Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.

Exagamglogene autotemcel (exa-cel), a one-time, ex vivo CRISPR/Cas9 gene-edited cell therapy, eliminated VOCs in 97% of participants with severe SCD in the phase 3 CLIMB SCD-121 trial. Here, we describe changes in patient-reported outcomes (PROs) in 30 adults and 12 adolescents with ≥16 months follow-up.

Exagamglogene autotemcel（exa-cel），一种一次性、体外CRISPR/Cas9基因编辑细胞疗法，在CLIMB SCD-121试验的第三阶段中，消除了97%严重SCD参与者的VOCs。在这里，我们描述了30名成人和12名青少年患者在≥16个月随访期间患者报告的结果（PROs）的变化。

Adult PROs include EuroQol Quality of Life Scale-5 dimensions-5 levels of severity (EQ-5D-5L), Functional Assessment of Cancer Therapy Bone Marrow Transplant (FACT-BMT), Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me), and 11-point pain Numerical Rating Scale (NRS).

成人患者报告的结果( PROs)包括欧洲五维生活质量量表-5维度-5级严重程度(EQ-5D-5L)、癌症治疗骨髓移植功能评估(FACT-BMT)、成人镰状细胞病生活质量测量信息系统(ASCQ-Me)以及11点疼痛数字评分量表(NRS)。

In adolescents, EuroQol Quality of Life Scale-5 dimensions-youth (EQ-5D-Y), Pediatric Quality of Life Inventory (PedsQL), and pain NRS were used.

在青少年中，使用了欧洲五维生活质量量表-5维度-青少年版(EQ-5D-Y)、儿童生活质量清单(PedsQL)和疼痛NRS。

Adult EQ-5D-5L US and UK healthy utility index and EQ visual analog scale (VAS) scores, which were lower than general population norm at baseline, substantially improved by month 6 after exa-cel infusion and were sustained through month 36, with similar improvements in FACT-General (FACT-G) total and bone marrow transplant subscale.

成人EQ-5D-5L美国和英国健康效用指数以及EQ视觉模拟量表(VAS)得分，在基线时低于一般人群标准，但在接受exa-cel输注后6个月内显著改善，并持续至36个月，同时FACT-General(Fact-G)总分和骨髓移植亚量表也有类似改善。

All FACT-G (physical, social/family, emotional, functional well-being) and ASCQ-Me (emotional, social functioning, stiffness, sleep impact) subscales showed clinically meaningful improvement, including ASCQ-Me pain subscales (impact, episode frequency, severity), with substantial decreases in pain episode frequency by month 6.

所有FACT-G（身体、社会/家庭、情感、功能福祉）和ASCQ-Me（情感、社会功能、僵硬、睡眠影响）亚量表均显示出临床上有意义的改善，包括ASCQ-Me疼痛亚量表（影响、发作频率、严重程度），疼痛发作频率在6个月内显著减少。

Mean pain NRS score decreased by month 6 and was sustained.

平均疼痛NRS评分在6个月内下降并得以维持。

Consistent with adults, adolescents had improvements in mean EQ-5D-Y VAS score, PedsQL score, and pain NRS.

与成人一致，青少年在平均EQ-5D-Y VAS评分、PedsQL评分和疼痛NRS方面都有所改善。

Exa-cel led to broad and clinically meaningful HRQoL benefits in adults and adolescents with SCD.

Exa-cel 在成年和青少年镰状细胞病（SCD）患者中带来了广泛且具有临床意义的健康相关生活质量（HRQoL）改善。

The CLIMB SCD-121 and CLIMB-131 trials were registered at www.clinicaltrials.gov as #NCT03745287 and #NCT04208529, respectively.

CLIMB SCD-121 和 CLIMB-131 试验分别在 www.clinicaltrials.gov 注册，注册编号分别为 #NCT03745287 和 #NCT04208529。