ABSTRACT
Cystic fibrosis (CF) has entered the era of variant-specific therapy, tailored to the genetic variants in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. CFTR modulators, the first variant-specific therapy available, have transformed the management of CF. The latest standards of care from the European CF Society (2018) did not include guidance on variant-specific therapy, as CFTR modulators were becoming established as a novel therapy. We have produced interim standards to guide healthcare professionals in the provision of variant-specific therapy for people with CF. Here we provide evidence-based guidance covering the spectrum of care, established using evidence from systematic reviews and expert opinion. Statements were reviewed by key stakeholders using Delphi methodology, with agreement (≥80%) achieved for all statements after one round of consultation. Issues around accessibility are discussed and there is clear consensus that all eligible people with CF should have access to variant-specific therapy.
Subject(s)
Cystic Fibrosis , Humans , Cystic Fibrosis/drug therapy , Cystic Fibrosis/genetics , Cystic Fibrosis Transmembrane Conductance Regulator/genetics , Standard of Care , Ion Transport , Signal Transduction , MutationABSTRACT
BACKGROUND: People with cystic fibrosis (CF) frequently experience isolation and are subjected to extensive complex treatment regimens which could be complemented by remote support. In the current research this is particularly relevant as the location, Tasmania, has the second highest incidence of CF in the world. This paper provides an overview of the evaluation of a pilot trial of an information system conceptualised and developed to assist people with CF, and their families, to enhance their skills and communication in relation to self-management for their condition. METHODS: The pilot involved people with CF ranging in age from 19 months to 52 years and their families. The primary outcome was the perceived usability of the online-symptom diary from the user's perspective. To assess perceived usability qualitative semi-structured interviews were conducted pre- and post-pilot and analysed using thematic coding. RESULTS: Participants initially and primarily perceived myCF as a system that would help others and enable peer support. Connectivity and involvement were highlighted as complex issues that needed consideration. CONCLUSION: There was an overall encouraging response to the pilot and indications that the use of information communication technology to complement health care delivery and facilitate self-care skills may be particularly suited to the Australian context where geographical distances and isolation provide a relative barrier to specialist care for chronic complex conditions.
Subject(s)
Cystic Fibrosis/pathology , Internet , Self Care , Adolescent , Adult , Child , Child, Preschool , Consumer Health Information , Humans , Infant , Information Systems , Middle Aged , Patient-Centered Care , Pilot Projects , Qualitative Research , Tasmania , User-Computer Interface , Young AdultABSTRACT
ICT use in cystic fibrosis management provides an alternative means of information supply to individuals, families, health care professionals and other stakeholders. The purpose of this paper is to present the evolution of a series of projects culminating in a project that translates the previous research into practice. In this paper the sequential nature of the projects will be detailed. The three projects explored are the Pathways Home for Respiratory Illness Project (Pathways Home), Enhancing Self-Efficacy for Self-Management in People with Cystic Fibrosis and the Tasmanian Community Fund Project (myCF pilot).
Subject(s)
Cystic Fibrosis/diagnosis , Cystic Fibrosis/therapy , Decision Support Systems, Clinical , Patient Participation , Self Care/methods , Adolescent , Adult , Humans , Young AdultABSTRACT
This paper reports on a research trial designed to evaluate the benefits of a health mentoring programme supported with a web and mobile phone based self-monitoring application for enhancing self-efficacy for self-management skills and quality of life for people with CF. This randomised, single-blind controlled trial evaluated two strategies designed to improve self-management behaviour and quality of life. Task-specific self-efficacy was fostered through mentorship and self-monitoring via a mobile phone application. Trial participants were randomised into one of three groups: Control, Mentor-only and Mentor plus mobile phone. Analysis and discussion focus on the experiences of participants through a methodology utilising descriptive statistics and semi-structured interviews. The results highlight the challenges of stimulating self-management behaviours particularly in adolescents and in the evaluation of the role of mobile applications in supporting them.
Subject(s)
Cystic Fibrosis/therapy , Remote Consultation/methods , Self Care , Adolescent , Adult , Cell Phone , Female , Humans , Internet , Male , Mentors , Middle Aged , Quality of Life , Tasmania , Telemedicine/methodsABSTRACT
INTRODUCTION: Marijuana or "bong" lung has been recently described. Subjects typically develop large peripheral paraseptal lung bullae and are predisposed to spontaneous pneumothoraces. The underlying mechanism for bullae formation is uncertain, but probably relates to direct lung toxicity and repeated barotrauma as the smoker performs frequent valsalva manoeuvres in an attempt to derive a greater drug effect. CASE PRESENTATION: We describe a case of probable "bong lung" occurring in a 23-year-old Caucasian man with cystic fibrosis who had a history of recurrent pneumothoraces and unusual findings on sputum cytology. CONCLUSION: Our case highlights the importance of questioning young adult cystic fibrosis patients about illicit drug use and the utility of sputum cytology and computed tomography scanning when patients present with pneumothoraces and deteriorations in clinical status.
