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Blood, Vol. 95 No. 2 (January 15), 2000:
pp. 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
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. Michallet,
A. N. Bekassy,
G. Krivan,
P. Di Bartolomeo,
C. Heilmann,
L. Zanesco,
J. Y. Cahn,
W. Arcese,
A. Bacigalupo, and
E. Gluckman for the Severe Aplastic
Anemia Working Party of the European Group for Blood and Marrow
Transplantation and the European Fanconi Anemia Registry
From the Department of Hematology, Bone Marrow Transplant Unit,
Hôpital Saint-Louis, Paris, France; Department of Hematology,
Hospital Civil, Curitiba, Brazil; Department of Hematology, Hammersmith
Hospital, London, UK; Department of Hematology, Hospital Maternal
Infantil Vall d'Hebron, Barcelona, Spain; Department of Pediatrics,
Leiden University Medical Centre, Leiden, The Netherlands; Division of
Hematology, Saint George's Hospital Medical School, London, UK;
Pediatric Clinic, Policlinico San Matteo, Pavia, Italy; Department of
Hematology, Cliniques Universitaires Saint Luc, Brussels, Belgium;
Paediatric Oncology Unit, Royal Victoria Infirmary, Newcastle upon
Tyne, UK; Department of Hematology, Huddinge Hospital, Huddinge,
Sweden; Department of Pediatrics, Ospedale Regina Margherita,
Turin, Italy; Oncology Unit, The New Children's Hospital,
Sydney, Australia; Pediatric Immuno-Hematology Unit, Hospital
Debrousse, Lyons, France; Bone Marrow Transplantation Unit
Pavillon E, Hospital Edouard Herriot, Lyons, France;
Department of Pediatrics, University Hospital, Lund, Sweden; Bone
Marrow Transplantation Unit, Saint Laszlo Hospital, Budapest, Hungary;
Department of Hematology, Ospedale Civile, Pescara, Italy; Pediatric
Clinic II, Rigshospitalet, Copenhagen, Denmark; Clinica Oncoematologica
Pediatrica e Centro Leucemie Infantili, Padua, Italy;
Department of Hematology, Hospital Jean Minjoz, Besançon, France;
GITMO - Roma, Ematologia, Universita "La Sapienza,"
Rome, Italy; and Department of Hematology, Ospedale San
Martino, Genoa, Italy.
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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