Evidence
This page contains a snapshot of featured content which highlights evidence addressing key clinical questions including areas of uncertainty. Please see the main topic reference list for details of all sources underpinning this topic.
BMJ Best Practice evidence tables
Evidence tables provide easily navigated layers of evidence in the context of specific clinical questions, using GRADE and a BMJ Best Practice Effectiveness rating. Follow the links at the bottom of the table, which go to the related evidence score in the main topic text, providing additional context for the clinical question. Find out more about our evidence tables.
This table is a summary of the analysis reported in a Cochrane Clinical Answer that focuses on the above important clinical question.
Confidence in the evidence is moderate or low to moderate where GRADE has been performed and the intervention may be more effective/beneficial than the comparison for key outcomes.
Population: People with cystic fibrosis aged 16 years and older (undergoing standard treatment including inhaled mucoactive agents and antibiotics)
Intervention: Web-based online platform (access to the CFHealthHub digital platform [website and smartphone application] including tailored, flexible support) plus use of electronic data‐logging nebulizers
Comparison: Usual care (use of electronic data‐logging nebulizers without access to the online platform)
| Outcome | Effectiveness (BMJ rating)? | Confidence in evidence (GRADE)? |
|---|---|---|
Treatment adherence (follow‐up: 12 months) | Favors intervention | Moderate |
Treatment burden (follow‐up: 12 months) | Favors intervention | Moderate |
Quality of life (follow‐up: 12 months): Cystic Fibrosis Questionnaire-revised (CFQ-R) subgroups (physical domain; emotional domain; social domain score; eating domain score; body image domain; respiratory domain; digestion domain) | No statistically significant difference | Moderate |
Forced expiratory volume in one second (FEV1% predicted; follow‐up: 12 months) | No statistically significant difference | Low |
Pulmonary exacerbations (follow‐up: 12 months) | See note ᵃ | Moderate |
Treatment‐related anxiety and depression (follow‐up: 12 months): subgroups (anxiety scores; depression scores) | No statistically significant difference | Moderate |
Adverse effects (follow‐up: 12 months): subgroups (all adverse events; serious adverse events) | No statistically significant difference | Moderate |
Note The Cochrane review which underpins this Cochrane Clinical Answer (CCA) noted that future research on the effect of digital technology on adherence should focus on both children and adults. ᵃ One RCT (607 participants) found no statistically significant difference between treatment groups for adjusted and unadjusted incidence rate ratios.
This evidence table is related to the following section/s:
This table is a summary of the analysis reported in a Cochrane Clinical Answer that focuses on the above important clinical question.
Confidence in the evidence is high or moderate to high where GRADE has been performed and there is no difference in effectiveness between the intervention and comparison for key outcomes.
Population: Adults and children with cystic fibrosis with a nonsense mutation in at least one allele of the CFTR gene
Intervention: Ataluren
Comparison: Placebo
| Outcome | Effectiveness (BMJ rating)? | Confidence in evidence (GRADE)? |
|---|---|---|
Change in % predicted forced expiratory volume (FEV)? (at 48 weeks) | No statistically significant difference ᵃ | High |
Change in forced vital capacity (at 48 weeks) | No statistically significant difference | GRADE assessment not performed for this outcome |
Sweat chloride (at 48 weeks) | No statistically significant difference | High |
Quality of life (at 48 weeks) | No statistically significant difference | High |
Mortality (at 48 weeks) | See note ᵇ | High |
Adverse events (≥ 10% of trial participants): acute kidney injury | Occurs more commonly with ataluren compared with placebo (favors comparison) | Moderate |
Adverse events (≥ 10% of trial participants): oropharyngeal pain | Occurs more commonly with placebo compared with ataluren (favors intervention) | Moderate |
Severity of adverse events | See note ᶜ | GRADE assessment not performed for this outcome |
Need for additional antibiotics, chest radiograph score | - | The RCT did not report these outcomes |
Note Ataluren may not be available in some countries, and where it is available, it may not be licensed for this indication. ᵃ This outcome was also reported at 8, 16, 24, 32 and 40 weeks and for a subgroup of patients not receiving long term inhaled tobramycin. The Cochrane Clinical Answer (CCA) noted that while ataluren improved lung function in some analyses, none of the results were clinically significant. See full CCA for more details. ᵇ Two RCTs (517 participants) reported that there were no deaths in either treatment group. ᶜ Two RCTs (512 participants) reported four different grades of adverse events (‘mild’, ‘moderate’, ‘severe’ and ‘life threatening or death’). No statistically significant difference between treatment groups was reported for the first two, while there was a decrease in severe adverse events with ataluren. No study participant experienced life threatening adverse events or death.
This evidence table is related to the following section/s:
Cochrane Clinical Answers
Cochrane Clinical Answers (CCAs) provide a readable, digestible, clinically focused entry point to rigorous research from Cochrane systematic reviews. They are designed to be actionable and to inform decision making at the point of care and have been added to relevant sections of the main Best Practice text.
- How do oral non‐steroidal anti‐inflammatory drugs affect outcomes in people with cystic fibrosis lung disease?
- How does the use of digital technology compare with usual care for monitoring inhaled therapy adherence in people with cystic fibrosis?
- What are the effects of digital technology for early identification of exacerbations in people with cystic fibrosis?
- What are the effects of ataluren for people with cystic fibrosis with a nonsense mutation in at least one allele of the CF transmembrane conductance regulator (CFTR) gene?
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