Systemic therapy for desmoid tumors

NSAID: nonsteroidal antiinflammatory drug; PLD: pegylated liposomal doxorubicin; DOXO: doxorubicin; DTIC: dacarbazine; TKI: tyrosine kinase inhibitor; MTX: methotrexate; VLB: vinblastine; PR: partial response; CR: complete response; SD: stable disease; PD: progressive disease; RT: radiation therapy; FAP: familial adenomatous polyposis; MRI: magnetic resonance imaging.
* The urgency to induce a response is based on the severity of symptoms, tumor location and underlying disorder (ie, FAP), and expected harm in the absence of rapid response.
¶ Includes most patients with a slowly growing intraabdominal desmoid in the setting of FAP that involves the mesentery or encases vessels and/or organs, and collagenous mesenteric desmoids with low contrast uptake on MRI.
Δ For patients with large, slowly growing intraabdominal/mesenteric desmoids in the setting of FAP, initial noncytotoxic systemic therapy is also an alternative to initial surgery or observation. This is a controversial area.
◊ At some institutions, a TKI is preferred over hormone therapy with or without an NSAID due to the higher response rate; options include sorafenib, pazopanib, and sunitinib. For patients with a mesenteric tumor and impaired function of the gastrointestinal tract, where oral absorption is unpredictable, and if there is concern for perforation with TKI therapy, systemic chemotherapy may be preferred.
§ TKIs may be reasonable first-line alternatives if cytotoxic chemotherapy is not feasible or because of patient preference; options include sorafenib, pazopanib, and sunitinib.
¥ Response may take >6 months to appear; we would not alter the treatment approach at 3 months unless there is progressive disease or worsening symptoms.