La place de l’amikacine liposomale dans la prise en charge des infections respiratoires à mycobactéries non tuberculeuses

Details

Supervisors

Gohy, Sophie

Faculty

Faculté de pharmacie et des sciences biomédicales

Degree label

Master [120] en sciences pharmaceutiques, à finalité spécialisée

Abstract

Pulmonary infections caused by non-tuberculous mycobacteria (NTM), particularly Mycobacterium avium complex (MAC), represent a growing clinical challenge due to their chronicity, therapeutic resistance, and diagnostic complexity. Among treatment-refractory forms, the introduction of Amikacin Liposome Inhalation Suspension (ALIS) has opened new therapeutic perspectives. This thesis critically reviews current literature, integrating randomized clinical trials and real-world evidence to evaluate the efficacy, safety, and limitations of ALIS in the management of refractory NTM pulmonary disease (NTM-PD). Findings indicate that ALIS demonstrates significant microbiological efficacy, particularly in improving sputum culture conversion, with an acceptable safety profile marked by predominantly local adverse effects. The liposomal formulation enables targeted pulmonary delivery while minimizing systemic toxicity, presenting a valuable option for patients who have failed conventional therapy. However, methodological heterogeneity, the lack of long-term data, and the risk of antimicrobial resistance highlight the need for cautious and personalized use. Through comparative analysis of pivotal studies (e.g., Griffith et al., 2018) and multicenter observational data (e.g., NTM-NET, 2024), this thesis explores the practical integration of ALIS as a consolidation or salvage therapy. It also addresses critical issues such as treatment access, reimbursement policies, and the limited availability of predictive biomarkers. The discussion underlines the importance of algorithm-based, individualized management and the need for phase III multicenter trials, pharmacogenomic approaches, and harmonization of international guidelines. In conclusion, ALIS represents a promising but still evolving tool in the therapeutic arsenal against refractory NTM-PD, whose future will depend on clinical validation, accessibility, and public health policy integration.