Failure of allogeneic bone marrow transplantation to correct Diamond-Blackfan anaemia despite haemopoietic stem cell engraftment

Bone Marrow Transplantation

Volumn 24, Issue 7, 1999, Pages 803-805

  Wynn, R F ^a   Grainger, J D ^a   Carr, T F ^a   Eden, O B ^a   Stevens, R F ^a   Will, A M ^a  

^a ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

Bone marrow transplantation; Diamond Blackfan anaemia

Indexed keywords

BUSULFAN; CYCLOPHOSPHAMIDE; CYCLOSPORIN;

ALLOGENIC BONE MARROW TRANSPLANTATION; ARTICLE; BLACKFAN DIAMOND ANEMIA; CASE REPORT; ERYTHROCYTE TRANSFUSION; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA MATCHING; HUMAN; HUMAN CELL; MALE; PRIORITY JOURNAL; PURE RED CELL ANEMIA; SCHOOL CHILD; TREATMENT FAILURE; VEIN OCCLUSION;

EID: 0032872463     PISSN: 02683369     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.bmt.1701982     Document Type: Article

References (23)

1. Ball SE, McGuckin CP, Jenkins G, Gordon-Smith EC. Diamond-Blackfan anaemia in the UK: analysis of 80 cases from a 20-year birth cohort. Br J Haematol 1996; 94: 645-653.
2. Glader BE, Guinan E, Lipton JM, Boxer L. Congenital bone marrow failure syndromes: diagnostic and therapeutic strategies. ASH 1998 Educational Supplement 1998; 384-403.
3. Gustavsson P, Willing TN, Van-Haeringen A et al. Diamond-Blackfan anaemia: genetic homogenecity for a gene on chromosome 19q23 restricted to 1.8 Mb. Nat Genet 1997; 16: 368-371.
4. McGuckin CP, Ball SE, Gordon-Smith EC. Diamond-Blackfan anaemia: three patterns of in vitro response to haemopoietic growth factors. Br J Haematol 1995; 89: 457-464.
5. McGuckin CP, Liu WM, Ball SE et al. Diamond-Blackfan anaemia: differential pattern of in vitro progenitor response to macrophage inflammatory protein 1-alpha. Br J Haematol 1996; 92: 280-286.
6. August CS, King E, Githens JH et al. Establishment of erythropoiesis following bone marrow transplantation in a patient with congenital hypoplastic anaemia (Diamond-Blackfan anaemia). Blood 1976; 48: 491-498.
7. Iriondo A, Garijo J, Baro J et al. Complete recovery of haemopoiesis following bone marrow transplant in a patient with unresponsive congenital hypoplastic anaemia (Blackfan-Diamond syndrome). Blood 1984; 64: 348-351.
8. Wiktor-Jedrzejczak W, Szczylik C, Pojda Z et al. Success of bone marrow transplant in congenital Diamond-Blackfan anaemia: a case report. Eur J Haematol 1987; 38: 204-206.
9. Lenarsky C, Weinberg K, Guinan E et al. Bone marrow transplantation for constitutional pure red cell aplasia. Blood 1998; 71: 226-229.
10. Gluckman E, Esperou H, Devergie A et al. Pediatric bone marrow transplantation for leukaemia and aplastic anaemia: report of 222 cases transplanted in a single centre. Nouv Rev Fr Hematol 1989; 31: 111-114.
11. Zintl F, Hermann J, Fuchs D et al. Correction of fatal genetic disease using bone marrow transplantation. Kinderarztl Prax 1991; 84: 515-520.
12. Greinix HT, Storb R, Sanders JE et al. Long term survival and cure after bone marrow transplantation for severe congenital hypoplastic anaemia (Diamond-Blackfan syndrome). Br J Haematol 1993; 84: 515-520.
13. Saunders EF, Olivieri N, Feedman MH. Unexpected complications after bone marrow transplantation in transfusion dependent children. Bone Marrow Transplant 1993; 12 (Suppl. 1): 88-90.
14. Mugishima H, Gale RP, Rowlings PA et al. Bone marrow transplant for Diamond-Blackfan anaemia. Bone Marrow transplant 1995; 15: 55-58.
15. Lee AC, Ha SY, Yuen KY, Lau YL. Listeria septicaemia complicating bone marrow transplantation for Diamond-Blackfan syndrome. Pediatr Hematol Oncol 1995; 12: 295-299.
16. McGuckin CP, Uhr MR, Ball SE, Gordon-Smith EC. In vitro progenitor analysis in a Diamond-Blackfan anaemia patient who responded once but not twice to interleukin-3 therapy, using short-term and long-term cultures and c-kit analysis. Br J Haematol 1996; 93: 319-325
17. Ladenstein R, Peters C, Minkov M et al. A single centre experience with allogenic stem cell transplantation for severe aplastic anaemia in childhood. Klin Pediatr 1997; 209: 201-208.
18. Wagner JE, Rosenthal J, Sweetman R et al. Successful transplantation of a HLA-matched and HLA mis-matched umbilical cord blood from unrelated donors: analysis of engraftment and acute graft-versus-host disease. Blood 1996; 88: 795-802
19. Bonno M, Azuma A, Nakano T et al. Successful haemopoietic reconstitution by transplant of umbilical cord blood cells in a transfusion dependent child with Diamond-Blackfan anaemia. Bone Marrow Transplant 1997; 19: 83-85.
20. Vettenrata K, Saarinen UM. Cord blood stem cell transplantation for Diamond-Blackfan anaemia. Bone Marrow Transplant 1997; 19: 507-508.
21. Morimoto T, Shikada M, Yabe H et al. Umbilical cord blood transplantation for a patient with Diamond-Blackfan syndrome. Rinsho Ketsuekei 1997; 38: 610-615.
22. Gluckman E, Rocha V, Boyer-Chammard A et al. Outcome of cord-blood transplantation from related and unrelated donors. New Engl J Med 1997; 337: 373-381.
23. Willig TN, Perignon JL, Gustavsson P et al. High adenosine deaminase level among healthy probands of Diamond-Blackfan anaemia (DBA) cosegregates with the DBA gene region on chromosome 19q13. The DBA Working Group of Société d'Immunologie Pediatrique (SHIP). Blood 1998; 92: 4422-4427.