3.5 Treatment Duration and Efficacy
The median duration of treatment on protocol was 1.5 months (range 0.4-7.2; Figure 1 ). Due to travel constraints, three patients were removed from protocol therapy but elected to continue to be treated as per the clinical trial protocol guidelines at their home institution (starred in Figure 1 ). These three patients were followed for an additional 30 days after being removed from protocol therapy and all were alive with disease at the end of this follow-up period.
Of the 12 patients, 10 (83%) patients were evaluable for objective response by investigator assessment (RECIST 1.1) and two (17%) were inevaluable. The two inevaluable patients did not complete disease evaluations before coming off protocol therapy due to toxicity and were classified as non-responders. Of the 10 patients evaluable for response, one patient with Ewing sarcoma had a partial response (PR), seven patients had stable disease (SD) and two patients had progressive disease (PD). Three patients with RECIST non-measurable disease were coded as having responses or non-complete response / non-progressive disease (non-CR/non-PD). Figure 2 shows a waterfall plot of the maximal change in tumor size from baseline for the five patients with measurable disease.