Goldschleger Eye Institute, Sheba Medical Center, Affiliated with The Sackler Faculty of Medicine, Tel Aviv University, Tel Aviv, Israel.
SIREDO Oncology Center (Care, Innovation and Research for Children, Adolescents and Young Adults with Cancer), Institut Curie, PSL University, Paris, France.
Acta Ophthalmol. 2021 May;99(3):334-341. doi: 10.1111/aos.14596. Epub 2020 Aug 24.
Localized orbital rhabdomyosarcoma (oRMS) has an overall favourable prognosis with more than 90% of survival. Little is known about the best strategy in recurrent/refractory (R/R) cases. The purpose is to examine the characteristics of patients with R/R-oRMS, focusing on local therapy.
This is bicentric retrospective study. Analysis is of young patients (<30 years) with R/R-oRMS who were treated from 1989 to 2018 at the Institut Curie and Gustave Roussy Cancer Campus, France.
Twenty-seven out of 162 patients (17%) with oRMS presented with R/R disease. 6 of these patients had alveolar RMS (22%), 3 of whom had initial parameningeal extension (11%). During first-line treatment, 18 patients (67%) had orbital radiotherapy. Median age at R/R was 10 years (ranges: 4-28) after a delay of 19 months from diagnosis (ranges: 3-40). Tumoral events were local relapses (22 cases), local progression (3 cases) or regional relapses (2 cases). Second-line treatments included chemotherapy (27 cases), radiotherapy (16 cases), surgery (exenteration; 8 cases) and metastasis/ nodal removal (3 cases). After a median follow-up of 99 months (range: 10-306), 4 patients died and 23 are in complete remission (CR) without treatment. One patient had subsequent relapse treated with exenteration and brachytherapy until a new tumour remission. Five-year event-free and overall survivals after first tumour event are, respectively, 84.4% (95% confidence interval: 71.5%-98.8%) and 85.8% (95% confidence interval: 72.1%-100.0%) CONCLUSION: R/R-oRMS is a rare situation. Second-line therapy is efficient in this location, sometime at the cost of lifesaving mutilating surgery. Second-line local therapy needs therefore to consider local radiotherapy if possible or complete wide surgery.
局部眼眶横纹肌肉瘤（oRMS）的总体预后良好，存活率超过 90%。对于复发性/难治性（R/R）病例，最佳治疗策略知之甚少。本研究旨在探讨 R/R-oRMS 患者的特征，重点关注局部治疗。
这是一项回顾性的、双中心研究。分析对象为 1989 年至 2018 年在法国居里研究所和古斯塔夫·鲁西癌症园区治疗的 R/R-oRMS 年轻患者（<30 岁）。
在 162 例 oRMS 患者中，有 27 例（17%）出现 R/R 疾病。其中 6 例为肺泡横纹肌肉瘤（22%），3 例有初始脑脊膜旁延伸（11%）。在一线治疗中，18 例（67%）患者接受了眼眶放射治疗。R/R 时的中位年龄为 10 岁（范围：4-28 岁），自诊断后延迟 19 个月（范围：3-40 个月）。肿瘤事件包括局部复发（22 例）、局部进展（3 例）或区域性复发（2 例）。二线治疗包括化疗（27 例）、放疗（16 例）、手术（根治性切除术；8 例）和转移/淋巴结切除（3 例）。中位随访 99 个月（范围：10-306 个月）后，4 例患者死亡，23 例患者无治疗的完全缓解（CR）。1 例患者随后复发，接受根治性切除术和近距离放疗，直至新肿瘤缓解。首次肿瘤事件后 5 年无事件生存率和总生存率分别为 84.4%（95%置信区间：71.5%-98.8%）和 85.8%（95%置信区间：72.1%-100.0%）。
R/R-oRMS 较为罕见。二线治疗在此部位有效，有时需要进行挽救生命的致残性手术。因此，二线局部治疗需要考虑局部放疗，如果可能的话，或进行广泛的根治性手术。
