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.