Targeted therapy

A small retrospective study of nine adult patients with metastatic alveolar soft part sarcoma treated with sunitinib reported partial responses in five patients and stable disease in two patients.[75][Level of evidence C3]

In another study, 15 adult patients with alveolar soft part sarcoma were treated with sunitinib. Five patients were treated with sunitinib for longer than 2 years.[76][Level of evidence C1]

In another study, 15 adult patients with alveolar soft part sarcoma were treated with sunitinib. Five patients were treated with sunitinib for longer than 2 years.[76][Level of evidence C1]

Six patients experienced partial responses.

In another study, 15 adult patients with alveolar soft part sarcoma were treated with sunitinib. Five patients were treated with sunitinib for longer than 2 years.[76][Level of evidence C1]

The median PFS was 19 months, and the median OS was 56 months.

In another study, 15 adult patients with alveolar soft part sarcoma were treated with sunitinib. Five patients were treated with sunitinib for longer than 2 years.[76][Level of evidence C1]

The 5-year OS rate was 49%.

In a pediatric phase II trial of cediranib, using 70% of the adult maximum tolerated dose in patients younger than 16 years, the following was reported:[77][Level of evidence B4]

In a pediatric phase II trial of cediranib, using 70% of the adult maximum tolerated dose in patients younger than 16 years, the following was reported:[77][Level of evidence B4]

Five of seven patients had stable disease for 14 months or longer.

An international group performed a double-blind, placebo-controlled, randomized, phase II trial of cediranib in adolescent and adult patients with alveolar soft part sarcoma.[78][Level of evidence A1]

An international group performed a double-blind, placebo-controlled, randomized, phase II trial of cediranib in adolescent and adult patients with alveolar soft part sarcoma.[78][Level of evidence A1]

Median percentage change in sum of target marker lesion diameters for the evaluable population was -8.3% (interquartile range [IQR], -26.5 to 5.9) for patients who received cediranib therapy, compared with 13.4% (IQR, 1.1–21.3) for patients who received the placebo (one-sided P = .0010).

An international group performed a double-blind, placebo-controlled, randomized, phase II trial of cediranib in adolescent and adult patients with alveolar soft part sarcoma.[78][Level of evidence A1]

The authors concluded that cediranib is an active agent in patients with alveolar soft part sarcoma.

In a phase II trial of cediranib, 15 of 43 adult patients (35%) with metastatic alveolar soft part sarcoma had partial responses.[79][Level of evidence C3]

In an open-label trial that evaluated the efficacy of pazopanib in six adult patients, one patient achieved a partial response and five patients had stable disease.[80]

Another trial included 30 adult patients who were treated with pazopanib.[81]

Another trial included 30 adult patients who were treated with pazopanib.[81]

One patient experienced a complete response, seven patients experienced partial responses, and 17 patients had stable disease.

Another trial included 30 adult patients who were treated with pazopanib.[81]

The median PFS was 13.6 months.

In one trial, adult patients with advanced sarcomas were treated with a combination of axitinib and pembrolizumab.[74]

In one trial, adult patients with advanced sarcomas were treated with a combination of axitinib and pembrolizumab.[74]

For the 12 patients with alveolar soft part sarcoma, the 3-month PFS rate was 73%.

In one trial, adult patients with advanced sarcomas were treated with a combination of axitinib and pembrolizumab.[74]

Six of eleven patients with evaluable disease had partial responses to axitinib.

In a phase II trial, 52 patients older than 2 years with advanced alveolar soft part sarcoma were treated with atezolizumab.[82]

In a phase II trial, 52 patients older than 2 years with advanced alveolar soft part sarcoma were treated with atezolizumab.[82]

Nineteen patients experienced a response, 18 had partial responses and 1 had a complete response.

In a phase II trial, 52 patients older than 2 years with advanced alveolar soft part sarcoma were treated with atezolizumab.[82]

Based on these data, the FDA approved the use of atezolizumab in children older than 2 years with unresectable or metastatic alveolar soft part sarcoma.