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.