Allogeneic stem cell transplantation for myelodysplastic syndromes in children: a report from the Spanish Working Party for Blood and Marrow Transplantation in Children (GETMON).
BACKGROUND
Experience with the use of allogeneic hemopoietic stem transplantation (AHSCT) in pediatric myelodisplastic syndrome (MDS) in Spain is reviewed.


METHODS
Twenty-four children with MDS were analyzed retrospectively. Median age of the patients was 10 years. Twenty patients received a bone marrow graft and 4 an unrelated cord blood (UCB) transplant; 12 bone marrow grafts were from a matched related donor (MRD) and 8 from a matched unrelated donor (MUD). Conditioning regimen consisted of chemotherapy alone in 17 patients (busulfan and cyclophosphamide +/- melfalan) Seven patients received TBI and cyclophosphamide.


RESULTS
Ten patients died from transplant-related toxicity and 4 had relapse or disease progression post-AHSCT. Nine patients are alive and event-free with a median follow-up of 120 months. EFS rate in the MRD group was 0.48 (SE 0.13) versus 0.25 (SE 0.12) in the MUD/UCB group (p = .07). Lansky score in survivors is >or=90%.


CONCLUSIONS
In this historical series of children with MDS, in spite of severe transplant-related toxicity, encouraging EFS outcomes have been achieved after AHSCT with good quality of life.
Muñoz
Arturo
A
Department of Pediatrics, Hospital Ramon y Cajal-University of Alcalá, Madrid, Spain. amvillatv@yahoo.es
Díaz-Heredia
Cristina
C
Badell
Isabel
I
Bureo
Encarna
E
Gómez
Pedro
P
Martínez
Ana
A
Verdeguer
Amparo
A
Pérez-Hurtado
José María
JM
Fernández-Delgado
Rafael
R
González-Vicent
Marta
M
Maldonado
María Soledad
MS
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D004359:Drug Therapy, Combination; D005260:Female; D006086:Graft vs Host Disease / Q000276:immunology / Q000517:prevention & control*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008297:Male; D009190:Myelodysplastic Syndromes / Q000276:immunology / Q000401:mortality / Q000601:surgery*; D011788:Quality of Life; D012189:Retrospective Studies; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous / Q000276:immunology*; D016896:Treatment Outcome