Abstract
Introduction Nontuberculous mycobacteria (NTM) cause chronic, debilitating pulmonary disease. Patient-reported outcomes provide measures of symptoms, functioning and treatment response. Here we describe the preliminary validation of the recently developed NTM Module.
Methods The study population included Northwest NTM Biobank patients in whom Mycobacterium avium complex (MAC) was isolated and who had ever met the 2007 American Thoracic Society/Infectious Diseases Society of America pulmonary disease criteria. The NTM Module was administered at enrolment and 12 months; a subset also completed the Quality of Life Questionnaire–Bronchiectasis (QOL-B). The NTM Module generates four domain scores (0–100; higher scores indicate better functioning) reflecting NTM-specific symptoms (NTM Symptoms, Body Image, Digestive Symptoms and Eating Problems). We described patient characteristics and mean scores, and evaluated psychometric properties, including response to treatment at 12 months, for each domain.
Results Overall, 203 patients with pulmonary MAC disease were included. Average enrolment scores ranged from 76 (NTM Symptoms) to 84 (Eating Problems). Ceiling effects were observed for Body Image (26% of participants) and Eating Problems (52%). Internal consistency (Cronbach's alpha) ranged from 0.67 (Digestive Symptoms) to 0.89 (Eating Problems). The intraclass correlation for test–retest reproducibility (n=27) ranged from 0.72 (Body Image) to 0.94 (Eating Problems). Patients starting treatment (n=35) had statistically significant increases in scores for NTM Symptoms (+5, p=0.04), Digestive Symptoms (+7, p=0.002), Body Image (+7, p=0.03) and QOL-B Respiratory Symptoms (n=25, +10, p=0.006). NTM Symptoms scores increased by 15 points (p=0.002) in the 16 patients with scores ≤80 at enrolment.
Conclusion The NTM Module generally performs well as a valid patient-reported outcome for pulmonary MAC disease and was responsive to MAC treatment.
Abstract
The NTM Symptom Module is a valid patient-reported outcome tool that can facilitate patient-centred care and may be used as an outcome in clinical trials to support labelling claims for regulatory bodies. http://bit.ly/2nwlPgi
Footnotes
Author contributions: E. Henkle had full access to all of the data in the study and takes responsibility for the integrity of the data and the accuracy of the data analysis. E. Henkle drafted the manuscript. G.P. Ranches, W. Plinke and H.K. Litvin contributed to data collection. K.L. Winthrop and A.L. Quittner contributed substantially to the study design, data analysis and interpretation, and the writing of the manuscript.
Conflict of interest: E. Henkle has nothing to disclose.
Conflict of interest: K.L. Winthrop reports grants and personal fees from Insmed Incorporated (consultant honoraria), outside the submitted work.
Conflict of interest: G.P. Ranches has nothing to disclose.
Conflict of interest: W. Plinke has nothing to disclose.
Conflict of interest: H.K. Litvin has nothing to disclose.
Conflict of interest: A.L. Quittner reports grants from Insmed Incorporated, during the conduct of the study; non-financial support from Insmed (travel funds to present a poster), personal fees from Aradigm (PRO consulting) and personal fees from Bayer (PRO consulting), outside the submitted work.
Support statement: This work was supported by two grants from the ATS Foundation Research Program: a partnered ATS Foundation/American Lung Association of the Mountain Pacific Junior Investigator Award (E. Henkle) and the DeSouza Research Award (DS-311495, K.L. Winthrop). The development of the NTM Symptoms Module was supported by Insmed Inc. and Behavioral Health Systems (A.L. Quittner). Sponsors involved in the initial development of the NTM Module had no role in the design, data collection, analysis or interpretation of this study. Funding information for this article has been deposited with the Crossref Funder Registry.
- Received July 2, 2019.
- Accepted September 23, 2019.
- Copyright ©ERS 2020