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National Guideline Alliance (UK). Cystic Fibrosis: Diagnosis and management. London: National Institute for Health and Care Excellence (NICE); 2017 Oct 25. (NICE Guideline, No. 78.)
| Term | Definition |
|---|---|
| Abstract | Summary of a study, which may be published alone or as an introduction to a full scientific paper. |
| ACT | Airway clearance techniques help to clear mucous from the lungs reducing the risk of infection and improve lung function. |
| Allocation concealment | The process used to prevent advance knowledge of group assignment in a randomised controlled trial (RCT). The allocation process should be impervious to any influence by the individual making the allocation, by being administered by someone who is not responsible for recruiting participants. |
| Alveolus (Alveoli) | The specialised part of the lung where oxygen enters the blood and carbon dioxide can leave. |
| Antimicrobial prophylaxis | Antimicrobial drugs administered to those without symptoms or positive cultures with the intention of preventing future infection/colonisation. |
| Applicability | How well the results of a study or NICE evidence review can answer a clinical question or be applied to the population being considered. |
| APRI | AST to platelet ratio index (APRI) is a liver fibrosis test. |
| Arm (of a clinical study) | Subsection of individuals within a study who receive one particular intervention, for example placebo arm. |
| Association | Statistical relationship between 2 or more events, characteristics or other variables. The relationship may or may not be causal. |
| Attrition bias | Systematic differences between comparison groups in withdrawals or exclusion of participants from a study. |
| Available case analysis | Analysis of data that is available for participants at the end of follow-up. |
| BAL | Bronchoalveolar lavage. The bronchi and alveoli are washed via bronchoscope with a small amount of fluid which is then collected for analysis. |
| Baseline | The initial set of measurements at the beginning of a study (after run-in period where applicable) with which subsequent results are compared. |
| Before-and-after study | A study that investigates the effects of an intervention by measuring particular characteristics of a population both before and after taking the intervention, and assessing any change that occurs. |
| Bias | Influences on a study that can make the results look better or worse than they really are. (Bias can even make it look as if a treatment works when it does not.) Bias can occur by chance, deliberately or as a result of systematic errors in the design and execution of a study. It can also occur at different stages in the research process, for example during the collection, analysis, interpretation, publication or review of research data. For examples see selection bias, performance bias, information bias, confounding factor and publication bias. |
| Bilirubin | A substance formed in liver by the breakdown of haemoglobin and excreted in bile. |
| Bronchus (Bronchi) | Small airways in the lung. |
| Cardiopulmonary exercise testing | Measurement of the function of the heart and lungs at rest and during exercise. |
| Carer (caregiver) | Someone who looks after family, partners or friends in need of help because they are ill, frail or have a disability. |
| Case series | Report of a number of cases of a given disease, usually covering the course of the disease and the response to treatment. There is no comparison (control) group of patients. |
| Case-control study | A study to find out the cause(s) of a disease or condition. This is done by comparing a group of patients who have the disease or condition (cases) with a group of people who do not have it (controls) but who are otherwise as similar as possible (in characteristics thought to be unrelated to the causes of the disease or condition). This means the researcher can look for aspects of their lives that differ to see if they may cause the condition. For example, a group of people with lung cancer might be compared with a group of people the same age that do not have lung cancer. The researcher could compare how long both groups had been exposed to tobacco smoke. Such studies are retrospective because they look back in time from the outcome to the possible causes of a disease or condition. |
| CFRD | Cystic fibrosis related diabetes. |
| Chronic pulmonary infection | Presence of pathogens on culture (colonisation) in the absence of worsening clinical symptoms/signs of respiratory disease. Antimicrobials may be administered to suppress or eradicate such pathogens with the intention of reducing future acute pulmonary exacerbations. |
| Cirrhosis | Progressive fibrous tissue overgrowth in an organ. |
| Clinical audit | A systematic process for setting and monitoring standards of clinical care. Whereas ‘guidelines’ define what the best clinical practice should be, ‘audit’ investigates whether best practice is being carried out. Clinical audit can be described as a cycle or spiral. Within the cycle there are stages that follow a systematic process of establishing best practice, measuring care against specific criteria, taking action to improve care and monitoring to sustain improvement. The spiral suggests that as the process continues, each cycle aspires to a higher level of quality. |
| Clinical effectiveness | How well a specific test or treatment works when used in the ‘real world’ (for example when used by a doctor with a patient at home), rather than in a carefully controlled clinical trial. Trials that assess clinical effectiveness are sometimes called management trials. Clinical effectiveness is not the same as efficacy. |
| Clinical efficacy | The extent to which an intervention is active when studied under controlled research conditions. |
| Clinician | A healthcare professional who provides patient care; for example a doctor, nurse or physiotherapist. |
| Cochrane Review | The Cochrane Library consists of a regularly updated collection of evidence based medicine databases including the Cochrane Database of Systematic Reviews (reviews of RCTs prepared by the Cochrane Collaboration). |
| Cohort study | A study with 2 or more groups of people – cohorts – with similar characteristics. One group receives a treatment, is exposed to a risk factor or has a particular symptom and the other group does not. The study follows their progress over time and records what happens. |
| Cohorting (cross-infection) | Grouping patients with positive cultures of the same pathogen(s). |
| Colonisation | Presence of pathogens on culture without signs of infection. See chronic pulmonary infection. |
| Community care | Care provided by community nurses, health visitors or school nurses of the region who look after people with CF and will administer treatments such as home IV antibiotics. It can include palliative services. |
| Comorbidity | A disease or condition that someone has in addition to the health problem being studied or treated. |
| Concealment of allocation | The process used to ensure that the person deciding to enter a participant into a randomised controlled trial does not know the comparison group into which that individual will be allocated. This is distinct from blinding and is aimed at preventing selection bias. Some attempts at concealing allocation are more prone to manipulation than others, and the method of allocation concealment is used as an assessment of the quality of a trial. |
| Confidence interval (CI) | There is always some uncertainty in research. This is because a small group of patients is studied to predict the effects of a treatment on the wider population. The confidence interval is a way of expressing how certain we are about the findings from a study, using statistics. It gives a range of results that is likely to include the ‘true’ value for the population. The CI is usually stated as ‘95% CI’, which means that the range of values has a 95 in 100 chance of including the ‘true’ value. For example, a study may state that “based on our sample findings, we are 95% certain that the ‘true’ population blood pressure is not higher than 150 and not lower than 110”. In such a case the 95% CI would be 110 to 150. A wide confidence interval indicates a lack of certainty about the true effect of the test or treatment – often because a small group of patients has been studied. A narrow confidence interval indicates a more precise estimate (for example if a large number of patients have been studied). |
| Confounding factor | Something that influences a study and can result in misleading findings if it is not understood or appropriately dealt with. For example, a study of heart disease may look at a group of people who exercise regularly and a group who do not exercise. If the ages of the people in the 2 groups are different, then any difference in heart disease rates between the 2 groups could be because of age rather than exercise. Therefore age is a confounding factor. |
| Consensus methods | Techniques used to reach agreement on a particular issue. Consensus methods may be used to develop NICE guidance if there is not enough good quality research evidence to give a clear answer to a question. Formal consensus methods include Delphi and nominal group techniques. |
| Continuous outcome | Data with a potentially infinite number of possible values within a given range. Height, weight and blood pressure are examples of continuous variables. |
| Control group | A group of people in a study who do not receive the treatment or test being studied. Instead, they may receive the standard treatment (sometimes called ‘usual care’) or a dummy treatment (placebo). The results for the control group are compared with those for a group receiving the treatment being tested. The aim is to check for any differences. Ideally, the people in the control group should be as similar as possible to those in the treatment group, to make it as easy as possible to detect any effects due to the treatment. |
| Corticosteroids | Anti-inflammatory medicines. |
| Cost–benefit analysis (CBA) | Cost-benefit analysis is one of the tools used to carry out an economic evaluation. The costs and benefits are measured using the same monetary units (for example UK pounds) to see whether the benefits exceed the costs. |
| Cost–consequence analysis (CCA) | Cost–consequence analysis is one of the tools used to carry out an economic evaluation. This compares the costs (such as treatment and hospital care) and the consequences (such as health outcomes) of a test or treatment with a suitable alternative. Unlike cost–benefit analysis or cost-effectiveness analysis, it does not attempt to summarise outcomes in a single measure (like the quality adjusted life year) or in financial terms. Instead, outcomes are shown in their natural units (some of which may be monetary) and it is left to decision-makers to determine whether, overall, the treatment is worth carrying out |
| Cost-effectiveness analysis (CEA) | Cost-effectiveness analysis is one of the tools used to carry out an economic evaluation. The benefits are expressed in non-monetary terms related to health, such as symptom-free days, heart attacks avoided, deaths avoided or life years gained (that is, the number of years by which life is extended as a result of the intervention). |
| Cost-effectiveness model | An explicit mathematical framework, which is used to represent clinical decision problems and incorporate evidence from a variety of sources in order to estimate the costs and health outcomes. |
| Cost-utility analysis (CUA) | Cost-utility analysis is one of the tools used to carry out an economic evaluation. The benefits are assessed in terms of both quality and duration of life, and expressed as quality adjusted life years (QALYs). |
| COX proportional hazard model | In survival analysis, a statistical model that asserts that the effect of the study factors (for example the intervention of interest) on the hazard rate (the risk of occurrence of an event) in the study population is multiplicative and does not change over time. |
| Credible interval (CrI) | The Bayesian equivalent of a confidence interval. |
| CT scan | Computerised tomography scan; computer-processed combinations of x-ray images producing cross-sectional images. |
| Decision analysis | An explicit quantitative approach to decision-making under uncertainty based on evidence from research. This evidence is translated into probabilities, and then into diagrams or decision trees which direct the clinician through a succession of possible scenarios, actions and outcomes. |
| Dichotomous outcomes | Outcome that can take 1 of 2 possible values, such as dead/alive, smoker/non-smoker, present/not present (also called binary data). |
| DIOS | Distal Intestinal Obstruction Syndrome. A blockage of the gut which occurs in older children and adults with cystic fibrosis. |
| Discounting | Costs and perhaps benefits incurred today have a higher value than costs and benefits occurring in the future. Discounting health benefits reflects individual preference for benefits to be experienced in the present rather than the future. Discounting costs reflects individual preference for costs to be experienced in the future rather than the present. |
| Dominance | A health economics term. When comparing tests or treatments, an option that is both less effective and costs more is said to be ‘dominated’ by the alternative. |
| Drop-out | A participant who withdraws from a trial before the end. |
| Economic evaluation | An economic evaluation is used to assess the cost effectiveness of healthcare interventions (that is, to compare the costs and benefits of a healthcare intervention to assess whether it is worth doing). The aim of an economic evaluation is to maximise the level of benefits – health effects – relative to the resources available. It should be used to inform and support the decision-making process; it is not supposed to replace the judgement of healthcare professionals. There are several types of economic evaluation: cost–benefit analysis, cost consequence analysis, cost-effectiveness analysis, cost-minimisation analysis and cost–utility analysis. They use similar methods to define and evaluate costs, but differ in the way they estimate the benefits of a particular drug, programme or intervention. |
| Effect (as in effect measure, treatment effect, estimate of effect, effect size) | A measure that shows the magnitude of the outcome in 1 group compared with that in a control group. For example, if the absolute risk reduction is shown to be 5% and it is the outcome of interest, the effect size is 5%. The effect size is usually tested, using statistics, to find out how likely it is that the effect is a result of the treatment and has not just happened incidentally. |
| Effectiveness | How beneficial a test or treatment is under usual or everyday conditions, compared with doing nothing or opting for another type of care. |
| Efficacy | How beneficial a test, treatment or public health intervention is under ideal conditions (for example in a laboratory), compared with doing nothing or opting for another type of care. |
| Enteric coated | Covered with a coating which protects against acid in the stomach. |
| Epidemiological study | The study of a disease within a population, defining its incidence and prevalence and examining the roles of external influences (for example infection, diet) and interventions. |
| EQ-5D (EuroQol 5 dimensions) | A standardised instrument used to measure health-related quality-of-life. It provides a single index value for health status. |
| Equivalence study | A trial designed to determine whether the response to 2 or more treatments differs by an amount that is clinically unimportant. This is usually demonstrated by showing that the true treatment difference is likely to lie between a lower and an upper equivalence level of clinically acceptable differences. |
| Eradication regimen, antibiotic | An antibiotic regimen aimed at eliminating a specific pulmonary pathogen such as S aureus or P aeruginosa in people with cystic fibrosis |
| Evidence | Information on which a decision or guidance is based. Evidence is obtained from a range of sources including randomised controlled trials, observational studies, expert opinion (of clinical professionals or patients). |
| Exclusion criteria (clinical study) | Criteria that define who is not eligible to participate in a clinical study. |
| Exclusion criteria (literature review) | Explicit standards used to decide which studies should be excluded from consideration as potential sources of evidence. |
| Extended dominance | If Option A is both more clinically effective than Option B and has a lower cost per unit of effect when both are compared with a do-nothing alternative, then Option A is said to have extended dominance over Option B. Option A is therefore more cost effective and should be preferred, other things remaining equal. |
| Extrapolation | An assumption that the results of studies of a specific population will also hold true for another population with similar characteristics. |
| Fibroscan | See Transient elastography |
| Fixed-effect model | In meta-analysis, a model that calculates a pooled effect estimate using the assumption that all observed variation between studies is caused by the play of chance. Studies are assumed to be measuring the same overall effect. |
| Follow-up | Observation over a period of time of an individual, group or initially defined population whose appropriate characteristics have been assessed in order to observe changes in health status or health-related variables. |
| Forest plot | A graphical representation of the individual results of each study included in a meta-analysis together with the combined meta-analysis result. The plot also allows readers to see the heterogeneity among the results of the studies. The results of individual studies are shown as squares centred on each study’s point estimate. A horizontal line runs through each square to show each study’s confidence interval. The overall estimate from the meta-analysis and its confidence interval are shown at the bottom, represented as a diamond. The centre of the diamond represents the pooled point estimate, and its horizontal tips represent the confidence interval. |
| Forns score | A non-invasive marker of liver fibrosis. |
| Generalisability | The extent to which the results of a study hold true for groups that did not participate in the research. See also external validity. |
| Gold standard | A method, procedure or measurement that is widely accepted as being the best available to test for or treat a disease. |
| GRADE, GRADE profile | A system developed by the GRADE Working Group to address the shortcomings of present grading systems in healthcare. The GRADE system uses a common, sensible and transparent approach to grading the quality of evidence. The results of applying the GRADE system to clinical trial data are displayed in a table known as a GRADE profile. |
| Haemophilus influenzae | A bacterium which is a common cause of respiratory infection in cystic fibrosis. |
| Haemoptysis | Coughing up blood. |
| Harms | Adverse effects of an intervention. |
| Hazard ratio | A hazard is the rate at which events happen, so that the probability of an event happening in a short time interval is the length of time multiplied by the hazard. Although the hazard may vary with time, the assumption in proportional hazard models for survival analysis is that the hazard in one group is a constant proportion of the hazard in the other group. This proportion is the hazard ratio. |
| Health economics | Study or analysis of the cost of using and distributing healthcare resources. |
| Health related quality of life (HRQoL) | A measure of the effects of an illness to see how it affects someone’s day-to-day life. |
| Heterogeneity | The term is used in meta-analyses and systematic reviews to describe when the results of a test or treatment (or estimates of its effect) differ. |
| HFCWO | High frequency chest wall oscillation; technique aimed at improving airway clearance |
| Home care (e.g. hospital at home) | Giving care at home instead of a hospital, provided by the relevant cystic fibrosis specialist (such as a specialist nurse, dietitian or psychologist). |
| Immunomodulatory dose | The use of a drug such as azithromycin prescribed at a lower dose than the minimum inhibitory dose. |
| Imprecision | Results are imprecise when studies include relatively few patients and few events and thus have wide confidence intervals around the estimate of effect. |
| Inclusion criteria (literature review) | Explicit criteria used to decide which studies should be considered as potential sources of evidence. |
| Incremental cost | The extra cost linked to using one test or treatment rather than another. Or the additional cost of doing a test or providing a treatment more frequently. |
| Incremental cost effectiveness ratio (ICER) | The difference in the mean costs in the population of interest divided by the differences in the mean outcomes in the population of interest for one treatment compared with another |
| Incremental net benefit (INB) | The value (usually in monetary terms) of an intervention net of its cost compared with a comparator intervention. The INB can be calculated for a given cost-effectiveness (willingness to pay) threshold. If the threshold is £20,000 per QALY gained then the INB is calculated as: (£20,000×QALYs gained) minus incremental cost. |
| Indirectness | The available evidence is different to the review question being addressed, in terms of population, intervention, comparison and outcome (PICO). |
| Intention-to-treat analysis (ITT) | An assessment of the people taking part in a clinical trial, based on the group they were initially (and randomly) allocated to. This is regardless of whether or not they dropped out, fully complied with the treatment or switched to an alternative treatment. Intention-to-treat analyses are often used to assess clinical effectiveness because they mirror actual practice: that is, not everyone complies with treatment and the treatment people receive may be changed according to how they respond to it. |
| Intervention | In medical terms this could be a drug treatment, surgical procedure, diagnostic or psychological therapy. Examples of public health interventions could include action to help someone to be physically active or to eat a more healthy diet. |
| Kappa statistic | A statistical measure of inter-rater agreement that takes into account the agreement occurring by chance. |
| LCI | Lung Clearance Index. A measure of lung function. |
| Length of stay | The total number of days a participant stays in hospital. |
| Licence | See ‘Product licence’. |
| Life years gained | Mean average years of life gained per person as a result of the intervention compared with an alternative intervention. |
| Likelihood ratio | The likelihood ratio combines information about the sensitivity and specificity. It tells you how much a positive or negative result changes the likelihood that a patient would have the disease. The likelihood ratio of a positive test result (LR+) is sensitivity divided by (1 minus specificity). |
| Loss to follow-up | Patients who have withdrawn from a clinical trial at the point of follow-up. |
| Malabsorption | A failure to absorb nutrients from the intestine. In cystic fibrosis this is due to the common occurrence of exocrine pancreatic insufficiency, so that there is a deficiency or absence of the pancreatic enzymes necessary to digest complex carbohydrates, proteins and fats (maldigestion) resulting in an inability to absorb these and other nutrients |
| Maldigestion | See malabsorption. |
| Markov model | A method for estimating long-term costs and effects for recurrent or chronic conditions, based on health states and the probability of transition between them within a given time period (cycle). |
| MDT | Multi-disciplinary team. A patient care team comprised of healthcare professionals of various specialties. |
| Mean | An average value, calculated by adding all the observations and dividing by the number of observations. |
| Mean difference | In meta-analysis, a method used to combine measures on continuous scales (such as weight), where the mean, standard deviation and sample size in each group are known. The weight given to the difference in means from each study (for example how much influence each study has on the overall results of the meta-analysis) is determined by the precision of its estimate of effect. |
| Meconium ileus | An obstruction of the small intestine at birth due to inspissated material in the gut lumen in infants with cystic fibrosis. |
| Meconium Ileus Equivalent | See DIOS |
| Median | The value of the observation that comes half-way when the observations are ranked in order. |
| Meta-analysis | A method often used in systematic reviews. Results from several studies of the same test or treatment are combined to estimate the overall effect of the treatment. |
| Microspheres | Enzyme granules contained within a pancreatin capsule. |
| Minimal important difference (MID) | Thresholds for clinical importance, which represent minimal important differences for benefit or for harm; for example the threshold at which drug A is less effective than drug B by an amount that is clinically important to patients. |
| MRI | Magnetic Resonance Imaging. Diagnostic imaging using magnetic fields and radio waves. |
| Mucoactive agent | See mucolytic agent. |
| Mucolytic agent | Drug affecting the viscosity of mucus, typically administered with the intention of making the removal of mucus through coughing easier. |
| Multivariate model | A statistical model for analysis of the relationship between 2 or more predictor (independent) variables and the outcome (dependent) variable. |
| Nebuliser | A small machine which converts liquid medication to a fine mist which can be breathed in to work directly in the lungs. |
| Net monetary benefit (NMB) | The value (usually in monetary terms) of an intervention net of its cost. The NMB can be calculated for a given cost-effectiveness (willingness to pay) threshold. If the threshold is £20,000 per QALY gained then the NMB is calculated as: (£20,000×QALYs gained) minus cost. |
| Network meta-analysis | Meta-analysis in which multiple treatments (that is, 3 or more) are being compared using both direct comparisons of interventions within RCTs and indirect comparisons across trials based on a common comparator. |
| Number needed to treat (NNT) | The average number of patients who need to be treated to get a positive outcome. For example, if the NNT is 4, then 4 patients would have to be treated to ensure 1 of them gets better. The closer the NNT is to 1, the better the treatment. For example, if you give a stroke prevention drug to 20 people before 1 stroke is prevented, the number needed to treat is 20. |
| Observational study | Individuals or groups are observed or certain factors are measured. No attempt is made to affect the outcome. For example, an observational study of a disease or treatment would allow ‘nature’ or usual medical care to take its course. Changes or differences in 1 characteristic (for example whether or not people received a specific treatment or intervention) are studied without intervening. There is a greater risk of selection bias than in experimental studies. |
| Odds ratio (OR) | Odds are a way to represent how likely it is that something will happen (the probability). An odds ratio compares the probability of something in 1 group with the probability of the same thing in another. An odds ratio of 1 between 2 groups would show that the probability of the event (for example a person developing a disease or a treatment working) is the same for both. An odds ratio greater than 1 means the event is more likely in the first group. An odds ratio less than 1 means that the event is less likely in the first group. Sometimes probability can be compared across more than 2 groups - in this case, 1 of the groups is chosen as the ‘reference category’, and the odds ratio is calculated for each group compared with the reference category. For example, to compare the risk of dying from lung cancer for non-smokers, occasional smokers and regular smokers, non-smokers could be used as the reference category. Odds ratios would be worked out for occasional smokers compared with non-smokers and for regular smokers compared with non-smokers. See also confidence interval, relative risk, risk ratio. |
| Odds ratio (OR) | Odds are a way to represent how likely it is that something will happen (the probability). An odds ratio compares the probability of something in 1 group with the probability of the same thing in another. An odds ratio of 1 between 2 groups would show that the probability of the event (for example a person developing a disease, or a treatment working) is the same for both. An odds ratio greater than 1 means the event is more likely in the first group. An odds ratio less than 1 means that the event is less likely in the first group. Sometimes probability can be compared across more than 2 groups – in this case, 1 of the groups is chosen as the ‘reference category’ and the odds ratio is calculated for each group compared with the reference category. For example, to compare the risk of dying from lung cancer for non-smokers, occasional smokers and regular smokers, non-smokers could be used as the reference category. Odds ratios would be worked out for occasional smokers compared with non-smokers and for regular smokers compared with non-smokers. |
| Opportunity cost | The loss of other healthcare programmes displaced by investment in or introduction of another intervention. This may be best measured by the health benefits that could have been achieved had the money been spent on the next best alternative healthcare intervention. |
| Outcome | The impact that a test, treatment, policy, programme or other intervention has on a person, group or population. Outcomes from interventions to improve the public’s health could include changes in knowledge and behaviour related to health, societal changes (for example a reduction in crime rates) and a change in people’s health and wellbeing or health status. In clinical terms, outcomes could include the number of patients who fully recover from an illness or the number of hospital admissions, and an improvement or deterioration in someone’s health, functional ability, symptoms or situation. Researchers should decide what outcomes to measure before a study begins. |
| Outreach care | A model of care in which the specialist multidisciplinary cystic fibrosis team provide outpatient clinics in local hospitals. |
| P value | The p value is a statistical measure that indicates whether or not an effect is statistically significant. For example, if a study comparing 2 treatments found that 1 seems more effective than the other, the p value is the probability of obtaining these results by chance. By convention, if the p value is below 0.05 (that is, there is less than a 5% probability that the results occurred by chance) it is considered that there probably is a real difference between treatments. If the p value is 0.001 or less (less than a 1% probability that the results occurred by chance), the result is seen as highly significant. If the p value shows that there is likely to be a difference between treatments, the confidence interval describes how big the difference in effect might be. |
| Pancreatin | An extract of animal pancreas; the general name for all pancreatic enzymes. |
| PEP | Positive expiratory pressure; a technique aimed at improving airway clearance. |
| Performance bias | Systematic differences between intervention groups in care provided apart from the intervention being evaluated. Blinding of study participants (both the recipients and providers of care) is used to protect against performance bias. |
| PERT | Pancreatic enzyme replacement therapy. |
| Placebo | A fake (or dummy) treatment given to participants in the control group of a clinical trial. It is indistinguishable from the actual treatment (which is given to participants in the experimental group). The aim is to determine what effect the experimental treatment has had – over and above any placebo effect caused because someone has received (or thinks they have received) care or attention. |
| Placebo effect | A beneficial (or adverse) effect produced by a placebo and not due to any property of the placebo itself. |
| Post-hoc analysis | Statistical analyses that are not specified in the trial protocol and are generally suggested by the data. |
| Power (statistical) | The ability to demonstrate an association when one exists. Power is related to sample size; the larger the sample size, the greater the power and the lower the risk that a possible association could be missed. |
| Primary care | Healthcare delivered outside hospitals. It includes a range of services provided by GPs, nurses, health visitors, midwives and other healthcare professionals and allied health professionals such as dentists, pharmacists and opticians. |
| Primary outcome | The outcome of greatest importance, usually the one in a study that the power calculation is based on. |
| Product licence | An authorisation from the MHRA to market a medicinal product. |
| Prognosis | A probable course or outcome of a disease. Prognostic factors are patient or disease characteristics that influence the course. Good prognosis is associated with low rate of undesirable outcomes; poor prognosis is associated with a high rate of undesirable outcomes. |
| Prophylactic antibiotics | Antibiotics used for the prevention of infection complications. |
| Prospective study | A research study in which the health or other characteristic of participants is monitored (or ‘followed up’) for a period of time, with events recorded as they happen. This contrasts with retrospective studies. |
| Pseudomonas aeruginosa | A bacterial infection which affects the lungs. |
| Publication bias | Publication bias occurs when researchers publish the results of studies showing that a treatment works well and don’t publish those showing it did not have any effect. If this happens, analysis of the published results will not give an accurate idea of how well the treatment works. This type of bias can be assessed by a funnel plot. |
| Pulmonary exacerbation | The sudden or recent worsening of clinical symptoms or signs. This is frequently caused by an acute pulmonary infection. |
| Pulmonary infection | In people with cystic fibrosis, this can be diagnosed based on symptoms or signs, or by identifying pathogens in respiratory secretion samples. |
| Pyrexia | A fever. |
| Sepsis | A whole-body inflammation caused by an infection. |
| Shared-care (Network CF Clinic) | When a local hospital cares for people with cystic fibrosis, with oversight, support and direct involvement from members of a specialist cystic fibrosis multidisciplinary team. |
| Specialist centre | This is a centre for the diagnosis and management of cystic fibrosis, and working with a multidisciplinary team approach. Cystic fibrosis specialist centres are commissioned by NHS England. |
| Spirometry | A lung function test measuring the volume and/or speed of air that can be inhaled and exhaled. |
| Stakeholder | An organisation with an interest in a topic that NICE is developing a NICE guideline or piece of public health guidance on. Organisations that register as stakeholders can comment on the draft scope and the draft guidance. Stakeholders may be: |
| Standard deviation (SD) | A measure of the spread or dispersion of a set of observations, calculated as the average difference from the mean value in the sample. |
| Staphylococcus aureus | A bacterial infection that can affect the lungs. |
| Steatorrhoea | Abnormal amount of fat in faeces due to malabsorption. |
| Subgroup analysis | An analysis in which the intervention effect is evaluated in a defined subset of the participants in a trial, or in complementary subsets. |
| Suppression regimen, antibiotic | An antibiotic regimen aimed at reducing the level of infection with a specific pulmonary pathogen such as S aureus or P aeruginosa in people with cystic fibrosis |
| Systematic review (SR) | A review in which evidence from scientific studies has been identified, appraised and synthesised in a methodical way according to predetermined criteria. It may include a meta-analysis. |
| Telemedicine | Providing clinical services remotely, using phone and video messaging to communicate with the patient. |
| Time horizon | The time span over which costs and health outcomes are considered in a decision analysis or economic evaluation. |
| Transient elastography | An ultrasound technique to measure tissue stiffness. |
| Treatment allocation | Assigning a participant to a particular arm of a trial. |
| UDCA | Ursodeoxycholic acid; drug administered with the intention of preventing progression of liver disease. |
| Ultrasound | Imaging technique using ultrasound (high frequency sound waves). |
| Univariate | Analysis which separately explores each variable in a data set. |
| Utility | In health economics, a ‘utility’ is the measure of the preference or value that an individual or society places upon a particular health state. It is generally a number between 0 (representing death) and 1 (perfect health). The most widely used measure of benefit in cost–utility analysis is the quality adjusted life year (QALY), but other measures include disability adjusted life years (DALYs) and healthy year equivalents (HYEs). |
- Glossary and abbreviations - Cystic FibrosisGlossary and abbreviations - Cystic Fibrosis
- Summary of identified studies - Cystic FibrosisSummary of identified studies - Cystic Fibrosis
- United States Agency for International DevelopmentUnited States Agency for International DevelopmentAn independent Federal agency established in 1961 as the focal point for economic matters affecting U.S. relations with developing countries.<br/>Year introduced: 2008MeSH
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