PROMIS Function Scores Are Lower in Patients Who Underwent More Aggressive Local Treatment for Desmoid Tumors

Summary

Desmoid tumors of the extremities often present with pain and functional limitation, but treatment can lead to morbidity and recurrence is common. In a retrospective review of consecutive patients treated at two institutions, we asked: (1) Is event-free survival (EFS) different between patients who undergo local treatment and those who do not for primary as well as for recurrent desmoid tumors? (2) What treatment-related factors are associated with worse Patient-reported Outcomes Measurement Information System (PROMIS) function scores at a minimum of 1 year after treatment? Between 1991 and 2017, 102 patients with desmoid tumors of the extremities (excluding those of the hands and feet) were treated at two institutions; of those, 85 patients with 90 tumors were followed clinically for at least 1 year (median [range] 59 months follow-up [12 to 293]) and were included in the present analysis. Complete survey data (minimum 1 year follow-up) were available for 46% (39 of 102) of patients with 40 tumors at a median of 125 months follow-up; only these patients were included in PROMIS data analyses. We analyzed 56 primary tumors (54 patients), and 101 discrete treatment episodes for recurrent tumors (88 patients). Among primary tumors, 5-year EFS was 44% (95% CI 24 to 80) for the systemic-only group versus 15% (95% CI 5 to 44) for the local treatment group (p = 0.087). Within the pooled recurrence treatment episode cohort, 5-year EFS after systemic-only treatment was 70% (95% CI 52 to 94) versus 56% among patients receiving any local treatment (95% CI 44 to 70; p = 0.46). PROMIS function scores were lowest among patients who underwent two or more resections (39 versus 51 versus 47 for ?2, 1, and 0 resections, respectively; p = 0.025); among those who received both surgery and radiation at any point, either concurrently or in separate treatment episodes, as compared with those who did not (39 versus 46; p = 0.047); and among those with higher levels of pain interference (38 versus 47 for pain interference scores > 50 versus < 50; p = 0.006). Patients treated with local modalities (surgery and/or radiation, with or without additional systemic therapy) did not experience improved EFS as compared with those treated without local modalities; this was the case for both the primary and the recurrent tumor cohorts. However, PROMIS function scores were lowest among patients who underwent two or more surgical interventions and among those treated with surgery and radiation at any time, suggesting that more aggressive local treatment may be associated with poorer long-term functional outcomes. Prospective collection of patient-reported outcomes data at multiple time points will allow for more direct correlations between treatment modality and impact on function and will help to elucidate the ideal management strategy for these benign but often-symptomatic tumors.