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Original Research: Chest Infections |

Preliminary Results of Bedaquiline as Salvage Therapy for Patients With Nontuberculous Mycobacterial Lung DiseaseBedaquiline for Nontuberculous Mycobacteria

Julie V. Philley, MD; Richard J. Wallace, Jr, MD, FCCP; Jeana L. Benwill, MD; Varsha Taskar, MD; Barbara A. Brown-Elliott, MS, MT(ASCP)SM; Foram Thakkar, MBBS; Timothy R. Aksamit, MD, FCCP; David E. Griffith, MD, FCCP
Author and Funding Information

From the Department of Medicine (Drs Philley, Wallace, Benwill, Taskar, Thakkar, and Griffith), the Mycobacteria/Nocardia Research Laboratory (Drs Wallace and Benwill and Ms Brown-Elliott), and the Department of Microbiology (Dr Wallace and Ms Brown-Elliott), University of Texas Health Science Center at Tyler, Tyler, TX; and the Department of Medicine (Dr Aksamit), Mayo Clinic, Rochester, MN.

CORRESPONDENCE TO: Julie V. Philley, MD, Department of Medicine, 11937 US Hwy 271, University of Texas Health Science Center at Tyler, Tyler, TX 75708; e-mail: Julie.Philley@uthct.edu


FUNDING/SUPPORT: The authors have reported to CHEST that no funding was received for this study.

Reproduction of this article is prohibited without written permission from the American College of Chest Physicians. See online for more details.


Chest. 2015;148(2):499-506. doi:10.1378/chest.14-2764
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BACKGROUND:  Bedaquiline is an oral antimycobacterial agent belonging to a new class of drugs called diarylquinolines. It has low equivalent minimal inhibitory concentrations for Mycobacterium tuberculosis and nontuberculous mycobacterial (NTM) lung disease, especially Mycobacterium avium complex (MAC) and Mycobacterium abscessus (Mab). Bedaquiline appears to be effective for the treatment of multidrug-resistant TB but has not been tested clinically for NTM disease.

METHODS:  We describe a case series of off-label use of bedaquiline for treatment failure lung disease caused by MAC or Mab. Only patients whose insurance would pay for the drug were included. Fifteen adult patients were selected, but only 10 (six MAC, four Mab) could obtain bedaquiline. The 10 patients had been treated for 1 to 8 years, and all were on treatment at the start of bedaquiline therapy. Eighty percent had macrolide-resistant isolates (eight of 10). The patients were treated with the same bedaquiline dosage as that used in TB trials and received the best available companion drugs (mean, 5.0 drugs). All patients completed 6 months of therapy and remain on bedaquiline.

RESULTS:  Common side effects included nausea (60%), arthralgias (40%), and anorexia and subjective fever (30%). No abnormal ECG findings were observed with a mean corrected QT interval lengthening of 2.4 milliseconds at 6 months. After 6 months of therapy, 60% of patients (six of 10) had a microbiologic response, with 50% (five of 10) having one or more negative cultures.

CONCLUSIONS:  This small preliminary report demonstrates potential clinical and microbiologic activity of bedaquiline in patients with advanced MAC or Mab lung disease but the findings require confirmation with larger studies.


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