT (EBMT) and the European Society for Immunodeficiency (ESID). Journal of Pediatrics 1999 Jun;134(6):740-8

Haddad E, Landais P, Friedrich W, Gerritsen B, Morgan G, Bertrand Y, Fasth A, Porta F, Cant A, Espanol T, Müller S, Veys P, Vossen J, Fischer A. Long term immune reconstitution and outcome after HLA-non-identical T-cell-depleted bone marrow transplantation for severe combined immuno-deficicency: An European retrospective study of 116 patients. Blood (in press).

Jabado N, de Graeff-Meeder ER, Cavazzano-Calvo M, Haddad E, Le Deist F, Benkerrou M, Dufourcq. R, Caillat S, Blanche S, Fischer A. Treatment of familial hemophagocytic lymphohistiocytosis with bone marrow transplantation from HLA genetically nonidentical donors. Blood 1997; 90: 4743-4748.

Porta F, Mazzolari E, Lanfranchi A, Pennacchio M, Fischer A. Immunological reconstitution after bone marrow transplantation. Bone Marrow Transplant 1996; 18: 148-150.

Jabado N, Le Deist F, Cant A, De Graeff-Meeders ER, Fasth A, Morgan G, Vellodi A, Hale G, Bujan W, Thomas C, Cavazzana-Calvo M, Wijdenes J, Fischer A. Bone marrow transplantation from genetically HLA-nonidentical donors in children with fatal inherited disorders excluding severe combined immunodeficiencies: use of two monoclonal antibodies to prevent graft rejection. Pediatr 1996; 98: 420-428.

Friedrich W. Bone marrow transplantation in immunodeficiency diseases. Ann Med 1996; 28: 115-119.

Thomas C, Le Deist F, Cavazzana-Calvo M, Benkerrou M, Haddad E, Blanche S, Hartmann W, Friedrich W, Fischer A. Results of allogeneic bone marrow transplantation in patients with leukocyte adhesion deficiency. Blood 1995; 86: 1629-1635.

Hoogerbrugge PM, Brouwer OF, Bordigoni P, Ringden O, Kapaun P, Ortega JJ, O,'Meara A, Cornu G, Souillet G, Frappaz D, Blanche S, Fischer A, for EBMT. Allogeneic bone transplantation for lysosomal stroage disease. Lancet 1995; 345: 1398-1402.

Gerritsen EJA Vossen JM, Fasth A, Friedrich W, Morgan G, Padmos A, Vellodi A, Porras O, O,'Meara A, Porta F, Bordigoni P, Cant A, Hermans J, Griscelli C, Fischer A. Bone marrow transplantation for autosomal recessive osteo petrosis. A report from the Working Party on Inborn Errors of the EBMT Group. J Pediatr 1994; 125: 896-902.

Gerritsen EJA Vossen JM, van Loo JHG. Autosomal recessive osteopetrosis: variability of findings at diagnosis and during the natural course. Pediatrics 1994; 93: 247-253.

Fischer A, Landais P, Friedrich W, Gerritsen B, Fasth A, Porta T, Vellodi A, Benkerrou M, Jais JP, Cavazzava-Calvo M, Souillet G, Bordigoni P, Morgan G, van Dijken P, Vossen J, Locatelli F, di Bartolomeo P. 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.

Vossen JM. Bone marrow transplantation in children. In: Bailliéres's Clinical Paediatrics 199; 31: 1029-1059.

Kriwit W, Shapiro E, Hoogerbrugge P, Moser PW. State of the art review: bone marrow transplantation treatment for storage diseases. Bone Marrow Transplant 1992; 10 (Suppl. 1): 87-97.

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July 2004

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