【摘要】 目的:观察口服Janus Kinase（JAK） 1/JAK₂抑制剂芦可替尼（ruxolitinib,捷恪卫）治疗原发性骨髓纤维化（PMF）、特发性血小板增多症继发骨髓纤维化（post-ET MF）患者的疗效,总结治疗3例患者的临床体会。方法:回顾性分析2例PMF、1例post-ET MF患者的临床特点,采用芦可替尼治疗的经过及临床疗效。结果:1例32岁男性PMF患者,MF-3级,羟基脲联合α-干扰素治疗4周,血象下降,Hb 48 g/L,WBC 1. 48×10⁹/L,PLT 41×10⁹/L,芦可替尼以5 mg/d开始,逐渐增加剂量,1个月后血象开始改善,PLT 112×10⁹/L,10个月Hb 109 g/L,WBC 4. 9×10⁹/L,脾脏缩小至正常。1例67岁男性PMF患者,巨脾,芦可替尼10 mg/次,2次/d,2个月后脾脏明显缩小,但患者自行停药2个月,脾脏再肿胀,厚9. 3 cm,Hb 100 g/L,PLT 89×10⁹/L,而WBC 42. 6×10⁹/L,重新口服芦可替尼,4个月后,血象基本正常,脾脏缩小,厚5. 8 cm。1例55岁女性,ET病史20年,间断羟基脲治疗,但继发了MF-3级（post-ET MF）,检测brc/ab L、JAK₂ V617F、CALR、MPL基因均阴性,用芦可替尼10 mg/次,2次/d,3周后血象急剧下降,直到停药13周后WBC、PLT开始恢复。结论:JAK抑制剂芦可替尼治疗JAK₂ V617F、MPL（exon10）基因阳性的PMF,可使肿大的脾脏明显缩小,改善生活质量。但应密切注意其不良反应,特别是极度减少的血细胞。更多还原

【Abstract】 Objective: To observe the curative effects of Janus kinase（ JAK） 1/JAK₂ inhibitor ruxolitinib（ Jakavi） in the treatment of patients with primary myelofibrosis（ PMF） or post essential thrombocythemia myelofibrosis（ post-ET MF）and summarize the clinical experience. Methods: The clinical characteristics,ruxolitinib treatment process and curative effects in 2 cases of PMF and 1 case of post-ET MF were retrospectively analyzed. A 32-year-old male patient was diagnosed as PMF,MF-3. After treatment with hydroxyurea and interferon alpha,his routine blood count began to drop: hemoglobin（ Hb） level was 48 g/L,white blood cell（ WBC） count was 1. 48 × 10⁹/L and platelet（ PLT） count was 41 × 10⁹/L. Afterwards,ruxolitinib（ 5 mg/day） was given. Gradually increasing the dosage of ruxolitinib,PLT was increased to 112 × 10⁹/L after a month. In the tenth month,Hb was 109 g/L,WBC was 4. 9 × 10⁹/L and the spleen volume was reduced to normal. A67-year-old male PMF patient with massive splenomegaly was treated with ruxolitinib（ 10 mg,twice every day）. The splenomegaly was significantly reduced after 2 months. However,in the next 2 months,he stopped taking ruxolitinib without the permission of doctors. The spleen size became larger again,with 9 cm thick. Hb was 100 g/L,PLT was 89 × 10⁹/L and WBC was 42. 6 × 10⁹/L. His routine blood count went back to normal after being retreated with ruxolitinib for four months.The spleen size was decreased to 5. 8 cm thick. A 55-year-old woman,with a history of ET for 20 years,discontinuously taking hydroxyurea,was diagnosed as MF-3（ post-ET MF） later. Assays for BCR/ABL,JAK₂ V617F,CALR and MPL were negative. After treatment with ruxolitinib（ 10 mg,twice every day） for 3 weeks,the routine blood count declined sharply.WBC and PLT began to recover until 13 weeks after drug withdrawal. Conclusions: Treatment with JAK₂ inhibitor ruxolitinib in PMF patients with positive JAK₂ V617F,MPL（ exon10） can alleviate anemia,reduce splenomegaly and improve the quality of life. But we should pay attention to its adverse reaction,especially severe blood cytopenia.更多还原