血细胞减少型骨髓纤维化（MF）患者与增生型克隆性疾病表现型患者相比，治疗选择更有限，预后更差。本文研究了在 RUX-MF 回顾性研究中纳入的886 例使用瑞洛司汀治疗的原发性/继发性 MF（PMF/SMF）患者的预后相关因素。血细胞减少的定义为：白细胞计数<4 × 109/L和/或血红蛋白<11/<10 g/dL（男性/女性）和/或血小板<100 × 109/L。在整体队列中，407例（45.9％）患者患有血细胞减少型 MF，其中249例（52.4％）为PMF。多变量分析表明，高分子风险突变（p=0.04）、中度2/高动态国际预测评分系统（p<0.001）和中度2/高红细胞增多症和Essential Thrombocythemia疾病的MF次发性预后模型（p<0.001）分别与整体队列、PMF和SMF中的血细胞减少型 MF 相关。与表现增生的患者相比，血细胞减少患者在开始治疗时（25.2 mg/日vs.30.2 mg/日，p<0.001）和总剂量上（23.6 mg/日vs.26.8 mg/日，p<0.001）都接受了较低平均瑞洛司汀，并在6个月时获得了较低的脾和症状反应率（26.5％vs。34.1％，p=0.04和59.8％vs。68.8％，p=0.008）。血细胞减少患者还具有更高的3个月血小板减少率（31.1％vs。18.8％，p<0.001），但贫血率较低（65.6％对57.7％，3个月时p=0.02，6个月时为56.6％vs。23.9％，p<0.001）。 在竞争风险分析后，瑞洛司汀中断的5年累积发生率分别为57％和38％在血细胞减少患者和增生型表现患者中（p<0.001），而白血病转化的累积发生率相似（p=0.06）。在动态国际预测评分系统分数调整的 Cox 回归分析中，血细胞减少患者的生存时间显著较短（p<0.001）。血细胞减少的 MF 患者使用瑞洛司汀作为单一治疗的成功率较低并且预后较差。应考虑为这些患者寻找替代治疗策略。

Patients with cytopenic myelofibrosis (MF) have more limited therapeutic options and poorer prognoses compared with patients with the myeloproliferative phenotype.Prognostic correlates of cytopenic phenotype were explored in 886 ruxolitinib-treated patients with primary/secondary MF (PMF/SMF) included in the RUX-MF retrospective study. Cytopenia was defined as: leukocyte count <4 × 109 /L and/or hemoglobin <11/<10 g/dL (males/females) and/or platelets <100 × 109 /L.Overall, 407 (45.9%) patients had a cytopenic MF, including 249 (52.4%) with PMF. In multivariable analysis, high molecular risk mutations (p = .04), intermediate 2/high Dynamic International Prognostic Score System (p < .001) and intermediate 2/high Myelofibrosis Secondary to Polycythemia Vera and Essential Thrombocythemia Prognostic Model (p < .001) remained associated with cytopenic MF in the overall cohort, PMF, and SMF, respectively. Patients with cytopenia received lower average ruxolitinib at the starting (25.2 mg/day vs. 30.2 mg/day, p < .001) and overall doses (23.6 mg/day vs. 26.8 mg/day, p < .001) and achieved lower rates of spleen (26.5% vs. 34.1%, p = .04) and symptom (59.8% vs. 68.8%, p = .008) responses at 6 months compared with patients with the proliferative phenotype. Patients with cytopenia also had higher rates of thrombocytopenia at 3 months (31.1% vs. 18.8%, p < .001) but lower rates of anemia (65.6% vs. 57.7%, p = .02 at 3 months and 56.6% vs. 23.9% at 6 months, p < .001). After competing risk analysis, the cumulative incidence of ruxolitinib discontinuation at 5 years was 57% and 38% in patients with cytopenia and the proliferative phenotype (p < .001), whereas cumulative incidence of leukemic transformation was similar (p = .06). In Cox regression analysis adjusted for Dynamic International Prognostic Score System score, survival was significantly shorter in patients with cytopenia (p < .001).Cytopenic MF has a lower probability of therapeutic success with ruxolitinib as monotherapy and worse outcome. These patients should be considered for alternative therapeutic strategies.© 2023 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.