Long-term immune reconstitution and clinical outcome after stem cell transplantation for severe T-cell immunodeficiency.
BACKGROUND
Currently, hematopoietic stem cell transplantation allows long-term survival in a high proportion of infants with congenital severe T-cell immunodeficiency. However, relatively little is known of their long-term quality of life.


OBJECTIVE
We sought to assess the long-term immune reconstitution and clinical status in children treated with stem cell transplantation for severe T-cell immunodeficiency.


METHODS
Immune function and clinical status have been analyzed in a cohort of 40 patients with severe T-cell immunodeficiency who are alive at a follow-up of at least 5 years after transplantation.


RESULTS
Most patients have attained normal T- and B-cell function. Weight and height were normal at last follow-up in most patients. Endocrine and severe neurologic abnormalities have been observed in 17.5% and 10% of the patients, respectively.


CONCLUSIONS
These data indicate that with current management strategies, stem cell transplantation can lead to long-term survival and good quality of life in the majority of patients with severe T-cell immunodeficiency.


CONCLUSIONS
Prompt recognition of congenital severe T-cell immunodeficiency, followed by stem cell transplantation, allows excellent perspectives of long-term survival and good quality of life for these otherwise fatal disorders.
Mazzolari
Evelina
E
Divisione di Oncoematologia Pediatrica, Ospedale dei Bambini, Spedali Civili.
Forino
Concetta
C
Guerci
Sara
S
Imberti
Luisa
L
Lanfranchi
Arnalda
A
Porta
Fulvio
F
Notarangelo
Luigi D
LD
D001327:Autoimmune Diseases / Q000209:etiology; D002657:Child Development; D002675:Child, Preschool; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects; D006801:Humans; D007223:Infant; D012677:Sensation; D016511:Severe Combined Immunodeficiency / Q000276:immunology / Q000401:mortality / Q000628:therapy*