Prader willi syndrome

Author: Sania Shafiq     Published: 3 years ago 3 Replies

>14 years old male , student of 9th class presented with c/o polyphagia , weight gain  and aggressive behaviour since 2 years of age. He also had history of  generalized skeletal pains and had two episodes of urinary retention in last 6 months relieved by catheterization.
> 0/E his weight was 72kg , esotropia  was present, Voice was hoarse with increased nasal tone ,thick skin was noted alongwith mild scoliosis .Sexual characteristics were underdeveloped for the age however he didnt have short stature.Rest of examination was unremarkable.
> BLIs were in range , Growth hormone levels were on  lower limit of normal range while thyroid function tests and serum cortisol were normal.MCUG did not reveal any abnormality.                  

>Prader willi syndrome was suspected  and karyotyping was advised.
FISH and molecular studies are in plan.
>What are major criterias for diagnosis of prader willi syndrome?
>What are indications of growth hormone replacement in Prader willi syndrome?

The diagnosis of Prader-Willi syndrome is highly likely in children younger than 3 years with 5 points (3 from major criteria) or in those older than 3 years with 8 points (4 from major criteria).

  • Major criteria (1 point each)

    • CNS - Infantile central hypotonia

    • GI - Infantile feeding problems and/or failure to thrive.

    • Nutrition - Rapid weight gain in children aged 1-6 years

    • Craniofacial - Characteristic facial features such as narrow bifrontal diameter, almond-shaped palpebral fissures, narrow nasal bridge, and down-turned mouth

    • Endocrine - Hypogonadism

    • Developmental - Developmental delay and/or mental retardation

  • Minor criteria (one half point each)

    • Neurologic - Decreased fetal movement and/or infantile lethargy

    • Pulmonary - Sleep disturbance and/or sleep apnea

    • Endocrine - Short stature for predicted height by mid adolescence

    • Dermatologic - Hypopigmentation

    • Orthopedic - Small hands and feet

    • Orthopedic - Narrow hands with straight ulnar border

    • Ophthalmologic – Esotropia and/or myopia

    • Dental - Thick viscous saliva

    • Otolaryngology - Speech articulation defects

    • Psychiatric - Skin picking

Maria, 3 years ago

In childhood no testing is required,The positive effects of growth hormone(GH) administration are detectable in all children with PWS, independent from the GH secretory status.

The benefits of GH treatment in children with PWS are , 

* positive action on linear growth, body composition, motor function, and psychomotor development. 

*The greater effects are observed in the first years of life, thus recommending the early institution of GH therapy (between 4 and 6 months of age) for PWS children.

*During childhood, long-term GH therapy allows the complete recovery from the growth defect, normalizing the final adult height, and is associated with an increase of lean body mass, adiponectin levels, and head circumference.

*GH administration is effective in increasing muscle strength and motor skills, as well as improving mental speed, flexibility, health-related quality of life, and cognition and behavioral issues

Italian criteria for determining GHD in PWS during transition phase:

(i) three or more pituitary hormone deficiencies, and

(ii) a peak GH level <4.1 μg/L after GHRH plus arginine test after a GH wash-out period prior to retesting of at least 1 month.

*The effects of GH during the transition period are mainly related to the maintenance of body proportions, cardiovascular function, and favorable metabolic indices, as well as to optimize quality of life.

Maria, 3 years ago

Thank you for sharing this interesting case.

This patient had a classical history of polyphagia. His father stated that he does not have any control over his eating. He is hungry all the time, takes very large boluses when he sees food so much so that sometimes the bolus gets stuck in his throat. Sometimes he takes it in without even chewing. At times he takes non-food items too.

He had small external genitalia, poorly developed secondary sexual characteristics, and esotropia. He had neuropsychiatric symptoms, growth hormone was low, and clinically he had hypogonadism too.

His father also stated that one of his siblings has the same problem.

Dr. Ahmed Farhan, 3 years ago

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