Ruiz-Mesa Catalina, Goldberg John M, Coronado Munoz Alvaro J, Dumont Sarah N, Trent Jonathan C
Department of Pediatrics, University of Miami/Holtz Children's Hospital at Jackson Health System, 1611 NW 12th Ave, Holtz Hospital 6th floor, Miami, FL, 33136, USA,
Curr Treat Options Oncol. 2015 Jun;16(6):27. doi: 10.1007/s11864-015-0342-8.
Rhabdomyosarcoma (RMS) is well known as a pediatric disease. Most of the knowledge, like biology, genetics, and treatments of this disease, comes from studies done in that age group. The two subtypes of RMS, embryonic RMS and alveolar RMS, that affect mainly the pediatric population are well described in the literature and that has had an impact on the improvement in overall survival during the past 20 years. RMS in the adult population has a low incidence, therefor the study of RMS in this group is challenging. Pleomorphic RMS is the subtype that mainly affects adults and its biology and genetics are not yet completely understood and described. The risk factors for this tumor and the differences among adults and children is also poorly understood. The treatments for adults that have RMS are not standardized having an impact on the overall survival. Pleomorphic RMS has, compared to other adult sarcomas, poor overall survival. Adult patients with RMS have poor prognosis. The standardization of treatments for the adult population is necessary as maybe new treatments for this specific group. There are new treatment options that are being studied mostly in pediatrics and young adults. Immunotherapy is currently proposed as an important treatment possibility including different techniques like vaccination, antigen-mediated therapy, and immune checkpoints. Even if we have a better understanding of RMS, there are still unanswered questions. The improvements seen in the pediatric population are encouraging, but there is still the need to enhance better therapies for adults with RMS.
横纹肌肉瘤(RMS)是一种广为人知的儿科疾病。关于这种疾病的大多数知识,如生物学、遗传学和治疗方法,都来自于对该年龄组的研究。RMS的两种主要影响儿科人群的亚型,胚胎型RMS和肺泡型RMS,在文献中有详细描述,这对过去20年总体生存率的提高产生了影响。成人RMS的发病率较低,因此对这一群体中RMS的研究具有挑战性。多形性RMS是主要影响成人的亚型,其生物学和遗传学尚未完全了解和描述。这种肿瘤的危险因素以及成人和儿童之间的差异也知之甚少。成人RMS的治疗方法尚未标准化,这对总体生存率产生了影响。与其他成人肉瘤相比,多形性RMS的总体生存率较低。成人RMS患者预后较差。成人治疗方法的标准化是必要的,因为这可能为这一特定群体带来新的治疗方法。目前正在研究的新治疗选择大多针对儿科和年轻成人。免疫疗法目前被认为是一种重要的治疗可能性,包括疫苗接种、抗原介导疗法和免疫检查点等不同技术。即使我们对RMS有了更好的了解,仍然存在一些未解决的问题。儿科人群中看到的改善令人鼓舞,但仍需要为成人RMS患者开发更好的治疗方法。