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Krabbe disease is a rare autosomal recessive genetic neurodegenerative disease caused by galactocerebrosidase (GALC) deficiency.
GALC deficiency can cause the accumulation of galactocerebrosides and its parent cytotoxic compound galactosylsphingosine, both of which are toxic to the central nervous system
The only effective treatment for Krabbe disease is hematopoietic stem cell transplantation (HSCT).
The only effective treatment for Krabbe's disease is hematopoietic stem cell transplantation (HSCT).
This study is a prospective longitudinal study designed to describe the long-term transplant prognosis of children with Krabbe disease who develops between 6 and 36 months of age .
Aims to describe the long-term transplant prognosis of children with Krabbe disease onset between 6 and 36 months of age
The Kaplan-Meier method was used for survival analysis.
Kaplan-Meier curve of overall survival
Kaplan-Meier curve of overall survivalFrom March 1997 to January 2020, a total of 19 children with late-onset Krabbe disease received HSCT treatment.
Compared with untreated children, children receiving transplantation have a higher survival probability, and their cognitive and language functions have been improved to a certain extent
Neurodevelopmental outcome of patients undergoing transplantation
Neurodevelopmental outcome of patients undergoing transplantationThe overall and fine motor development are affected the most, with varying results.
Asymptomatic children benefit the most from transplantation, and all aspects of development are normal or nearly normal
In summary, the study found that HSCT can prolong the life of children with late-onset Krabbe disease and improve their functional capabilities, especially those who have received transplantation before symptoms appear .
HSCT can prolong the life of children with late-onset Krabbe disease and improve their functional capacity, especially those who have received transplantation before symptoms appear
Original source:
Original source:Isabel C.
org/10.
1182/blood.
2020005477" target="_blank" rel="noopener">Long-term neurodevelopmental outcomes of hematopoietic stem cell transplantation for late-infantile Krabbe disease
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