Improvements in health-related quality of life in patients with transfusion-dependent β-thalassemia after exagamglogene autotemcel.

Exagamglogene autotemcel (exa-cel) is a one-time, ex vivo CRISPR-Cas9 gene-edited cell therapy for TDT shown in a phase 3 clinical trial to result in transfusion independence in most participants.

Exagamglogene autotemcel（exa-cel）是一种一次性、体外CRISPR-Cas9基因编辑细胞疗法，用于TDT，在一项III期临床试验中显示，大多数参与者实现了输血独立。

Here, we describe changes in patient-reported outcome (PRO) measures after exa-cel infusion in 54 participants (adults, n = 35; adolescents, n = 19), who had ≥16 months of follow-up.

在此，我们报告了54名受试者（成人，n=35；青少年，n=19）在接受exa-cel输注后，患者报告的结果（PRO）指标的变化情况，这些受试者有≥16个月的随访期。

In adults, PRO measures included the EuroQol Quality of Life Scale-5 dimensions-5 levels of severity (EQ-5D-5L) and the Functional Assessment of Cancer Therapy Bone Marrow Transplant (FACT-BMT).

在成人中，PRO指标包括欧洲五维健康量表-5级严重程度（EQ-5D-5L）和癌症治疗功能评估-骨髓移植（FACT-BMT）。

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

在青少年中，使用了欧洲五维健康量表-5维度-青少年版(EQ-5D-Y)和儿童生活质量清单(PedsQL)工具。

At baseline, mean EQ-5D-5L visual analog scale (VAS) and US and UK health utility index scores in adults were in line with baseline scores reported for adults with TDT.

在基线时，成人平均的欧洲五维健康量表-5级视觉模拟评分(VAS)以及美国和英国的健康效用指数得分与成人TDT患者的基线得分相符。

After exa-cel infusion, all 3 scores improved, exceeding established minimal clinically important differences (MCIDs) at month 48.

在exa-cel输注后，所有3个评分均有所改善，在第48个月时超过了既定的最小临床重要差异（MCIDs）。

Mean FACT-General (FACT-G) score and bone marrow transplant subscale score improved through month 48, also exceeding MCIDs, with improvements in all 4 FACT-G subscales (physical, social/family, emotional, and functional well-being).

平均FACT-General（FACT-G）评分和骨髓移植子量表评分在第48个月时有所改善，也超过了MCIDs，所有4个FACT-G子量表（身体、社会/家庭、情感和功能良好）均有所改善。

Consistent with HRQoL improvements in adults, adolescents had increases from baseline at month 24 in mean EQ-5D-Y VAS score and PedsQL total score, with sustained improvements in both physical and psychosocial health subcomponents.

与成人健康相关生活质量（HRQoL）的改善一致，青少年在24个月时的基线平均EQ-5D-Y VAS评分和PedsQL总分有所增加，身体和心理社会健康的子成分均持续改善。

These results indicate exa-cel leads to broad, durable, and clinically meaningful improvements in HRQoL in adults and adolescents with TDT.

这些结果表明，exa-cel可导致患有输血依赖性地中海贫血（TDT）的成人和青少年在健康相关生活质量（HRQoL）方面得到广泛、持久且具有临床意义的改善。

These trials were registered at www.ClinicalTrials.gov as #NCT03655678 and #NCT04208529.

这些试验已在www.ClinicalTrials.gov上注册，注册号为#NCT03655678和#NCT04208529。