anurajms/meta_analysis huggingface dataset:
primary outcome:
Immunosuppressive agents for treating IgA nephropathy
ESKD requiring kidney replacement therapy (KRT) (dialysis or kidney transplantation)Complete remission: defined by a reduction in urinary protein excretion to less than 1 g/24 hours in three consecutive daily samples or as defined by the investigatorsDoubling of SCrSCr (µmol/L)Estimated or measured glomerular filtration rate (GFR) (either creatinine clearance (CrCl) (mL/min) or Cockcroft clearance (mL/min/1.73 m2)Urinary protein excretion (g/24 hours)
primary outcome:
Sclerotherapy versus sham or no intervention for primary prophylaxis of oesophageal variceal bleeding in children with chronic liver disease or portal vein thrombosis
All‐cause mortality.Upper gastrointestinal bleeding.Serious adverse events and liver‐related morbidity (i.e. proportion of participants who developed ascites, hepatorenal syndrome, hepatocellular carcinoma, or hepatic encephalopathy). A serious adverse event, defined according to the International Conference on Harmonisation (ICH) Guidelines for Good Clinical Practice (ICH‐GCP 1997), was any untoward medical occurrence that resulted in death, was life‐threatening, required hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability or incapacity, or was a congenital anomaly or birth defect. All other adverse events were considered non‐serious adverse events.Health‐related quality of life, determined exclusively by means of validated scales, classifications, and measurement systems such as the Paediatric Quality of Life Inventory (PedsQL), Child Health Questionnaire (CHQ), and DISABKIDS.
primary outcome:
Probiotics for maintenance of remission in ulcerative colitis
Relapse (clinical, endoscopic, histopathological or radiological), as defined by the authors of the primary studies.
primary outcome:
Home fortification of foods with multiple micronutrient powders for health and nutrition in children under two years of age
Anaemia (defined as haemoglobin values lower than 110 g/L)Iron deficiency (as defined by trialists)Haemoglobin concentration (g/L)Iron status (as defined by trialists)Weight for age (z scores)All‐cause mortality
primary outcome:
Timing of hypertonic saline inhalation for cystic fibrosis
Lung function, i.e., change in lung function (measured in litres or % predicted). If change values are unavailable, final post‐treatment values will be used. Values in litres and in % predicted will be analysed separately.forced expiratory volume at one second (FEV1)forced vital capacity (FVC)Patient‐reported outcomes, using validated scales or subjective reporting measuresmeasures of QoLsymptom scores (including cough, tolerability, subjective ease of clearance, or treatment satisfaction)
primary outcome:
Different unfractionated heparin doses for preventing arterial thrombosis in children undergoing cardiac catheterization
Arterial thrombotic eventsMajor bleeding eventsfatal bleeding;bleeding that is symptomatic and occurs in a critical area or organ (such as intracranial, intraspinal, intraocular, retroperitoneal, pericardial, in a non‐operated joint, or intramuscular with compartment syndrome), assessed in consultation with the surgeon;extrasurgical site bleeding causing a fall in hemoglobin level of 20 g/L (1.24 mmol/L) or more, or leading to transfusion of whole blood or red cells with a temporal association within 24 to 48 h of the bleeding;surgical site bleeding that requires a second intervention (open, arthroscopic, endovascular) or a hemarthrosis of sufficient size as to interfere with rehabilitation by delaying mobilization or causing delayed wound healing, resulting in prolonged hospitalization or a deep wound infection;surgical site bleeding that is unexpected and prolonged or sufficiently large to cause hemodynamic instability, as assessed by the surgeon. There should be an associated fall in the hemoglobin level of at least 20 g/L (1.24 mmol/L), or a transfusion indicated by the bleeding with a temporal association within 24 h of the bleeding.Minor bleeding events: overt or macroscopic evidence of bleeding not encompassed in the above definition for major bleeding
primary outcome:
Ibuprofen for the treatment of patent ductus arteriosus in preterm or low birth weight (or both) infants
Failure of permanent PDA closure within one week of administration of the first dose of ibuprofen (PDA diagnosed either clinically or by ECHO criteria).
primary outcome:
Early versus late erythropoietin for preventing red blood cell transfusion in preterm and/or low birth weight infants
Use of one or more red blood cell (RBC) transfusions.
primary outcome:
Surfactant for pulmonary haemorrhage in neonates
All‐cause mortality during initial hospital stay.Mortality due to PH during initial hospitalisation.All‐cause mortality during the neonatal period.
primary outcome:
Intravenous immunoglobulin for preventing infection in preterm and/or low birth weight infants
Sepsis, one or more episodes (clinical signs and symptoms of sepsis and positive blood culture for bacteria or fungi).
primary outcome:
Paracetamol (acetaminophen) for prevention or treatment of pain in newborns
Behavioural (cry duration, proportion of time crying, facial actions).Physiological (heart rate, respiratory rate, saturation of peripheral oxygen in the blood (SpO₂), transcutaneous oxygen and carbon dioxide (gas exchange measured across the skin — TcpO₂, TcpCO₂).Validated composite pain scores.Combination of these.
primary outcome:
Oral Janus kinase inhibitors for maintenance of remission in ulcerative colitis
The primary outcome was the proportion of participants who failed to maintain clinical remission (as defined by the included studies).
primary outcome:
Interventions for chronic pruritus of unknown origin
Patient‐ or parent‐reported pruritus intensity, as measured by a visual analogue scale (Pereira 2017), the Itch Severity Scale (Majeski 2007), or another validated, or commonly used, scaleAdverse events, including pruritus exacerbation, skin irritation, or other agent‐specific adverse events
primary outcome:
Probiotics for people with cystic fibrosis
Pulmonary exacerbation defined using consensus criteria, e.g. Fuch's criteria (Fuchs 1994)number of pulmonary exacerbationsduration of antibiotic therapy (any route) for pulmonary exacerbations (days)Inflammatory biomarkers (mean change from baseline and post treatment absolute mean)intestinalcalprotectin (µg/g)M2‐PK (U/mL)rNO (µmol/L)serumC‐reactive protein (CRP) (mg/L)cytokines (pg/mL))sputumC‐reactive protein (mg/L)cytokines (pg/mL)Adverse eventsserious adverse reactions defined as any untoward medical occurrence which results in death, is life‐threatening, requires hospitalisation or prolongation of hospitalisation, or results in persistent or significant disability or incapacity (e.g. sepsis (bacteraemia))adverse reaction defined as any untoward medical occurrence with reasonable causal relationships (e.g. nausea and abdominal bloating)mortality (all causes)
primary outcome:
Improving vaccination uptake among adolescents
Adolescent vaccination coverage, that is, the proportion of adolescents who have received the recommended dose(s) of the vaccine(s) studied.
primary outcome:
Hypertonic saline versus other intracranial pressure–lowering agents for people with acute traumatic brain injury
Death at final follow‐up (grouped by period of reporting e.g. short term (while in ICU); long term (at six months)
primary outcome:
Anti‐inflammatory medications for obstructive sleep apnoea in children
AHI as measured by polysomnographySerious adverse events
primary outcome:
Sclerotherapy versus beta‐blockers for primary prophylaxis of oesophageal variceal bleeding in children and adolescents with chronic liver disease or portal vein thrombosis
All‐cause mortality.Upper gastrointestinal bleeding.Serious adverse events and liver‐related morbidity (i.e. proportion of participants who developed ascites, hepatorenal syndrome, hepatocellular carcinoma, or hepatic encephalopathy). A serious adverse event, defined according to the International Conference on Harmonisation (ICH) Guidelines for Good Clinical Practice (ICH‐GCP 1997), was any untoward medical occurrence that resulted in death, was life‐threatening, required hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability or incapacity, or was a congenital anomaly or birth defect. All other adverse events were considered non‐serious adverse events.Health‐related quality of life, determined exclusively by validated scales, classification, and measurement systems such as the Paediatric Quality of Life Inventory (PedsQL), Child Health Questionnaire (CHQ), and the Europena DISABKIDS tool (assesses health‐related quality of life in children and adolescents with a chronic condition).
primary outcome:
Topical antiseptics for chronic suppurative otitis media
Resolution of ear discharge or 'dry ear' (whether otoscopically confirmed or not), measured at:between one week and up to two weeks;two weeks to up to four weeks; andafter four weeks.Health‐related quality of life using a validated instrument for CSOM (e.g. Chronic Otitis Media Questionnaire (COMQ)‐12 (Phillips 2014a; Phillips 2014b; van Dinther 2015), Chronic Otitis Media Outcome Test (COMOT)‐15 (Baumann 2011), Chronic Ear Survey (CES) (Nadol 2000)).Ear pain (otalgia) or discomfort or local irritation.
primary outcome:
Drug treatment for spinal muscular atrophy types II and III
Change in disability score (e.g. Gross Motor Function Measure (GMFM), Hammersmith Functional Motor Score (HFMS), Motor Function Measure (MFM) and SMA Functional Rating Scale (SMAFRS)) as determined by the original study authors.
primary outcome:
Antibiotics versus topical antiseptics for chronic suppurative otitis media
Resolution of ear discharge or 'dry ear' (whether otoscopically confirmed or not), measured at:between one week and up to two weeks;two weeks to up to four weeks; andafter four weeks.Health‐related quality of life using a validated instrument for CSOM (e.g. Chronic Otitis Media Questionnaire (COMQ)‐12 (Phillips 2014a; Phillips 2014b; van Dinther 2015), Chronic Otitis Media Outcome Test (COMOT)‐15 (Baumann 2011), Chronic Ear Survey (CES) (Nadol 2000)).Ear pain (otalgia) or discomfort or local irritation.
primary outcome:
Caregiver involvement in interventions for improving children's dietary intake and physical activity behaviors
Children's dietary intake (i.e. total energy intake, percentage of total energy intake from fat, percentage of total energy intake from saturated fat, percentage of total energy intake from trans fat, percentage of total energy from carbohydrates, percentage of total energy from protein, sodium intake, fruit and vegetable intake, sugar‐sweetened beverage [SSB] intake, and water intake), as measured by instruments such as 24‐hour dietary recalls and food frequency questionnairesChildren's physical activity levels (i.e. total physical activity, time spent in moderate to vigorous physical activity [MVPA], time spent in light physical activity [LPA], time spent in weight‐bearing physical activity, steps), as measured by instruments such as accelerometers, pedometers, and physical activity questionnairesAdverse effects (as defined by trial authors), such as family conflict or disordered eating or activity behaviors
primary outcome:
Topical antibiotics for chronic suppurative otitis media
Resolution of ear discharge or 'dry ear' (whether otoscopically confirmed or not), measured at:between one week and up to two weeks;two weeks to up to four weeks; andafter four weeks.Health‐related quality of life using a validated instrument for CSOM (e.g. Chronic Otitis Media Questionnaire (COMQ)‐12 (Phillips 2014a; Phillips 2014b; van Dinther 2015), Chronic Otitis Media Outcome Test (COMOT)‐15 (Baumann 2011), Chronic Ear Survey (CES) (Nadol 2000).Ear pain (otalgia) or discomfort or local irritation.
primary outcome:
Decompressive craniectomy for the treatment of high intracranial pressure in closed traumatic brain injury
Neurological outcome at 6 or 12 months evaluated with the dichotomized Glasgow Outcome Scale (GOS) (Jennett 1975 or subsequent versions) and categorized into ’good’ or ’bad’ outcomes. Patients with a good recovery or moderate disability were included in the good outcome group while those who were severely disabled, remained in a vegetative state, or died were included in the bad outcome group. The introduction in 1981 of the extended version of the GOS (GOS‐E) changed the traditional 5‐point scale to an 8‐point scale (Jennett 1981). The main difference between the original and the extended scale is that the latter separates patients in the moderate and severe disability categories into two subcategories: 'upper' and 'lower' on the basis of a structured interview (Wilson 1998). We, the review authors, are aware that thinking regarding this ’traditional’ dichotomy has changed, and that ongoing controversies regarding what should be considered a good or favorable outcome involve value judgements about the quality of life of the survivors. As a result of this, we decided to present outcome data in a variety of formats, to help to contextualize the choices that patients, families and clinicians need to face in the informed consent process for DC (see Appendix 1; Appendix 2)Mortality: we included one month mortality in our protocol to avoid problems in evaluation of medium‐term outcomes in contemporary clinical trials in which loss to follow‐up is common (Sahuquillo 2002b). If DC is effective, a significant reduction in short‐term mortality can be expected. However, mortality at one month is rarely reported in clinical trials, so we reported longer‐term data for this outcome as well.
primary outcome:
Tonsillectomy for periodic fever, aphthous stomatitis, pharyngitis and cervical adenitis syndrome (PFAPA)
Proportion of children whose symptoms have completely resolved.Complications of surgery (haemorrhage and number of days of postoperative pain).
primary outcome:
Racecadotril for acute diarrhoea in children
Failure of oral rehydration, defined as any of the following: worsening or no improvement of dehydration; recurring dehydration; need for unscheduled intravenous fluid infusion; participants initially without dehydration subsequently developing dehydration; or need for hospital admission in outpatients.Duration of diarrhoea, defined as the time period in days from randomization or start of treatment to the resolution of diarrhoea.Number of stools, defined as the number of stools or bowel movements within 48 hours after administration of interventions.
primary outcome:
Equivalent schedules of intradermal fractional dose versus intramuscular full dose of inactivated polio vaccine for prevention of poliomyelitis
Paralytic poliomyelitisSeroconversion rateGeometric mean titres of antibodies for wild poliovirus types 1, 2, and 3Any vaccine‐related adverse event after each dose (early or late events; e.g. injection site reactions, pyrexia, haematomas, or gastroenteritis, etc.), measured by the number of participants in an arm having an adverse event/total number of participants in that arm
primary outcome:
Treatments for preventing recurrence of infection with Pseudomonas aeruginosa in people with cystic fibrosis
Time to next isolation of PA (identified by any method, e.g. sputum culture (spontaneous or induced), BAL or OP culture and as defined by the trial investigators)
primary outcome:
Prophylactic antibiotics for penetrating abdominal trauma: duration of use and antibiotic choice
Abdominal surgical site infection
primary outcome:
Interventions for managing halitosis
For the primary outcomes in this review we considered self‐expressed (perceived) (Greenman 2004) and organoleptic test (OLT) (human nose) assessments of halitosis using any validated malodour intensity scale.
primary outcome:
Drug treatment for spinal muscular atrophy type I
Time from birth until death or full‐time ventilation (a requirement for 16 hours of ventilation per day regardless of whether this is with a tracheostomy, tube or mask)
primary outcome:
Iron therapy for preoperative anaemia
Proportion of participants who received a blood transfusion
primary outcome:
Primary closure versus delayed or no closure for traumatic wounds due to mammalian bite
Time to complete wound healingProportion of wounds healed within 7 days, 10 days and 14 days (from occurrence of bite)Proportion of wounds which were infection‐free in 7 days, 10 days and 14 days; where wound infection was defined as purulent discharge, or erythema associated with pus, or cellulitis, or infection as evidenced by tissue/swab microbial culture (or as defined by the trial authors)
primary outcome:
Corticosteroids for treating sepsis in children and adults
28‐Day all‐cause mortality
primary outcome:
Beginning reading interventions for children and adolescents with intellectual disability
Phonologic awareness (e.g. ability to blend individual phonemes into a word), measured by standardized measures such as the Comprehensive Test of Phonological Processing (CTOPP; Wagner 1999) or Test of Phonological Awareness (TOPA; Torgesen 1994).Sound‐symbol correspondence (e.g. ability to produce phonemes correctly when presented with isolated graphemes).Word reading (e.g. ability to pronounce a written word correctly), measured by assessments of reading efficiency such as the Sight Word Efficiency subscale of the Test of One Word Reading Efficiency (TOWRE; Torgesen 1999) or the Word Identification subscale of the Woodcock Reading Mastery Test (WRMT; Woodcock 1987).Decoding (e.g. ability to use sound‐symbol knowledge to decode novel words or pseudo‐words), measured by assessments of reading efficiency such as the Phonemic Decoding Efficiency subscale of the TOWRE (Torgesen 1999), or the Word Attack subscale of the WRMT (Woodcock 1987); or curriculum‐based measures of non‐word reading fluency (NWF) such as Dynamic Indicators of Basic Early Literacy Skills (DIBELS; Good 2002).Adverse events (e.g. problem behavior, social stigmatism).
primary outcome:
Sequential inactivated (IPV) and live oral (OPV) poliovirus vaccines for preventing poliomyelitis
Paralytic polio, measured as change in level and change in slope by ITS.
primary outcome:
Parent training programmes for managing infantile colic
Change in duration of crying (post‐intervention versus baseline). Data could have been continuous (e.g. hours per day), or dichotomous (e.g. reduction under a predefined threshold, as determined by the study authors)Crying time at completion of study period (endpoint). Data could have been continuous (e.g. hours per day), or dichotomous (e.g. reduction under a predefined threshold, as determined by the trial authors)Adverse effects, including choking, apparent life‐threatening events (dichotomous outcome)
primary outcome:
Intravenous immunoglobulins for epilepsy
Seizure freedom: proportion of participants with complete cessation of seizures during the treatment period.Satisfactory seizure control: proportion of participants with at least 50% or greater reduction in seizure frequency at the end of the study compared with the baseline.
primary outcome:
Video feedback for parental sensitivity and attachment security in children under five years
Parental sensitivity, measured by, for example, the Ainsworth Sensitivity Scale (ASS; Ainsworth 1974), the Child‐Adult Relationship Experimental Index (CARE‐Index; Crittenden 2001), the Parental Sensitivity Assessment Scale (PSAS; Hoff 2004), Coding Interactive Behaviour (CIB; Feldman 1998), the Emotional Availability (EA) scales (Biringen 2000a), Global Ratings Scales (GRS) of mother‐infant interaction (Murray 1996), Maternal Behaviour Q‐sort (MBQS; Pederson 1999), or Nursing Child Assessment Teaching Scale (NCATS; Sumner 1994).Parental reflective functioning, measured by, for example, the Parent Development Interview (PDI; unpublished manuscript by Aber 1985), or the PDI‐Revised (PDI‐R; unpublished manuscript by Slade 2004).Attachment security, measured by, for example, the Attachment Q‐sort (AQS; Waters 1985; Waters 1987), or the Strange Situation Procedure (SSP; Ainsworth 1978).Adverse effects. We acknowledge that a worsening of any of our primary outcomes listed above would be considered an adverse effect. However, we also considered the effects of the intervention on parental anxiety and stress, measured by, for example, the Parenting Stress Index (PSI; Abidin 1995), or the Parenting Stress Scale (PSS; Berry 1995).
primary outcome:
Positive expiratory pressure physiotherapy for airway clearance in people with cystic fibrosis
Forced expiratory volume at one second (FEV1) (change in FEV1 between baseline and post‐intervention; L and per cent (%) predicted values are both stated wherever possible)Number of respiratory exacerbations between baseline and post‐intervention (respiratory exacerbations must have been defined either by symptoms or by initiation of antibiotics for respiratory symptoms after medical assessment)Direct measures of mucus clearance (mucus transport rate or mucociliary clearance rate as assessed by radioactive tracer)
primary outcome:
Fluorides for preventing early tooth decay (demineralised lesions) during fixed brace treatment
The primary outcome was the difference in the proportion of participants in the experimental and comparison groups with new DLs on the day the fixed appliance was removed.This could be assessed directly from the participant (clinically) or preferably from start and finish photographs or fluorescent images of the teeth immediately after the active fixed brace was removed. If the number of DLs was not recorded at the start of treatment, the outcome was the presence or absence of DLs at the end of orthodontic treatment, again assessed directly from the participant or indirectly from photographs or fluorescent images of the teeth. It is important that the assessment was carried out on the day the appliance was removed (or as close to this as possible), because the white marks will tend to heal (remineralise) after the brace is removed. The rate at which the DLs remineralise can be variable between individuals and is another potential confounding factor.
primary outcome:
Human milk‐derived fortifier versus bovine milk‐derived fortifier for prevention of mortality and morbidity in preterm neonates
Necrotizing enterocolitis as defined by stage 2 or stage 3 of modified Bell’s Stage (Walsh 1986), or all‐cause mortality prior to hospital discharge and first year of life.
primary outcome:
Intranasal corticosteroids for non‐allergic rhinitis
Disease severity as measured by patient‐reported symptom score (such as a total nasal symptom score (TNSS) or visual analogue scale (VAS)).Significant adverse event: epistaxis.
primary outcome:
Interventions to reduce Staphylococcus aureus in the management of eczema
(a) Global degree of improvement in symptoms or signs, or both, as rated by the participant or medical practitioner, i.e. percentage with good or excellent improvement. We included both short‐term (less than or equal to a month) improvement and long‐term (more than one month) benefit.
(b) Improvement in Quality of Life questionnaires.
primary outcome:
Stopping enteral feeds for prevention of transfusion‐associated necrotising enterocolitis in preterm infants
Number of infants with necrotising enterocolitis (as defined by modified Bell Stage II or III (Bell 1978), a modified Bell staging system, or investigator‐defined variations of the Bell staging system) within 48 hours after transfusionNumber of infants with necrotising enterocolitis (as defined by modified Bell Stage II or III (Bell 1978), a modified Bell staging system, or investigator‐defined variations of the Bell staging system): any episode(s) after the first blood transfusion. We planned to consider infants with one or more episodes of NEC as an eventMortality to 44 weeks' postmenstrual age
primary outcome:
Medical and surgical interventions for the treatment of urinary stones in children
Stone‐free rate.Serious adverse events or complications of treatment.Secondary procedures for residual fragments.
primary outcome:
Botulinum toxin type A in the treatment of lower limb spasticity in children with cerebral palsy
Gait analysis, including observational gait analysis, assessed by subjective assessments and standardised scales such as the Physician Rating Scale (PRS) and the Edinburgh Visual Gait Score (Koman 1994; Read 2003), as well as instrumented gait analysis, including spatio‐temporal parameters, kinematic and kinetic data.Function, measured (preferably) by validated scales, including the Gross Motor Function Measure (GMFM; Russell 1989), the mobility domain of the Pediatric Evaluation of Disability Inventory (PEDI; Feldman 1990), and the performance domain of the Canadian Occupational Performance Measure (COPM; Law 1990).
primary outcome:
Valproate for acute mania
1. Efficacy (dichotomous): We defined response to treatment as a 50% or greater reduction in mean score on the Young Mania Rating Scale (YMRS) ‐ or any other equivalent standardised rating scale ‐ from baseline.

2. Tolerability (dichotomous): We assessed tolerability using the number of participants experiencing adverse events of any nature. In order to avoid missing any relatively rare or unexpected but important side effects, in the data extraction phase, we collected information on all side‐effects data reported in the studies and discussed ways to summarise them post hoc. We extracted descriptive data for adverse effect profiles from all available studies. In cases where reporting was inconsistent, we combined terms describing similar side effects (Caddy 2015; McCloud 2015). For example, we combined 'dry mouth’, 'reduced salivation’, and 'thirst’ into 'dry mouth’.No data on primary outcomes were available for this comparison.No data on primary outcomes were available for this comparison.No data on primary outcomes were available for this comparison.
primary outcome:
Antioxidant supplementation for lung disease in cystic fibrosis
Lung function testsforced expiratory volume in one second (FEV1) (% predicted or L)forced vital capacity (FVC) (% predicted or L)Quality of life (QoL) (using validated measurement tools only)
primary outcome:
Inhaled steroids with and without regular formoterol for asthma: serious adverse events
All‐cause mortality.All‐cause non‐fatal SAEs.
primary outcome:
Interventions to improve disposal of child faeces for preventing diarrhoea and soil‐transmitted helminth infection
Diarrhoea episodes among individuals, whether or not confirmed by microbiological examination. We defined an episode according to the case definitions used in each reviewed study. A third of the included studies used the WHO definition, which is the passage of three or more loose or liquid stools per day or more than usual for the individual (WHO 2013), while others used other definitions, which are defined in the results section. We treated this outcome as dichotomous, whether an individual had one or more episodes of diarrhoea.Infection with one or more of the following species of STHs: Ascaris lumbricoides (round worm), Trichuris trichiura (whip worm), Ancylostoma duodenale , orNecator americanus (hookworm). We defined infection as the presence of eggs, or juvenile nematodes, or both in the stools of the participants. We included any accepted diagnostic techniques.
primary outcome:
Conservative interventions for treating functional daytime urinary incontinence in children
Number of children no longer experiencing daytime urinary incontinence: child‐ or carer‐reported
primary outcome:
Calcium supplementation commencing before or early in pregnancy, for preventing hypertensive disorders of pregnancy
Pre‐eclampsia (gestational hypertension and proteinuria, as defined below)Pre‐elampsia or pregnancy loss or stillbirth (or a combination) at any gestational ageSevere maternal morbidity and mortality index: one or more of secondary outcomes marked # below
primary outcome:
Public health deworming programmes for soil‐transmitted helminths in children living in endemic areas
Growth, as measured by the following indicators:weightheightHaemoglobinFormal tests of cognition
primary outcome:
Oral non‐steroidal anti‐inflammatory drug therapy for lung disease in cystic fibrosis
Objective measures of lung function (measured as absolute change in % predicted from baseline compared to control and post‐treatment change in % predicted compared to control):forced expiratory volume in one second (FEV1)forced vital capacity (FVC)forced expiratory flow 25%‐75% (FEF25%‐75%)
primary outcome:
Respiratory muscle training in children and adults with neuromuscular disease
Measures of lung capacity (e.g. total lung capacity (TLC), forced vital capacity (FVC)) over the short term (less than 3 months), medium term (greater than 3 months but less than 1 year), and long term (greater than 1 year).
primary outcome:
Homeopathy for treatment of irritable bowel syndrome
The primary outcome was global improvement of symptoms (patient‐reported or clinician‐evaluated or both) as measured by a global IBS symptom score (e.g. IBS Severity Scoring System (IBS‐SSS), Adequate Relief Measure, GI Symptom Rating Scale, Functional Bowel Disorder Severity Index or IBS Symptom Questionnaire).
primary outcome:
Altering the availability or proximity of food, alcohol, and tobacco products to change their selection and consumption
Measures of unconstrained selection (with or without purchasing) or consumption of the manipulated food, alcohol, or tobacco product(s). We anticipated encountering a range of measures of these outcome constructs amongst included studies, and present the following examples of likely measures below.1. Selection of a product (a) without purchase, or b) with purchase.Assessment of the amounts of products (e.g. food, drink, alcohol, or tobacco products), energy or substances (e.g. saturated fat, alcohol, carbon monoxide) selected, measured in applicable natural units (e.g. kilojoules, grams). Depending on the study setting, a product may be selected with or without this involving a purchase, that is a transfer of money to the vendor. In cases where there is no purchasing, selection may be comparable to typical purchasing (e.g. products being selected in a restaurant or bar where there is no charge for them) or it may be behaviour that necessarily precedes consumption in that context, such as serving an amount of a food product onto a plate or pouring an amount of drink into a glass.2. Consumption (intake) of a product.As per selection, assessment of the amounts of products (e.g. food, drink, alcohol, or tobacco products), energy, or substances (e.g. saturated fat, alcohol, carbon monoxide) consumed, measured in applicable natural units (e.g. kilojoules, grams).
primary outcome:
Once‐daily versus multiple‐daily dosing with intravenous aminoglycosides for cystic fibrosis
Lung function measurementsforced expiratory volume in one second (FEV1)forced vital capacity (FVC)forced expiratory flow in mid expiration (FEF25‐75%)
primary outcome:
Fetal biometry for guiding the medical management of women with gestational diabetes mellitus for improving maternal and perinatal health
large‐for‐gestational age (as defined by authors)caesarean deliveryperinatal mortalityComposite outcome neonatal morbidity or mortality (defined as one or more of the following: fetal or neonatal death, shoulder dystocia, bone fracture, and nerve palsy)hypertensive disorders of pregnancy (including pre‐eclampsia, pregnancy‐induced hypertension, eclampsia)development of type 2 diabetes in the mother
primary outcome:
Chlorhexidine bathing of the critically ill for the prevention of hospital‐acquired infection
Hospital‐acquired infections, including bloodstream infections; central‐line associated bloodstream infections; ventilator‐associated pneumonia; catheter‐associated urinary tract infections; multidrug‐resistant organisms (MDROs), e.g. Methicillin‐resistant Staphylococcus aureus (MRSA), vancomycin‐resistant Enterococci (VRE).
primary outcome:
Contact tracing strategies in household and congregate environments to identify cases of tuberculosis in low‐ and moderate‐incidence populations
Contacts with latent tuberculosis infection out of all contacts screened.
primary outcome:
Sulthiame add‐on therapy for epilepsy
50% or greater reduction in seizure frequency. We selected this outcome as it is commonly reported in studies assessing the efficacy of AEDs.
primary outcome:
TNF‐α blockers for the treatment of Kawasaki disease in children
Treatment resistance: persistent or recurrent fever (38.0 °C or higher) between 36 hours and two weeks after completion of TNF‐α blocker, without other causes.Incidence of coronary artery abnormalities (CAAs): measured by echocardiography or coronary angiography, or both, within three months of KD diagnosis (Ayusawa 2005; Newburger 2004).Incidence of any adverse effects after treatment initiation. Adverse effects of TNF‐α blockers have been identified through both clinical trials and postmarketing surveillance. These include: injection site reactions, infusion reactions, neutropenia, infections, demyelinating disease, heart failure, cutaneous reactions, malignancy, and induction of autoimmunity.
primary outcome:
Prebiotics for the prevention of hyperbilirubinaemia in neonates
Neonatal hyperbilirubinaemia: incidence of hyperbilirubinaemia at any time during the first ten days of life, considered as the primary outcome. Hyperbilirubinaemia is defined as follows:for term and late preterm neonates (gestational age ≥ 35 weeks) as total bilirubin (TB) level eligible for phototherapy, as described in the guidelines of the American Academy of Pediatrics 2004 (Figure 1), or as absolute TB level ≥ 15 mg/dL; andfor preterm neonates (< 35 weeks’ gestation) as TB level that is eligible for phototherapy, as described in the guidelines of the American Academy of Pediatrics 2004 (Figure 2), or as absolute TB level > 1% of body weight.Acute bilirubin encephalopathy: defined as a clinical syndrome, in the presence of severe hyperbilirubinaemia, that ranges from the initial phase of lethargy, hypotonia, reduced movement, and poor sucking and suckling to the advanced phase of deep stupor, hypertonia, inability to feed, and shrill cry or seizures (Canadian Pediatric Society 2007).
primary outcome:
eHealth interventions for people with chronic kidney disease
Time intervals at which outcome assessment takes place may affect the effect of the intervention programs. We considered all time frames used by authors.1. Clinical parameters2. Patient‐centred parameters3. Cost effectiveness4. Potential harms
primary outcome:
Azathioprine and 6‐mercaptopurine for maintenance of surgically‐induced remission in Crohn's disease
The primary outcome measure was clinical relapse as defined by the primary studies.
primary outcome:
Protein hydrolysate versus standard formula for preterm infants
Infants with at least one episode of feed intolerance that resulted in cessation of, or reduction in, enteral feeding (enteral feeds were reduced or ceased for longer than four hours), or mean number of episodes of feed intolerance during the trial period, or bothInfants with at least one episode of necrotising enterocolitis (modified Bell stage 2/3) (Walsh 1986) (unless indicated for use following necrotising enterocolitis)
primary outcome:
Interventions for preventing obesity in children
zBMI score/BMIPrevalence of overweight and obesityWeight and heightPonderal indexPer cent fat contentSkin‐fold thickness
primary outcome:
Buffered solutions versus 0.9% saline for resuscitation in critically ill adults and children
Overall (in‐hospital) mortalityAcute kidney injury (AKI) during hospitalization as defined by risk of renal dysfunction, injury to the kidney, failure of kidney function, loss of kidney function, and end‐stage kidney disease (RIFLE), or by Acute Kidney Injury Network (AKIN) criteria
primary outcome:
Community first responders for out‐of‐hospital cardiac arrest in adults and children
Survival at hospital dischargeNeurological function at hospital discharge, measured by cerebral performance category (CPC)
primary outcome:
Carbamazepine versus phenytoin monotherapy for epilepsy: an individual participant data review
Time to treatment failure for any treatment‐related reason (continued seizures, side effects, non‐compliance or the initiation of additional add‐on treatment)Time to treatment failure due to adverse events (i.e. side effects)Time to treatment failure due to lack of efficacy (i.e. continued seizures)
primary outcome:
Genetic testing for prevention of severe drug‐induced skin rash
The incidence of severe drug‐induced skin rash (defined as skin rash with systemic symptoms including fever and multiple organ involvement).Long‐term sequelae (any of the following: ophthalmologic, cutaneous, or liver damage, etc.) up to 12 months after the severe drug‐induced skin rash.
primary outcome:
Interventions for morphea
Global improvement of disease activity or damage, assessed by a medical practitioner or by participants.Adverse effects of the interventions, including local and systemic reactions.
primary outcome:
Singing as an adjunct therapy for children and adults with cystic fibrosis
QoL measured by validated instruments, e.g. Cystic Fibrosis Questionnaire‐Revised (CFQ‐R) (Quittner 2009), St. George's Respiratory Questionnaire (Jones 1991), Pediatric Quality of Life InventoryTM (PedsQLTM) (Varni 1999)Respiratory muscle functionmaximal inspiratory pressure (MIP)maximal expiratory pressure (MEP)cough peak flow (Table 1)
primary outcome:
Inositol in preterm infants at risk for or having respiratory distress syndrome
DeathNeonatal death (death < 28 days postnatal age)Death during hospital stay (added as an outcome in 2015)Infant death (death during the first year of life)Type 1 ROP and death before determination of ROP outcome (added as an outcome in 2019)All cause mortality (outcomes collected up to 55 weeks' PMA (added as an outcome in 2019)All‐cause mortality (outcomes collected through the first event; death, hospital discharge, hospital transfer or 120 days after birth (added as an outcome in 2019))
primary outcome:
Interventions for treating neuropathic pain in people with sickle cell disease
Self‐reported pain relief of:30% or greater (moderate);50% or greater (substantial).Patient Global Impression of Change (PGIC):much or very much improved (moderate);very much improved (substantial).
primary outcome:
Inhaled corticosteroids for cystic fibrosis
Objective measures of lung functionabsolute values and change (L and % predicted) of forced expiratory volume in one second (FEV1)absolute values and change (L and % predicted) of the forced vital capacity (FVC)
primary outcome:
Pharmacological interventions for prevention and treatment of upper gastrointestinal bleeding in newborn infants
All‐cause mortality to near‐term age or discharge.Any upper gastrointestinal bleeding in infants at risk of upper gastrointestinal bleeding.Duration of upper gastrointestinal bleeding in infants with upper gastrointestinal bleeding (days).
primary outcome:
Computer and mobile technology interventions to promote medication adherence and disease management in people with thalassemia
Medication adherencedirectly observed adherence rates with individuals recording their daily iron chelator use on their cell phonesradiological markers of iron overload (MRI of liver R2* and MRI of heart T2*)laboratory markers of iron overload (serum ferritin levels)iron chelation adherence rates by pharmacy records (i.e. medication possession ratio)self‐reported iron chelator adherence rate
primary outcome:
Banked preterm versus banked term human milk to promote growth and development in very low birth weight infants
Short‐term growth parameters: time to regain birth weight (days of life), weight gain (grams/day), length gain (centimetres/week), head growth (centimetres/week) at discharge;Longer‐term growth parameters (following discharge from hospital): weight gain (grams/week), length gain (centimetres/week), head growth (centimetres/week) at four months' follow‐up;Neurodevelopmental outcomes at term corrected age and at 18 to 24 months using validated assessment tools.
primary outcome:
Radix Sophorae flavescentis versus other drugs or herbs for chronic hepatitis B
All‐cause mortality.Proportion of participants with one or more serious adverse events; that is, any untoward medical occurrence that resulted in death, was life threatening, required hospitalisation or prolongation of existing hospitalisation, resulted in persistent or significant disability or incapacity, or was a congenital anomaly or birth defect (ICH‐E2A 1994; ICH‐GCP E6(R2) 2016).Health‐related quality of life: any scale used by trialists to assess the participants' reporting of their quality of life.
primary outcome:
Social skills training for attention deficit hyperactivity disorder (ADHD) in children aged 5 to 18 years
Social skills in school or at home, measured at post‐treatment and longest follow‐up, by well‐established and validated instruments such as the Social Skills Rating System (SSRS; Gresham 1990) or Conners' Behaviour Rating Scales (CBRS;Conners 2008a).Emotional competencies in school or at home, measured at post‐treatment and longest follow‐up, by well‐established and validated instruments such as the Emotion Regulation Checklist (ERC; Hannesdottir 2017).General behaviour in school or at home, measured at post‐treatment and longest follow‐up, by well‐established and validated instruments such as the Achenbach Child Behavior Checklist (Achenbach 1991).
primary outcome:
Oral 5‐aminosalicylic acid for maintenance of surgically‐induced remission in Crohn's disease
The primary outcome measure was clinical relapse as defined by the primary studies. We accepted the authors’ definitions of what constitutes a clinical relapse.
primary outcome:
Probiotics for preventing acute otitis media in children
Proportion of children with AOM (in each group) (i.e. the number of children experiencing one or more episodes of AOM during the treatment).Severity of AOM.Adverse events (e.g. gastrointestinal side effects).
primary outcome:
Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I
Glycosaminoglycan (GAG) levels in urineRespiratory function as assessed by pulmonary function tests (e.g. forced expiratory volume at one second (FEV1), force vital capacity (FVC))Endurance performance as assessed by endurance test in the form of the six‐minute walk test (6MWT)
primary outcome:
Inhaled corticosteroids in children with persistent asthma: effects of different drugs and delivery devices on growth
Linear growth velocity (cm/year), obtained by measuring height with a stadiometer at a number of time points during the study, and performing linear regression of height over time (Price 2002).
primary outcome:
Lipid emulsions for parenterally fed preterm infants
Physical growth:days to regain birth weight;growth rate (g/kg/day) during study period and hospital stay (Fenton 2017).Parenteral nutrition‐associated liver disease (PNALD), defined as conjugated bilirubin 2 mg/dL or greater (or 34.2 µmol/L or greater) with or without raised liver enzymes (alanine aminotransferase (ALT) greater than 45 IU/L, alkaline phosphatase greater than 420 IU/L) in the absence of other causes (Christensen 2007; Hojsak 2016; Robinson 2008), in preterm infants without PNALD at study entry.PNALD/cholestasis (any definition)*Resolution of PNALD, defined as conjugated bilirubin less than 2 mg/dL (34.2 µmol/L), in preterm infants with established PNALD (Lam 2014).
primary outcome:
Lipid emulsions for parenterally fed term and late preterm infants
Parenteral nutrition‐associated liver disease (PNALD) (conjugated bilirubin ≥ 2 mg/dL (34.2 µmol/L)) with or without raised liver enzymes (alanine aminotransferase (ALT) greater than 45 IU/L, alkaline phosphatase (ALP) greater than 420 IU/L) in the absence of other causes (Christensen 2007; Robinson 2008), in late preterm and term infants without PNALD at study entry.PNALD/cholestasis (any definition).*Resolution of PNALD (conjugated bilirubin less than 2 mg/dL (34.2 µmol/L)), in late preterm and term infants with established PNALD (Lam 2014).Physical growth:days to regain birth weight;growth rate (g/kg/day) during study period and hospital stay (Fenton 2017).
primary outcome:
Lithium for acute mania
Efficacy outcomes: response (categorical). Number of participants who responded to treatment, where treatment response was defined as a decrease in score on the Young Mania Rating Scale (YMRS), or any other equivalent standardised rating scale, of at least 50% from baseline to the end of the study.Efficacy outcomes: response (continuous). Mean endpoint scores or mean change scores in manic symptoms (YMRS or other equivalent standardised rating scale) from baseline to the end of the study.Efficacy outcomes: remission (categorical). Number of participants who achieved remission by the end of the study out of the total number of randomised participants. We defined remission as a YMRS score of 12 or less (or equivalent on other validated mania rating scales).Acceptability (categorical): overall number of participants who dropped out during the study as a proportion of the total number of randomised participants.Acceptability: adverse events (categorical). We evaluated adverse events using the following outcome measures.Total number of participants who experienced at least one side effect between first treatment dose and end of study.Total number of participants who experienced the following specific side effects between the first treatment dose and the end of the study (BNF 2016).DepressionManiaWeight gainAkathisiaHeadacheSomnolenceDizzinessInsomniaDiarrhoeaNauseaVomitingDry mouthPainExtra‐pyramidal side effectsTremorConstipationFeverRashAttempted suicideAnorexiaInfectionWeight lossAgitationConvulsions or seizuresDyspepsiaPsychosisSuicidal ideationBlood disordersHyperprolactinaemiaThyroid disordersArthralgiaRhinitisPruritisRenal impairmentSexual dysfunction
primary outcome:
Interventions for promoting participation in shared decision‐making for children and adolescents with cystic fibrosis
Presence of shared decision‐making measured by the change in score of any validated toolthe Observing Patient Involvement 12‐item (OPTION) Scale (Elwyn 2003)the Observer‐based Measure Observer 5‐item (OPTION) Scale (Elwyn 2013a)decision‐making instrument facilitation antecedents (e.g. the Preparation for Decision‐making Scale (Graham 1995))decision process (e.g. the Rochester Participatory Decision‐making Scale (Shields 2005))Quality of life (QoL) as measured by e.g. the Cystic Fibrosis Questionnaire‐Revised (CFQ‐R) (Bryon 2005; Quittner 2005; Quittner 2009; Quittner 2012) or the CF disease‐specific (HRQoL) questionnaire (Gee 2000)Adverse effect such as longer consultation time, increased frequency of hospital admissions, longer hospital stay, increased costs or unanticipated adverse effects as reported by study authors
primary outcome:
Ready‐to‐use therapeutic food (RUTF) for home‐based nutritional rehabilitation of severe acute malnutrition in children from six months to five years of age
Recovery, during and beyond the intervention period, as defined by study authorsDeterioration or relapse, during and beyond the intervention period, as defined by study authorsMortality, during and beyond the intervention period
primary outcome:
Enteral lactoferrin for the treatment of sepsis and necrotizing enterocolitis in neonates
All‐cause mortality during hospital stay and at 28 days.
primary outcome:
Preventive lipid‐based nutrient supplements given with complementary foods to infants and young children 6 to 23 months of age for health, nutrition, and developmental outcomes
Stunting (moderate: height/length‐for‐age (HFA) < −2 standard deviations (SD); severe: HFA < −3 SD)Wasting (moderate: weight‐for‐height/length (WFH) < −2 SD; severe: WFH < −3 SD)Underweight (moderate: weight‐for‐age (WFA) < −2 SD; severe: WFA < −3 SD)Anaemia (as defined by trialists)Psychomotor development outcomes (as defined by trialists)Neuro‐developmental outcomes (as defined by trialists)Any adverse effects, including allergic reactions, as diagnosed by clinical assessment (atopic dermatitis, urticaria, oedema (oral), ophthalmic pruritus, allergic rhinitis, asthma, anaphylaxis)
primary outcome:
Family therapy approaches for anorexia nervosa
Remission (by DSM or ICD or trialist‐defined cut‐off on standardised scale measure for remission versus no remission)All‐cause mortality