Department of Leukemia, The University of Texas MD Anderson Cancer Center, Houston, Texas.
Cancer. 2020 Oct 1;126(19):4322-4331. doi: 10.1002/cncr.33094. Epub 2020 Jul 22.
Circulating blasts (peripheral blood [PB] blasts) ≥1% have long been considered an unfavorable feature for patients with primary myelofibrosis. Whether further quantification of PB blasts and their correlation with bone marrow (BM) blasts have incremental value with regard to patient prognostication is unclear. Similarly, the role of the JAK1/JAK2 inhibitor ruxolitinib (RUX) is not well defined in patients who have increased blasts.
The authors retrospectively studied 1316 patients with myelofibrosis who presented at their institution between 1984 and 2018 and had available PB and BM blasts.
The PB blast percentage influenced overall survival (OS) only among patients who had BM blasts <5%, with a median OS of 64 months for patients with 0% PB blasts, 48 months for those with 1% to 3% PB blasts, and 22 months for those with 4% PB blasts (P < .01). Patients who had 4% PB blasts and 5% to 9% BM/PB blasts had clinical features similar to those of patients who had 10% to 19% blasts. Although the OS of the former patients was longer than in patients who had 10% to 19% blasts, it was not statistically different (median OS: 22, 26, and 13 months, respectively; P > .05). Forty-four percent of patients received RUX throughout their disease course. All patients who had <10% blasts (PB or BM) and received treatment with RUX had superior OS compared with those who did not receive RUX within the same group. PB blasts ≥4% and BM blasts ≥5% were significant for predicting inferior survival in multivariate analysis.
The current results provide comprehensive insight into the role of peripheral blasts in patients with myelofibrosis and indicates that patients who have PB blasts ≥4% have an unfavorable prognosis. RUX provides a survival benefit to patients who have PB blasts <10%.
外周血原始细胞（PB 原始细胞）≥1%长期以来一直被认为是原发性骨髓纤维化患者的不良特征。进一步量化 PB 原始细胞及其与骨髓（BM）原始细胞的相关性是否对患者预后有增量价值尚不清楚。同样，JAK1/JAK2 抑制剂芦可替尼（RUX）在 PB 原始细胞增多的患者中的作用也没有得到很好的定义。
作者回顾性研究了 1984 年至 2018 年间在其机构就诊的 1316 例骨髓纤维化患者，这些患者均有 PB 和 BM 原始细胞数据。
PB 原始细胞比例仅影响 BM 原始细胞<5%的患者的总生存（OS），0%PB 原始细胞患者的中位 OS 为 64 个月，1%至 3%PB 原始细胞患者的中位 OS 为 48 个月，4%PB 原始细胞患者的中位 OS 为 22 个月（P<.01）。PB 原始细胞为 4%且 BM/PB 原始细胞为 5%至 9%的患者具有与 PB 原始细胞为 10%至 19%的患者相似的临床特征。虽然前者的 OS 长于 PB 原始细胞为 10%至 19%的患者，但差异无统计学意义（中位 OS：分别为 22、26 和 13 个月；P>.05）。44%的患者在整个病程中接受了 RUX 治疗。所有 PB 原始细胞<10%（PB 或 BM）且接受 RUX 治疗的患者的 OS 均优于同一组中未接受 RUX 治疗的患者。多变量分析显示，PB 原始细胞≥4%和 BM 原始细胞≥5%是预测生存不良的显著因素。
目前的结果提供了对骨髓纤维化患者外周原始细胞作用的全面了解，并表明 PB 原始细胞≥4%的患者预后不良。RUX 为 PB 原始细胞<10%的患者提供了生存获益。
