ACHONDROPLASIA 

TOPIC: ACHONDROPLASIA

Etiology

  1. Description:

Characteristics of ACHONDROPLASIA :

  • small stature (may not be present in infants due to them already being small),
  • short limbs and rhizomatic disproportion (present in arms, variable in severity
  • macrocephaly (large head at birth and remains that way for entire life),
  • midfacial retrusion, (underdevelopment of cartilaginous bones of face, results in flattening of entire midface)
  • small chest, (overly compliant ribs, resulting in paradoxical movement with inspiration)
  • thoracolumbar kyphosis, (not present at birth, nearly all infants develop this)
  • lumbar hypertoroids, (exaggerated lordosis, “swayback”, starts when walking begins)
  • limited elbow extension, (stiff elbows, gets worse with age)
  • short fingers and trident configuration of the hands
  • hypermobile hips and knees
  • bowing of the mesial segment of the legs (arises during early childhood)
  • hypotonia (most infants with this are hypotonic, described as “floppy”

Anatomy involved: long bones of arms and legs (bone tissue does not develop properly)

 

Effects on the normal structure and function: results in shortened bones, short stature, results in swayback, bowed legs

  1. Incident Rate/Statistics (What are the national statistics, Does the pathology affect a particular demographic group?)
  • Most common form of disproportionate short stature, occurs in every 15,000 to one in 40,000 live births
  • Affects males and females equally
  • Affects all demographic groups equally

 

  1. Method of ACHONDROPLASIA diagnosis:
  • Requires radiographic assessment (of entire body, hemi-survey, or pelvis and femora)
  • Molecular testing (as needed, very rare this happens, this testing occurs if uncertainty remains after clinical and radiologic assessment)
  • Genetic testing (for individuals who may be too young to make a diagnosis, or people with atypical symptoms)
  1. Treatment/Medication (What is the success rate of drugs?)
  • No treatment that will make it go away but these things can help the problem
  • children born with it need to have weight, height, and head circumference monitored
  • support for social adjustment
  • problems with lower limbs (clonus, hyperreflexia, central hypopnea)
  • surgery (called suboccipital decompression) performed to decrease pressure on brain

 

  1. Prognosis of ACHONDROPLASIA

-What happens if patient receives treatment?

-What happens if patient goes untreated?

Websites:

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6318916/

https://www.genome.gov/Genetic-Disorders/Achondroplasia

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