Table 6.

Targeted Treatment of PIK3CA-Related Overgrowth Spectrum

TreatmentDosageIndicationMechanism
Alpelisib
(VIJOICE®) 1		Treatment regimen in persons age 2 to <18 yrs:
  • Initial dose 50 mg taken orally 1x/day 2 w/food
  • In those age ≥6 yrs, dose may be ↑ to 125 mg 1x/day after 24 wks of treatment. 3
Treatment regimen in persons ≥18 yrs: 250 mg taken orally 1x/day 1 w/food		The treatment of adult & pediatric patients age ≥2 yrs w/severe manifestations of PROS who require systemic therapyPI3K inhibitor

PROS = PIK3CA-related overgrowth spectrum

1.

This medication has been approved specifically for the reduction of overgrowth, vascular lesions, and other functional complications. To date, it is unknown whether this drug has any efficacy in treating the neurologic manifestations of PROS (as, e.g., in MCAP syndrome).

2.

The tablet should be taken at approximately the same time every day

3.

A dose reduction back to 50 gm/day may be considered in those with an adverse reaction on the higher dose.

From: PIK3CA-Related Overgrowth Spectrum

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