Ferrari Andrea, Dileo Palma, Casanova Michela, Bertulli Rossella, Meazza Cristina, Gandola Lorenza, Navarria Pierina, Collini Paola, Gronchi Alessandro, Olmi Patrizia, Fossati-Bellani Franca, Casali Paolo G
Pediatric Oncology Unit, Istituto Nazionale Tumori, Milan, Italy.
Cancer. 2003 Aug 1;98(3):571-80. doi: 10.1002/cncr.11550.
The goal of the current study was to clarify treatment outcomes for adult patients with rhabdomyosarcoma (RMS). Published series have reported definitively worse results for adults with RMS compared with children with RMS. This finding casts doubt on whether RMS is the same disease in adults as it is in children.
Of 190 patients with RMS who were age 18 years or older and whose cases were recorded over a 25-year span in the pathology database of the Istituto Nazionale Tumori (Milan, Italy), 171 could be analyzed retrospectively for treatment outcome. The authors attempted to stratify patients according to the degree to which they had been treated appropriately, based on current treatment guidelines for childhood RMS.
The overall rate of response to chemotherapy was 85%. For the entire series, 5-year event-free survival and 5-year overall survival (OS) were 28% and 40%, respectively. Among the 110 patients with embryonal, alveolar, or 'not otherwise specified' RMS, 5-year OS was 46%; however, 5-year OS was 61% for patients within this group (39% of the total) who had high scores for appropriate treatment.
The current series parallels other published series in that it confirms the finding of a relatively poor long-term outcome for adult patients with RMS. However, for patients whose treatment adhered to the current guidelines for treatment of children, outcome was similar to what has been reported in pediatric series. In addition, the rate of response to chemotherapy for the entire series was similar to the rate typically observed among children. These findings suggest that adults and children with RMS should receive similar treatment. Treatment protocols adopted from pediatric programs but tailored to adults could increase adults' chances of receiving appropriate treatment; prospective studies are needed to test this idea.
本研究的目的是阐明成年横纹肌肉瘤（RMS）患者的治疗结果。已发表的系列研究报告称，成年RMS患者的结果明显比儿童RMS患者差。这一发现使人怀疑RMS在成人和儿童中是否为同一种疾病。
在意大利米兰国立肿瘤研究所病理数据库中记录的190例18岁及以上的RMS患者中，171例可进行回顾性治疗结果分析。作者试图根据当前儿童RMS治疗指南，将患者按接受适当治疗的程度进行分层。
化疗的总体缓解率为85%。在整个系列中，5年无事件生存率和5年总生存率（OS）分别为28%和40%。在110例胚胎型、肺泡型或“未另行规定”的RMS患者中，5年OS为46%；然而，在该组（占总数的39%）中接受适当治疗评分高的患者，5年OS为61%。
本系列研究与其他已发表的系列研究一致，证实了成年RMS患者长期预后相对较差的发现。然而，对于治疗遵循当前儿童治疗指南的患者，其结果与儿科系列研究报告的结果相似。此外，整个系列的化疗缓解率与儿童中通常观察到的缓解率相似。这些发现表明，成年和儿童RMS患者应接受相似的治疗。采用儿科方案但针对成人进行调整的治疗方案可能会增加成人接受适当治疗的机会；需要进行前瞻性研究来验证这一想法。
