Efficacy in Pediatric Patients
Not an actual patient.
Primary analysis
In EPIK-P1
Overgrowth response in pediatric patients1,2
Response, as determined by BICR, was defined as:
The proportion of patients achieving a ≥20% reduction from baseline in the sum of measurable target lesion volume (1 to 3 lesions) confirmed by at least 1 subsequent imaging assessment, provided that:
None of the individual target lesions had a ≥20% increase from baseline,
Nontarget lesions had not progressed, and
There were no new lesions, as determined by BICR
†Patients without any response assessment at Week 24 were considered nonresponders.
Longer-term results
EPIK-P1 + EPIK-P3
Improvements in pediatric PROS signs and symptoms through the longer-term analysis3
Pediatric patients (<18 years of age)
In the longer-term pooled analysis of EPIK-P1 and EPIK-P3, pediatric patients experienced improvements in fatigue (100.0%, 28/28), vascular malformations (92.0%, 23/25), disseminated intravascular coagulation (89.5%, 17/19), lipomatosis (88.2%, 15/17), limb asymmetry (76.5%, 13/17), scoliosis (64.7%, 11/17), and other signs and symptoms.
EPIK-P1 + EPIK-P3
VIJOICE and surgeries in pediatric patients3
Pediatric patients (<18 years of age)
Three pediatric patients required progression-related surgery during the longer-term evaluation
Twelve pediatric patients (30.8%) underwent PROS-related surgeries after treatment initiation; 15 surgeries were performed
Since the start of the analysis, 3 patients underwent surgeries due to disease progression
Six surgeries were performed due to disease improvement
Seven surgeries were performed for other reasons