If Active Surveillance is the Standard of Care for Desmoid Patients, When Should Intervention be Considered?

Summary

In this issue, experts at Royal Marsden Hospital report the outcomes for a large cohort of patients with desmoid tumor (DT) who did not undergo intervention at the time of their initial diagnosis. In this vein, the current report describes 584 DT patients during an 18-year period, 168 of whom were managed with active surveillance. Most of these patients did not need any form of additional treatment during the follow-up period. The main indications in those treated were tumor progression, pain, or both, which occurred within 2.5 years after diagnosis. Active surveillance dominated this large series, especially in the latter part of the study period. Indications for upfront surveillance in this series included: no/limited symptoms, slowly growing tumor, no involvement of relevant structures, and/or patient preference. The Royal Marsden cohort was composed of young patients (median age, 42.2 years; range, 11–86 years) with a median tumor size of 5.9 cm and included all disease sites [abdominal wall (36%), extremity (30%), chest wall (18%), intrabdominal (9%), and other (6%)]. Overall, 36% of the patients progressed (most commonly in the upper extremity and chest wall), 36% remained stable, and 27% had partial or complete regression, in keeping with outcomes of initial observation in other institutional series. The only variable that univariate analysis identified with disease progression was age younger than 50 years at diagnosis. In contrast, variables associated with intervention after a period of observation shown by multivariable analysis were: size (??7 cm; p?<?0.05; hazard ratio [HR], 1.8; range, 1.1–2.8), pain (p?<?0.01; HR, 2.1; range, 1.3–3.3), stable disease (p?<?0.01; HR, 2.1; range, 1.3–3.3), and most strikingly, tumor progression (p?<?0.01; HR, 10.5; 4.1–26.7). Although site also was not statistically significant for intervention, the majority of upper-extremity (58%) and chest wall (57%) patients required treatment, consistent with their clinically appreciated aggressive biology. Following methotrexate plus vinca agent chemotherapy, 8 systemic therapy for DT has remained enigmatic, but possibly more sophisticated and convenient. Pazopanib, sorafenib, and possibly gamma-secretase inhibitors offer patients oral therapy with excellent opportunity for disease control. However, one wonders about the emotional impact on patients to continue returning on a regular basis to a (cancer) center for ongoing CT or MRI scans and follow-up assessment with an oncologic provider. Recently, we reported higher levels of anxiety in individuals with DT even compared with sarcoma, which in fact did not ease with treatment, and continued throughout surveillance. At our center, we have the tools to offer routine distress and symptom screening for all patients—however screening falls short unless resources are available to aid in treating patient anxiety. In a culture of active surveillance, treatment for anxiety, distress, and symptoms still needs to be addressed.