A Pragmatic Trial of a 6-Month Strategy for Rifampicin-Resistant Tuberculosis.

Methods

We conducted a phase 3, open-label, pragmatic, randomized, controlled noninferiority trial in South Africa to assess a 6-month treatment strategy for pulmonary rifampicin-resistant tuberculosis.

我们在南非进行了一项3期、开放标签、实用、随机、非劣效试验，以评估一种针对肺部利福平耐药结核病的6个月治疗策略。

Participants with pulmonary rifampicin-resistant tuberculosis who were 6 years of age or older were randomly assigned to a regimen consisting of bedaquiline, linezolid, delamanid, and levofloxacin or clofazimine or both for 6 months (trial-strategy group) or the 9-month standard-of-care treatment regimen that was current in South Africa (control group).

年龄在6岁或以上的肺部利福平耐药性结核病患者被随机分配到一个由贝达喹啉、利奈唑胺、德拉马尼和左氧氟沙星或氯法齐明或两者组成的治疗方案，持续6个月（试验策略组）或接受南非当时现行的9个月标准治疗方案（对照组）。

Persons who were pregnant or breastfeeding and those who had fluoroquinolone-resistant tuberculosis were included in the trial population.

孕妇或哺乳期妇女以及对氟喹诺酮类药物耐药的结核病患者被纳入试验人群。

Treatment in both groups was adjusted on the basis of results of second-line drug susceptibility testing.

两组的治疗方案根据二线药物敏感性测试结果进行了调整。

The primary efficacy end point was a successful outcome (cure or completion of treatment) at the end of treatment and at 76 weeks after randomization.

主要疗效终点是在治疗结束时以及随机化后76周的成功结果（治愈或完成治疗）。

The noninferiority margin was 10 percentage points.

非劣效性边界为10个百分点。

The primary safety end point was an adverse eventof grade 3 or higher.

主要安全性终点为3级或更高级别的不良事件。

Results

Among the 432 persons who were screened, 403 underwent randomization; 203 were assigned to the trial-strategy group, and 200 to the control group.

在432名接受筛查的个体中，有403人进行了随机分组；203人被分配到试验策略组，200人被分配到对照组。

A successful outcome was observed in 174 of 202 participants (86.1%) in the trial-strategy group and in 172 of 200 (86.0%) in the control group.

在试验策略组的202名参与者中，有174人（86.1%）观察到成功的结果，在对照组的200名参与者中，有172人（86.0%）观察到成功的结果。

The adjusted risk difference was -0.2 percentage points (95% confidence interval[CI], -6.9 to 6.5; P = 0.001 for noninferiority).

调整后的风险差异为-0.2个百分点（95%置信区间[CI]，-6.9至6.5；非劣效性检验P=0.001）。

Adverse events of grade 3 or higher occurred during treatment in 63 of 202 participants (31.2%) in the trial-strategy group and in 74 of 200 (37.0%) in the control group; 10 participants in each group died.

在试验策略组的202名参与者中有63人（31.2%）和对照组的200名参与者中有74人（37.0%）在治疗期间发生了3级或更高级别的不良事件；每组各有10名参与者死亡。

Conclusions

Among participants in South Africa with rifampicin-resistant tuberculosis, the 6-month trial strategy was noninferior to the standard-of-care strategy with respect to a successful outcome.

在南非的利福平耐药结核病患者中，6个月试验策略在成功结果方面不劣于标准治疗策略。

The safety profiles of the two strategies were similar.

两种策略的安全性特征相似。

(Funded by the U.S.

(由美国国际开发署和其他机构资助; BEAT结核病临床试验注册号, NCT04062201。)

Agency for International Development and others; BEAT Tuberculosis ClinicalTrials.gov number, NCT04062201.).

国际开发署和其他机构; BEAT结核病临床试验注册号，NCT04062201。