Long-term outcomes for patients with desmoid fibromatosis treated with radiation therapy: a 10-year update and re-evaluation of the role of radiation therapy for younger patients

We reviewed the records of 209 consecutive patients with desmoid fibromatosis treated with RT, either alone or as combined modality therapy (CMT) with surgery, at our institution from 1965 to 2015. Median follow-up time was 98 months (range, 1-509 months). The 5-year and 10-year LC was 71% and 69%, respectively. 59 patients (28%) experienced a local recurrence at a median time of 23 months (interquartile range, 15-38 months). Among all patients, on multivariable analysis, adjusting for anatomic site, size, age, treatment era (>2005 vs. ≤ 2005), treatment approach (RT alone vs. CMT), and an interaction between age and treatment, we found only age ≤30 yrs (HR 2.94, P=0.005, 95% CI 1.38-6.27) and large tumor size >10cm (HR 2.51, P=0.03, 95% CI 1.09-5.78) to be correlated with poorer LC. Notably, for patients receiving RT alone the 5-year LC was 43% for patients ≤30 yrs vs 75% for >30 yrs (P<0.001). On multivariable analyses, for patients receiving RT alone, the only factor associated with inferior LC was age ≤30 yrs (HR 2.87, P=0.001, 95% CI 1.51-5.47). The same was true for patients treated with CMT; age ≤30 yrs was the only factor associated with inferior LC (HR 5.36, P=0.01, 95% CI 1.40-20.58). Among all patients with desmoid fibromatosis, RT is an effective local therapy for tumor control. However, young patients ≤ 30 yrs have notably high rates of local recurrence regardless of treatment strategy, which requires further study. Treatment decisions should be risk-adapted by large referral centers with multidisciplinary expertise in desmoid management.

We present 3 cases of desmoid fibromatosis post pancreatectomy for pancreatic adenocarcinoma. All cases occurred within 3 years of diagnosis of pancreatic cancer, with subsequent extensive diagnostic work-up to rule out metastatic disease. No relationship between pancreatic cancer and desmoid fibromatosis is documented in the literature, with a postulated connection via mutations on the Wnt/APC/Beta-catenin pathway.

Pediatric desmoid tumor (PDT) is rare and has a high local recurrence rate. The purpose of the present study was to analyze clinical risk factors of local recurrence in PDT patients. We reviewed clinical data of 66 PDT patients from 2004 to 2015. All patients underwent macroscopically complete resection, and some recurrent tumors were prescribed radiotherapy. Factors such as sex, age at presentation, location, and proximity to nerves or vasculature were analyzed. The local recurrence rate and recurrence-free survival were analyzed with these factors. The median follow-up time was 6.6 years. Thirty-six (55%) patients had local recurrence. Age, sex, tumor site, tumor size, and proximity to nerves/vasculature had a significant impact on prognosis in univariate analysis. Radiotherapy decreased the local recurrence rate. In multivariate analysis, younger age, tumor location in buttocks, larger tumor, and proximity to important nerves/vasculature were independent risk factors for poor prognosis. Favorable therapeutic strategies could be selected according to the preoperative prognostic risk factors. Radiotherapy should be considered for local recurrence of PDT.

Desmoid tumors (also referred to as aggressive fibromatosis) are connective tissue neoplasms that can arise in any anatomical location and infiltrate the mesentery, neurovascular structures, and visceral organs. There is no standard of care. In this double-blind, phase 3 trial, we randomly assigned 87 patients with progressive, symptomatic, or recurrent desmoid tumors to receive either sorafenib (400-mg tablet once daily) or matching placebo. Crossover to the sorafenib group was permitted for patients in the placebo group who had disease progression. The primary end point was investigator-assessed progression-free survival; rates of objective response and adverse events were also evaluated. With a median follow-up of 27.2 months, the 2-year progression-free survival rate was 81% (95% confidence interval [CI], 69 to 96) in the sorafenib group and 36% (95% CI, 22 to 57) in the placebo group (hazard ratio for progression or death, 0.13; 95% CI, 0.05 to 0.31; P<0.001). Before crossover, the objective response rate was 33% (95% CI, 20 to 48) in the sorafenib group and 20% (95% CI, 8 to 38) in the placebo group. The median time to an objective response among patients who had a response was 9.6 months (interquartile range, 6.6 to 16.7) in the sorafenib group and 13.3 months (interquartile range, 11.2 to 31.1) in the placebo group. The objective responses are ongoing. Among patients who received sorafenib, the most frequently reported adverse events were grade 1 or 2 events of rash (73%), fatigue (67%), hypertension (55%), and diarrhea (51%). Among patients with progressive, refractory, or symptomatic desmoid tumors, sorafenib significantly prolonged progression-free survival and induced durable responses.

This original report describes the occurrence of a desmoid tumor in the site of renal transplantation, being evaluated as a differential diagnosis, although rare, of primary renal neoplasm of the graft. The understanding of the possibility of this tumor occurrence, simulating primary renal neoplasia (rapid growth and locoregional aggressiveness), would avoid, as in this case, unnecessary graft removal, sparing patients from evolution to dialytic chronic renal disease again.

Desmoid tumors are exceedingly rare within the larynx and cause significant morbidity due to their locally aggressive and infiltrative nature. Surgery is the mainstay of treatment with previous reports describing total and near-total laryngectomy for cure. We present a case of recurrent glottic desmoid tumor managed with hemilaryngectomy and reconstructed with temporoparietal free tissue, rib, and buccal grafts. Three-dimensional modeling was utilized to optimize aerodigestive function after laryngeal reconstruction.