Prospective evaluation of nontuberculous mycobacteria disease in cystic fibrosis: The design of the PREDICT study.

Autor: Martiniano SL; Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO 80045, USA. Electronic address: stacey.martiniano@childrenscolorado.org., Caceres SM; Department of Medicine, National Jewish Health, Denver, CO 80206, USA., Poch K; Department of Medicine, National Jewish Health, Denver, CO 80206, USA., Rysavy NM; Department of Medicine, National Jewish Health, Denver, CO 80206, USA., Lovell VK; Department of Medicine, National Jewish Health, Denver, CO 80206, USA., Armantrout E; Department of Medicine, National Jewish Health, Denver, CO 80206, USA., Jones M; Department of Medicine, National Jewish Health, Denver, CO 80206, USA., Anthony M; Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO 80045, USA., Keck A; Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO 80045, USA., Nichols DP; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA., Vandalfsen JM; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA., Sagel SD; Department of Pediatrics, Children's Hospital Colorado, University of Colorado School of Medicine, Aurora, CO 80045, USA., Wagner B; Department of Biostatistics and Informatics, Colorado School of Public Health University of Colorado Anschutz Medical Campus Aurora Colorado, USA., Xie J; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA., Weaver K; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA., Heltshe SL; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, Washington, USA., Daley CL; Department of Medicine, National Jewish Health, Denver, CO 80206, USA; Department of Medicine, University of Colorado School of Medicine, Aurora, CO 80045, USA., Davidson RM; Center for Genes, Environment, and Health, National Jewish Health, Denver, CO 80206, USA., Nick JA; Department of Medicine, National Jewish Health, Denver, CO 80206, USA; Department of Medicine, University of Colorado School of Medicine, Aurora, CO 80045, USA.
Jazyk: angličtina
Zdroj: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2024 Jan; Vol. 23 (1), pp. 50-57. Date of Electronic Publication: 2023 Sep 04.
DOI: 10.1016/j.jcf.2023.08.007
Abstrakt: Background: Nontuberculous mycobacteria (NTM) are an important cause of airway infections in people with cystic fibrosis (pwCF). Isolation of NTM from respiratory specimens of pwCF do not mandate treatment in the absence of clinical and radiologic features of NTM pulmonary disease (NTM-PD), as some pwCF clear the infection without treatment and others do not appear to progress to NTM-PD despite persistent infection. An evidence-based protocol to standardize diagnosis of NTM-PD is needed to systematically identify pwCF who may benefit from treatment.
Methods: In this multicenter observational study, eligible pwCF who are 6 years of age and older and who have had a recent positive NTM culture are systematically evaluated for NTM-PD. Participants are identified based on positive NTM culture results obtained during routine clinical care and following enrollment are evaluated for NTM-PD and CF-related comorbidities. Participants are followed in PREDICT until they meet NTM-PD diagnostic criteria and are ready to initiate NTM treatment, or until study termination. Active participants who have not met these criteria are re-consented every 5 years to enable long-term participation.
Results: The primary endpoint will summarize the proportion of participants who meet the NTM-PD diagnosis definition. The time from enrollment to NTM-PD diagnosis will be derived from Kaplan-Meier estimates.
Conclusion: A prospective protocol to identify NTM-PD in pwCF will test if this standardized approach defines a cohort with signs and symptoms associated with NTM-PD, to assist with clinical decision making and to build a framework for future therapeutic trials.
Trial Registration: ClinicalTrials.gov Identifier: NCT02073409.
Competing Interests: Declaration of Competing Interest The authors declare that they have no competing or conflicting interests. The authors have no financial or other relationship with other people or organizations that may inappropriately influence their work
(Copyright © 2023 The Authors. Published by Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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