Treatment Response
Of patients determined to be evaluable for response, 85.7% (96/112)
demonstrated a treatment response at 3 months and 89.1% (114/128) by 6
months. The vincristine treatment group had higher RadR at 3 months
compared to sirolimus (72.7% vs 20%, p=0.03), but equal at 6 months
(76.9% vs 68%, p=0.7). There were no other significant differences in
overall response, HemR, or ClinR between the sirolimus and vincristine
groups at 3 or 6 months (Table 3). Similarly, there were no significant
differences in overall response, RadR, HemR, or ClinR across all
treatment groups (Supplemental Table 2) except RadR in the surgical
resection group at 3 months only.
Based on previously published data showing improved outcomes in patients
with KHE treated with combination of sirolimus and steroids versus
sirolimus alone, we also compared these groups in our cohort (Table
4).31 Small patient numbers precluded statistical
comparisons, but 11 of 15 (73.3%) patients who received sirolimus plus
steroid had response by 3 months compared to only 1 of 4 (25%) patients
who received sirolimus. Treatment responses were not significantly
different amongst those who received sirolimus and steroid in comparison
to those who received vincristine and steroid.