In this analysis we describe the effectiveness of first-line ibrutinib in 747 patients with chronic lymphocytic leukemia (CLL) and TP53 aberrations in a nationwide study with a 100% capture of patients who received the study drug. Median age was 71 years (range 32–95). An estimated treatment persistence rate of 63.4% (95% CI 60.0%-67.0%) and survival rate of 82.6% (95% CI 79.9–85.4%) were recorded at 24 months. Disease progression or death were the reasons for discontinuation in 182/397 patients (45.8%). A higher risk of treatment discontinuation was found to be associated with age, ECOG-PS and pre-existing heart disease, whereas ECOG ≥ 1, age ≥ 70 years and male sex were associated with an increased risk of death. Median post-progression overall survival (OS) was 12.2 months (95% CI 9.2–22.0). Post-discontinuation median OS in patients who discontinued ibrutinib for other reasons was not reached (95% CI 42.3 months – NA). Ibrutinib was an effective first-line treatment for CLL and TP53 aberrations in patients treated at large academic centers and community practice hospitals. Clinical characteristics at baseline may influence the effectiveness of ibrutinib, whereas the experience of prescribing centers and multi-hit or single-hit TP53 aberrations had no impact on outcome in this high-risk population.
在本项全国性研究中，我们通过对接受研究药物的患者实现100%覆盖，描述了747例伴有TP53畸变的慢性淋巴细胞白血病（CLL）患者接受一线伊布替尼治疗的有效性。患者中位年龄为71岁（范围32-95岁）。24个月时估计治疗持续率为63.4%（95% CI 60.0%-67.0%），生存率为82.6%（95% CI 79.9–85.4%）。在397例中止治疗的患者中，182例（45.8%）因疾病进展或死亡而停药。研究发现，较高的停药风险与年龄、ECOG体能状态评分（ECOG-PS）和既存心脏病相关，而ECOG评分≥1分、年龄≥70岁和男性则与死亡风险增加相关。疾病进展后的中位总生存期（OS）为12.2个月（95% CI 9.2–22.0）。因其他原因停用伊布替尼的患者，停药后中位OS尚未达到（95% CI 42.3个月–不可评估）。对于在大型学术中心和社区医院接受治疗的CLL伴TP53畸变患者，伊布替尼是一种有效的一线治疗方案。基线临床特征可能影响伊布替尼疗效，而处方中心的经验以及TP53畸变属多重打击或单一打击，对此高危人群的预后均无显著影响。
肿瘤(癌症)患者之家