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Amikacin liposome inhalation suspension clinical benefit–risk assessment for refractory Mycobacterium avium complex lung disease

ABSTRACT: Mycobacterium avium complex (MAC) is the leading cause of nontuberculous mycobacterial lung disease, which can be associated with progressive lung damage and increased mortality [1]. Patients with MAC lung disease have substantial disease burden and limited treatment options [1]. Up to 40% of patients experience failure, with lengthy multidrug treatments, relapse or reinfection [2]. For patients with treatment-refractory MAC lung disease (persistent MAC-positive sputum despite ≥6 months of guideline-based therapy (GBT)), international guidelines recommend the addition of amikacin liposome inhalation suspension (ALIS) to GBT regimens [3]. In clinical trials, patients with treatment-refractory MAC lung disease had improved culture conversion with ALIS+GBT versus GBT [4, 5]. Marraset al. report a low number needed to treat and high number needed to harm supporting addition of amikacin liposome inhalation suspension to guideline-based treatments in patients with treatment-refractory Mycobacterium avium complex lung diseasehttps://bit.ly/3tPFW7D

SUBMITTER: Marras T

PROVIDER: S-EPMC9271752 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:IntroductionThis study aimed to gain insight from patients with refractory Mycobacterium avium complex lung disease (MAC-LD) into strategies used to manage adverse events (AEs) associated with amikacin liposome inhalation suspension (ALIS).MethodsWe conducted semi-structured interviews with US patients with refractory MAC-LD prescribed ALIS in a real-world setting. Interview transcripts were analyzed and coded to identify patterns in participants' descriptions of their ALIS treatment experiences, including AEs and their disruptiveness, and AE mitigation strategies, including participants' ratings of strategies' effectiveness. Concept saturation was also assessed.ResultsTwenty participants (mean age 48.7 years; 80% women; mean ALIS duration 5.45 months) were interviewed. At the time of the interview, 15 participants (75%) had received ALIS for > 1 month and 13 (65%) were currently receiving ALIS. Participants described 44 unique AE mitigation strategies, which can be categorized into three groups: prepare for treatment; prevent increased emergence of AEs; and persist on treatment by mitigating AEs. Common strategies (reported by ≥ 50% of participants) included use of educational materials from the patient support program, localized management of throat irritation, and symptom management to reduce fatigue. Evidence of concept saturation was observed: no new strategies were identified in the last five interviews, which suggests the sample was robust enough to identify all mitigation strategies likely to be used by the broader patient population.ConclusionsThis real-world study identified a diverse set of potential AE mitigation strategies intended to help individual patients prepare for ALIS treatment, prevent the increased emergence of certain AEs, and mitigate the impact of AEs on treatment persistence. Developing a comprehensive accounting of the types of mitigation strategies in use among patients in real-world settings can inform future investigation of the effectiveness of such strategies, and support evidence-based recommendations for treatment management.

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Amikacin liposome inhalation suspension clinical benefit–risk assessment for refractory Mycobacterium avium complex lung disease

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