Department of Biostatistics and Epidemiology, Gustave Roussy, Villejuif, France.
Oncostat U1018, Inserm, University Paris-Saclay, Labeled Ligue Contre Le Cancer, Villejuif, France.
Qual Life Res. 2021 Jul;30(7):2021-2032. doi: 10.1007/s11136-021-02794-5. Epub 2021 Mar 2.
Tyrosine kinase inhibitors (TKIs) have dramatically improved the prognosis of chronic myeloid leukemia (CML). We aimed to assess health state utility and quality of life (QoL) in French patients with CML in real-life setting, to study the determinants of utility score and to compare health-related QoL values to general population norms.
We conducted a cross-sectional study in 412 patients with CML. Data were collected by electronic survey. Three patient-reported outcomes questionnaires were used: EORTC QLQ-C30, EORTC QLQ-CML24 and EuroQol EQ-5D-3L. Health state utility values were computed using the French value set. We computed deviations from reference norms from the general population. We studied the determinants of health utility score using multiple regression models.
The mean utility score (SD) was 0.72 (0.25) in the chronic phase and 0.84 (0.21) in treatment-free remission, with marked variations by gender. Patients with CML had a deviation from the reference norm of -0.15 on average (SD: 0.25). In terms of QoL, social functioning, role functioning and cognitive functioning were notably impacted with a mean difference of -16.0, -13.1 and -11.7 respectively. Fatigue, dyspnea and pain were the symptoms with the highest deviation from general population norms (mean difference of 20.6, 14.0 and 8.3 respectively). In the multiple regression analysis, fatigue was the most important independent predictor of the utility score.
Although TKIs prevent the disease from progressing and even allow remission without treatment, QoL in patients with CML is notably altered. The utility scores deteriorate with CML symptoms.
酪氨酸激酶抑制剂（TKI）显著改善了慢性髓性白血病（CML）的预后。我们旨在评估真实环境下法国 CML 患者的健康状态效用和生活质量（QoL），研究效用评分的决定因素，并将健康相关 QoL 值与一般人群的正常值进行比较。
我们对 412 名 CML 患者进行了横断面研究。数据通过电子调查收集。使用了三个患者报告的结局问卷：EORTC QLQ-C30、EORTC QLQ-CML24 和 EuroQol EQ-5D-3L。使用法国价值集计算健康状态效用值。我们计算了与一般人群参考值的偏差。我们使用多元回归模型研究了健康效用评分的决定因素。
慢性期的平均效用评分（SD）为 0.72（0.25），无治疗缓解期为 0.84（0.21），性别差异显著。CML 患者的平均参考值偏差为-0.15（SD：0.25）。在 QoL 方面，社会功能、角色功能和认知功能受到明显影响，平均差异分别为-16.0、-13.1 和-11.7。疲劳、呼吸困难和疼痛是与一般人群正常值偏差最大的症状（平均差异分别为 20.6、14.0 和 8.3）。在多元回归分析中，疲劳是效用评分的最重要独立预测因素。
尽管 TKI 可防止疾病进展，甚至在无需治疗的情况下实现缓解，但 CML 患者的 QoL 明显改变。CML 症状会使效用评分恶化。
