Health-related quality of life (HRQL) assessments in a 52-week, double-blind, randomized, placebo-controlled phase III study of resmetirom (MGL-3196) in patients with metabolic dysfunction-associated steatohepatitis (MASH) and fibrosis.

We assessed health-related quality of life (HRQL) in patients with MASH treated with resmetirom.

我们评估了接受resmetirom治疗的MASH患者的健康相关生活质量（HRQL）。

approach_and_results

Patients with MASH/NASH without cirrhosis and with confirmed/suspected fibrosis were enrolled in a 54-month double-blindrandomized placebo-controlledphase III clinical trial with serial biopsy assessments at baseline and week 52 (MAESTRO-NASH, NCT03900429).

在一项为期54个月的双盲随机安慰剂对照的III期临床试验中，招募了无肝硬化且有确认/疑似纤维化的MASH/NASH患者，该试验在基线和第52周进行了连续活检评估（MAESTRO-NASH，NCT03900429）。

HRQL was assessed using Chronic Liver Disease Questionnaire-NASH (CLDQ-NAFLD) and Liver Disease Quality of Life (LDQOL).

使用慢性肝病问卷-NASH（CLDQ-NAFLD）和肝病生活质量（LDQOL）评估健康相关生活质量（HRQL）。

Baseline HRQL score changes by treatment group (resmetirom 80 mg, resmetirom 100 mg, or placebo) and histological response (improvement of fibrosis without worsening of NAFLD activity score or resolution of MASH/NASH without worsening of fibrosis) were compared after 52 weeks.

在治疗组（resmetirom 80 mg、resmetirom 100 mg或安慰剂）和组织学反应（纤维化改善而NAFLD活动评分未恶化或MASH/NASH消解而纤维化未恶化）之间，比较了52周后的基线HRQL评分变化。

Included were 966 intention-to-treatpatients: 323 received resmetirom 100 mg, 322 resmetirom 80 mg, and 321 placebo.

共有966名意向治疗患者被纳入研究：323名接受resmetirom 100 mg治疗，322名接受resmetirom 80 mg治疗，321名接受安慰剂。

By weeks 24 and 52, patients receiving 80 or 100 mg resmetirom experienced HRQL improvement in CLDQ-NAFLD Worry domain (mean +0.21 to +0.24, p < 0.05).

在第24周和第52周，接受80毫克或100毫克resmetirom治疗的患者在CLDQ-NAFLD担忧领域经历了健康相关生活质量（HRQL）的改善（平均值+0.21至+0.24，p < 0.05）。

At week 52, subjects who met histologic endpoints after treatment with resmetirom (100 mg and 80 mg pooled) experienced HRQL improvement in CLDQ-NAFLD Worry +0.46 (41% met minimal clinically important difference [MCID]), LDQOL domains: Role Emotional +3.0 (28% met MCID), Health Distress +8.1 (38% MCID), Stigma +3.5 (39% MCID), and total LDQOL +2.2 (35% MCID) (all p < 0.05).

在第52周，经过resmetirom（100毫克和80毫克联合）治疗后达到组织学终点的受试者，在CLDQ-NAFLD担忧领域经历了健康相关生活质量（HRQL）的改善，改善值为+0.46（41%达到最小临床重要差异[MCID]），LDQOL领域：角色情感+3.0（28%达到MCID），健康困扰+8.1（38%达到MCID），污名感+3.5（39%达到MCID），以及LDQOL总分+2.2（35%达到MCID）（所有p < 0.05）。

Similar improvements were noted in histologic responders from 100 mg or 80 mg resmetirom groups when separated-no improvements in placebo or nonresponders.

在100毫克或80毫克resmetirom组中，组织学反应者也观察到了类似的改善，而在安慰剂组或无反应者中没有改善。

Baseline F3 histologic responders had similar/more pronounced HRQL improvements.

基线时F3组织学反应者在健康相关生活质量（HRQL）的改善上相似或更为显著。

Conclusions

Patients with MASH/NASH with fibrosis improvement or the resolution of MASH with resmetirom experienced clinically meaningful and statistically significant HRQL improvements.

具有MASH/NASH和纤维化改善或MASH消解的患者，在使用resmetirom后经历了临床上有意义且统计学上显著的健康相关生活质量(HRQL)改善。