Prospective Analysis of urINe LAM to Eliminate NTM Sputum Screening (PAINLESS) study: Rationale and trial design for testing urine lipoarabinomannan as a marker of NTM lung infection in cystic fibrosis

• DOI: 10.1371/journal.pone.0309191
• Publication Date: 2025-03-10T20:08:18Z
• Authors: Kara M. Calhoun; Emily Armantrout; Katie Poch; Silvia Caceres; Valerie K. Lovell; Marion Jones; Kenneth C. Malcolm; Brian Vestal; Emily Wheeler; Noel Rysavy; Jordan Manzer; Ibrahim Aboellail; Delphi Chatterjee; Jerry A. Nick
• Keywords: Lipoarabinomannan; Sputum culture; Nontuberculous Mycobacteria; Surrogate endpoint; Clinical endpoint

Background Routine screening for nontuberculous mycobacterial (NTM) lung disease is dependent on sputum cultures. This particularly challenging in the cystic fibrosis (CF) population due to reduced production and low culture sensitivity. Biomarkers of infection that do not rely may lead earlier diagnosis, but validation trials require a unique prospective design. Purpose The rationale this trial investigate utility urine lipoarabinomannan (LAM) as test identify people with CF new positive NTM culture. We hypothesize LAM sensitive, non-invasive high negative predictive value individuals relatively risk having Study design prospective, single-center, non-randomized observational study adults CF, 3 years cultures, no known history Patients are followed two year-long periods primary endpoint being within year result secondary result. implementation includes remote consent sample collection accommodate changes from COVID-19 pandemic. Conclusions report describes an aimed at using biomarker assist diagnosis pwCF. If successful, could be used adjunct traditional cultures routine screening, replace low-risk unable produce sputum.