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The Desmoid Tumor Research Foundation

July 2019

Clinical benefit of methotrexate plus vinorelbine chemotherapy for desmoid fibromatosis (DF) and correlation of treatment response with MRI

Author Information

Ingley KM(1)(2), Burtenshaw SM(3), Theobalds NC(2), White LM(4), Blackstein ME(1)(2), Gladdy RA(3)(5), Thipphavong S(6)(4), Gupta AA(1)(2).

(1)Dept of Medical Oncology and Hematology, Princess Margaret Cancer Centre, Toronto, ON. (2)Div of Medical Oncology and Hematology, Sinai Health System, Toronto, ON.(3)Div of General Surgery, Sinai Health System, Dept of Surgery, Univ of Toronto, Toronto, ON. (4)Dept of Medical Imaging, Univ of Toronto, Toronto, ON. (5)Dept of Surgical Oncology, Princess Margaret Cancer Centre, Dept of Surgery, Univ of Toronto, Toronto, ON. (6)Toronto Joint Dept of Medical Imaging, Univ Health Network, Sinai Health System and Women's College Hospital, Toronto, ON.

Publication

Cancer Med

Summary

Desmoid fibromatosis (DF) is a rare fibroblastic proliferation that was historically treated with surgery. We report (a) outcomes using low-dose chemotherapy, methotrexate (MTX), and vinorelbine (VNL) for patients with progressing disease (PD) and (b) whether tumor volume (Vtumor ) and T2 signal on magnetic resonance imaging (MRI) are more reflective of treatment response compared with maximum tumor dimension (Dmax ) defined by RECIST1.1. Patients with biopsy-proven DF, treated with MTX/VNL from 1997 to 2015 were reviewed. MRI for a subset of patients was independently re-evaluated for response by RECIST, Vtumor , and quantitative T2 hyperintensity. Among 48 patients treated for a median 19 months MTX/VNL, only nine (19%) had previous surgery. RECIST-based overall response rate was complete response (CR) 20 (42%) + partial response (PR) 19 (39%), stable disease (SD) 8 (17%), for a clinical benefit rate of 98%. The median progression-free survival (PFS) was 120 months, (95%CI 84-155 months). Thirty-six (75%) patients had not progressed at a median 38 months from treatment completion. Most common grade 1/2 toxicities included nausea (n = 12, 25%) and fatigue (n = 9,19%) with no grade 3/4 toxicities. In 22 patients with serial MRIs, there was a decrease in Dmax mean by 30%, Vtumor by 76%, and in 19/22 (86%) a decrease in T2 signal intensity. Low-dose MTX/VNL for a defined duration has high efficacy with sustained benefit and minimal toxicity for treating DF. Vtumor and T2 signal might better predict treatment response than RECIST.

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