Escolar et al developed a staging system for children with infantile Krabbe disease based on clinical progression. This can help to identify patients likely to benefit from umbilical cord blood transplantation (UCBT). The authors are from the University of North Carolina at Chapel Hill and Duke University.
Enzyme defect: galactocerebrosidase
Patient selection: infantile
Clinical Findings
Clinical Stage
normal development without neurologic signs
Stage 1
normal development with minor, inconclusive neurologic findings
Stage 1
definite neurologic findings that are mild
Stage 2
moderate to severe neurologic disease
Stage 3
advanced neurologic deterioration
Stage 4
An infant in Stage 1 or 2 is a candidate for UCBT and has a good chance for a good survival if the transplant is performed if transplanted during the first weeks of life.
An infant in Stage 3 or 4 is not because of the rate of severe disability or death.
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