Zöllner Stefan K, Amatruda James F, Bauer Sebastian, Collaud Stéphane, de Álava Enrique, DuBois Steven G, Hardes Jendrik, Hartmann Wolfgang, Kovar Heinrich, Metzler Markus, Shulman David S, Streitbürger Arne, Timmermann Beate, Toretsky Jeffrey A, Uhlenbruch Yasmin, Vieth Volker, Grünewald Thomas G P, Dirksen Uta
Pediatrics III, University Hospital Essen, 45147 Essen, Germany.
West German Cancer Center (WTZ), University Hospital Essen, 45147 Essen, Germany.
J Clin Med. 2021 Apr 14;10(8):1685. doi: 10.3390/jcm10081685.
Ewing sarcoma, a highly aggressive bone and soft-tissue cancer, is considered a prime example of the paradigms of a translocation-positive sarcoma: a genetically rather simple disease with a specific and neomorphic-potential therapeutic target, whose oncogenic role was irrefutably defined decades ago. This is a disease that by definition has micrometastatic disease at diagnosis and a dismal prognosis for patients with macrometastatic or recurrent disease. International collaborations have defined the current standard of care in prospective studies, delivering multiple cycles of systemic therapy combined with local treatment; both are associated with significant morbidity that may result in strong psychological and physical burden for survivors. Nevertheless, the combination of non-directed chemotherapeutics and ever-evolving local modalities nowadays achieve a realistic chance of cure for the majority of patients with Ewing sarcoma. In this review, we focus on the current standard of diagnosis and treatment while attempting to answer some of the most pressing questions in clinical practice. In addition, this review provides scientific answers to clinical phenomena and occasionally defines the resulting translational studies needed to overcome the hurdle of treatment-associated morbidities and, most importantly, non-survival.
尤因肉瘤是一种极具侵袭性的骨与软组织癌症，被视为易位阳性肉瘤范例的典型代表：这是一种遗传上相对简单的疾病，具有特定且具有新形态学潜在治疗靶点，其致癌作用在数十年前就已明确界定。从定义上讲，这种疾病在诊断时就已存在微转移病灶，对于出现大转移灶或复发病例的患者预后很差。国际合作在前瞻性研究中确定了当前的治疗标准，即进行多周期全身治疗并结合局部治疗；这两种治疗方式都伴有显著的发病率，可能给幸存者带来沉重的心理和身体负担。尽管如此，如今非靶向化疗药物与不断发展的局部治疗方式相结合，为大多数尤因肉瘤患者带来了切实的治愈机会。在本综述中，我们聚焦于当前的诊断和治疗标准，同时尝试回答临床实践中一些最紧迫的问题。此外，本综述为临床现象提供科学解答，并偶尔确定为克服与治疗相关的发病率这一障碍，尤其是非生存问题所需开展的转化研究。
