Background Respiratory infection with nontuberculous mycobacteria (NTM) in children with cystic fibrosis (CF) has increased in prevalence. The condition is difficult to diagnose and treatments are complex with limited evidence to guide practice. This study describes the approaches to diagnosis, management and consequences of treatment in a multicentre cohort of children with CF in the UK.
Methods Retrospective data were collected from 11 CF specialist centres from patients less than 17 years old, treated for NTM infection between 2006 and 2017. Descriptive statistics were used to describe the clinical characteristics of children treated. Treatment regimens, adverse events and success of treatment, with respect to lung function and culture conversion, were evaluated.
Results Data from 70 patients treated for NTM pulmonary disease were collated (60 Mycobacterium abscessus complex (MABSC); 10 M. avium complex (MAC)). Older age and previous diagnosis of allergic bronchopulmonary aspergillosis were all significantly associated with NTM. There was a wide variance in drug choice and side effects were reported with all agents. NTM eradication occurred in 80% of patients with MAC and 48% with MABSC, with variable outcomes on lung function.
Conclusions Diagnosis and treatment of NTM infection in children with CF is challenging. Treatment success is not guaranteed, particularly for MABSC. Large clinical trials are urgently required to evaluate treatment regimes and their suitability and efficacy in children.
- cystic fibrosis
Data availability statement
Data are available on reasonable request. Data are available upon reasonable request.
Statistics from Altmetric.com
GLS and MFT are joint first authors.
MB and PM are joint senior authors.
Collaborators Francis Gilchrist (Royal Stoke University Hospital, Francis.Gilchrist@uhnm.nhs.uk); Chris Hine (Royal Stoke University Hospital, Chris.Hine@uhnm.nhs.uk); Anirban Maitra (Manchester Royal Children’s Hospital, email@example.com); Joanne Gooch (Manchester Royal Children’s Hospital, Joanne.Gooch@mft.nhs.uk); Louise Turnball (Manchester Royal Children’s Hospital, firstname.lastname@example.org); Alan Smyth (Nottingham University Hospitals, email@example.com); Adnan Zafar (Nottingham University Hospitals, firstname.lastname@example.org); Noreen West (Sheffield Children’s NHS Foundation Trust, Noreen.email@example.com); Ina Aldag (Sheffield Children’s NHS Foundation Trust, firstname.lastname@example.org); Gill Leonard (Alder Hey Children's NHS Foundation Trust, Liverpool, email@example.com); Kevin W Southern (Alder Hey Children's NHS Foundation Trust, Liverpool, firstname.lastname@example.org); Erol Gaillard (Leicester Royal Infirmary, email@example.com); Molla Imaduddin (Ahmed Leicester Royal Infirmary, Molla.Ahmed@nhs.net); Deepa Patel (Leicester Royal Infirmary, Deepa.firstname.lastname@example.org); Maya Desai (Birmingham Women's and Children's NHS Foundation Trust, email@example.com); Benjamin Davies (Birmingham Women's and Children's NHS Foundation Trust, Benjamin.firstname.lastname@example.org); Julian Forton (The Children's Hospital for Wales, Cardiff, Julian.email@example.com); Juliette Oakley (The Children's Hospital for Wales, Cardiff, firstname.lastname@example.org); Steve Cunningham (The University of Edinburgh, email@example.com); Andrew Fall (Royal Hospital for Sick Children, Edinburgh, Andrew.firstname.lastname@example.org); Anne Devenny (Royal Hospital for Children, Glasgow, email@example.com); Jane Wilkinson (Royal Hospital for Children, Glasgow, firstname.lastname@example.org); David Galloway (Great North Children's Hospital, Newcastle upon Tyne, email@example.com).
Contributors Conception and design: PM, MB and MFT. Acquisition of data: GLS, PM, MB, MFT, RJL and all members of the NTM collaborators group. Analysis and interpretation of data: GLS, PM, MB, MFT, RJL and NZA. Manuscript preparation: GLS, PM, MFT, MB and NZA. Manuscript review: all authors including NTM collaborators group. PM acts as guarantor for the study.
Funding This work was supported by Alder Hey NHS Foundation Charity and Newcastle upon Tyne Hospitals NHS Charity who part funded the database. MB is supported by a Medical Research Council (MRC) Clinician Scientist Fellowship [MR/M008797/1]. The research was supported by the Newcastle upon Tyne Hospitals NHS Charity, National Institute for Health Research Newcastle Biomedical Research Centre based at Newcastle Hospitals NHS Foundation Trust and Newcastle University. GLS is a (National Institute of Health Research) NIHR Academic Clinical Lecturer.
Disclaimer The views expressed are those of the authors and not necessarily those of the NHS, the NIHR or the Department of Health.
Competing interests MB: not related to this study, has been CI on investigator-led research grants from Pfizer and Roche Diagnostics; speaker fees paid to Newcastle University from Novartis, Roche Diagnostics and TEVA; travel expenses to educational meetings from Boehringer Ingelheim and Vertex. MFT: not related to this study, investigator-led research grant from Pfizer.
Provenance and peer review Not commissioned; externally peer reviewed.
Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.
If you wish to reuse any or all of this article please use the link below which will take you to the Copyright Clearance Center’s RightsLink service. You will be able to get a quick price and instant permission to reuse the content in many different ways.