VASE
12 evidence topics · 2 sources
Objective
Location and study date
Study design
Eligibility criteria
Treatment
Systemic sirolimus treatment
| Field | Quoted value |
|---|---|
| Starting dose in children younger than 12 years, mg/m² twice daily | “0.8” |
| Starting dose in older participants | “2 mg/day” |
| Planned treatment duration | “2 years” |
Study outcomes
Rationale for using patient-reported symptoms and functional limitation as the primary endpoint
Statistical analysis description
Number of participants (Planned and analyzed)
Description of analysis sets
Interim statistical approach
Results
Participant disposition
Completion of the planned 2-year treatment period in the interim report
| Outcome | Quoted count |
|---|---|
| Participants completing treatment | “61” |
Baseline characteristics
Efficacy results
Systemic sirolimus: clinical improvement in the interim population
Post-trial personalized-regimen cohort
Health-related quality of life and patient-reported outcomes
Safety results
Adverse-event rates after switching to personalized intermittent or on-demand regimens
Study limitations
Summary
VASE was nonrandomized and included heterogeneous slow-flow vascular malformations. Its interim symptom and safety analysis did not establish a QTORIN-specific or microcystic-specific effect. The authors identify limited pediatric enrollment, small diagnostic subgroups, and limited genetic characterization. The later personalized-regimen analysis included 30 selected adults who had previously completed two years of sirolimus and experienced recurrent symptoms. Its retrospective design and selection of prior responders limit causal comparison of dosing schedules and extrapolation to children or treatment-naive participants.