Imetelstat improves patient-reported outcomes and quality of life in lower-risk myelodysplastic syndromes: results from the phase III IMerge study.

Thus, reduction in RBC transfusion dependency (TD) is a primary therapeutic goal. Imetelstat is a first-in-class, competitive telomerase inhibitor approved to treat certain adult patients with LRMDS with RBC-TD anemia who have not responded to, have lost response to, or are ineligible for erythropoiesis-stimulating agents.

因此，减少RBC输血依赖（TD）是主要的治疗目标。Imetelstat是一种首创的、竞争性的端粒酶抑制剂，已获批准用于治疗某些对红细胞生成刺激剂无反应、失去反应或不适合的成年LR-MDS患者，这些患者有RBC-TD贫血。

In the phase III IMerge study (clinicaltrials.gov identifier: NCT02598661), treatment with imetelstat resulted in clinically meaningful, statistically significant increases in the primary endpoint of ≥8-week RBC transfusion independence (TI) versus placebo.

在III期IMerge研究（临床试验注册号：NCT02598661）中，与安慰剂相比，imetelstat治疗在主要终点≥8周红细胞输血独立性（TI）方面带来了具有临床意义的统计学显著增加。

Because patients with LR-MDS experience detrimental effects on numerous facets of QOL (physical, emotional, social, and functional), these exploratory analyses assessed patient-reported outcomes using the Functional Assessment of Chronic Illness Therapy-Fatigue, Quality of Life in Myelodysplasia Scale, and Functional Assessment of Cancer Therapy-Anemia questionnaires as part of the phase III IMerge study.

由于低危骨髓增生异常综合征（LR-MDS）患者在身体、情感、社交和功能等多个生活方面受到负面影响，这些探索性分析使用慢性疾病功能评估-疲劳、骨髓增生异常综合征生活质量量表和癌症治疗功能评估-贫血问卷作为III期IMerge研究的一部分，评估了患者报告的结果。

Nominal P values were reported.

报告了名义P值。

Fewer imetelstat-treated patients experienced deterioration in fatigue levels and more imetelstat-treated patients experienced sustained improvement in fatigue levels and QOL versus placebo.

与安慰剂相比，接受imetelstat治疗的患者疲劳程度恶化的较少，且疲劳程度和生活质量持续改善的患者更多。

In the imetelstat group, 8-week, 24-week, and 1-year RBC-TI responders had sustained improvements in predefined significance thresholds versus non-responders for fatigue (70%, 73%, and 88%, respectively, vs. 37%, 41%, and 44%, respectively; P<0.001, P=0.004, and P=0.002) and QOL across different measures of response (43-53% vs. 21-30%; P≤0.0126).

在imetelstat组中，8周、24周和1年RBC-TI反应者在疲劳（分别为70%、73%和88% vs. 37%、41%和44%；P<0.001、P=0.004和P=0.002）和生活质量（QOL）的不同测量指标上（43-53% vs. 21-30%；P≤0.0126）与非反应者相比，持续改善至预设的显著性阈值。

These results suggest that treatment with imetelstat may be associated with improvement in QOL beyond fatigue while sustaining RBC-TI in patients with LR-MDS with RBC-TD anemia.

这些结果表明，imetelstat治疗可能与改善LR-MDS患者的QOL相关，而不仅仅是疲劳，同时在RBC-TD贫血患者中维持RBC-TI。