[Severe acquired aplastic anemia: historical outcome of patients treated by allogeneic bone marrow transplantation from matched sibling donors. A study by the Spanish Group for Bone Marrow Transplantation in Children (GETMON)].
BACKGROUND
Allogeneic haematopoietic stem-cell transplantation is the treatment of choice for acquired aplastic anaemia in children. Experience with this approach from Spanish Working Party for Bone Marrow Transplantation in Children in two sequential time periods (1982-1990 and 1991-2004) is reported.


METHODS
Sixty two consecutive patients with a median age of 10 years were transplanted; 18 in the 1982-1990 period and 44 in the 1991-2004 period. Conditioning regimen consisted mainly of irradiation and cyclophosphamide in the first period (72 % of patients) and cyclophosphamide +/- anti-thymocyte globulin (62 %) in the second. Graft versus host disease prophylaxis consisted of cyclosporine in most patients (57/62).


RESULTS
Fifty one patients are alive and disease-free at a median follow-up of 127 months. Five years probability of event-free survival is 82 %. The survival increased from 61 % to 91 % during the two time periods. Eleven patients died from graft failure or rejection (3), acute or chronic graft versus host disease and infection (4) or multi-organ failure (4). Univariate analysis identified two significant prognostic factors: interval diagnostic/transplant and time period of transplant (for both p = 0.03).


CONCLUSIONS
This experience corroborates that allogeneic haematopoietic stem-cell transplantation is the best treatment for severe acquired aplastic anaemia, with a current disease-free survival of 90 % of patients.
Muñoz Villa
A
A
Hospital Ramon y Cajal, Universidad de Alcalá, Madrid, Spain. amvillatv@yahoo.es
Díaz de Heredia
C
C
Díaz González
M A
MA
Badell Serra
I
I
Martínez Rubio
A
A
González Valentín
M A
MA
Dasí Carpio
M A
MA
Gómez Pérez
P
P
Bureo Dacal
E
E
Olivé Oliveras
T
T
Pérez Hurtado
J M
JM
Maldonado Regalado
M S
MS
D000741:Anemia, Aplastic / Q000175:diagnosis* / Q000188:drug therapy / Q000628:therapy*; D000970:Antineoplastic Agents / Q000627:therapeutic use; D016026:Bone Marrow Transplantation / Q000379:methods*; D002648:Child; D016572:Cyclosporine / Q000627:therapeutic use; D005260:Female; D006084:Graft Rejection / Q000517:prevention & control; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D008297:Male; D012720:Severity of Illness Index; D035781:Siblings; D013030:Spain; D014019:Tissue Donors; D014184:Transplantation, Homologous
D000970:Antineoplastic Agents; D007166:Immunosuppressive Agents; D016572:Cyclosporine