Patients with treatment-refractory Mycobacterium avium complex (MAC) lung disease receiving treatment with amikacin liposome inhalation suspension and guideline therapy had increased culture conversion compared with guideline therapy alone, according to the results of a recent study published in the journal CHEST.
Participants in the CONVERT study (ClinicalTrials.gov Identifier: NCT02344004) with treatment-refractory MAC lung disease were randomly assigned 2:1 to receive either amikacin liposome inhalation suspension added to guideline-based therapy or guideline-based therapy alone as a control. The primary study outcome was the increased culture conversion by 6 months. Secondary endpoints included adverse event profiles and culture durability after 3 months without treatment.
Among the 492 patients at 127 sites in North America, Europe, Australasia, and Asia who were screened, 336 patients were included in the trial; 224 received amikacin liposome inhalation suspension plus guideline-based therapy and 112 received guideline-based therapy alone. A total of 75 participants achieved culture conversion within 6 months, 65 of whom were in the amikacin liposome inhalation suspension group. However, 55.4% of converters in amikacin liposome inhalation suspension group compared with 0% in the guideline-based therapy alone group had sustained and durable conversion 3 months after treatment ended.
At the end of treatment, 80.0% of converters in the amikacin liposome inhalation suspension plus guideline-based therapy group were culture negative, 7.7% had MAC-positive sputum that met the definition of relapse, 4.6% had MAC-positive sputum that met the definition of reinfection, and 7.7% were missing culture data. A total of 30.0% of patients in the guideline-based therapy alone group were culture negative, 30.0% had MAC-positive sputum that met the definition of relapse, 10% had MAC-positive sputum that met the definition of reinfection, and 30.0% were missing culture data.
“These results indicate that the culture conversion reported by month 6 in the pivotal phase 3 CONVERT trial is predictive of both a sustained and durable effect and that amikacin liposome inhalation suspension is an effective treatment for those with guideline-based therapy-refractory pulmonary MAC,” the study authors wrote. “Treatment with amikacin liposome inhalation suspension for up to 16 months did not result in new safety signals or changes to the established safety profile of amikacin liposome inhalation suspension.”
Disclosure: This research was supported by Insmed Incorporated. Please see the original reference for a full list of disclosures.
Griffith DE, Thomson R, Flume PA, et al; for the CONVERT Study Group. Amikacin liposome inhalation suspension for refractory MAC lung disease: sustainability and durability of culture conversion and safety of long-term exposure. CHEST. Published online April 19, 2021. doi:10.1016/j.chest.2021.03.070