Local Treatment of Extremity Desmoid Tumors Does Not Improve Survival, Function
Key findings
- This retrospective study of 85 patients with extremity desmoid tumors compared those treated with local modalities (surgery and/or radiation, with or without additional systemic therapy) and those who were treated only systemically or with observation
- In both the primary and recurrent tumor cohorts, patients treated only with local modalities did not exhibit improved five-year event-free survival compared with those treated without local modalities
- Patient-reported function scores were lowest among patients who underwent two or more surgeries and those treated with both surgery and radiation
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Different physicians and different specialties vary considerably in how they treat desmoid tumors of the extremities, but the usual management is aggressive surgical resection, often paired with radiation. More recent reports, including European consensus guidelines, suggest that the first-line approach to these very locally aggressive but ultimately benign tumors should be with observation or systemic treatment.
The best approach is unclear because previous large studies have evaluated only the conventional oncologic metrics of survival and recurrence. In the first study of patient-reported outcomes, Orthopedic Surgeons Erik T. Newman, MD, and Santiago Lozano Calderon, MD, PhD, both in the Orthopaedic Oncology Service at Massachusetts General Hospital and the Mass General Cancer Center, and colleagues found that aggressive local treatment may have a negative impact on both function and survival. Their report appears in Clinical Orthopaedics and Related Research.
Study Design
The researchers studied 85 patients (90 tumors) who were treated for desmoid fibromatosis of the upper or lower extremities between 1991 and 2017 at two affiliated quaternary-care cancer centers. Patients with tumors of the axilla and buttock/hip girdle were included. A minimum of 12 months of follow-up was required, and the median follow-up was 59 months.
During the study period, there was no formal treatment algorithm in place at the institutions. Of the 56 primary tumors (54 patients), 36 had local treatment only (surgery alone in 35 patients, surgery plus radiation in one), 14 patients had systemic treatment only, five had local plus systemic treatment and one was observed. (For purposes of survival analysis, observation was categorized as systemic treatment.)
There were 101 treatment episodes for recurrent tumors: 60 episodes of local treatment, 24 of systemic treatment, 15 of local plus systemic and two of observation.
The research team attempted to contact all patients to administer the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function Short Form 10a, Upper Extremity Short Form 7a version 2.0 (where applicable) and Pain Interference Short Form 8a. Complete survey data were available for 41 patients (48%).
Event-free Survival
Event-free survival (EFS) did not differ between patients who had local treatment (surgery and/or radiation) compared with those who did not.
- Primary tumor cohort: The five-year EFS rate was 15% for local treatment vs. 44% for systemic-only treatment (P = NS)
- Recurrence cohort: 56% for local treatment vs. 70% for systemic-only treatment (P = NS)
PROMIS Scores
The average PROMIS function scores were lowest for patients who underwent two or more surgical resections (39 for ≥2 resections, 51 for one resection and 47 for 0 resections; P = .03) and for those who received both surgery and radiation, either concurrently or in separate treatment episodes (39 vs. 46; P = .047). Patients with PROMIS pain interference scores below the population-based norm had significantly better function scores than those with higher levels of pain interference (47 vs. 38, P = .006). PROMIS function scores were not influenced by age, location, tumor volume, tumor depth, nerve involvement or disease status at final oncologic follow-up.
A New Treatment Paradigm at Mass General
In light of these findings, the current practice for the treatment of desmoid tumors at Mass General is to favor systemic options aimed at curbing symptoms and tumor progression. All new patients with these tumors are evaluated in a multidisciplinary clinic by a surgeon, a radiation oncologist and a medical oncologist.
While acknowledging that the natural history of desmoid tumors isn't well understood, the researchers say they suspect desmoid tumors may tend to "burn out," regardless of treatment modality. In several studies, the wait-and-see approach has been associated with disease regression or stability in at least half of patients.
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