来那度胺
在小型 II 期试验中尚未证明来那度胺(一种沙利度胺衍生物)比沙利度胺更有效,并且如果沙利度胺无效,它也无效。[43]Tefferi A, Cortes J, Verstovsek S, et al. Lenalidomide therapy in myelofibrosis with myeloid metaplasia. Blood. 2006;108:1158-1164.http://bloodjournal.hematologylibrary.org/content/108/4/1158.longhttp://www.ncbi.nlm.nih.gov/pubmed/16609064?tool=bestpractice.com
伊马替尼
在 II 期试验中,伊马替尼(一种酪氨酸激酶抑制剂)没有显示任何有意义的临床疗效。[44]Tefferi A, Mesa RA, Gray LA, et al. Phase 2 trial of imatinib mesylate in myelofibrosis with myeloid metaplasia. Blood. 2002;99:3854-3856.http://bloodjournal.hematologylibrary.org/cgi/content/full/99/10/3854http://www.ncbi.nlm.nih.gov/pubmed/11986248?tool=bestpractice.com
依那西普
在一项小型试验中,依那西普(一种抗肿瘤坏死因子疗法)改善了约 60% 患者的全身症状。[45]Steensma DP, Mesa RA, Li CY, et al. Etanercept, a soluble tumor necrosis factor receptor, palliates constitutional symptoms in patients with myelofibrosis with myeloid metaplasia: results of a pilot study. Blood. 2002;99:2252-2254.http://bloodjournal.hematologylibrary.org/cgi/content/full/99/6/2252http://www.ncbi.nlm.nih.gov/pubmed/11877307?tool=bestpractice.com然而它没有显示改善血球减少症的临床疗效。
硼替佐米
在 I 期和 II 期试验中,对硼替佐米(一种蛋白酶体抑制剂)治疗没有临床反应。[46]Barosi G, Rosti V. Novel strategies for patients with chronic myeloproliferative disorders. Curr Opin Hematol. 2009;16:129-134.http://www.ncbi.nlm.nih.gov/pubmed/19468275?tool=bestpractice.com
JAK2 抑制剂
一些使用不同的 JAK2 抑制剂治疗原发性骨髓纤维化患者 (PMF) 的初期试验报告了该类药物可以缩小脾脏体积,改善全身症状。[28]Verstovsek S, Kantarjian H, Mesa RA, et al. Safety and efficacy of INCB018424, a JAK1 and JAK2 inhibitor, in myelofibrosis. N Engl J Med. 2010;363:1117-1127.http://www.nejm.org/doi/full/10.1056/NEJMoa1002028#t=articlehttp://www.ncbi.nlm.nih.gov/pubmed/20843246?tool=bestpractice.comMyeloproliferative Disorders Research ConsortiumMPN Research Foundation