Treatment outcome definitions in nontuberculous mycobacterial pulmonary disease: an NTM-NET consensus statement

Abstract

Nontuberculous mycobacterial pulmonary diseases (NTM-PD) are increasingly recognised as opportunistic infections of humans. These chronic pulmonary infections have two main presentations. The first is a fibro-cavitary disease, that occurs in patients with pre-existing pulmonary diseases, such as chronic obstructive pulmonary disease, bronchiectasis, previous tuberculosis or other structural lung disease. The second presentation is a nodular- bronchiectatic disease of primarily the lingula and middle lobe that tends to affect a middle- aged and elderly female population [1]. Treatment of NTM-PD requires long-term administration of complex multidrug therapies that are species-specific. Currently recommended regimens are supported by a very limited evidence base [2, 3]. The increasing incidence of NTM-PD has sparked increased interest in performing prospective randomised clinical trials [4]. One of the drawbacks of the existing case series and clinical trials is that they have applied different outcome measures [5]. This hampers meta-analyses, which are important in these still understudied infectious diseases. To enhance the quality and interpretability of the results of future trials and retrospective cohort studies, we aimed to formulate clear and broadly acceptable outcome definitions for NTM-PD treatment.