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Pancytopenia-developmental delay syndrome

ORPHA:401764· ICD-10 D61.0

Definition

A rare constitutional aplastic anemia characterized by progressive trilineage bone marrow failure (with hypocellularity), developmental delay with learning disabilities, and microcephaly. Mild facial dysmorphism and hypotonia have also been reported.

Prevalence
<1 / 1 000 000
Inheritance
Autosomal recessive
Age of onset
Adolescent