Adverse Events
Adverse events evaluated in the cohort included multi-organ failure, serious infection, and significant bleeding (Supplemental Table 4). Rates of multi-organ failure and significant bleeding were low across all treatment groups, except combined Siro+VCR treatment group (p=0.002). Infection rates were increased in the vincristine only group compared to sirolimus (24% vs 11.8%, p=0.033). Other adverse events included ptosis secondary to vincristine in 2 patients (one primarily treated with steroids, VCR added later), major airway compromise in 1 patient requiring intubation (Siro+VCR), necrotizing enterocolitis in 1 patient (primary treatment VCR), biliary tract obstruction due to tumor in 1 patient (surgical resection), and severe lymphedema in one Siro patient.