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Regressive spondylometaphyseal dysplasia

ORPHA:448267· ICD-10 Q77.8

Definition

A rare, primary bone dysplasia characterized by mild short stature, rhizomelic shortening of the arms and legs, bowing of long bones with widened and irregular metaphyses, thoracolumbar kyphosis, and metacarpal shortening. A marked improvement of the radiologic skeletal features is typical. Pelger-Huet anomaly (i.e. dumbbell shape bilobed nuclei of neutrophils) is a characteristic hematological feature of this disease.

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