Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: A study on behalf of the European Group for Blood and Marrow Transplantation

Blood

Volumn 95, Issue 2, 2000, Pages 422-429

  Guardiola, Ph ^a   Pasquini, R ^b   Dokal, I ^c   Ortega, J J ^d   Van Weel Sipman, M ^e   Marsh, J C W ^f   Ball, S E ^g   Locatelli, F ^h   Vermylen, C ⁱ   Skinner, R ^j   Ljungman, P ^k   Miniero, R ^l   Shaw, P J ^m   Souillet, G ⁿ   Mfchatlel, M ^o   Bekassy, A N ^p   Krivan, G ^q   Di Bartolomeo, P ^r   Heilmann, C ^s   Zanesco, L ^t   Cahn, J Y ^u   Arcese, W ^v   Bacigalupo, A ^a   Gluckman, E ^a   more..

^a SAINT LOUIS HOSPITAL   (France)

^b Department of Hematology   (Brazil)

^c HAMMERSMITH HOSPITAL   (United Kingdom)

^d HOSPITAL MATERNO INFANTIL VALL D HEBRON   (Spain)

^e LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

^f ST GEORGE S HOSPITAL   (United Kingdom)

^g FONDAZIONE IRCCS POLICLINICO SAN MATTEO   (Italy)

^h CLINIQUES UNIVERSITAIRES SAINT LUC   (Belgium)

ⁱ ROYAL VICTORIA INFIRMARY   (United Kingdom)

^j KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

^k REGINA MARGHERITA CHILDREN S HOSPITAL   (Italy)

^l CHILDREN S HOSPITAL AT WESTMEAD   (Australia)

^m HÔPITAL DEBROUSSE   (France)

ⁿ EDOUARD HERRIOT HOSPITAL   (France)

^o LUND UNIVERSITY HOSPITAL   (Sweden)

^p Szent Laszlo Hospital   (Hungary)

^q Ospedale Civile   (Italy)

^r Rigshospitalet   (Denmark)

^s Clinica Oncoematologica Pediatrica e Centra Leucémie Infantili   (Italy)

^t UNIVERSITY HOSPITAL JEAN MINJOZ   (France)

^u SAPIENZA UNIVERSITY OF ROME   (Italy)

^v UNIVERSITY OF GENOA   (Italy)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ASPARTATE AMINOTRANSFERASE; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN A; METHOTREXATE;

ADOLESCENT; ADULT; ARTICLE; BONE MARROW TRANSPLANTATION; CHILD; CLINICAL TRIAL; FANCONI ANEMIA; FEMALE; GRAFT VERSUS HOST REACTION; HLA MATCHING; HUMAN; HUMAN CELL; HUMAN TISSUE; INFANT; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; NEWBORN; PREDICTION; PRIORITY JOURNAL; SURVIVAL; TREATMENT OUTCOME;

ADOLESCENT; ADULT; AGE OF ONSET; CHILD; CHILD, PRESCHOOL; EUROPE; FANCONI ANEMIA; FEMALE; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCOMPATIBILITY TESTING; HUMANS; MALE; SURVIVAL ANALYSIS; TRANSPLANTATION, HOMOLOGOUS; TREATMENT OUTCOME;

EID: 12944315003     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (38)

1. D'Andréa AD, Grompe M. Molecular biology of Fanconi anemia: implications for diagnosis and therapy. Blood. 1997;90:1725-1736.
2. Young NS, Alter BR Clinical features of Fanconi's anemia. In: Young NS, Alter BP, eds. Aplastic Anemia Acquired and Inherited. Philadelphia, PA: Saunders; 1994:275-309.
3. Gampietro PF, Verlander PC, Davis JG, Auerbach AD. Diagnosis of Fanconi anemia in patients without congenital malformations: an International Fanconi Anemia Registry Study. Am J Med Genet. 1997;68:58-61.
4. Berger R, Bemheim A, Gluckman E. In vitro effect of cyclophosphamide metabolites on chromosomes of Fanconi's anaemia patients. Br J Haematol. 1980:45:565-568.
5. Auerbach AD, Woman SR. Susceptibility of Fanconi fibroblasts to chromosome damage by carcinogens. Nature. 1976:261:494-496.
6. Seyschab H, Friedl R, Sun Y, Schindler D, Hoehn H, Hentze S, et al. Comparative evaluation of diepoxybutane sensitivity and cell cycle blockage in the diagnosis of Fanconi anemia. Blood. 1995; 85:2233-2237.
7. Miglierina R, Le Coniat M, Gendron M, Berger R. . Diagnosis of Fanconi's anemia by flow cytometry. Nouv Rev Fr Hematol. 1990:32:391-393.
8. Butturini A, Gale RP, Verlander PC, Adler-Brecher B, Gillio AP, Auerbach AD. Hématologie abnormalities in Fanconi anemia: an International Fanconi Anemia Registry study. Blood. 1994;84: 1650-1655.
9. Guinan EC, Lopez KD, Huhn RD, Felser JM, Nathan DG. Evaluation of granulocyte-macrophage colony-stimulating factor for treatment of pancytopenia in children with Fanconi anemia. J Pediatr. 1994;124:144-150.
10. Kemahli S, Canatan D, Uysal Z, Akar N, Cin S, Arcasoy A. GM-CSF in the treatment of Fanconi's anaemia. Br J Haematol. 1994:87:871-872.
11. Rackoff WR, Orazi A, Robinson CA, Cooper RJ, Alter BP, Freedman MH, et al. Prolonged administration of granulocyte colony-stimulating factor (filgrastim) to patients with Fanconi anemia: a pilot study. Blood. 199S;88:1588-1593.
12. Scagni P, Saracco P, Timeus F, Farinasso L, Dall'Aglio M, Bosa EM, et al. Use of recombinant granulocyte colony-stimulating factor in Fanconi's anemia. Haematologica. 1998:83:432-437.
13. Gluckman E, Auerbach AD, Horowitz MM, Sobocinski KA, Ash RC, Bortin MM, et al. Bone marrow transplantation for Fanconi anemia. Blood. 1995:86:2856-2862.
14. Socie G, Devergie A, Girinski T, Piel G, Ribaud P, Esperou H, et al. Transplantation for Fanconi's anaemia: long-term follow-up of fifty patients transplanted from a sibling donor after low-dose cyclophosphamide and thoraco-abdominal irradiation for conditioning. Br J Haematol. 1998;103: 249-255.
15. Socie G, Gluckman E, Raynal B, Petit T, Landman J, Devergie A, et al. Bone marrow transplantation for Fanconi anemia using low-dose cyclophosphamide/thoracoabdominal irradiation as conditioning regimen: chimerism study by the polymerase chain reaction. Blood. 1993:82:22492256.
16. Flowers ME, Zanis J, Pasquini R, Deeg HJ, Ribeiro R, Longton G, et al. Marrow transplantation (or Fanconi anaemia: conditioning with reduced doses of cyclophosphamide without radia' ' tion. Br J Haematol. 1996:92:699-706.
17. Kohli-Kumar M, Morris C, DeLaat C, Sambrano J, Masterson M, Mueller R, et al. Bone marrow transplantation in Fanconi anemia using matched sibling donors. Blood. 1994:84:2050-2054.
18. Zanis-Neto J, Ribeiro RC, Medeiros C, Andrade RJ, Ogasawara V, Hush M, et al. Bone marrow transplantation for patients with Fanconi anemia: a study of 24 cases from a single institution. Bone Marrow Transplant. 1995:15:293-298.
19. Davies SM, Khan S, Wagner JE, Arthur DC, Auerbach AD, Ramsay NK, et al. Unrelated donor bone marrow transplantation for Fanconi anemia. Bone Marrow Transplant. 1996:17:43-47.
20. Zwaan ChM, Van Weel-Sipman MH, Fibbe WE, Oudshoorn M, Vossen JMJJ. Unrelated donor bone marrow transplantation in Fanconi anaemia: the Leiden experience. Bone Marrow Transplant. 1998:21:447-453.
21. Massumoto C, Moraes JR, Moraes ME, Macedo MC, Medeiros R, Chamone D, et al. Unrelated peripheral blood stem cell transplantation for Fanconi anemia. Bone Marrow Transplant. 1997;19: 299-300.
22. Glucksberg H, Storb R, Fefer A, Buckner CD, Neiman PE, Clift RA, et al. Clinical manifestations of graft-versus-host disease in human recipient of marrow from HLA-matched sibling donors. Transplantation. 1974:18:295-304.
23. Thomas E, Storb R, Clift RA, Feier A, Johnson FL, Neiman PE, et al. Bone-marrow transplantation. N Engl J Med. 1975:292:832-843.
24. Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Slat Assoc. 1958:53:457.
25. Cox DR. Regression models and life tables. J R StatSoc.J972;34:187.
26. Cook D. Weisberg S. Residuals and Influence in Regression. London: Chapman and Hall. 1982.
27. Weissfeld W. A multicolinearity diagnostic for models fitted to censored data. Commun StatistTheory Meth. 1989:18:2073-
28. Schoenfeld D. Partial residuals for the proportional hazards model. Biometrika. 1982:69:51.
29. Harrell FE Jr, Lee KL, Mark DB. Multivariable prognostic models: issues in developing models, evaluating assumptions and adequacy, and measuring and reducing errors. Stat Med. 1996;15: 361-337.
30. Gillio AP, Verlander PC, Batish SD, Giampietro PF, Auerbach AD. Phenotypic consequences of mutations in the Fanconi anemia FAC gene: an International Fanconi Anemia Registry study. Blood. 1997:90:105-110.
31. Yamashita T, Wu N, Kupfer G, Cortess C, Joenje H, Grompe M, et al. Clinical variability of Fanconi anemia (type C) results from expression of an amino terminal truncated Fanconi anemia complementation group C polypeptide with partial activity. Blood. 1996;87:4424-4432.
32. Verlander PC, Kaporis A, Liu Q, Zhang Q. Seligsohn U, Auerbach AD. Carrier frequency of the IVS4 + 4 AT mutation of the Fanconi anemia gene FAC in the Ashkenazi Jewish population. Blood. 1995;86:4034-4038.
33. Carreras E, Bertz H, Arcese W, Vernant JP, Tomas JF, Hagglund H, et at. Incidence and outcome of hepatic veno-occlusive disease after blood or marrow transplantation: a prospective cohort study of the European Group for Blood and Marrow Transplantation. Blood. 1998;92: 3599-3604.
34. Gluckman E, Rocha V, Boyer-Chammard A, Locatelli F, Arcese W, Pasquini R, et al. Outcome of cord-blood transplantation from related and unrelated donors: Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. N Engl J Med. 1997:337:373-381.
35. Morrison VA, Haake RJ, Weisdorf DJ. Non-Candida fungal infections after bone marrow transplantation: risk factors and outcome. Am J Med. 1994;96:497-503.
36. Humar A, Wood S, Lipton J, et al. Effect of cytomegalovirus infection on 1-year mortality rates among recipients of allogeneic bone marrow transplants. Clin Infect Dis. 1998:26,606-610.
37. Sasazuki T, Juji T, Morishima Y, Kinukawa N, Kashiwabara H, Inoko H, et al, for the Japan Marrow Donor Program. Effect of matching of class I HLA alleles on clinical outcome after transplantation of hematopoietic stem ceils from an unrelated donor. N Engl JMed. 1998:339:1177-1185.
38. Petersdorf EW, Gooley TA, Anasetti C, Martin PJ, Smith AG, Mickelson EM, et al. Optimizing outcome after unrelated marrow transplantation by comprehensive matching of HLA class I and II alleles in the donor and recipient. Blood. 1998:92: 3515-3520.