Haploidentical stem cell transplantation in a boy with chronic granulomatous disease
Allergol. immunopatol
; 46(4): 385-388, jul.-ago. 2018.
Article
in English
| IBECS
| ID: ibc-177870
Responsible library:
ES1.1
Localization: BNCS
ABSTRACT
Chronic granulomatous disease is a primary immunodeficiency caused by mutations in any one of the five components of the NADPH oxidase in phagocytic leucocytes. This causes impaired microbial killing, which leads to severe life-threatening bacterial and fungal infections. Currently, allogenic hematopoietic stem cell transplantation (HSCT) is the only curative treatment for chronic granulomatous disease, although gene therapy may provide a new therapeutic option for the treatment of patients with CGD. Haploidentical HSCT provides a potentially curative treatment option for patients who lack a suitably HLA-matched donor, but only a few cases have been reported in the literature. Herein, we report a boy with X-linked chronic granulomatous disease treated successfully by haploidentical HSCT with post-transplant cyclophosphamide using a treosulfan-based conditioning regimen
RESUMEN
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Collection:
National databases
/
Spain
Database:
IBECS
Main subject:
Hematopoietic Stem Cell Transplantation
/
Granulomatous Disease, Chronic
Limits:
Child
/
Humans
/
Male
Language:
English
Journal:
Allergol. immunopatol
Year:
2018
Document type:
Article
Institution/Affiliation country:
Hospital Clínico Universitario de Santiago/Spain