Chronic Cholangiopathy Associated with Primary Immune Deficiencies Can Be Resolved by Effective Hematopoietic Stem Cell Transplantation.
J Pediatr
; 209: 97-106.e2, 2019 06.
Article
en En
| MEDLINE
| ID: mdl-30850087
ABSTRACT
OBJECTIVES:
To investigate effects and outcome of hematopoietic stem cell transplantation (HSCT) on sclerosing cholangitis, in pediatric patients with different primary immunodeficiencies (PIDs). STUDYDESIGN:
From databases in 2 tertiary centers for immunodeficiencies and liver disease, we have identified children with PIDs and sclerosing cholangitis, who have paired clinical, radiologic, and histologic information before and after HSCT and studied their clinical progress and outcome.RESULTS:
Seven of 13 children (53.8%) died at a median interval of 4 months (range, 3 months-5 years) after HSCT. However, 6 surviving children (46.2%) with different PIDs and less severe cholangiopathies showed an improvement in markers of liver injury within months of successful unrelated reduced intensity conditioning HSCT. The repeated native liver biopsy, performed in 4 patients at a median of 96 (range, 4-144) months post-HSCT, showed a considerable improvement. Biochemical markers of liver function in the survivors completely normalized after a median of 13 months (range, 2-48). All patients continue to have a mildly dilated extrahepatic biliary system on ultrasonography with no intrahepatic ductal changes on magnetic resonance cholangiography after a follow-up of median 18 years (range, 2-20).CONCLUSIONS:
Effective HSCT has the potential to improve biochemical and histologic features of cholangiopathy in children with PIDs, presumably by clearance of chronic infection following establishment of immune competence. However, careful patient selection is critical as advanced liver injury is often associated with serious complications and mortality.Palabras clave
Texto completo:
1
Banco de datos:
MEDLINE
Asunto principal:
Colangitis Esclerosante
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Causas de Muerte
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Trasplante de Células Madre Hematopoyéticas
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Enfermedades de Inmunodeficiencia Primaria
Tipo de estudio:
Diagnostic_studies
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Etiology_studies
/
Incidence_studies
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Observational_studies
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Prognostic_studies
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Risk_factors_studies
Límite:
Child, preschool
/
Female
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Humans
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Infant
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Male
País como asunto:
Europa
Idioma:
En
Año:
2019
Tipo del documento:
Article