Alveolar soft part sarcomas in young patients: The French national NETSARC+ network experience

Background and aims: 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.