Edahiro Yoko, Gotoh Akihiko, Inano Tadaaki, Tsutsui Miyuki, Tsukune Yutaka, Yasuda Hajime, Komatsu Norio
Department of Hematology, Juntendo University School of Medicine.
Rinsho Ketsueki. 2018;59(3):323-325. doi: 10.11406/rinketsu.59.323.
Primary myelofibrosis (PMF) is commonly associated with anemia. IMiD immunomodulatory drugs including thalidomide and lenalidomide have been shown to be effective in improving anemia associated with PMF. However, because of adverse events, their use has been restricted. Herein we report the case of a 67-year-old male patient with transfusion-dependent PMF treated with the immunomodulatory drug pomalidomide in a clinical trial. Significant improvements in anemia and thrombocytopenia were observed with pomalidomide, and the patient recovered from transfusion dependence for 8 months. Although phase 3 trial failed to show the superiority of pomalidomide over placebo, pomalidomide may have some benefit in selected patients with transfusion-dependent PMF.
原发性骨髓纤维化（PMF）通常与贫血相关。包括沙利度胺和来那度胺在内的免疫调节药物（IMiD）已被证明对改善与PMF相关的贫血有效。然而，由于不良事件，它们的使用受到了限制。在此，我们报告一例67岁男性输血依赖型PMF患者在一项临床试验中接受免疫调节药物泊马度胺治疗的病例。泊马度胺治疗后贫血和血小板减少症有显著改善，患者摆脱输血依赖达8个月。尽管3期试验未能显示泊马度胺优于安慰剂，但泊马度胺可能对某些输血依赖型PMF患者有益。
