Efficacy and safety of the all-oral bedaquiline-containing regimen as treatment for pediatric multidrug/rifampicin-resistant tuberculosis: a multicenter, retrospective, cohort study

ABSTRACT

Objective

The study aimed to observe the efficacy and safety of an all-oral bedaquiline (BDQ)-containing regimen for pediatric multidrug/rifampicin-resistant tuberculosis (MDR/RR-TB) through a multicenter, retrospective study in China.

Methods

In the study, pediatric patients receiving all-oral BDQ-containing regimen (BDQ group) with clinical matched control group were included, the control group received an injection-containing regimen. The treatment outcomes and the incidence of adverse events (AEs) were compared and analyzed.

Results

79 pediatric patients were enrolled, including 37 cases in BDQ group and 42 cases in the control group, the median age was 12 {8–16} and 11 {9–15} in both groups respectively. Favorable treatment outcome and cure rate in BDQ group were significantly higher than those in control group (100%vs 83.3%, p 0.03; 94.6%vs 63.3%, p 0.00). Median time of sputum culture conversion in BDQ group was significantly shorter than that in the control group (4 weeks vs 8 weeks, p 0.00). The incidence of AEs in the BDQ group was significantly less than that in the control group (48.6% vs 71.4%, p 0.03). No AEs leading to treatment discontinuation of BDQ occurred.

Conclusions

The all-oral BDQ-containing regimens may be effective and safe in the Chinese pediatric population.

KEYWORDS:

• RR-TB
• MDR-TB
• pediatric tuberculosis
• bedaquiline
• treatment outcome
• sputum culture conversion
• adverse events

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Acknowledgments

The authors wish to thank all participants for their efforts.

Ethics statement

This retrospective study was performed in accordance with the Declaration of Helsinki and approved by the Ethics Committee of the Shanghai Pulmonary Hospital (Approval No K20–430). The present study was a retrospective study, the informed consent was waived signed, however, the privacy of each participant was fully protected.

Author contribution statement

WS, MY: Data statistics, writing the manuscript; JZ, HC, HW and CF: Data collation and statistics; LF and CY: Research design and revised the manuscript. All authors read and approved the final submitted version.

Additional information

Funding

The study was supported by National Natural Science Foundation of China [82170006]; the Shanghai Science and Technology Development Foundation, China [21Y11901000,20ZR1446700]; Clinical Research Foundation of Shanghai Pulmonary Hospital [SKPY2021003]. The funders had no role in the study design, data collection, and analysis, decision to publish, or preparation of the manuscript.