Abstract 1511P
Background
There is a lack of consensus in management of desmoid type fibromatosis (DTF). Sorafenib is preferred for the management of symptomatic patients. In this study, we aim to assess the clinical efficacy and adverse effects of sorafenib therapy in DTF patients.
Methods
Patients age > 18 years with a histological diagnosis of DTF and who received sorafenib were prospectively followed. The primary objective was objective response rate (ORR). Response assessment was based on RECIST 1.1 criteria. Adverse effects were graded according to NCI CTCAE criteria. Progression free survival (PFS) was calculated by plotting Kaplan Meir curves and compared using log-rank test.
Results
A total of 104 patients were eligible for the current analysis. The median age of the population was 32 (range, 18-81) years and 66.35 % of patients were females. Limbs (53.85 %) and abdominal wall/mesentery (21.15 %) were the most commonly involved sites. The most common presenting symptoms were swelling (88%), pain (84%), and restriction of movements (56%). Two-thirds (66.6%) of patients had already received some form of treatment. At the time of analysis, 68 patients were continuing sorafenib. Only 4.8 % had progressed, while 10.5 % stopped therapy due to toxicity and 17.3 % due to other reasons. ORR was 46.1 % and stable disease was seen in 31.7 %. The most common all-grade toxicity was hand-foot syndrome (89.4%) followed by fatigue (79.8 %), alopecia (70.1%), and diarrhea (48.0%). Beta-catenin mutation testing was performed in 15 patients and any mutations were detected in 8 patients.
Conclusions
Sorafenib has good activity in DTF but it is associated with significant toxicity.
Clinical trial identification
Editorial acknowledgement
Legal entity responsible for the study
The authors.
Funding
Has not received any funding.
Disclosure
All authors have declared no conflicts of interest.