Rhabdomyosarcoma in adolescent and young adult patients: current perspectives.
Rhabdomyosarcoma in adolescent and young adult patients: current perspectives.
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DOI：
10.2147/ahmt.s44582
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发表时间：
2014
期刊：
影响因子：
--
通讯作者：
Huh WW
中科院分区：
文献类型：
--
作者：
Egas-Bejar D;Huh WW
横纹肌肉瘤（RMS）是一种间质来源的恶性肿瘤，是儿童和青少年颅外恶性实体瘤中第三常见的肿瘤。然而，在成人中，RMS占所有实体瘤恶性肿瘤的<1%。胚胎和肺泡组织学变异在儿科患者中更常见，而多形性变异在儿童中罕见，在成人中更常见。胚胎和肺泡变体的研究进展提高了我们对RMS中涉及的某些基因和生物学途径的理解，但对其他变体的了解要少得多。综合治疗包括手术和化疗加或不加放射治疗是RMS治疗的主要手段。基于术前检查的儿科患者风险分层的改进（原发肿瘤部位、肿瘤大小、区域淋巴结受累、转移的存在）和术后参数（切除的完整性或存在残留疾病或转移）允许在不同的研究和治疗试验中对患者进行治疗分配，导致在过去40年中5年存活率从25%-70%增加。然而，对于成年患者，在很大程度上由于罕见的疾病和缺乏共识的最佳治疗，临床结果仍然很差。儿童RMS与成人RMS之间的不同结局涉及许多因素，例如缺乏成人RMS患者的标准化治疗方案以及晚期表现的患病率增加。现在有越来越多的幸存者，我们可以欣赏这些患者的治疗后遗症，如骨生长异常，内分泌疾病和不孕症。风险分层的改进导致临床试验使用较低剂量的化疗或放疗，目的是降低副作用的发生率，而不影响生存结局。
Rhabdomyosarcoma (RMS), a malignant tumor of mesenchymal origin, is the third most common extracranial malignant solid tumor in children and adolescents. However, in adults, RMS represents <1% of all solid tumor malignancies. The embryonal and alveolar histologic variants are more commonly seen in pediatric patients, while the pleomorphic variant is rare in children and seen more often in adults. Advances in the research of the embryonal and alveolar variants have improved our understanding of certain genes and biologic pathways that are involved in RMS, but much less is known for the other variants. Multimodality therapy that includes surgery and chemotherapy with or without radiation therapy is the mainstay of treatment for RMS. Improvements in the risk stratification of the pediatric patients based on presurgical (primary tumor site, tumor size, regional lymph node involvement, presence of metastasis) and postsurgical parameters (completeness of resection or presence of residual disease or metastasis) has allowed for the treatment assignment of patients in different studies and therapeutic trials, leading to increases in 5-year survival from 25%–70% over the past 40 years. However, for adult patients, in great part due to rarity of the disease and the lack of consensus on optimal treatment, clinical outcome is still poor. Many factors have been implicated for the differing outcomes between pediatric RMS versus adult RMS, such as the lack of standardized treatment protocols for adult RMS patients and the increased prevalence of advanced presentations. Now that there are increased numbers of survivors, we can appreciate the sequelae from therapy in these patients, such as bone growth abnormalities, endocrinopathies, and infertility. Improvements in risk stratification have led to clinical trials using lower doses of chemotherapy or radiation therapy with the intention of decreasing the incidence of side effects without compromising survival outcome.