Preliminary validation of the NTM module: a patient-reported outcome measure for patients with pulmonary nontuberculous mycobacterial disease
- Emily Henkle1⇑,
- Kevin L. Winthrop1,
- Gregory P. Ranches2,
- Wesley Plinke1,
- Hana K. Litvin1 and
- Alexandra L. Quittner3
- 1OHSU-PSU School of Public Health, Portland, OR, USA
- 2Division of Pulmonary & Critical Care Medicine, OHSU, Portland, OR, USA
- 3Nicklaus Children's Research Institute, Nicklaus Children's Hospital, Miami, FL, USA
- Emily Henkle, PhD, MPH, 3181 SW Sam Jackson Park Road, Mailcode GH104, Portland, OR 97239. E-mail henkle{at}ohsu.edu
Abstract
Introduction Nontuberculous mycobacteria (NTM) cause chronic, debilitating pulmonary disease. Patient-reported outcomes (PRO) 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 who isolated Mycobacterium avium complex (MAC) and ever met 2007 ATS/IDSA pulmonary disease criteria. The NTM Module was administered at enrolment and 12 months; a subset also completed the QOL-Bronchiectasis (QOL-B). The NTM Module generates four domain scores (0–100; higher scores=better functioning) reflecting NTM-specific symptoms (NTM Symptoms, Body Image, Digestive Symptoms, and Eating Problems). We described patient characteristics, mean scores, and evaluated psychometric properties, including responsivity 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%) and Eating Problems (52%). Internal consistency (Cronbach's alpha) ranged from 0.67 (Digestive Symptoms) to 0.89 (Eating Problems). The intra-class 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 15 points (p=0.002) in the 16 patients with scores ≤80 at enrolment.
Conclusion The NTM Module generally performs well as a valid PRO for pulmonary MAC disease and was responsive to MAC treatment.
Footnotes
This manuscript has recently been accepted for publication in the European Respiratory Journal. It is published here in its accepted form prior to copyediting and typesetting by our production team. After these production processes are complete and the authors have approved the resulting proofs, the article will move to the latest issue of the ERJ online. Please open or download the PDF to view this article.
Conflict of interest: Dr. Winthrop reports grants and personal fees from Insmed Incorporated, outside the submitted work.
Conflict of interest: Dr. Ranches has nothing to disclose.
Conflict of interest: Dr. Quittner reports grants from Insmed Incorporated, during the conduct of the study; non-financial support from Insmed, personal fees from Aradigm, personal fees from Bayer, outside the submitted work.
Conflict of interest: Mr. Plinke has nothing to disclose.
Conflict of interest: Ms. Litvin has nothing to disclose.
Conflict of interest: Dr. Henkle has nothing to disclose.
This is a PDF-only article. Please click on the PDF link above to read it.
- Copyright ©ERS 2019