Desmoid tumor of the small intestine is a locally aggressive mesenchymal neoplasm. When initial systemic medical therapy does not achieve adequate disease control, a defined next-line approach is available, informed by clinical evidence.
This protocol applies after failure of systemic medical treatment — which may include tyrosine kinase inhibitors (sorafenib, pazopanib, imatinib, or sunitinib), or cytotoxic chemotherapy (such as methotrexate plus vinblastine or vinorelbine, or an anthracycline-containing regimen) — where objective tumor response or stable disease on imaging and symptomatic improvement were not achieved.
The next step involves an oral agent from the γ-secretase inhibitor class, used in patients whose disease has progressed through or become refractory to prior therapies. The full eligibility criteria, regimen, and monitoring requirements are available in the complete protocol.
Treatment aims for objective tumor response — including partial response with measurable tumor regression on imaging — alongside a clinically meaningful reduction in symptom burden.