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Curr Treat Options Oncol. 2013 Sep;14(3):454-64. doi: 10.1007/s11864-013-0239-3.
Sarcoma is a disease that includes many different subtypes that can present with a wide range of differing clinical findings, prognosis, and treatment options. For certain subsets (e.g., Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, gastrointestinal stromal tumor [GIST]), extensive clinical trials have delineated effective treatment regimens often involving combination therapy, including surgery, radiation, systemic chemotherapy, and small molecular inhibitors of tyrosine kinases (as in the case of GIST). For nearly all patients with sarcoma who have relapsed or developed metastasis, the therapeutic benefit of chemotherapy has reached a plateau and as such new treatment approaches are needed to move this field forward. We recommend that all patients have the opportunity to participate in clinical trials where available. Recently, in our clinic we have started to increase our use of molecular testing and DNA sequencing studies to help identify potential treatment options for patients. One area of evolving basic and clinical research in sarcomas is the field of epigenetic therapeutics. The enclosed article reviews the basics of epigenetics and highlights some completed and ongoing clinical trials of epigenetic treatments in sarcoma. We anticipate in the future that diagnostic platforms will be developed to help clinicians determine if an epigenetic therapy could be effective for an individual patient with sarcoma.
肉瘤是一种包含许多不同亚型的疾病,其临床表现、预后和治疗选择差异很大。对于某些亚组(例如尤文肉瘤、骨肉瘤、横纹肌肉瘤、胃肠道间质瘤[GIST]),广泛的临床试验已经确定了有效的治疗方案,通常包括联合治疗,包括手术、放疗、全身化疗和酪氨酸激酶的小分子抑制剂(如 GIST)。对于几乎所有复发或发生转移的肉瘤患者,化疗的治疗益处已达到平台期,因此需要新的治疗方法来推动这一领域的发展。我们建议所有有机会的患者都有机会参加临床试验。最近,在我们的诊所,我们开始增加使用分子检测和 DNA 测序研究来帮助确定患者的潜在治疗选择。肉瘤中一个不断发展的基础和临床研究领域是表观遗传学治疗学。本文综述了表观遗传学的基础知识,并强调了一些已完成和正在进行的肉瘤表观遗传学治疗的临床试验。我们预计,未来将开发诊断平台,以帮助临床医生确定针对个体肉瘤患者的表观遗传学治疗是否有效。