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.