Department of Orthopaedic Surgery, Okayama University Graduate School of Medicine, Dentistry, and Pharmaceutical Sciences, 2-5-1 Shikata-cho, Kita-ku, Okayama, Japan.
Department of Musculoskeletal Oncology, National Cancer Center Hospital, 5-1-1 Tsukiji, Chuo-ku, Tokyo, Japan.
BMC Cancer. 2022 Aug 15;22(1):891. doi: 10.1186/s12885-022-09968-5.
Alveolar soft part sarcoma (ASPS) is a rare histological subtype of soft-tissue sarcoma, which remains refractory to conventional cytotoxic chemotherapy. We aimed to characterize ASPS and investigate whether the oncological outcome has improved over the past decade.
One hundred and twenty patients with newly diagnosed ASPS from 2006 to 2017, identified from the Bone and Soft-Tissue Tumor Registry in Japan, were analyzed retrospectively.
The study cohort comprised 34 (28%) patients with localized ASPS and 86 (72%) with metastatic disease at presentation. The 5-year disease-specific survival (DSS) was 68% for all patients and 86% and 62% for localized and metastatic disease, respectively (p = 0.019). Metastasis at presentation was the only adverse prognostic factor for DSS (hazard ratio [HR]: 7.65; p = 0.048). Patients who were > 25 years (80%; p = 0.023), had deep-seated tumors (75%; p = 0.002), and tumors > 5 cm (5-10 cm, 81%; > 10 cm, 81%; p < 0.001) were more likely to have metastases at presentation. In patients with localized ASPS, adjuvant chemotherapy or radiotherapy did not affect survival, and 13 patients (45%) developed distant metastases in the lung (n = 12, 92%) and brain (n = 2, 15%). In patients with metastatic ASPS (lung, n = 85 [99%]; bone, n = 12 [14%]; and brain n = 9 [11%]), surgery for the primary or metastatic site did not affect survival. Prolonged survival was seen in patients who received pazopanib treatment (p = 0.045), but not in those who received doxorubicin-based cytotoxic chemotherapy. Overall, improved DSS for metastatic ASPS has been observed since 2012 (5-year DSS, from 58 to 65%) when pazopanib was approved for advanced diseases, although without a statistically significant difference (p = 0.117).
The national study confirmed a unique feature of ASPS with frequent metastasis to the lung and brain but an indolent clinical course. An overall trend toward prolonged survival after the introduction of targeted therapy encourages continuous efforts to develop novel therapeutic options for this therapeutically resistant soft-tissue sarcoma.
腺泡状软组织肉瘤（ASPS）是一种罕见的软组织肉瘤组织学亚型，对传统细胞毒性化疗仍具有抗药性。我们旨在对 ASPS 进行特征描述，并研究过去十年间肿瘤学预后是否有所改善。
回顾性分析了 2006 年至 2017 年间从日本骨骼和软组织肿瘤登记处确诊的 120 例新发 ASPS 患者。
研究队列包括 34 例（28%）局限性 ASPS 和 86 例（72%）转移性疾病患者。所有患者的 5 年疾病特异性生存率（DSS）为 68%，局限性和转移性疾病分别为 86%和 62%（p=0.019）。初诊时发生转移是影响 DSS 的唯一不良预后因素（风险比[HR]：7.65；p=0.048）。年龄>25 岁（80%；p=0.023）、肿瘤位于深部（75%；p=0.002）和肿瘤>5cm（5-10cm，81%；>10cm，81%；p<0.001）的患者更有可能在初诊时发生转移。局限性 ASPS 患者中，辅助化疗或放疗对生存无影响，13 例（45%）患者在肺部（n=12，92%）和脑部（n=2，15%）发生远处转移。转移性 ASPS 患者（肺部，n=85[99%]；骨骼，n=12[14%]；脑部，n=9[11%]）中，手术治疗原发性或转移性肿瘤部位并不能影响生存。接受帕唑帕尼治疗的患者生存时间延长（p=0.045），而接受多柔比星为基础的细胞毒性化疗的患者则不然。总体而言，自 2012 年帕唑帕尼获批用于晚期疾病以来，转移性 ASPS 的 5 年 DSS 有所改善（从 58%提高至 65%），尽管差异无统计学意义（p=0.117）。
这项全国性研究证实了 ASPS 的一个独特特征，即常向肺部和脑部转移，但临床病程呈惰性。在引入靶向治疗后，生存时间总体呈延长趋势，这鼓励我们继续努力为这种治疗抵抗的软组织肉瘤开发新的治疗选择。
