anurajms/meta_analysis huggingface dataset:
primary outcome:
Duodenum‐preserving pancreatic resection versus pancreaticoduodenectomy for chronic pancreatitis
All‐cause mortality.Short‐term mortality (in‐hospital mortality or mortality within three months).Long‐term mortality (maximal follow‐up).Treatment‐related serious adverse events (within three months). We accepted the following definitions of serious adverse events.Clavien‐Dindo classification (Clavien 2009; Dindo 2004): Grade III or greater.International Conference on Harmonisation ‐ Good Clinical Practice (ICH‐GCP) guideline (ICH‐GCP 1996): serious adverse events defined as any untoward medical occurrence that resulted in death, was life‐threatening, required hospitalisation or prolongation of existing hospitalisation, or resulted in persistent or significant disability/incapacity.Individual complications such as anastomotic leak requiring re‐operation that can clearly be classified as Grade III or greater with the Clavien‐Dindo classification (Clavien 2009; Dindo 2004), or as a serious adverse event with the ICH‐GCP classification.Clinically significant pancreatic fistulas (Type B or Type C International Study Group on Pancreatic Fistula Definition (ISGPF)) (Bassi 2005).Health‐related quality of life following surgery (using any validated scale such as EQ5D or 36‐item Short Form (SF‐36)) (EuroQol 2014; Ware 2014). EQ5D assesses the quality of life under five domains, namely mobility, self care, usual activities, pain or discomfort, and anxiety or depression (EuroQol 2014). SF‐36 assesses the quality of life under eight sections, namely vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health (Ware 2014).Short‐term (four weeks to three months).Medium‐term (three months to five years).Long‐term (more than five years).
primary outcome:
Pregabalin for decreasing pancreatic pain in chronic pancreatitis
Health‐related quality of life post‐treatment (using any validated scale, such as EQ‐5D or SF‐36; EuroQol 2014; Ware 2014). The EQ‐5D assesses the quality of life under five domains: mobility, self care, usual activities, pain or discomfort, and anxiety or depression (EuroQol 2014). The SF‐36 assesses the quality of life under eight sections: vitality, physical functioning, bodily pain, general health perceptions, physical role functioning, emotional role functioning, social role functioning, and mental health (Ware 2014).Short‐term (two weeks to three months).Medium‐term (three months to five years).Long‐term (more than five years).Treatment‐related serious adverse events such as heart or renal failure (within three months). We accepted the following definitions of serious adverse events.ICH‐GCP International Conference on Harmonisation ‐ Good Clinical Practice guideline (ICH‐GCP 1996): serious adverse events defined as any untoward medical occurrence that results in death, is life‐threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation, or results in persistent or significant disability or incapacity.Other variations of ICH‐GCP classifications such as the US Food and Drug Administration (FDA) classification (FDA 2006), or the Medicines and Healthcare products Regulatory Agency (MHRA) classification (MHRA 2013).Opiate requirement (in terms of the total amount required during the follow‐up period, and only from trials where opiates were not used routinely).Short‐term (two weeks to three months).Medium‐term (three months to five years).Long‐term (more than five years).
primary outcome:
Enhanced recovery protocols for major upper gastrointestinal, liver and pancreatic surgery
Mortality.Short‐term mortality (in‐hospital mortality or mortality within three months).Long‐term mortality (for patients undergoing surgery for cancer).Serious adverse events (within three months). We accepted the following definitions of serious adverse events.Clavien‐Dindo classification (Clavien 2009; Dindo 2004): grade III or higher.International Conference on Harmonisation–Good Clinical Practice (ICH‐GCP) guideline (ICH‐GCP 1996): serious adverse events defined as any untoward medical occurrences that result in death, are life threatening, require inpatient hospitalisation or prolongation of existing hospitalisation and result in persistent or significant disability/incapacity.Individual complications that could clearly be classified as grade III or higher by the Clavien‐Dindo classification (Clavien 2009; Dindo 2004), or as a serious adverse event by the ICH‐GCP classification.Health‐related quality of life (using any validated scale).Short‐term (until three months).Medium‐term (three months to one year).
primary outcome:
Antibiotic prophylaxis for surgical site infection in people undergoing liver transplantation
All‐cause mortality.Surgical site infection, as defined by the Centers for Disease Control and Prevention (CDC) (Appendix 1).Adverse events: serious adverse events. A serious adverse event, defined according to the International Conference on Harmonisation (ICH) Guidelines for Good Clinical Practice (ICH‐GCP 1997), is any untoward medical occurrence that at any dose results in death, is life‐threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation or results in persistent or significant disability or incapacity, or in a congenital anomaly or birth defect. We will consider all other adverse events as non‐serious.Quality of life.
primary outcome:
Etrolizumab for induction of remission in ulcerative colitis
The primary outcome measure was the proportion of patients achieving clinical remission as defined by the primary studies and expressed as a percentage of the number of patients randomized (intention‐to‐treat analysis).
primary outcome:
Surgery for complete (full‐thickness) rectal prolapse in adults
Number of people with recurrent full‐thickness rectal prolapseNumber of people with residual mucosal prolapseNumber of people with faecal incontinence (or incontinence score)Number of people with constipation (or delayed gut transit time)
primary outcome:
Treatment for dysphagia (swallowing difficulties) in hereditary ataxia
The presence or absence of aspiration or penetration of fluid or food matter during swallowing, as determined by functional imaging techniques (for example, videofluoroscopy swallowing studies (VFSS)), and assessed using validated protocols such as the Penetration Aspiration Scale (PAS) (Rosenbek 1996).
primary outcome:
Laparoscopic fundoplication surgery versus medical management for gastro‐oesophageal reflux disease (GORD) in adults
Health‐related quality of life (HRQoL) (using any validated scale).Short‐term (four weeks to 12 months).Medium‐term (one year to five years).Long‐term (five years or more).GORD‐specific quality of life (QoL)Short‐term (four weeks to 12 months).Medium‐term (one year to five years).Long‐term (five years or more).Serious adverse events (within three months of cessation of treatment ‐ for surgery this period refers to three months after index surgery). We will accept the following definitions of serious adverse events.ICH‐GCP International Conference on Harmonisation ‐ Good Clinical Practice guideline (ICH‐GCP 1996): serious adverse events defined as any untoward medical occurrence that results in death, is life‐threatening, requires inpatient hospitalisation or prolongation of existing hospitalisation, results in persistent or significant disability/incapacity.Other variations of ICH‐GCP classifications such as Food and Drug Administration (FDA) classification (FDA 2006), Medicines and Healthcare products Regulatory Agency (MHRA) classification (MHRA 2013).
primary outcome:
Azathioprine or 6‐mercaptopurine for maintenance of remission in Crohn's disease
The primary outcome was maintenance of remission (as defined by the included studies, e.g. CDAI < 150) at study endpoint.
primary outcome:
Oral budesonide for induction of remission in ulcerative colitis
The primary outcome was induction of remission of active ulcerative colitis. Clinical remission was defined by the primary studies and was expressed as the percentage of patients randomised (intention‐to‐treat analysis).
primary outcome:
Noninvasive positive pressure ventilation for acute respiratory failure following upper abdominal surgery
Hospital mortality (measured starting at the date of randomization up to the time of discharge from hospital).Rate of tracheal intubation, (measured in number of events occurred since the date of randomization up to the time of discharge from hospital).
primary outcome:
Preoperative inspiratory muscle training for postoperative pulmonary complications in adults undergoing cardiac and major abdominal surgery
PPCs. We defined clinically significant PPCs as two or more items in the grade 2 complications or one item in the grade 3 or 4 complications (Appendix 1).All‐cause mortality within 30 days during postoperative period.Evidence of adverse events from IMT.
primary outcome:
Interventions for heartburn in pregnancy
Relief of heartburn (complete or partial relief measured by frequency of heartburn)
primary outcome:
Type I interferons for induction of remission in ulcerative colitis
Primary outcome
The primary outcome was induction of remission of UC. Remission was defined by the primary studies (see disease activity indices described under 'Types of participants') and was expressed as the percentage of patients randomised (intention‐to‐treat analysis).Secondary outcomes
The secondary outcomes included:
1. Time to remission;
2. The mean change in the disease activity index score;
3. Clinical, histological or endoscopic improvement as defined by the authors;
4. Improvement in quality of life as defined by a validated quality of life tool; and
5. Adverse events associated with IFN therapy for the treatment of UC.
Four different outcome measures were used to evaluate the safety of type I IFNs:
primary outcome:
Methotrexate for maintenance of remission in ulcerative colitis
The primary outcome measure was the occurrence of clinical or endoscopic relapse as defined by the primary studies.
primary outcome:
Unfractionated or low‐molecular weight heparin for induction of remission in ulcerative colitis
The primary outcome measure was the number of patients achieving clinical remission and off steroids as defined by the studies and expressed as a percentage of the number of patients randomized (intention‐to‐treat analysis). Secondary outcomes measures included:
a) Endoscopic remission as defined by the authors;
b) Clinical, histological or endoscopic improvement as defined by the authors;
c) The occurrence of adverse events;
d) The occurrence of bleeding; and
e) Improvements in quality of life as measured by a validated instrument.
primary outcome:
Post‐pyloric versus gastric tube feeding for preventing pneumonia and improving nutritional outcomes in critically ill adults
Pneumonia.Mortality.Percentage of total nutrition delivered to the participant, which is calculated by comparing the mean estimated caloric need versus the mean actual calories delivered to the participant.Time required to achieve the full nutritional target: time from participant enrolment in the study to time the nutritional goal rate was reached and continued successfully for four hours.
primary outcome:
Plugs for containing faecal incontinence
1. Patient symptoms:2. Physical measures:3. Patient satisfaction:4. Health status measures:5. Costs6. Other outcomes:
primary outcome:
Antifibrinolytic amino acids for upper gastrointestinal bleeding in people with acute or chronic liver disease
Mortality.Failure to control bleeding.Serious adverse events. We used the International Conference on Harmonisation (ICH) Guidelines for Good Clinical Practice's definition of a serious adverse event (ICH‐GCP 1997), 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. We considered all other adverse events as non‐serious.
primary outcome:
Budesonide for induction of remission in Crohn's disease
Primary outcome
The primary outcome was induction of remission of CD. This was defined by a CDAI < 150 or PDAI <10 by 8 to 16 weeks of therapy and was expressed as the percentage of patients randomised (intention‐to‐treat analysis).Secondary outcomes
The secondary outcomes that were documented included:
1. Time to remission;
2. The mean change in the CDAI;
3. Clinical, histologic, or endoscopic improvement as defined by the authors;
4. Improvement in quality of life as defined by a validated quality of life tool; and
5. Adverse events associated with budesonide therapy for the treatment of CD.
The different outcome measures used to evaluate the safety of budesonide included:
a. The percentage of patients experiencing adverse events (including changes in adrenal function);
b. The percentage of patients withdrawing from the study due to adverse events;
c. The percentage of patients undergoing surgery; and
d. Mortality expressed as a percentage.
primary outcome:
Water infusion versus air insufflation for colonoscopy
Cecal intubation rateAdenoma detectionNumber of participants with at least one adenoma detected (adenoma detection rate)Number of adenomas detected per participant
primary outcome:
Endoscopic injection of cyanoacrylate glue versus other endoscopic procedures for acute bleeding gastric varices in people with portal hypertension
All‐cause mortality at maximum follow‐up (see Differences between protocol and review).Bleeding‐related mortality: number of people who died from uncontrolled variceal bleeding at medium term (approximately one month) (see Differences between protocol and review).Failure of intervention: number of people in which the intervention was unable to control active or acute bleeding within 24 hours, triggering a need to change treatment or repeat endoscopy (active: endoscopy evidence of current bleeding; acute: endoscopy evidence of recent bleeding stigmata without current bleeding) (see Differences between protocol and review).Re‐bleeding: number of people in which the intervention was unable to prevent re‐bleeding at short term (approximately one week) (see Differences between protocol and review).Adverse events:number of people with pulmonary embolism caused by cyanoacrylate (measured by radiological and clinical criteria) or with cyanoacrylate embolism in other organs such as brain and spleen;number of people who developed septicaemia after intervention;number of people with other serious adverse effects according to the International Conference on Harmonization Guidelines (ICH‐GCP 1997) (see Differences between protocol and review).
primary outcome:
Enteral nutrition formulations for acute pancreatitis
All‐cause mortality.Systemic inflammatory response syndrome (SIRS), defined by two or more of the following criteria: pulse rate > 90 beats per minute; respiratory rate > 20 per minute or arterial partial pressure of carbon dioxide (PaCO2) < 32mmHg; body temperature > 38ºC or < 36ºC; white cell count > 12,000 or < 4000 cells per mm3 (Buter 2002).Multiple organ dysfunction syndrome, as defined by the Modified Marshall Scoring System, by which organ failure is defined as a score ≥ 2 for at least one of the three organ systems (Banks 2012).Adverse events.
primary outcome:
Endoscopic or surgical intervention for painful obstructive chronic pancreatitis
Pain relief: proportion of participants achieving pain relief compared with the situation prior to intervention. Since pain is a subjective outcome and many different scores are used, we did not use a strict definition of pain relief. We classified the observed pain relief as either complete or partial, defining partial pain relief as a decrease in pain of at least 50% compared with baseline, without achieving complete pain relief. We conducted analyses for complete and partial pain relief separately and for total proportion of participants experiencing pain relief (both partial and complete).Major postinterventional complications, including intra‐abdominal abscess, ileus‐necessitating surgery, pancreatitis flare‐up, bleeding, anastomotic leakage, sepsis, abdominal fascial dehiscence (Platzbauch), and myocardial infarction.Mortality.
primary outcome:
Treatment for cryoglobulinemic and non‐cryoglobulinemic peripheral neuropathy associated with hepatitis C virus infection
Neuropathy associated with HCV most often has primarily sensory features (Yoon 2011). Therefore, our primary outcome was change in sensory impairment at the end of the follow‐up period (at least six months after treatment initiation). Sensory impairment could have been evaluated using any validated sensory neuropathy scale (including the Sensory Sum Score (Merkies 2000) and the Utah Early Neuropathy Scale (Singleton 2008)) or quantitative sensory testing.
primary outcome:
Tranexamic acid for upper gastrointestinal bleeding
Mortality.Adverse events. We defined serious adverse events as all adverse events considered serious by study participants or investigators.
primary outcome:
Hepatitis B vaccination during pregnancy for preventing infant infection
Incidence of hepatitis B virus infection in infants.
primary outcome:
Tolerability of selective cyclooxygenase 2 inhibitors used for the treatment of rheumatological manifestations of inflammatory bowel disease
The primary outcome was the proportion of patients with disease exacerbation as defined by the included studies.
primary outcome:
Non‐nutritive sucking for gastro‐oesophageal reflux disease in preterm and low birth weight infants
GOR as diagnosed by pH metry, multiple intraluminal impedance, or endoscopy within two hours postprandial (after eating a meal).
primary outcome:
Epinephrine injection versus epinephrine injection and a second endoscopic method in high‐risk bleeding ulcers
Rebleeding rates (persistent bleeding and recurrent bleeding) as confirmed by endoscopy and further clinically significant bleeding as defined according to the criteria established in each study.
primary outcome:
Interventions for treating postpartum constipation
Pain or straining on defecation.Participant‐reported relief of constipation symptoms.Stool frequency.
primary outcome:
Budesonide for maintenance of remission in Crohn's disease
The primary outcome measure was the number of patients maintaining remission following initiation of maintenance therapy. Where remission rates were not reported or available from study authors, remission rates were defined as the opposite proportion (1 ‐ relapse rate, or 100% ‐ relapse rate percentage).Secondary outcomes included:
primary outcome:
Vedolizumab for induction and maintenance of remission in ulcerative colitis
The primary outcome measures were the proportion of patients who failed to achieve clinical remission and the proportion of patients who relapsed as defined by the included studies. Secondary outcomes included the proportion of patients who failed to have a clinical response, failure to enter endoscopic remission, failure to have an endoscopic response, endoscopic relapse, disease‐specific quality of life, adverse events, withdrawal due to adverse events and serious adverse events.
primary outcome:
Methotrexate for induction of remission in refractory Crohn's disease
The primary outcome was the proportion of patients who failed to enter clinical remission and withdraw from steroids.
primary outcome:
Antimicrobial drugs for treating cholera
Duration of diarrhoea: from the time of initiation of the study drug until the end of diarrhoea as defined in the study.Stool volume: from the time of initiation of the study drug until end of diarrhoea as defined in the study.
primary outcome:
Antimicrobial prophylaxis for colorectal surgery
The abdominal wound has the greatest risk of infection; most commonly from contamination by endogenous colorectal bacteria (contained within the patient's bowel) during surgical procedures (Pollock 1987). In this review, therefore, the rate of surgical wound infection was used as the primary and sole outcome measure to assess the relative effectiveness of antimicrobial prophylaxis in colorectal surgery. The definition and diagnosis of surgical wound infections often varies between published studies, but has the common feature of pus expressed from the surgical incision. We included only abdominal wound infections, because they are more reliably identified than, for example, perineal infections.
primary outcome:
Botulinum toxin for upper oesophageal sphincter dysfunction in neurological swallowing disorders
Positive change to oral intake status (Yes or No)Reduction or elimination of aspiration or laryngeal penetration of food or fluids, or both, as rated on objective assessment (videofluoroscopy, fibreoptic examination of swallowing safety (FEES) (Yes or No)Adverse events including increase in swallowing problems, compromised medical health, negative psychological consequences, negative social consequences, hospitalisation, death (Yes or No)Client or carer satisfaction, or both, with intervention (Yes or No)
primary outcome:
Dopamine agents for hepatic encephalopathy
Mortality (all‐cause).All cause non‐fatal serious adverse events.Morbidity. This outcome measure was assessed on the basis of the number of participants who showed no improvement in manifestations of hepatic encephalopathy as defined by the authors of included trials.
primary outcome:
Early versus delayed laparoscopic cholecystectomy for acute gallstone pancreatitis
All‐cause mortality.Other serious adverse events, defined as any event that would increase mortality, is life‐threatening, requires inpatient hospitalisation, results in a persistent or significant disability, or any important medical event which might have jeopardised the participant or required intervention to prevent it (ICH‐GCP 1996).Overall quality of life (using any validated measurement scale such as EQ5D or SF‐36). If quality of life was reported at multiple time points in the same trial, we planned to use the latest time point within six months after onset of acute pancreatitis.
primary outcome:
Abdominal lift for laparoscopic cholecystectomy
Mortality.Serious adverse events: adverse events were defined as any untoward medical occurrence not necessarily having a causal relationship with the treatment, but resulting in a dose reduction or discontinuation of treatment (ICH‐GCP 1997); serious adverse events were defined as any event that would increase mortality, was life‐threatening, required inpatient hospitalisation, resulted in a persistent or significant disability, or any important medical event which might have jeopardised the patient or required intervention to prevent it.Quality of life.
primary outcome:
Opioids for acute pancreatitis pain
Number of participants showing improvements in pain intensity as defined by the trialist.Number of participants requiring supplementary analgesia (offered when trial drug intervention fails to relieve pain and following trial protocol).
primary outcome:
Laparoscopic repair for perforated peptic ulcer disease
Septic and other abdominal and extra‐abdominal complications, defined according to the Centers for Disease Control (CDC) classification (Garner 1996) and recorded as 'number of complications' and 'at least one abdominal complication'.We considered the following abdominal complications: intra‐abdominal abscess; anastomosis leakage; secondary peritonitis; surgical‐site infection; prolonged ileus for more than 72 hours without recovery of bowel movement, clinically‐determined; and incisional hernia.
primary outcome:
Open Mesh versus non‐Mesh for groin hernia repair
1. Duration of operation (minutes)
2. 'Opposite' method initiated
3. Conversion to 'opposite' method after procedure started
4. Haematoma
5. Seroma
6. Wound/superficial infection
7. Serious complications (mesh/deep infection, vascular injury, visceral injury)
8. Length of postoperative hospital stay (days)
9. Time to return to usual activities
10. Pain persisting around one year after surgery (and at least for more than three months)
11. Numbness persisting around one year after surgery (and at least for more than three months)
12. Hernia recurrence
13. Known death
primary outcome:
Antioxidant supplementation for sickle cell disease
Frequency of crisis (defined as the number of painful episodes within a given period of time)Severity of pain (as reported by study authors; for example, as number needing opioid analgesics or non‐steroidal anti‐inflammatory drugs (NSAIDs))QoL of participants living with SCD and their caregivers (using a validated form; for example, the 36‐item Short Form Health Survey (SF‐36) questionnaire)
primary outcome:
Nebuliser systems for drug delivery in cystic fibrosis
Treatment time (for single nebulised treatment)Quality of life (QoL) (as measured by e.g. the Cystic Fibrosis Questionnaire (CF‐Q) (Henry 2003) or Cystic Fibrosis Quality of Life (CFQoL) (Gee 2000), both validated measures of QoL in people with CF)Deposition (as measured by radio labelling or by serum, sputum or urine levels of the studied medication) (post hoc change to consider sputum and urine levels)
primary outcome:
Interventions for improving adherence to airway clearance treatment and exercise in people with cystic fibrosis
Adherenceelectronic monitoring (% of completed treatments)confirmed observed participation by parent or carer or healthcare professional (% of completed treatments)self‐reported diary (% of completed treatments)
primary outcome:
Digital technology for delivering and monitoring exercise programs for people with cystic fibrosis
Physical activity (measured objectively with devices such as pedometers, accelerometers, or activity monitors or subjectively using self‐report and validated questionnaires [e.g. International Physical Activity Questionnaire (IPAQ)])Participation in physical activity (defined as number of steps, time spent in physical activity [e.g. minutes per day or week], energy expenditure [e.g. kilocalories or joules per day or week])Adherence to exercise training (defined as the amount of completed exercise divided by the amount of prescribed exercise)Intensity of physical activity (e.g. MET)Self‐management behaviorAbility of the individual to fit treatment requirements for CF into their everyday activities (e.g. monitoring symptoms, monitoring of energy expenditure, communicating about illness or aspects of care)Measures of self‐efficacy, coping, problem‐solving, or independencePulmonary exacerbationsTime to subsequent exacerbationNumber of pulmonary exacerbations (per participant per month, if available)
primary outcome:
Interventions for the long‐term prevention of hereditary angioedema attacks
HAE attacks (number of attacks per person, per population) and change in number of HAE attacksMortalitySerious adverse events, such as hepatic dysfunction, hepatic toxicity and deleterious changes in blood tests (e.g. glucose tolerance, thyroid hormones, lipids, lipoproteins)
primary outcome:
Strategies for screening for familial hypercholesterolaemia in primary care and other community settings
Diagnosis of definite FH at the end of study follow‐up, defined by a positive genetic mutation test or clinical phenotype typical of FH (as defined by diagnostic criteria)Diagnosis of possible and probable FH (as defined by diagnostic criteria) at the end of study follow‐upAdverse events at the end of study follow‐up (e.g. drug adverse events, hospitalisations, all‐cause mortality)
primary outcome:
Embolisation for pulmonary arteriovenous malformation
Initial occlusion as determined by angiogram immediately after embolisationLong‐term occlusion as determined bychest radiography (standard posteroanterior and lateral chest radiographs)contrast “bubble” echocardiographyradionuclide shunt study and pulse oximetry (SaO₂)measured in up‐right positionmeasured in supine positioncomputed tomography (CT)helical CT pulmonary angiogram with 3D reconstruction for assessment of PAVM perfusion (post hoc change)helical CT without contrast media for assessment of PAVM morphology onlyAll causes mortality secondary to PAVM if death was due to any cause, including:cerebral abscessstrokehaemoptysishaemothorax
primary outcome:
Long‐acting inhaled bronchodilators for cystic fibrosis
Change in FEV1 from baseline (L and per cent (%) predicted)Participant‐reported outcomes including QoL using standardised and validated QoL scores (e.g. CFQ‐R (Quittner 2009)) and symptom scores (e.g. Respiratory and Systemic Symptoms Questionnaire (RSSQ), Respiratory Symptom Score (RSS) (Goss 2007))Adverse effects (especially those associated with long‐acting inhaled bronchodilators i.e. for beta‐2 agonists tremor, nervous tension, headache, peripheral dilatation and palpitation, increased heart rate, dry mouth, increased wheeze and shortness of breath; for LAMAs dry mouth, gastrointestinal motility disorder (constipation and diarrhoea), nausea, gastro‐oesophageal reflux disease, dysphagia, tachycardia)frequency of adverse effectsseverity of adverse effects, e.g. mild or moderate or severe (where it has been reported)
primary outcome:
Enzyme replacement therapy for infantile‐onset Pompe disease
Cardiac function (as measured by the left ventricular mass z score (LVM‐Z), fractional shortening and ejection fraction).Time to ventilation.Survival.
primary outcome:
Beta‐blockers for preventing aortic dissection in Marfan syndrome
All‐cause mortality (including mortality attributed to Marfan syndrome)All non‐fatal serious adverse events (including all‐cause hospitalisations, cardiovascular events such as aortic dissection or rupture, and cardiovascular surgery)
primary outcome:
Prophylactic levosimendan for the prevention of low cardiac output syndrome and mortality in paediatric patients undergoing surgery for congenital heart disease
All‐cause mortality within 30 days.Time to death (censored after three months).LCOS, defined as two or more of the following: (a) blood lactate higher than 3 mmol/L (27 mg/dL) or increase in blood lactate of at least 2 mmol/L (18 mg/dL) from baseline, prior to administration of levosimendan, (b) central venous oxygen saturation less than 50% in biventricular physiology without shunts, (c) increase in arterial to central venous oxygen saturation difference by at least 20% from baseline, prior to administration of levosimendan, (d) urine output less than 1 mL/kg/h, (e) peripheral skin temperature to core body temperature difference of more than 7°C, (f) cardiac index less than 2.2 L/min/m².
primary outcome:
Non‐invasive ventilation for cystic fibrosis
MortalityQuality of life (QoL)Symptoms of sleep‐disordered breathing
primary outcome:
Non‐surgical interventions for treating heavy menstrual bleeding (menorrhagia) in women with bleeding disorders
Menstrual blood loss (MBL)*objective assessment of blood loss (mL)subjective assessment of blood lossparticipant’s satisfaction as regards reduction in blood loss (very satisfied, satisfied, not sure, not satisfied)participant's perception of blood loss (reduced, same, increased)indirect measures of blood lossduration of loss (days)number of sanitary padspictorial bleeding assessment charts (PBACs)Adverse effects
primary outcome:
Pneumococcal vaccines for cystic fibrosis
Quality of life (validated scores e.g. Cystic Fibrosis Questionnaire‐Revised version (CFQ‐R) (Quittner 2009) and Cystic Fibrosis Quality of Life Questionnaire (CFQoL) (Gee 2000)).Exacerbation of respiratory disease over the period of the studypulmonary exacerbation (as defined in trial protocol)need for hospitalisationneed for additional antibiotic therapyoralintravenousInfection with pneumococcus (invasive pneumococcal disease of vaccine‐specific serotype)
primary outcome:
Intravenous alpha‐1 antitrypsin augmentation therapy for treating patients with alpha‐1 antitrypsin deficiency and lung disease
Mortality.Harms of the intervention.
primary outcome:
Enzyme replacement therapy for Anderson‐Fabry disease
Changes in globotriaosylceramide (Gb3) concentration in plasma and tissue (i.e. endothelial cells)All‐cause deathPain (measured by the McGill Pain Questionnaire*)Acroparaesthesia (measured by the intensity and duration of acroparaesthesia as reported by the individual)Fabry crises
primary outcome:
Palivizumab for prophylaxis against respiratory syncytial virus infection in children with cystic fibrosis
Need for hospitalisation with respiratory syncytial virus (RSV) infection based on clinical diagnosis or validated laboratory diagnosis or bothMortality
primary outcome:
Topical cystic fibrosis transmembrane conductance regulator gene replacement for cystic fibrosis‐related lung disease
Respiratory exacerbations (assessed by need for additional oral or intravenous antibiotics)*oral antibiotics (days or episodes)intravenous antibiotics (days or episodes)Lung function testing (absolute values or percent predicted for age, sex and height)forced expiratory volume at one second (FEV1)forced vital capacity (FVC)relative change in FEV1 or FVCother relevant lung function tests (for example, infant lung function tests and lung clearance index)Survival (either as a binary outcome or time to event)*
primary outcome:
Neuraminidase inhibitors for the treatment of influenza infection in people with cystic fibrosis
Need for hospitalisation (directly related to influenza illness)Time to alleviation of constitutional symptoms (fever and associated symptoms) (post hoc change ‐ see Differences between protocol and review)Respiratory functionforced expiratory volume in one second (FEV1) as per cent of predicted for age, sex and heightforced vital capacity (FVC) as per cent of predicted for age, sex and height
primary outcome:
Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)
Z scores forheightweightSix‐minute walk test (6MWT)
primary outcome:
Anti‐inflammatory drugs and analgesics for managing symptoms in people with cystic fibrosis‐related arthritis
Joint involvementnumber of swollen jointsnumber of joints with limited range of movementnumber of painful joints; pain assessed, for example, using visual analogue scalesPhysical function (to be assessed using instruments including disease‐specific instruments (for example, Health Assessment Questionnaire (HAQ), Child Health Assessment Questionnaire (CHAQ), Child Health Questionnaire (CHQ) and Juvenile Arthritis Functional Status Index (JASI)), generic instruments and visual analogue scales (VAS). The Steinbrocker classification should also be considered.Quality of Life (to be measured using various disease‐specific and generic instruments including CHQ, JAQQ, PedsQL and Quality of My Life Questionnaire (QoMLQ), AIMS, Nottingham Health Profile (NHP), Sickness Impact Profile (SIP), Short Form Health Survey 36 (SF36) and EuroQol).
primary outcome:
Home versus hospital intravenous antibiotic therapy for cystic fibrosis
Lung functionchange in per cent predicted or absolute change in forced expiratory volume in one second (FEV1)change in forced vital capacity (FVC)any other lung function parametersLung infection (conversion of sputum from culture positive to culture negative, reduction in colony forming units (CFU) counts for P. aeruginosa and other micro‐organisms)Improvement of clinical score, by validated instruments
primary outcome:
Ascorbic acid for the treatment of Charcot‐Marie‐Tooth disease
Change in impairment after at least 12 months.We accepted any measure of impairment made with a validated scale, such as the MRC sum score, Neuropathy Impairment Score (NIS) (Dyck 2005b), Total Neuropathy Score (Cornblath 1999), CMT Neuropathy score (Shy 2005a), or CMT neuropathy score version 2 (Murphy 2011).High‐quality evidence found no evidence of clinically significant differences between ascorbic acid and placebo at 12 months, on any of the validated measures for the primary outcome. Using the CMTNS (scale 0 to 36 points), the mean difference (MD) was ‐0.37; 95% CI ‐0.83 to 0.09; five studies; N = 533 (see Analysis 1.1; Figure 3). The CMTES (scale 0 to 28 points), which represents the CMTNS without the electrophysiological parameters, showed a significant benefit for ascorbic acid in one trial (Micallef 2009), but the meta‐analysis of all three trials that reported this outcome parameter found no significant difference (MD ‐0.15; 95% CI ‐0.57 to 0.27; three trials; N = 460; Analysis 1.2). For both outcome parameters, Pareyson 2011 dominated the meta‐analysis as it is the largest of the trials. The one study using the NIS reported no significant difference (MD 1.91; 95% CI ‐2.24 to 6.06; N = 110; Lewis 2013; Analysis 1.3). No study reported the MRC score, TNS, or CMTNS v2.There was moderate‐quality evidence for the primary outcome that ascorbic acid treatment had no clinically meaningful effect over placebo using any of the validated outcome parameters (CMTNS in three studies, N = 388; CMTES in two studies, N = 337; Analysis 2.1; Analysis 2.2; Figure 5). It should be noted that these comparisons are strongly dominated by Pareyson 2011 because of its larger number of participants.The trial did not report the CMTNS, CMTES or CMTNS v2 because they are not validated for children. A paediatric CMT scale has been recently developed (Burns 2012), but was not available at the time of the study.
primary outcome:
Oral steroids for long‐term use in cystic fibrosis
Objective lung function testsforced expiratory volume at one second (FEV1), forced vital capacity (FVC), forced expiratory flow from 25% to 75% of vital capacity (FEF25-75) (% predicted) absolute change from baselineFEV1, FVC, FEF25-75 (% predicted) post‐treatmentNumber of days of intravenous antibiotics for respiratory exacerbationsHospitalisation rates for respiratory exacerbations
primary outcome:
Vaccines for preventing infection with Pseudomonas aeruginosa in cystic fibrosis
Time to chronic P. aeruginosa infectionPulmonary functionforced expiratory volume in one second (FEV1) as per cent of predicted for age, sex and heightforced vital capacity (FVC) as per cent of predicted for age, sex and heightMortality
primary outcome:
Intravenous antibiotics for pulmonary exacerbations in people with cystic fibrosis
Lung functionforced expiratory volume at one second (FEV1)forced vital capacity (FVC)Time to next exacerbation (although as there is no agreed definition of pulmonary exacerbations, we shall accept the individual clinicians' diagnosis, acknowledging the inherent difficulties that this poses)Quality of life (e.g. Cystic Fibrosis Questionnaire‐Revised (CFQ‐R) (Quittner 2009))
primary outcome:
Prophylactic milrinone for the prevention of low cardiac output syndrome and mortality in children undergoing surgery for congenital heart disease
Total mortality within 30 daysTime to death (censored after three months)Low cardiac output syndrome as defined by authors of individual studies and typically based on two or more of the following:
primary outcome:
Surgical interventions for treating pectus excavatum
Quality of life (QoL) ‐ we considered QoL in its broader definition that encompasses overall QoL, symptoms and side effects that may or may not reflect or affect QoL. Data from either the patient or another person by proxy (Fayers 2007)the generic health‐related quality of life questionnaires, the Child Health Questionnaire CHQ‐CF87 (child version), and CHQ‐PF50 (parent version)Keith and Schalock's quality of life model (Schalock 1989)aesthetic appearance post‐surgery (body image functioning attributable to pectus excavatum) (Lawson 2003)patient reported outcomes (PROs)or any other validated questionnairePulmonary functionmaximum voluntary ventilation (MVV)forced expiratory volume measured over one second (FEV1)vital capacity (VC)forced vital capacity (FVC)as otherwise assessed by the investigatorsCardiac function (we will consider the echocardiographic method, transthoracic or transesophageal)left ventricular ejection fraction (LVEF)right ventricular ejection fraction (RVEF)left ventricular end‐diastolic volume (LVEDV)right ventricular end‐diastolic volume (RVEDV)as otherwise assessed by the investigators
primary outcome:
Sodium channel blockers for cystic fibrosis
Respiratory exacerbations (to be measured using a validated method)*Lung function testing (absolute values or percent predicted for age, sex and height)forced expiratory volume at one second (FEV1)forced vital capacity (FVC)other relevant lung function tests (for example, infant lung function tests)Adverse events*mild (examples: sore throat; hoarse voice; dry mouth)moderate (examples: wheeze; cough; local allergic reaction)severe (examples: coughing up blood (haemoptysis); collapsed lung (pneumothorax); chest infection; systemic allergic reactions)Survival (either as a binary outcome or time to event)*
primary outcome:
Vaccines for preventing influenza in people with cystic fibrosis
Lung function measurement expressed as forced expiratory volume in one second (FEV1) and forced vital capacity (FVC) as per cent of predicted for age, sex and heightShwachman score (outcome data under point one to five following vaccination will be grouped into those measured at 0 to 3 months, 3 to 6 months, 6 to 12 months and then annually thereafter)Days spent in hospital and number of hospital admission due to respiratory exacerbation
primary outcome:
Interventions for promoting physical activity in people with cystic fibrosis
Participation in physical activity (change from baseline where possible, measured either subjectively, e.g. by an activity diary, or objectively using a monitoring device e.g. a pedometer)intensity of physical activity (measured or estimated using objective measures e.g. activity monitoring, or self‐report of physical activity e.g. International Physical Activity Questionnaire (IPAQ)time spent in physical activity (measured in minutes per week, sessions per week, etc)energy expenditure (in calories or joules)step count (using a monitoring device such as a pedometer)Health‐related quality of life measured by generic or disease specific assessments, or both
primary outcome:
Nebulized and oral thiol derivatives for pulmonary disease in cystic fibrosis
Pulmonary function testing (PFT)forced expiratory volume in one second (FEV1) per cent predicted (change from baseline and absolute data)forced vital capacity (FVC) per cent predicted (change from baseline and absolute data)
primary outcome:
Single‐incision sling operations for urinary incontinence in women
Subjective cure or improvement of urinary incontinence
primary outcome:
Surgical approach to hysterectomy for benign gynaecological disease
Return to normal activitiesSatisfaction and quality of lifeIntraoperative visceral injuryBladder injuryUreter injuryUrinary tract (bladder or ureter) injuryBowel injuryVascular injuryMajor long‐term complicationsFistulaPelvic‐abdominal painUrinary dysfunctionBowel dysfunctionPelvic floor condition (prolapse)Sexual dysfunction
primary outcome:
Combined oral contraceptive pill for primary dysmenorrhoea
Difference in pain between the treatment and control groups, measured with Visual Analogue Scale (VAS) or another scale, preferably at the end of treatment (continuous data).Difference in the number of women experiencing pain relief between the treatment and control groups at the end of treatment (dichotomous data).Adverse events from treatment (incidence and type of adverse events).
primary outcome:
Surgery for women with apical vaginal prolapse
1. Awareness of prolapse: any affirmative response to questions relating to awareness of prolapse or vaginal bulge, or any affirmative response to question three of Pelvic floor distress inventory (PFDI‐20) “Do you usually have a bulge or something falling out that you can see or feel in the vaginal area?”2. Repeat surgery:
2.1 repeat surgery for prolapse;
2.2 repeat surgery for stress urinary incontinence.2.3 repeat surgery for mesh exposure if relevant.3. Any recurrent prolapse Defined as any stage 2 or greater vaginal prolapse (Pelvic Organ Prolapse Quantification (POPQ): prolapse ‐ 1 cm above the hymen or below).
primary outcome:
Statins for women with polycystic ovary syndrome not actively trying to conceive
Resumption of menstrual regularity (i.e. initiation of menses or significant shortening of cycles, number of women with resumption of normal menstrual cycle (between 21 and 34 days), or as defined by study authors).Resumption of spontaneous ovulation documented by biochemical methods (i.e. evidence of serum progesterone in the luteal range for the reference laboratory; or rise in basal body temperature of more than 0.4 ºC, as measured on a basal body temperature chart, for 10 days or more).
primary outcome:
Gonadotropin‐releasing hormone analogues for endometriosis
Overall pain, defined by using both quantitative measures such as visual analogue scales or categorical outcomes, at the end of treatment and at three, six, nine, 12, 18 and 24 months' follow‐up, where possibleThe objective measurement of bone mineral density (BMD), including dual‐energy photon absorptiometry (DPA), dual‐energy X‐ray absorptiometry (DEXA), single‐energy photon absorptiometry (SPA), single‐energy X‐ray absorptiometry (SXA) and quantitative computed tomography (QCT). Measurements taken at the lumbar spine and femoral head will be considered, whilst those at the distal forearm will be excluded because these measurements are of cortical bone which is less affected by GnRHa therapy (Whitehouse 1990; Ylikorkala 1990). Bone density measurements at the end of treatment and in the follow‐up period will be included. Measurements will be grouped according to the anatomical location of measurements and the timing of measurements.
primary outcome:
Oral anticholinergic drugs versus placebo or no treatment for managing overactive bladder syndrome in adults
Mean change from baseline in condition‐specific quality of life (e.g. measured using the OAB‐Q‐SF or King's Health Questionnaire).Perception of cure or improvement (patient‐reported using a validated instrument, e.g. the Patient Perception of Bladder Condition (PPBC). The PPBC was used as the primary tool for meta‐analysis).Mean number of urinary urgency episodes per 24 hours (patient‐reported, e.g. by patient diary).
primary outcome:
Immediate versus delayed postpartum insertion of contraceptive implant and IUD for contraception
Rate of insertion of contraceptive implants and IUDs at the first postpartum visit (four to six weeks postpartum).
primary outcome:
Hormone therapy for uterine and endometrial development in women with premature ovarian insufficiency
Uterine volume: measured by ultrasound (to reflect reproductive potential); calculated by using the formula: volume (mL) = transverse width x sagittal width x length x 0.5.
primary outcome:
Interventions to prevent or treat heavy menstrual bleeding or pain associated with intrauterine‐device use
Volume of menstrual blood loss: outcome measures for bleeding included quantitative measurements of blood loss (Hallberg 1964), semi‐quantitative assessments of blood loss by PBAC scores (Higham 1990; Reid 2000), or IUD discontinuation (Hubacher 2006) and number of sanitary pads used for bleeding. Participant perceptions of bleeding being more abundant than usual using categorical scales or menstrual diary cards could also be included.Duration of bleeding: duration of bleeding in number of bleeding or spotting daysPainful menstruation: level of pain could be reported on Likert scales, pain scores, qualitatively or any pain reduction
primary outcome:
Medical methods for first trimester abortion
The main outcome measure was failure to achieve complete abortion two weeks after medical abortion.Sonography was performed to verify abortion completion. If the sonographer reported that the uterus was empty, with no products of conception identified, complete success and efficacy of the drug were recorded.
primary outcome:
Immediate versus delayed postabortal insertion of contraceptive implant
Initiation rate of contraceptive implant, evaluated at four to six weeks after abortionUtilization rate of contraceptive implant, six months after abortionUtilization rate of contraceptive implant, 12 months after abortionFailure rate of medical abortion including overall failure rate, require extra medication, and require surgerySide effectsBleeding, including mean days of bleeding and proportion of irregular bleedingSide effects other than bleeding