Gaps in the evidence for treatment decisions in cystic fibrosis: a systematic review

Abstract

IntroductionCystic fibrosis (CF) is a multisystem disorder. Treatment is complex and evidence for treatment decisions may be absent. Characterising gaps in the research evidence will highlight treatment uncertainties and help prioritise research questions. We systematically identified the evidence gaps for treatment decisions in CF.MethodsWe searched for systematic reviews and guidelines on treatment interventions in CF. Two researchers identified eligible reviews with arbitration from a third. Using a structured framework, we extracted and characterised evidence gaps.ResultsThere were 73 reviews and 21 guidelines that met our inclusion criteria. From these, we identified 148 evidence gaps across a range of treatment areas. We found 111 evidence gaps through systematic reviews and a further 37 from guidelines. The reason for an evidence gap could only be reliably characterised for systematic reviews. In most cases, there was more than one explanation—most commonly few or no trials (97/111 evidence gaps). Other important factors leading to evidence gaps were small sample size (49/111), inadequate duration of follow-up (38/111) or intervention (37/111) and factors relating to outcomes (35/111). Evidence gaps from both systematic reviews and guidelines fell into the following categories: Respiratory (91); Gastrointestinal (20); PhysiotherapyandExercise (16); Musculoskeletal (6); Endocrine (4); Basic defect of CF (8); Psychosocial (2); Ears, Nose and Throat (1).ConclusionsWe have compiled an up-to-date list of treatment uncertainties in CF and the reasons for these uncertainties. These can be used as a resource to aid researchers and funders when planning future trials.PROSPERO registration numberPre-results; CRD42015030111.

Adapting the James Lind Alliance priority setting process to better support patient participation: an example from cystic fibrosis

“…We set aside any questions which did not relate to treatment uncertainties and those which had already been answered. Prior to commencing this analysis we undertook a systematic review of gaps in the evidence for treatment decisions in CF which helped with this process [1]. We sorted and grouped the resulting questions to produce a shorter list of questions which could go through to the ranking survey.…”

“…Cystic Fibrosis (CF) is a life limiting inherited multi-system disorder with a high treatment burden but relatively little good quality evidence to guide treatment decisions [1]. In the UK, 10,000 people have CF [2] and 70,000 adults and children worldwide [3] are affected by the condition.…”

Adult Care in Cystic Fibrosis

“…The burden of care for many adults with CF is enormous, [119][120][121] In a recent patient-driven program through the James Lind Alliance, identified treatment burden as the primary area that people with CF want further research on. 122,123 In CF, the care treatment burden is often cumulative. It is difficult for patients and health care professionals to stop treatments.…”

Efficacy of Glutathione for Patients With Cystic Fibrosis: A Meta-analysis of Randomized-Controlled Studies

“…Glutathione represents the first-line defense of the lung against oxidative stress-induced damage and improves the functional activity of different immune cells. [10][11][12] The reduction in glutathione levels is found in the airways of patients with cystic fibrosis. 13 Several studies have investigated the efficacy of inhaled glutathione for patients with cystic fibrosis, and the results confirm the positive impact of inhaled glutathione on pulmonary function in these subjects.…”