预测真性红细胞增多症后和原发性血小板增多症后骨髓纤维化患者生存的临床-分子预后模型

摘要(中文译文)

真性红细胞增多症（PV）和原发性血小板增多症（ET）是具有不同进展为PV后及ET后骨髓纤维化风险的骨髓增殖性肿瘤，后者以下统称为继发性骨髓纤维化（SMF）。目前尚未确定用于SMF风险分层的特异性工具。为建立预测生存的预后模型，我们研究了685例具有JAK2、CALR和MPL注释的SMF患者。整个队列的中位生存期为9.3年（95% CI：8-未达到-NR-）。通过惩罚Cox回归，我们确定了生存的负性预测因素，并根据β风险系数分配分值：血红蛋白水平<11 g/dl、循环原始细胞⩾3%及CALR未突变基因型各分配2分，血小板计数<150×10⁹/l及体质性症状各分配1分，年龄每增加1岁分配0.15分。继发于PV和ET的骨髓纤维化预后模型（MYSEC-PM）将SMF患者分为四个具有不同生存期的风险类别（P<0.0001）：低危（中位生存期NR；133例患者）、中危-1（9.3年，95% CI：8.1-NR；245例患者）、中危-2（4.4年，95% CI：3.2-7.9；126例患者）和高危（2年，95% CI：1.7-3.9；75例患者）。最后，我们发现MYSEC-PM是SMF决策中最合适的工具，可用于临床和试验环境。

Abstract

Polycythemia vera (PV) and essential thrombocythemia (ET) are myeloproliferative neoplasms with variable risk of evolution into post-PV and post-ET myelofibrosis, from now on referred to as secondary myelofibrosis (SMF). No specific tools have been defined for risk stratification in SMF. To develop a prognostic model for predicting survival, we studied 685 JAK2, CALR, and MPL annotated patients with SMF. Median survival of the whole cohort was 9.3 years (95% CI: 8-not reached-NR-). Through penalized Cox regressions we identified negative predictors of survival and according to beta risk coefficients we assigned 2 points to hemoglobin level <11 g/dl, to circulating blasts ⩾3%, and to CALR-unmutated genotype, 1 point to platelet count <150 × 10/l and to constitutional symptoms, and 0.15 points to any year of age. Myelofibrosis Secondary to PV and ET-Prognostic Model (MYSEC-PM) allocated SMF patients into four risk categories with different survival (P<0.0001): low (median survival NR; 133 patients), intermediate-1 (9.3 years, 95% CI: 8.1-NR; 245 patients), intermediate-2 (4.4 years, 95% CI: 3.2-7.9; 126 patients), and high risk (2 years, 95% CI: 1.7-3.9; 75 patients). Finally, we found that the MYSEC-PM represents the most appropriate tool for SMF decision-making to be used in clinical and trial settings.

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