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Updated: Feb 4, 2026

Establishment of a Primary Culture of Patient-derived Soft Tissue Sarcoma
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Establishment of a Primary Culture of Patient-derived Soft Tissue Sarcoma

Published on: April 11, 2018

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A Phase II Trial of Pazopanib in Patients with Metastatic Alveolar Soft Part Sarcoma

Miso Kim1, Tae Min Kim2, Bhumsuk Keam1

  • 1Department of Internal Medicine, Seoul National University Hospital, Seoul National University Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.

The Oncologist
|September 27, 2018
PubMed
Abstract

Insights

Pazopanib demonstrated modest efficacy in treating metastatic alveolar soft part sarcoma (ASPS). This antiangiogenic drug showed manageable toxicity, supporting further investigation in larger clinical trials for ASPS patients.

Area of Science:

  • Oncology
  • Medical Science
  • Pharmacology

Background:

  • Alveolar soft part sarcoma (ASPS) is a rare malignancy driven by a specific genetic translocation (t(X;17)(p11.2;q25)) leading to angiogenesis.
  • Antiangiogenic therapies are a therapeutic strategy for ASPS due to the upregulation of angiogenesis-related genes.

Purpose of the Study:

  • To evaluate the efficacy and safety of pazopanib in patients with metastatic ASPS.
  • To assess overall response rate (ORR), progression-free survival (PFS), and overall survival (OS) as primary and secondary endpoints.

Main Methods:

  • An open-label, single-arm, multicenter Phase II clinical trial.
  • Pazopanib 800 mg administered once daily to patients with metastatic ASPS.
  • Biomarker analysis included 68Ga-RGD PET scans and NanoString gene expression profiling.

Main Results:

  • One partial response (16.7% ORR) and stable disease in five patients.
  • Median PFS was 5.5 months; median OS was not reached.
  • The treatment was generally well-tolerated, with one case of grade 3 diarrhea being the most significant toxicity.

Conclusions:

  • Pazopanib exhibits modest antitumor activity in metastatic ASPS.
  • The drug presents a manageable toxicity profile for ASPS patients.
  • Further prospective studies with larger cohorts are needed to confirm pazopanib's benefit in ASPS.

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