Reichardt Peter, Pink Daniel
Helios-Klinikum Berlin-Buch, Sarkomzentrum Berlin-Brandenburg, Helios-Klinikum Bad Saarow, Sarkomzentrum Berlin-Brandenburg, Klinik für Innere Medizin C der Universitätsmedizin Greifswald.
Dtsch Med Wochenschr. 2021 Feb;146(3):157-161. doi: 10.1055/a-1170-7731. Epub 2021 Jan 29.
Soft tissue sarcomas are rare tumors that represent a major challenge due to varying clinical presentations and often interdisciplinary treatment concepts. Gold standard for the treatment of localized resectable soft tissue sarcomas is complete surgical removal. So far, multimodality treatment does not represent a clininal standard. However, several newer analyses and studies suggest that a subgroup of patients seems to derive an overall survival benefit from perioperative chemotherapy. In metastatic soft tissue sarcoma systemic therapy is the treatment of choice. Most active drugs are the anthracyclines and ifosfamide. Combination chemotherapy has improved both response rate and progression-free survival at the costs of increased toxicity in comparison to single agent therapy but without impact on overall survival in first-line therapy. In pretreated patients, treatment options consist of trabectedin, pazopanib, gemcitabine plus docetaxel or DTIC, and eribulin. Recent data have shown that histiotype-specific treatment options including targeted therapy represent a major improvement for several sarcoma subtypes.In GIST, imatinib is the gold standard for patients with advanced or metastatic disease. In imatinib refractory or intolerant patients, sunitinib in an individualized treatment schedule is recommended. Regorafenib has been approved for third-line therapy. Recently, avapritinib has been approved for treatment of patients with the so far resistant D842V mutation in the PDGFRA exon 18. Ripretinib has shown very promising activity in forth and further lines of therapy and is already approved in the US. The use of adjuvant imatinib therapy in patients with completely resected localized GIST with a high risk of recurrence has significantly improved overall survival with a treatment duration of 3 years. These results have now been confirmed with a 10 years follow-up analysis.
软组织肉瘤是罕见肿瘤，因其临床表现多样且治疗理念常涉及多学科，所以构成了一项重大挑战。局限性可切除软组织肉瘤的治疗金标准是完整手术切除。到目前为止，多模式治疗并非临床标准。然而，一些更新的分析和研究表明，有一部分患者似乎能从围手术期化疗中获得总生存益处。对于转移性软组织肉瘤，全身治疗是首选治疗方法。最有效的药物是蒽环类药物和异环磷酰胺。与单药治疗相比，联合化疗虽以增加毒性为代价提高了缓解率和无进展生存期，但对一线治疗的总生存期无影响。对于经治患者，治疗选择包括曲贝替定、帕唑帕尼、吉西他滨加多西他赛或达卡巴嗪，以及艾瑞布林。最近的数据表明，包括靶向治疗在内的组织学类型特异性治疗选择是几种肉瘤亚型的重大进展。在胃肠道间质瘤（GIST）中，伊马替尼是晚期或转移性疾病患者的金标准。对于伊马替尼难治或不耐受的患者，建议采用个体化治疗方案使用舒尼替尼。瑞戈非尼已被批准用于三线治疗。最近，阿伐替尼已被批准用于治疗血小板衍生生长因子受体A（PDGFRA）外显子18中具有迄今耐药的D842V突变的患者。瑞普替尼在四线及后续治疗中显示出非常有前景的活性，并且已在美国获批。在复发风险高的完全切除的局限性GIST患者中使用辅助伊马替尼治疗，治疗持续时间为3年，显著改善了总生存期。这些结果现已通过10年的随访分析得到证实。
