芦可替尼治疗骨髓纤维化阶段性疗效及安全性评估

Phased efficacy and safety of ruxolitinib in the treatment of myelofibrosis

目的评价芦可替尼治疗骨髓纤维化(myelofibrosis,MF)患者的疗效及安全性。方法回顾MPN症状评估表(MPN-SAF-TSS)评价症状,B超评估脾脏大小,观察骨髓纤维化及JAK2V617F基因突变负荷变化和不良反应情况,同时探讨芦可替尼对患者免疫功能的影响。结果服用芦可替尼12个月,51.67%的MF患者总症状评分降低50%以上;脾脏中位长厚径分别由基线的(16.65±3.42)、(5.86±1.54)cm,缩短至(14.93±3.02)、(5.32±1.29)cm,差异有统计学意义(P<0.001);92.86%患者骨髓纤维化稳定或改善,45例患者合并JAK2V617F等位基因突变,基因突变负荷中位差异无统计学意义(P=0.600)。血液学不良反应主要为贫血(68.09%)和血小板减少(40.43%);非血液学不良反应主要观察到感染的发生(23.33%);用计学意义(P=0.020)。结论随访1年的结果显示芦可替尼用于治疗骨髓纤维化患者是安全且有效的。

Objective To explore the efficacy and safety of ruxolitinib in the treatment of myelofibrosis(MF).Methods This study enrolled 60 cases with MF,aged over 18 years and diagnosed with PMF,PPV,or PET in Nanfang Hospital from June 2017 and May 2019.All patients received ruxolitinib over 12 months.The symp⁃toms were evaluated by MPN Symptom Assessment Form⁃Total symptom score(MPN⁃SAF⁃TSS)and B⁃ultrasound was used to assess the size of spleen.The improvement of bone marrow fibrosis,JAK2V617 gene mutation burden and adverse events were observed and the effect of ruxolitinib on lymphocytes was analyzed.Results The total symptom score reduced at least 50%in 51.67%of the patients.The median length and thickness of the spleen were shortened and reduced from(16.65±3.42)cm and(5.86±1.54)cm at baseline to(14.93±3.02)cm and(5.32±1.29)cm respectively(P<0.001).A total of 92.86%of the patients were with stable or improved bone marrow fibrosis;45 patients with JAK2V617F gene mutation and the median mutation burden decreased by 0.90%(P=0.600).The most common hematologic adverse events were anemia(68.09%)and thrombocytopenia(40.43%)and infection(23.33%)was the frequently⁃seen non⁃hematologic adverse event.CD3+,CD4+,CD8+,and NK cells were reduced after the treatment,and the difference in terms of NK cells before and after the treatment was statistically significant(P=0.020).Conclusion Ruxolitinib is safe and effective for the treatment of patients with myelofibrosis.