TriangleHEALTH PROFESSIONAL · SOURCE READING
Tyrosine kinase inhibitors
Source: Childhood Soft Tissue Sarcoma Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 21, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
Targeted therapy has been used to treat children and adults with desmoid-type fibromatosis.
Evidence (sorafenib):
An international, prospective, phase III, double-blind study was conducted through the National Clinical Trials Network to evaluate the efficacy of sorafenib in patients with unresectable progressive or symptomatic desmoid tumors. Eighty-seven patients were enrolled (aged 18–72 years). Patients were randomly assigned in a 2:1 fashion to receive either sorafenib or placebo. Crossover to sorafenib was permitted after disease progression.[2][Level of evidence B1]
Source links and citations
An international, prospective, phase III, double-blind study was conducted through the National Clinical Trials Network to evaluate the efficacy of sorafenib in patients with unresectable progressive or symptomatic desmoid tumors. Eighty-seven patients were enrolled (aged 18–72 years). Patients were randomly assigned in a 2:1 fashion to receive either sorafenib or placebo. Crossover to sorafenib was permitted after disease progression.[2][Level of evidence B1]
The objective response rate was 33% (95% confidence interval [CI], 20%–48%) in the sorafenib arm and 20% (95% CI, 8%–38%) in the placebo arm.
An international, prospective, phase III, double-blind study was conducted through the National Clinical Trials Network to evaluate the efficacy of sorafenib in patients with unresectable progressive or symptomatic desmoid tumors. Eighty-seven patients were enrolled (aged 18–72 years). Patients were randomly assigned in a 2:1 fashion to receive either sorafenib or placebo. Crossover to sorafenib was permitted after disease progression.[2][Level of evidence B1]
The median time to objective response was 9.5 months for patients treated with sorafenib and 13.3 months for patients who received the placebo.
An international, prospective, phase III, double-blind study was conducted through the National Clinical Trials Network to evaluate the efficacy of sorafenib in patients with unresectable progressive or symptomatic desmoid tumors. Eighty-seven patients were enrolled (aged 18–72 years). Patients were randomly assigned in a 2:1 fashion to receive either sorafenib or placebo. Crossover to sorafenib was permitted after disease progression.[2][Level of evidence B1]
The 2-year PFS rate was 81% for patients treated with sorafenib, compared with 36% for patients who received the placebo.
Evidence (pazopanib):
One small series included six patients (aged 3–21 years) with desmoid-type fibromatosis who were treated with pazopanib.[29]
Source links and citations
One small series included six patients (aged 3–21 years) with desmoid-type fibromatosis who were treated with pazopanib.[29]
Symptomatic improvement and stable disease were reported in all patients.
A randomized noncomparative study included adult patients with desmoid tumors who were treated with either pazopanib or methotrexate/vinblastine.[30]
Source links and citations
A randomized noncomparative study included adult patients with desmoid tumors who were treated with either pazopanib or methotrexate/vinblastine.[30]
About 84% of the patients who received pazopanib had no progression at 6 months.
Publication references
Read the original reference and check its publication notices.
Preserved source evidence · Not medical advice