Activity of @entity2857 against XXXX /deep @entity81 .

@entity1 @entity137 @entity82 @entity5 @entity765 @entity2857 @entity81 @entity204
BACKGROUND: @entity765 (deep @entity81 ) are clonal connective tissue @entity5 that do not metastasize, but have a significant risk of local recurrence, and are associated with morbidity and occasionally mortality. Responses of desmoid @entity1 to @entity2857 on an expanded access program led us to review our experience. METHODS: After Institutional Review Board (IRB) approval, we reviewed data for 26 @entity1 with @entity765 treated with @entity2857 . @entity2857 was administered at 400 mg oral daily and adjusted for @entity137 . RESULTS: @entity2857 was the first-line therapy in 11/26 @entity1 and the remaining 15/26 had received a median of 2 prior lines of therapy. Twenty-three of 26 @entity1 had shown evidence of progressive disease by imaging, whereas 3 @entity1 had achieved maximum benefit or @entity137 with chemotherapy. Sixteen of 22 (   70%) @entity1 reported significant improvement of symptoms. At a median of 6 months (2-29) of treatment, the best response evaluation criteria in @entity5 (RECIST) 1.1 response included 6/24 (25%) @entity1 with partial response (PR), 17/24 (70%) with stable disease, and 1 with progression and @entity204 . Twelve of 13 (92%) @entity1 evaluated by MRI had > 30% decrease in T2 signal intensity, an indirect metric for increased @entity82 and loss of cellularity. Eighty percent of @entity1 with radiological benefit had extra-abdominal desmoids. DISCUSSION: @entity2857 is active against @entity765 . A prospective, randomized clinical trial of @entity2857 against other active agents is warranted. Loss of MRI T2 signal may be a useful surrogate for defining responses, but requires validation by examination of @entity5 pathology.
