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

Ph Guardiola, R. Pasquini, I. Dokal, J. J. Ortega, M. Van Weel-Sipman, J. C W Marsh, S. E. Ball, F. Locatelli, C. Vermylen, R. Skinner, P. Ljungman, R. Miniero, P. J. Shaw, G. Souillet, M. Mfchatlel, A. N. Bekassy, G. Krivan, P. Di Bartolomeo, C. Heilmann, L. ZanescoJ. Y. Cahn, W. Arcese, A. Bacigalupo, E. Gluckman

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Abstract

Allogeneic stem cell transplantation is the only treatment that can restore a normal hematopoiesis in Fanconi anemia (FA). In this retrospective multicenter study, we analyzed the results of this approach using HLA-matched unrelated bone marrow donors, and tried to identify covariates predicting the outcome of the transplant. From January 1985 to June 1998, 69 FA patients were transplanted with unrelated HLA-matched donors. Patients' characteristics before and after transplant were provided by the European group blood and marrow transplant registry and were analyzed in collaboration with the European Fanconi Anemia Registry. The 3-year probability of survival was 33%. Extensive malformations, a positive recipient cytomegalovirus serology, the use of androgens before transplant, and female donors were associated With a worse outcome. Primary graft failures were observed more frequently when female donors were used, mainly because the grafts contained lower nucleated cell doses per kilogram of recipient body weight compared with grafts coming from male donors. The probability of grade III-IV acute graft-versus-host disease (GVHD) was 34%. Elevated serum alanine/aspartate transaminases before transplantation; limb, urogenital tract, or nephrologic malformations; and non-T-cell-depleted grafts were predictors of severe acute GVHD. This study shows the dramatic impact of preexisting congenital malformations on the outcome of FA patients transplanted with HLA-matched unrelated donors. If the use of T-cell depletion has led to a dramatic reduction of acute GVHD incidence, no significant outcome improvement was observed with this approach, mainly because of an increased risk of graft failure.

Original languageEnglish
Pages (from-to)422-429
Number of pages8
JournalBlood
Volume95
Issue number2
Publication statusPublished - Jan 15 2000

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Fanconi Anemia
Unrelated Donors
Stem Cell Transplantation
Stem cells
Grafts
Transplants
Tissue Donors
Graft vs Host Disease
Registries
Bone Marrow
T-cells
Blood Group Antigens
Aspartate Aminotransferases
Hematopoiesis
Alanine
Serology
Androgens
Alanine Transaminase
Cytomegalovirus
Bone

ASJC Scopus subject areas

  • Hematology

Cite this

Guardiola, P., Pasquini, R., Dokal, I., Ortega, J. J., Van Weel-Sipman, M., Marsh, J. C. W., ... Gluckman, E. (2000). 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, 95(2), 422-429.

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. / Guardiola, Ph; Pasquini, R.; Dokal, I.; Ortega, J. J.; Van Weel-Sipman, M.; Marsh, J. C W; Ball, S. E.; Locatelli, F.; Vermylen, C.; Skinner, R.; Ljungman, P.; Miniero, R.; Shaw, P. J.; Souillet, G.; Mfchatlel, M.; Bekassy, A. N.; Krivan, G.; Di Bartolomeo, P.; Heilmann, C.; Zanesco, L.; Cahn, J. Y.; Arcese, W.; Bacigalupo, A.; Gluckman, E.

In: Blood, Vol. 95, No. 2, 15.01.2000, p. 422-429.

Research output: Contribution to journalArticle

Guardiola, P, Pasquini, R, Dokal, I, Ortega, JJ, Van Weel-Sipman, M, Marsh, JCW, Ball, SE, Locatelli, F, Vermylen, C, Skinner, R, Ljungman, P, Miniero, R, Shaw, PJ, Souillet, G, Mfchatlel, M, Bekassy, AN, Krivan, G, Di Bartolomeo, P, Heilmann, C, Zanesco, L, Cahn, JY, Arcese, W, Bacigalupo, A & Gluckman, E 2000, '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, vol. 95, no. 2, pp. 422-429.
Guardiola, Ph ; Pasquini, R. ; Dokal, I. ; Ortega, J. J. ; Van Weel-Sipman, M. ; Marsh, J. C W ; Ball, S. E. ; Locatelli, F. ; Vermylen, C. ; Skinner, R. ; Ljungman, P. ; Miniero, R. ; Shaw, P. J. ; Souillet, G. ; Mfchatlel, M. ; Bekassy, A. N. ; Krivan, G. ; Di Bartolomeo, P. ; Heilmann, C. ; Zanesco, L. ; Cahn, J. Y. ; Arcese, W. ; Bacigalupo, A. ; Gluckman, E. / 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. In: Blood. 2000 ; Vol. 95, No. 2. pp. 422-429.
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abstract = "Allogeneic stem cell transplantation is the only treatment that can restore a normal hematopoiesis in Fanconi anemia (FA). In this retrospective multicenter study, we analyzed the results of this approach using HLA-matched unrelated bone marrow donors, and tried to identify covariates predicting the outcome of the transplant. From January 1985 to June 1998, 69 FA patients were transplanted with unrelated HLA-matched donors. Patients' characteristics before and after transplant were provided by the European group blood and marrow transplant registry and were analyzed in collaboration with the European Fanconi Anemia Registry. The 3-year probability of survival was 33{\%}. Extensive malformations, a positive recipient cytomegalovirus serology, the use of androgens before transplant, and female donors were associated With a worse outcome. Primary graft failures were observed more frequently when female donors were used, mainly because the grafts contained lower nucleated cell doses per kilogram of recipient body weight compared with grafts coming from male donors. The probability of grade III-IV acute graft-versus-host disease (GVHD) was 34{\%}. Elevated serum alanine/aspartate transaminases before transplantation; limb, urogenital tract, or nephrologic malformations; and non-T-cell-depleted grafts were predictors of severe acute GVHD. This study shows the dramatic impact of preexisting congenital malformations on the outcome of FA patients transplanted with HLA-matched unrelated donors. If the use of T-cell depletion has led to a dramatic reduction of acute GVHD incidence, no significant outcome improvement was observed with this approach, mainly because of an increased risk of graft failure.",
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AU - Guardiola, Ph

AU - Pasquini, R.

AU - Dokal, I.

AU - Ortega, J. J.

AU - Van Weel-Sipman, M.

AU - Marsh, J. C W

AU - Ball, S. E.

AU - Locatelli, F.

AU - Vermylen, C.

AU - Skinner, R.

AU - Ljungman, P.

AU - Miniero, R.

AU - Shaw, P. J.

AU - Souillet, G.

AU - Mfchatlel, M.

AU - Bekassy, A. N.

AU - Krivan, G.

AU - Di Bartolomeo, P.

AU - Heilmann, C.

AU - Zanesco, L.

AU - Cahn, J. Y.

AU - Arcese, W.

AU - Bacigalupo, A.

AU - Gluckman, E.

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