Efficacy and safety of luspatercept in non-transfusion-dependent β-thalassemia: long-term results from the BEYOND study.

Luspatercept was previously shown to improve hemoglobin levels in patients with NTDT in the phase 2, randomized, double-blind, placebo-controlledBEYOND trial (ClinicalTrials.gov identifier: NCT03342404).

在先前的2期、随机、双盲、安慰剂对照的BEYOND试验中（ClinicalTrials.gov注册号：NCT03342404），已证实Luspatercept能够提高非输血依赖性β-地中海贫血（NTDT）患者的血红蛋白水平。

Here, we report long-term efficacy and safety results from the final analysis of BEYOND spanning an additional 26 months (∼2.2 years) of follow-up.

在这里，我们报告了BEYOND研究的最终分析结果，该研究额外增加了26个月（约2.2年）的随访时间，以评估长期疗效和安全性。

Median treatment duration was 202.8 weeks for luspatercept and 61.1 weeks for placebo.

对于luspatercept的中位治疗持续时间为202.8周，而安慰剂组为61.1周。

Overall, 94.8% and 22.4% of patients in the luspatercept and placebo arms, respectively, achieved a mean hemoglobin increase from baseline ≥1.0 g/dL during any 12-week interval, with mean durations of response of 1136.0 and 203.3 days, respectively.

在任何12周间隔期间，94.8%和22.4%的患者在luspatercept和安慰剂组分别实现了从基线起平均血红蛋白增加≥1.0 g/dL，平均反应持续时间分别为1136.0天和203.3天。

Patient-reported tiredness and weakness showed sustained improvement with luspatercept treatment.

患者报告的疲劳和虚弱症状在luspatercept治疗下显示出持续改善。

The most common treatment-emergent adverse events in the luspatercept group were headache (45.8% vs 20.4% with placebo), bone pain (43.8% vs 6.1%), back pain (39.6% vs 12.2%), and arthralgia (38.5% vs 16.3%).

在使用luspatercept的患者中，最常见的治疗后不良事件是头痛（45.8%对比安慰剂组的20.4%）、骨痛（43.8%对比6.1%）、背痛（39.6%对比12.2%）和关节痛（38.5%对比16.3%）。

Treatment-emergent extramedullary hematopoiesis events were reported in 12 (9.0%) and 2 (4.1%) patients receiving luspatercept and placebo, respectively, although differences in treatment exposures prevented informative comparisons.

在接受luspatercept和安慰剂治疗的患者中，分别有12例（9.0%）和2例（4.1%）报告了治疗后发生的髓外造血事件，尽管治疗暴露的差异使得无法进行有信息的比较。

Of the 4 patients receiving luspatercept who reported thromboembolic events, all had >1 risk factor.

在接受luspatercept治疗的4名报告血栓栓塞事件的患者中，所有人都有超过1个风险因素。

These results show that luspatercept led to a sustained increase in hemoglobin levels in patients with NTDT for up to ∼4.6 years of treatment, with a consistent safety profile and no new safety findings.

这些结果表明，luspatercept在长达约4.6年的治疗期间，使非输血依赖性地中海贫血（NTDT）患者的血红蛋白水平持续增加，且安全性一致，没有新的安全发现。

Luspatercept is a valuable treatment option for patients with NTDT, addressing the need for effective long-term treatment of anemia.

Luspatercept 是一种宝贵的治疗选择，适用于非输血依赖性地中海贫血（NTDT）患者，满足了有效长期治疗贫血的需求。

This trial was registered at www.clinicaltrials.gov as #NCT03342404.

该试验已在 www.clinicaltrials.gov 注册，注册号为 #NCT03342404。