骨髓原始细胞比例对骨髓纤维化患者的临床意义及芦可替尼治疗的影响。
Clinical Significance of Bone Marrow Blast Percentage in Patients With Myelofibrosis and the Effect of Ruxolitinib Therapy.
机构信息
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.
Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, TX.
出版信息
Clin Lymphoma Myeloma Leuk. 2021 May;21(5):318-327.e6. doi: 10.1016/j.clml.2020.12.024. Epub 2021 Jan 6.
BACKGROUND
The effect of bone marrow (BM) blasts on the outcome of patients with myelofibrosis (MF) is poorly understood, unless they are ≥ 10% and represent a more aggressive accelerated phase. Similarly, the role of the JAK inhibitor, ruxolitinib (RUX), has not been assessed in correlation with BM blasts.
PATIENTS AND METHODS
Herein, we present clinical characteristics and outcomes of 1412 patients with MF stratified by BM blasts and therapy.
RESULTS
Seven percent and 4% of patients had 5% to 9% and ≥ 10% BM blasts, respectively. Forty-four percent of patients were treated with RUX throughout their disease course. Overall survival (OS) differed among patients with 0% to 1%, 2% to 4%, and 5% to 9% BM blasts, with median OS of 64, 48, and 22 months, respectively (P < .001). Patients with 5% to 9% BM blasts had similar OS as patients with ≥ 10% BM blasts (22 vs. 14 months; P = .73). All patients with < 10% blasts who were treated with RUX showed superior OS to patients who did not receive RUX.
CONCLUSIONS
Our results indicate that patients with MF with ≥ 5% BM blasts represent a high-risk group with adverse clinical characteristics and inferior outcome. However, they still appear to derive substantial survival benefit from therapy with RUX.
背景
骨髓（BM）原始细胞对骨髓纤维化（MF）患者结局的影响尚不清楚，除非原始细胞≥10%，并代表更具侵袭性的加速期。同样，JAK 抑制剂芦可替尼（RUX）的作用也尚未与 BM 原始细胞相关联进行评估。
患者与方法
在此，我们根据 BM 原始细胞和治疗方法，对 1412 例 MF 患者的临床特征和结局进行了分层。
结果
分别有 7%和 4%的患者存在 5%9%和≥10%的 BM 原始细胞。44%的患者在整个疾病过程中接受了 RUX 治疗。BM 原始细胞为 0%1%、2%4%和 5%9%的患者总生存（OS）不同，中位 OS 分别为 64、48 和 22 个月（P<0.001）。BM 原始细胞为 5%~9%的患者 OS 与原始细胞≥10%的患者相似（22 个月与 14 个月；P=0.73）。所有接受 RUX 治疗且原始细胞<10%的患者 OS 均优于未接受 RUX 治疗的患者。
结论
我们的结果表明，MF 患者的 BM 原始细胞≥5%代表了具有不良临床特征和预后不良的高危人群。然而，他们似乎仍能从 RUX 治疗中获得显著的生存获益。
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