Abstract：
OBJECTIVE: Alveolar soft part sarcoma (ASPS) is an ultra-rare chemo-resistant sarcoma in children, occurring preferentially in young adults. We aimed to describe and compare its clinical presentation and behaviour in children and young adults to determine whether the same therapeutic strategy should be addressed for both populations.
METHODS: National retrospective multicentre study of children (0-18 years) vs. young adults (19-30 years) included in the \"ConticaBase\" sarcoma database, treated for ASPS between 2010 and 2019 with pathology reviewed via the NETSARC + network.
RESULTS: Overall, 45 patients were identified, 19 children (42%) and 26 young adults (58%). All ASPS diagnoses were confirmed with TFE3 rearrangement by immunohistochemistry or FISH. All clinical characteristics were balanced between both populations with frequent metastases at diagnosis (8/19 vs. 10/26). The therapeutic strategy was based on surgery (17/19 vs. 21/26), radiotherapy (8/19 vs. 12/26) ± systemic treatment (8/19 vs. 9/26). In patients with initially localized disease, metastatic relapse occurred only in adults (8/16), whereas metastatic progression was present in both metastatic groups (5/8 vs. 8/10). After a median follow-up of 5.2 years (range, 0.2-12.2), 5-year EFS was 74% [95%CI, 56-96] vs. 47% [30-74] (p = 0.071) respectively, and 5-year OS was 95% [85-100] vs. 85% [70-100] (p = 0.84). For localized tumours, 5-year MFS was 100% [100-100] vs. 60% [39-91] (p = 0.005). The 5-year OS of all patients with metastasis at diagnosis was 80.2% (62.2%-100%).
CONCLUSIONS: ASPS appears to have the overall same clinical characteristics, but a more aggressive behaviour in young adults than in children. However, despite frequent metastases at diagnosis, long-term survival is high in both groups. Overall, the same therapeutic strategies may be considered for both populations.
METHODS: National retrospective multicentre study of children (0-18 years) vs. young adults (19-30 years) included in the \"ConticaBase\" sarcoma database, treated for ASPS between 2010 and 2019 with pathology reviewed via the NETSARC + network.
RESULTS: Overall, 45 patients were identified, 19 children (42%) and 26 young adults (58%). All ASPS diagnoses were confirmed with TFE3 rearrangement by immunohistochemistry or FISH. All clinical characteristics were balanced between both populations with frequent metastases at diagnosis (8/19 vs. 10/26). The therapeutic strategy was based on surgery (17/19 vs. 21/26), radiotherapy (8/19 vs. 12/26) ± systemic treatment (8/19 vs. 9/26). In patients with initially localized disease, metastatic relapse occurred only in adults (8/16), whereas metastatic progression was present in both metastatic groups (5/8 vs. 8/10). After a median follow-up of 5.2 years (range, 0.2-12.2), 5-year EFS was 74% [95%CI, 56-96] vs. 47% [30-74] (p = 0.071) respectively, and 5-year OS was 95% [85-100] vs. 85% [70-100] (p = 0.84). For localized tumours, 5-year MFS was 100% [100-100] vs. 60% [39-91] (p = 0.005). The 5-year OS of all patients with metastasis at diagnosis was 80.2% (62.2%-100%).
CONCLUSIONS: ASPS appears to have the overall same clinical characteristics, but a more aggressive behaviour in young adults than in children. However, despite frequent metastases at diagnosis, long-term survival is high in both groups. Overall, the same therapeutic strategies may be considered for both populations.
摘要：
目的：肺泡软组织肉瘤（ASPS）是一种罕见的儿童化疗耐药肉瘤，优先发生在年轻人中。我们旨在描述和比较其在儿童和年轻人中的临床表现和行为，以确定是否应针对这两个人群采用相同的治疗策略。
方法：全国儿童回顾性多中心研究（0-18岁）与包括在“ConticaBase”肉瘤数据库中的年轻人（19-30岁），2010年至2019年间接受ASPS治疗，并通过NETSARC+网络进行病理学审查。
结果：总体而言，确定了45名患者，19名儿童（42％）和26名年轻人（58％）。所有ASPS诊断均通过免疫组织化学或FISH进行TFE3重排确认。在诊断时频繁转移的两个人群之间，所有临床特征均保持平衡（8/19vs.10/26).治疗策略基于手术（17/19vs.21/26),放射治疗（8/19vs.12/26）±全身治疗（8/19vs.9/26).在最初患有局限性疾病的患者中，转移性复发仅发生在成人（8/16），而在两个转移组中都存在转移性进展(5/8vs.8/10).经过5.2年的中位随访（范围，0.2-12.2),5年期EFS为74%[95CI，56-96]与分别为47%[30-74](p=0.071)，5年OS为95%[85-100]vs.85%[70-100](p=0.84)。对于局部肿瘤，5年MFS为100％[100-100]vs.60%[39-91](p=0.005)。所有诊断为转移的患者的5年OS为80.2%(62.2%-100%)。
结论：ASPS似乎具有总体相同的临床特征，但是年轻人比儿童更具攻击性。然而,尽管在诊断时经常发生转移，两组的长期生存率都很高.总的来说,对于这两个人群,可以考虑采用相同的治疗策略.
方法：全国儿童回顾性多中心研究（0-18岁）与包括在“ConticaBase”肉瘤数据库中的年轻人（19-30岁），2010年至2019年间接受ASPS治疗，并通过NETSARC+网络进行病理学审查。
结果：总体而言，确定了45名患者，19名儿童（42％）和26名年轻人（58％）。所有ASPS诊断均通过免疫组织化学或FISH进行TFE3重排确认。在诊断时频繁转移的两个人群之间，所有临床特征均保持平衡（8/19vs.10/26).治疗策略基于手术（17/19vs.21/26),放射治疗（8/19vs.12/26）±全身治疗（8/19vs.9/26).在最初患有局限性疾病的患者中，转移性复发仅发生在成人（8/16），而在两个转移组中都存在转移性进展(5/8vs.8/10).经过5.2年的中位随访（范围，0.2-12.2),5年期EFS为74%[95CI，56-96]与分别为47%[30-74](p=0.071)，5年OS为95%[85-100]vs.85%[70-100](p=0.84)。对于局部肿瘤，5年MFS为100％[100-100]vs.60%[39-91](p=0.005)。所有诊断为转移的患者的5年OS为80.2%(62.2%-100%)。
结论：ASPS似乎具有总体相同的临床特征，但是年轻人比儿童更具攻击性。然而,尽管在诊断时经常发生转移，两组的长期生存率都很高.总的来说,对于这两个人群,可以考虑采用相同的治疗策略.
