Efficacy in the Full Population
Not actual patients.
Primary analysis
VIJOICE shrunk overgrowth (EPIK-P1)1
Two additional patients experienced a response, but the response was not confirmed by BICR.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 that1:
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
DURABLE RESPONSE
The median duration of response was not reached as of the data cutoff. The median time to censoring for this analysis was 63 weeks (range: 1 day to 187 weeks).1,2
Patients maintained response1,2
†Patients without any response assessment at Week 24 were considered nonresponders.
Longer-term results
In an exploratory analysis of EPIK-P1 + EPIK-P3,
Improvements in the most common PROS signs and symptoms3
Percentage of patients with improvements at the end of the pooling period in the most commonly reported signs and symptoms3
Improvement was defined based on CTCAE grade reduction or resolution of the event. Percentages were calculated based on the number of patients who reported the event at baseline.3
In EPIK-P1 + EPIK-P3
Functionality assessment through the longer-term analysis2,3
Performance status was recorded at baseline for 47 patients (33 pediatric patients; 14 adult patients) using ECOG, Karnofsky, and Lansky assessments.2
For the ECOG† scale, improvement is defined as a decrease by at least 1 point and worsening is defined as an increase by at least 1 point. For the Karnofsky and Lansky‡ scales, improvement is defined as an increase by at least 20 points, worsening is defined as a decrease by at least 20 points, and stable is defined as a change in score between -20 and 20 points.2,3
No other scales were reported for change in score. Change in score is only applicable if both index and post-index date information are available. Patients may have started with Lansky at index and switched to Karnofsky at a later time.2,3
†ECOG scales assess level of functioning by measuring a patient’s daily activity, physical ability, and ability to care for oneself.4
‡Lansky and Karnofsky scales assess a patient’s ability to perform normal activities of daily living and functional status.4,5