Bone marrow transplantation in children with hereditary disorders

Current Opinion in Pediatrics

Volumn 8, Issue 1, 1996, Pages 42-44

  Gluckman, Elaine ^a  

^a SAINT LOUIS HOSPITAL   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ALBERS SCHOENBERG DISEASE; BONE MARROW TRANSPLANTATION; COMBINED IMMUNODEFICIENCY; FANCONI ANEMIA; GENE MUTATION; HUMAN; HURLER SYNDROME; IMMUNE DEFICIENCY; INBORN ERROR OF METABOLISM; MUCOPOLYSACCHARIDOSIS; PATHOPHYSIOLOGY; PRIORITY JOURNAL; REVIEW; THALASSEMIA; TREATMENT OUTCOME; UMBILICAL CORD BLOOD; WISKOTT ALDRICH SYNDROME;

BONE MARROW TRANSPLANTATION; CHILD; FANCONI ANEMIA; FETAL BLOOD; HUMANS; IMMUNE SYSTEM DISEASES; SEVERE COMBINED IMMUNODEFICIENCY;

EID: 0029997538     PISSN: 10408703     EISSN: None     Source Type: Journal    
DOI: 10.1097/00008480-199602000-00009     Document Type: Review

References (17)

1. Rosen FS, Cooper MD, Wedgwood JP: The primary immunodeficiencies. N Engl J Med 1995, 337:431-440. Characterization, molecular analysis, and functional analysis of six immune deficiencies.
2. Gratwohl A, Hermans J: Bone marrow transplantation in Europe 1992: report from the European Group for Bone Marrow Transplantation (EBMT). Bone Marrow Transplant 1994, 13:5-10.
3. Thomas C, De Saint Basile G, Le Daist F, Theophile D, Benkerrou M, Haddad E, Blanche S, Fischer A: Brief report: correction of X-linked hyper-IgM syndrome by allogeneic bone marrow transplantation. N Engl J Med 1995, 333:426-429. A report of the first case treated by allogeneic BMT.
4. Klein C, Philippe N, Le Daist F, Fraitag S, Prost C, Durandy A, Fishcher A, Griscelli C: Partial albinism with immunodeficiency (Griscelli syndrome). J Pediatr 1994, 125:886-895.
5. Klein C, Cavazzana-Calvo M, Le Deist F, Jabado N, Benkerrou M, Blanche S, Lisowska-Grospierre B, Griscelli C, Fischer A: Bone marrow transplantation in major histocompatibllity complex class II deficiency: a single-center study of 19 patients. Blood 1995, 85:580-587.
6. Tjonnfjord GE, Steen R, Veiby OP, Friedrich W, Egeland T: Evidence for engraftment of donor-type multipotent CD34+ cells in a patient with selective T-lymphocyte reconstitution after bone marrow transplantation for B-SCID. Blood 1994, 84:3584-3589. A study of hematopoietic chimerism on CD34+ cells after BMT.
7. Bacchetta R, Parkman R, McMahon M, Weinberg K, Bigler M, de Vries JE, Roncarolo MG: Dysfunctional cytokine production by host-reactive T cell clones isolated from a chimeric severe combined Immunodeficiency patient transplanted with haploidentical bone marrow. Blood 1995, 85:1944-1953. A study of immunologic reconstitution after BMT by selecting T-cell clones.
8. Van-Leeuwen JE, Van-Tol MJ, Joosten AM, Schellekens PT, Van-den-Bergh RL, Waaijer JL, Oudeman-Gruber NJ, van-der-Weijden-Ragas CP, Roos MT, Gerritsen EJ, et al.: Relationship between patterns of engraftment in peripheral blood and immune reconstitution after allogeneic bone marrow transplantation for (severe) combined immunodeficiency. Blood 1994, 84:3936-3947. In this study of immune reconstitution in 31 patients who underwent transplantation for SCID, the authors studied engraftment in diverse cell subsets and evaluated the influence of mixed chimerism on immune reconstitution.
9. Sottini A, Quiros-Roldan E, Notarangelo LD, Malagoli A, Primi D, Imberti L: Engrafted maternal T cells in a severe combined immunodeficiency patient express T cell receptor variable beta segments characterized by a restricted VDJ junctional diversity. Blood 1995, 85:2105-2113. A study of the T-cell repertoire of engrafted maternal T cells in a patient with SCID.
10. Field RE, Buchanan JA, Copplemans MG, Aichroth PM: Bone marrow transplantation in Hurler's syndrome. J Bone Joint Surg Br 1994, 76:975-981.
11. Bergstrom SK, Quinn JJ, Greenstein R, Ascensao J: Long term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review. Bone Marrow Transplant 1994, 14:653-658.
12. Gerritsen EJ, Vossen JM, Fasth A, Friedrich W, Morgan G, Padmos A, Vellodi A, Porras O, O'Meara A, Porta F, et al.: Bone marrow transplantation for autosomal recessive osteopetrosis: a report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group. J Pediatr 1994, 125:896-902. This is a comprehensive review of the results of BMT for osteopetrosis in Europe.
13. Liu JM, Buchwald M, Walsh E, Young NS: Fanconi anemia and novel strategies for therapy. Blood 1994, 84:3995-4007. A review of the current knowledge of the mechanism, methods of diagnosis, gene localization, and treatment of Fanconi's anemia.
14. Gluckman E, Auerbach AD, Horowitz MM, Sobocinski A, Ash RC, Bortin MM, Butturini A, Camitta BM, Champlinj RE, Friedrich W, et al.: Bone marrow transplantation for Fanconi anemia. Blood 1995, 86:2856-2862.
15. Issaragrisil S, Visuthisakchal S, Suvatte V, Tanphaichitr VS, Chandanayingyoung D, Schreiner T, Kanokpongsakdi N, Piankijagum: A brief report: transplantation of cord blood stem cells into a patient with severe thalassemia. N Engl J Med 1995, 332:367-369. One case of successful cord blood transplantation in a patient with thalassemia, with a study of the establishment of chimerism.
16. Wagner JE, Kernan NA, Steinbuch M, Broxmeyer HE, Gluckman E: Allogeneic sibling umbilical cord blood transplantation in children with malignant and non malignant disease. Lancet 1995, 346:214-219. A survey of the results of cord blood transplantation worldwide.
17. Hanley ME, Nolta JA, Parkman R, Kohn DB: Umbilical cord blood cell transduction by retroviral vectors: preclinical studies to optimize gene transfer. Blood Cells 1994, 20:539-546. A study of retroviral gene transfer on umbilical cord blood hematopoietic stem cells. Successful transfer in three patients with adenosine deaminase deficiency of cord blood transfected at birth and reinfused.