Search review questions

Red blood cell dehydration · adults and children with sickle cell disease

Quality of life: Days of work lost per crisis · adults and children with sickle cell disease

Number of other sickle‐related events: · adults and children with sickle cell disease

Vitamin A deficiency disorders · children and adults with CF

Growth and nutritional status:z‐score for height · children and adults with CF

Mortality · children and adults with CF

Respiratory outcomes: FEV1 (% predicted): · children and adults with CF

Respiratory outcomes: Antibiotic days per patient in past 3 months · children and adults with CF

Adverse events · children and adults with CF

Appearance or exacerbation of gastroesophageal reflux episode: clinical test · infants and young children with cystic fibrosis

Appearance or exacerbation of gastroesophageal reflux episodes: pulmonary events · infants and young children with cystic fibrosis

Peripheral oxygen saturation (%) · infants and young children with cystic fibrosis

Number of exacerbations of upper respiratory tract symptoms · infants and young children with cystic fibrosis

Number of days on antibiotics for acute exacerbations · infants and young children with cystic fibrosis

Chest X‐ray scores · infants and young children with cystic fibrosis

Pulmonary function test (FVC) % predicted · infants and young children with cystic fibrosis

Outcomes1 · 117 children with homozygous sickle cell (SS) disease

Folate concentration Serum folate levels, measured between 6 and 12 months after entry to the study · 117 children with homozygous sickle cell (SS) disease

Haemoglobin concentration Haematological analyses were performed in the 100/115 (87%) children in whom baseline (within 2 months of entry to study) and 1 year (between 10 and 14 months after entry) · 117 children with homozygous sickle cell (SS) disease

Adverse events ‐ acute splenic sequestration Clinical events experienced by children during the 1 year period commencing at entry to the trial · 117 children with homozygous sickle cell (SS) disease

Adverse events ‐ painful episodes Clinical events experienced by children during the 1 year period commencing at entry to the trial · 117 children with homozygous sickle cell (SS) disease

Adverse events ‐ minor Infections Clinical events experienced by children during the 1 year period commencing at entry to the trial · 117 children with homozygous sickle cell (SS) disease

Adverse events ‐ major infections Clinical events experienced by children during the 1 year period commencing at entry to the trial · 117 children with homozygous sickle cell (SS) disease

Adverse events ‐ dactylitis Clinical events experienced by children during the 1 year period commencing at entry to the trial · 117 children with homozygous sickle cell (SS) disease

Major bleeding requiring transfusion of packed red blood cells · People on continuous VKA treatment undergoing minor oral surgery or dental extractions (eligibility included DOACs and… · 7 days

Number of immediate and delayed postoperative bleeding episodes requiring intervention · People on continuous VKA treatment undergoing minor oral surgery or dental extractions (eligibility included DOACs and… · Immediate (<24 h) and delayed (24 h to 10 days); bleeds occurred 1, 3 or 5 days postoperatively

Any postoperative complication except bleeding (e.g. wound infection) · People on continuous VKA treatment undergoing minor oral surgery or dental extractions (eligibility included DOACs and… · 7 days

Number of participants with postoperative bleedings requiring intervention · People on continuous VKA treatment undergoing minor oral surgery or dental extractions (eligibility included DOACs and… · 7 days

Amount of postoperative blood loss (mL) · People on continuous VKA treatment undergoing minor oral surgery or dental extractions (eligibility included DOACs and…

Change of hepatocellular enzymes1 · adults and children with cystic fibrosis‐related liver disease

Abnormally large livers reduced to within normal limits, as measured by ultrasound · adults and children with cystic fibrosis‐related liver disease

Need for liver transplantation · adults and children with cystic fibrosis‐related liver disease

Mortality · adults and children with cystic fibrosis‐related liver disease

Nutritional status: change in weight (kg) · adults and children with cystic fibrosis‐related liver disease

Development of portal hypertension · adults and children with cystic fibrosis‐related liver disease

Improved abnormal biliary excretion · adults and children with cystic fibrosis‐related liver disease

Change in weight (kg):1 at 12 months · adults and children with cystic fibrosis

Change in height (cm):1 at 12 months · adults and children with cystic fibrosis

Change in total calories (Kcal/day): at 12 months · adults and children with cystic fibrosis

Change in total protein (g/day): at 12 months · adults and children with cystic fibrosis

Change in total fat (g/day): at 12 months · adults and children with cystic fibrosis

Adverse events: · adults and children with cystic fibrosis

Change in lung function ‐ FEV₁ (% predicted): at 12 months · adults and children with cystic fibrosis

Mortality at any time point · people with transfusion‐dependent thalassemia

Serum ferritin (ng/mL): mean change from baseline and at end of study · people with transfusion‐dependent thalassemia

LIC (mg/g) evaluated by MRI R2*: mean change from baseline · people with transfusion‐dependent thalassemia

Satisfaction · people with transfusion‐dependent thalassemia

Adherence: discontinuations · people with transfusion‐dependent thalassemia

Renal failure · people with transfusion‐dependent thalassemia

Mortality at any time point · people with transfusion‐dependent thalassaemia