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Vol. 53. Issue 6.
Pages 513-519 (1 December 2000)
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Vol. 53. Issue 6.
Pages 513-519 (1 December 2000)
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Trasplante de progenitores hematopoyéticos de sangre de cordón umbilical en niños
Transplantation of umbilical cord blood hematopoietic progenitor cells in children
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I. Badell Serraa,
Corresponding author
ibadell@hsp.santpau.es

Correspondencia:Dra. I. Badell Serra. Servicio de Pediatría. Hospital de la Santa Creu i Sant Pau. Avda. Sant Antoni M. Claret, 167. 08025 Barcelona.
, T. Olivé Oliverasb, L. Madero Lópezc, A. Muñoz Villad, A. Martínez Rubioe, A. Verdeguer Mirallesf, C. Díaz de Heredia Rubiob, M.A. Díaz Perezc, J. Cubells Rieróa, M.S. Maldonado Regaladod, J.J. Ortega Aramburub, por el Grupo Español para el Trasplante de Medula Ósea en niños (GETMON)
a Servicios de Pediatría.Hospital de la Santa Creu i Sant Pau y
b Hospital Vall d’Hebron. Barcelona.
c Hospital Niño Jesús,
d Hospital Ramón y Cajal y
e Hospital La Paz. Madrid.
f Hospital La Fe. Valencia
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Objetivo

Estudio retrospectivo de los resultados del trasplante de progenitores hematopoyéticos de sangre de cordón umbilical en España.

Pacientes y métodos

Veintiocho niños con edad media de 6,5 años y peso medio de 25 kg recibieron un trasplante de sangre de cordón umbilical entre julio de 1994 y mayo de 1998 en distintos centros pertenecientes al Grupo Español para el Trasplan-te de Medula Ósea en niños (GETMON).

El donante fue en 2 pacientes un hermano HLA-idéntico, en otros 2 pacientes un familiar no idéntico y en los 24 restantes un donante no emparentado. Entre éstos, la identidad antigénica HLA (A, B y DR) 6/6 sólo se observaba en 3 pacientes. Los trasplantes se realizaron en su mayoría por leucemia (21 pacientes, 75%) y en fase avanzada. Los restantes 7 pacientes se trasplantaron por una enfermedad genética, en su mayoría inmunodeficiencia congénita. El tratamiento de acondicionamiento incluyó irradiación corporal total en 10 pacientes y poliquimioterapia en los restantes. La profilaxis de la enfermedad del injerto contra huésped aguda se realizó con ciclosporina en todos los casos añadiendo corticoides o metotrexato en los trasplantes sin identidad HLA. La media de células perfun-didas fue de 53,4×106/kg.

Resultados

El fallo de implante de la sangre de cordón umbilical se observó en 9 pacientes. Presentaron enfermedad del injerto contra huésped aguda superior al grado II 18 pacientes (64,3%). Ocho (28,6%) presentaron EICH grave. La supervivencia actuarial libre de enfermedad (SLE) de la serie global fue del 34,4±9% a 3 años, con una media de seguimiento de 16,6 meses. Se observó una mejor SLE en las enfermedades congénitas, con una SLE del 71±17% y también en los pacientes que recibieron trasplante de sangre de cordón umbilical con una identidad HLA A, B y DR 6/6, en los que la SLE fue del 66±19%.

Conclusiones

Los mejores resultados se obtuvieron en las enfermeda-des genéticas. Se ha observado una correlación inversa en-tre la SLE y la disparidad antigénica HLA. La incidencia relativamente alta de la enfermedad injerto contra huésped aguda en esta serie, podría relacionarse con la escasa precisión de la tipificación HLA efectuada en algunos pacientes.

Palabras clave:
Trasplante de progenitores hematopoyéticos
Trasplan-te de sangre de cordón umbilical
Leucemia
Inmunodefi-ciencia congénita
Niños
Objective

Retrospective study of the outcome of cord blood trans-plantation (CBT) in children in Spain.

Patients and method

Twenty-eight patients (mean age 6.5 years; mean weight 25 kg) received a CBT between July 1994 and May 1998 in several centres of the Spanish Pediatric Bone Marrow Transplant Group. In 2 patients the donor was an identical human leukocyte antigen (HLA)-sibling and in two the donor was a mismatched family donor. In 24 patients the donor was unrelated, and 21 of these received an HLA-mismatched CBT. Twenty-one patients (75%) received a CBT for leukemia mainly in advanced phase. Seven patients were transplanted for genetic disease. Of these, five had congenital immunodeficiency. The conditioning treatment included total body irradiation in ten patients and combined chemotherapy in the remaining patients. In all patients graft-versus-host disease (GVHD) prophylaxis was performed with cyclosporine, and corticosteroids or methotrexate were added in patients with HLA-mismatched donors. The mean number of nucleated cells infused was 53.4×106/kg.

Results

Graft failure was observed in nine patients. Eighteen patients (64.3%) developed grade II-IV acute GVHD. Eight patients (28.6%) developed severe GVHD. Actuarial event-free survival (EFS) of all the patients was 34.4±69% at 3 years, with a mean followup of 16.6 months. EFS was more favorable in patients with genetic disease (71±17%) and in those with an HLA (A, B and DR) identical donor (6/6) (66±19%).

Conclusions

The most favorable results were obtained in patients with genetic diseases. We observed an inverse correlation between EFS and patients with HLA-identical donors. The high incidence of severe acute GVHD could have been related to a lack of accuracy in the HLA-typography of some patients.

Key words:
Stem cell transplantation
Cord blood transplantation
Leukemia
Congenital immunodeficiency
Children
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Copyright © 2000. Asociación Española de Pediatría
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