Efficacy in Adult Patients
Not an actual patient.
Primary analysis
In EPIK-P1
Overgrowth response in adult 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 adult PROS signs and symptoms through the longer-term analysis3
Adult patients (≥18 years of age)
In the longer-term pooled analysis of EPIK-P1 and EPIK-P3, adult patients experienced improvements in fatigue (100.0%, 14/14), limb asymmetry (100.0%, 12/12), vascular malformations (92.3%, 12/13), pain (90.0%, 9/10), disseminated intravascular coagulation (80.0%, 8/10), and other signs and symptoms.
EPIK-P1 + EPIK-P3
VIJOICE and surgeries in adult patients3
Adult patients (≥18 years of age)
No adult patients required any PROS-related surgery during the longer-term evaluation
Two adult patients (11.1%) underwent PROS-related surgeries after treatment initiation, with 3 surgeries performed
One of the 3 surgeries was due to disease improvement; the other 2 were for other reasons