We evaluated the chronic lymphocytic leukemia International Prognostic Index (CLL-IPI) in patients with CLL treated first line with targeted drugs (n = 991) or chemoimmunotherapy (n = 1256). With a median observation time of 40.5 months, the 3-year progression-free survival (PFS) rates for targeted drug–treated patients varied by CLL-IPI risk group: 96.5% (low), 87.6% (intermediate), 82.4% (high), and 78.7% (very high). Differences between consecutive CLL-IPI risk groups were observed for intermediate vs low and high vs intermediate, but not very high vs high. CLL-IPI factors β2-microglobulin, immunoglobulin heavy variable (IGHV) status, and status each retained prognostic value for PFS. The 3-year overall survival (OS) rates by CLL-IPI risk groups were 100%, 96%, 93.9%, and 89.4%, respectively, with no differences between consecutive risk groups. Age, Binet stage, β2-microglobulin, and status each retained prognostic value for OS. In chemoimmunotherapy patients (median observation time, 66.9 months), 3-year PFS rates for CLL-IPI risk groups were 78.1%, 51.4%, 40.1%, and 16.5%, respectively; corresponding 3-year OS rates were 97.4%, 93.1%, 81.8%, and 57.3%. In a matched-pair analysis, PFS differences in targeted therapies (n = 812) vs chemoimmunotherapy (n = 812) across all risk groups and OS differences in all but patients at low risk were demonstrated. The CLL-IPI maintains its prognostic value in predicting PFS outcomes with targeted drugs, but its impact in predicting survival appears diminished. Targeted therapies showed enhanced outcomes over chemoimmunotherapy, highlighting their effectiveness across various risk groups. Our findings support ongoing assessment of prognostic tools in CLL treatment evolution. These trials were registered at as #NCT02345863, #NCT02401503, #NCT02689141, #NCT02445131, #NCT02758665, #NCT02950051, #NCT02242942, #NCT00262782, #NCT00281918, and #NCT01010061.

中文翻译：

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靶向治疗时代重新评估慢性淋巴细胞白血病国际预后指数

我们评估了接受一线靶向药物 (n = 991) 或化学免疫疗法 (n = 1256) 治疗的 CLL 患者的慢性淋巴细胞白血病国际预后指数 (CLL-IPI)。中位观察时间为 40.5 个月，靶向药物治疗患者的 3 年无进展生存 (PFS) 率因 CLL-IPI 风险组而异：96.5%（低）、87.6%（中）、82.4%（高）和 78.7%（非常高）。观察到连续 CLL-IPI 风险组之间的中度与低度以及高度与中度之间的差异，但高度与高度之间的差异不是很高。 CLL-IPI 因素 β2-微球蛋白、免疫球蛋白重变异 (IGHV) 状态以及状态均保留了 PFS 的预后价值。 CLL-IPI 风险组的 3 年总生存率 (OS) 分别为 100%、96%、93.9% 和 89.4%，连续风险组之间没有差异。年龄、Binet 分期、β2-微球蛋白和状态均保留了 OS 的预后价值。在化学免疫治疗患者中（中位观察时间为 66.9 个月），CLL-IPI 风险组的 3 年 PFS 率分别为 78.1%、51.4%、40.1% 和 16.5%；相应的 3 年 OS 率分别为 97.4%、93.1%、81.8% 和 57.3%。在配对分析中，证明了所有风险组中靶向治疗 (n = 812) 与化学免疫治疗 (n = 812) 的 PFS 差异以及除低风险患者外的所有患者的 OS 差异。 CLL-IPI 在预测靶向药物 PFS 结局方面保持其预后价值，但其在预测生存方面的影响似乎减弱。靶向疗法比化学免疫疗法显示出更好的结果，凸显了它们对不同风险群体的有效性。我们的研究结果支持对 CLL 治疗进展中预后工具的持续评估。这些试验注册为#NCT02345863、#NCT02401503、#NCT02689141、#NCT02445131、#NCT02758665、#NCT02950051、#NCT02242942、#NCT00262782、#NCT00281918 和#NCT01010 061.