索拉非尼
索拉非尼是一种批准用于肾脏细胞癌的抗血管生成酪氨酸激酶抑制剂,一项 II 期研究表明索拉非尼在治疗恶性血管内皮瘤方面有一定的作用。[70]Maki RG, D'Adamo DR, Keohan ML, et al. Phase II study of sorafenib in patients with metastatic or recurrent sarcomas. J Clin Oncol. 2009 Jul 1;27(19):3133-40.http://ascopubs.org/doi/full/10.1200/JCO.2008.20.4495?url_ver=Z39.88-2003&rfr_id=ori%3Arid%3Acrossref.org&rfr_dat=cr_pub%3Dpubmed&http://www.ncbi.nlm.nih.gov/pubmed/19451436?tool=bestpractice.com 一项用索拉非尼治疗 51 例恶性血管内皮瘤和 L-肉瘤(脂肪肉瘤和平滑肌肉瘤)患者的进一步 II 期研究发现,肿瘤大小没有改变。[71]von Mehren M, Rankin C, Goldblum JR, et al. Phase 2 Southwest Oncology Group-directed intergroup trial (S0505) of sorafenib in advanced soft tissue sarcomas. Cancer. 2012 Feb 1;118(3):770-6.https://onlinelibrary.wiley.com/doi/full/10.1002/cncr.26334http://www.ncbi.nlm.nih.gov/pubmed/21751200?tool=bestpractice.com 许多人表示,这些 RECIST(实体瘤反应评估标准)标准对生物疗法来说可能还不够。[72]Choi H, Charnsangavej C, Faria SC, et al. Correlation of computed tomography and positron emission tomography in patients with metastatic gastrointestinal stromal tumor treated at a single institution with imatinib mesylate: proposal of new computed tomography response criteria. J Clin Oncol. 2007 May 1;25(13):1753-9.http://ascopubs.org/doi/full/10.1200/JCO.2006.07.3049?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%3dpubmedhttp://www.ncbi.nlm.nih.gov/pubmed/17470865?tool=bestpractice.com 在该项涉及 51 例患者的 II 期研究中,尽管肿瘤没有明确的减小,但是 75% 的恶性血管内皮瘤患者都出现病情稳定和 5 个月的中位无进展生存期,L-肉瘤患者则为 2-3 个月。[71]von Mehren M, Rankin C, Goldblum JR, et al. Phase 2 Southwest Oncology Group-directed intergroup trial (S0505) of sorafenib in advanced soft tissue sarcomas. Cancer. 2012 Feb 1;118(3):770-6.https://onlinelibrary.wiley.com/doi/full/10.1002/cncr.26334http://www.ncbi.nlm.nih.gov/pubmed/21751200?tool=bestpractice.com
贝伐珠单抗
贝伐单抗是一种通常与化疗药物联合应用治疗多种类型肿瘤的抗血管生成药。 病理报告提示其在头皮及面部有恶性血管内皮瘤、软组织腺泡状肉瘤以及复发的儿科型肉瘤。[73]Verschraegen CF, Arias-Pulido H, Lee SJ, et al. Phase IB study of the combination of docetaxel, gemcitabine, and bevacizumab in patients with advanced or recurrent soft tissue sarcoma: the Axtell regimen. Ann Oncol. 2012 Mar;23(3):785-90.https://academic.oup.com/annonc/article-lookup/doi/10.1093/annonc/mdr299http://www.ncbi.nlm.nih.gov/pubmed/21746804?tool=bestpractice.com[74]Mir O, Boudou-Rouquette P, Larousserie F, et al. Durable clinical activity of single-agent bevacizumab in a nonagenarian patient with metastatic alveolar soft part sarcoma. Anticancer Drugs. 2012 Aug;23(7):745-8.http://www.ncbi.nlm.nih.gov/pubmed/22331039?tool=bestpractice.com[75]Hingorani P, Eshun F, White-Collins A, et al. Gemcitabine, docetaxel, and bevacizumab in relapsed and refractory pediatric sarcomas. J Pediatr Hematol Oncol. 2012 Oct;34(7):524-7.http://www.ncbi.nlm.nih.gov/pubmed/23007339?tool=bestpractice.com 已知有血管不良反应,且已有致命性肠穿孔的病例报道,因此在做出加用贝伐珠单抗的决定时应谨慎。[76]Cassier PA, Labidi-Galy SI, Heudel P, et al. Therapeutic pipeline for soft-tissue sarcoma. Expert Opin Pharmacother. 2011 Nov;12(16):2479-91.http://www.ncbi.nlm.nih.gov/pubmed/21913865?tool=bestpractice.com
Aldoxorubicin
Aldoxorubicin 由通过链接器绑定到白蛋白上的多柔比星组成。 在一项针对一线化疗开展的 IIb 期随机临床试验中,Aldoxorubicin 治疗后的肿瘤无进展生存期是单独使用多柔比星的两倍(5.6 个月对比 2.7 个月,p = 0.02)。 重度中性粒细胞减少症在使用 Aldoxorubicin 时更为常见,在发热性中性粒细胞减少症中无增加。 总体生存率未报告。[77]Chawla SP, Papai Z, Mukhametshina G, et al. First-line aldoxorubicin vs doxorubicin in metastatic or locally advanced unresectable soft-tissue sarcoma: a phase 2b randomized clinical trial. JAMA Oncol. 2015 Dec;1(9):1272-80.https://jamanetwork.com/journals/jamaoncology/fullarticle/2436837http://www.ncbi.nlm.nih.gov/pubmed/26378637?tool=bestpractice.com 一项备受期待的 III 期研究正在进行。
有生长因子或自体骨髓/干细胞移植支持的高剂量化疗药物进行化疗
目前还无法将有生长因子支持的高剂量化疗药物化疗推荐用作不能动手术的局部晚期或转移性肿瘤患者的一线处理,且使用自体骨髓或干细胞移植支持的高剂量化疗药物进行化疗的数据尚不充分。 应鼓励情况合适的患者参加评估新方法或药物的临床试验。[78]Verma S, Younus J, Haynes AE, et al. Dose-intensive chemotherapy with growth factor or autologous bone marrow or stem-cell transplant support in first-line treatment of advanced or metastatic adult soft tissue sarcoma: a clinical practice guideline. Curr Oncol. 2008 Apr;15(2):80-4.http://www.current-oncology.com/index.php/oncology/article/view/162/217;http://www.ncbi.nlm.nih.gov/pubmed/18454188?tool=bestpractice.com[79]Verma S, Younus J, Stys-Norman D, et al. Dose-intensive chemotherapy with growth factor or autologous bone marrow/stem cell transplant support in first-line treatment of advanced or metastatic adult soft tissue sarcoma: a systematic review. Cancer. 2008 Mar 15;112(6):1197-205.https://onlinelibrary.wiley.com/doi/full/10.1002/cncr.23302http://www.ncbi.nlm.nih.gov/pubmed/18224666?tool=bestpractice.com