Sai Satoshi, Imamura Yoshinori, Kiyota Naomi, Jimbo Naoe, Toyoda Masanori, Funakoshi Yohei, Chayahara Naoko, Hyogo Yasuko, Takenaka Kei, Suto Hirotaka, Minami Hironobu
Department of Medical Oncology/Hematology, Kobe University Hospital and Graduate School of Medicine, Kobe City, Hyogo 650-0017, Japan.
Cancer Center, Kobe University Hospital, Kobe City, Hyogo 650-0017, Japan.
Mol Clin Oncol. 2021 Jan;14(1):6. doi: 10.3892/mco.2020.2168. Epub 2020 Nov 9.
Intimal sarcoma of the pulmonary artery (PAIS) is a rare disease with a poor prognosis. Pazopanib, which has been indicated in metastatic non-adipocytic soft-tissue sarcomas and is expected to be active in PAIS, is a multi-kinase inhibitor that targets the tyrosine kinase activity of vascular endothelial growth factor receptor (VEGFR), platelet-derived growth factor receptor (PDGFR) and stem cell factor receptor. The present study reports findings related to two cases of PAIS with PDGF and VEGF expression following treatment with pazopanib. A case with a moderate to strong expression of PDGFR-α and -β presented a long-term stable disease when treated with pazopanib (progression-free survival, 5.8 months). In a second case with a weak expression of PDGFR-α and -β, the disease progressed rapidly on pazopanib (progression-free survival, 1.1 months). VEGFR-2 was not expressed in the tumors of both cases. The level of PDGFR expression in the tumor tissue may therefore be predictive of pazopanib efficacy.
肺动脉内膜肉瘤（PAIS）是一种罕见疾病，预后较差。帕唑帕尼已被批准用于转移性非脂肪细胞性软组织肉瘤，预计对PAIS也有效，它是一种多激酶抑制剂，可靶向血管内皮生长因子受体（VEGFR）、血小板衍生生长因子受体（PDGFR）和干细胞因子受体的酪氨酸激酶活性。本研究报告了两例PAIS患者在接受帕唑帕尼治疗后PDGF和VEGF表达情况的相关发现。一名PDGFR-α和-β呈中度至强表达的患者在接受帕唑帕尼治疗时出现长期疾病稳定（无进展生存期为5.8个月）。在第二例PDGFR-α和-β呈弱表达的患者中，疾病在帕唑帕尼治疗后迅速进展（无进展生存期为1.1个月）。两例患者的肿瘤中均未检测到VEGFR-2表达。因此，肿瘤组织中PDGFR的表达水平可能预测帕唑帕尼的疗效。
