Is Watchful Waiting a Viable Strategy in Mycobacterium Avium Complex Pulmonary Disease? Insights From a Propensity-matched Cohort Study

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Abstract Introduction According to recent guidelines, initiating treatment in patients with Mycobacterium avium complex pulmonary disease (MAC-PD) is recommended. However, watchful waiting may be preferable in some cases, particularly when side effects are a concern or the disease remains mild and progresses slowly. This study compared the clinical outcomes of MAC-PD patients to further assess the benefits of early treatment. Method This retrospective study analyzed MAC-PD patients at Seoul National University Bundang Hospital from May 1st, 2003, through October 31st, 2021. Using 1:1 propensity score matching, we controlled for factors influencing early treatment initiation and long-term outcomes, such as sex, body mass index, age and presence of cavity. Patients were divided into an early treatment group (treatment within 60 days of diagnosis) and a watchful waiting group (follow-up without treatment or treatment initiation after 60 days post-diagnosis if necessary). Key outcomes included time to culture conversion, rate of adverse reactions, all-cause mortality, and mortality from respiratory cause or infection. Results Out of 592 MAC-PD patients, propensity score matching yielded 102 matched pairs, assigned equally to each group. The proportion of patients with a BACES score of 4 or 5 was 13.8% in the early treatment group and 19.6 % in the watchful waiting group. In the watchful waiting group, 55 of 102 (53.9%) eventually received treatment at a median of 4.89 months (interquartile range [IQR] 2.86-22.54) post-diagnosis, while remaining 47 (46.1%) continued without treatment for a median of 5.78 years (IQR 2.73-11.42). Notably, early treatment was linked to a shorter time to culture conversion (hazard ratio (HR) 3.07, 95% confidence interval (CI) [1.68-5.61]) but also a higher incidence of adverse events (64.7% versus 41.2%, P<0.001). There were no significant differences in all-cause mortality and mortality from respiratory cause or infection between groups (HR for early treatment: 0.95 with 95% CI [0.51-1.78]) and 0.64 with 95% CI [0.25-1.64]), respectively). Similar outcomes were observed for respiratory or infection-related mortality in subgroups with larger cavities (≥2cm) or initial positive sputum smears (HR for early treatment: 0.80 with 95% CI [0.24-1.87]). Conclusion Although early treatment for MAC-PD does not markedly affect long-term mortality, it may benefit patients who can tolerate potential side effects. Watchful waiting remains a feasible option, underscoring the need for personalized treatment strategies in MAC-PD to balance the benefits of early intervention against the risks of adverse events.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.