Speciality
Condition
Studies selecting outcomes for use in clinical trials of interventions to treat specific illnesses or clinical features of underlying conditions
Disease Activity
Physical consequences Adverse
Functional status
Social outcome/ family
Resource utilisation
of disease – damage/
effects of
outcome, including
complications
therapy
Quality of Life
Study
Critical care:
Sepsis
(Goldstein)[1]
Gastroenterology:
Crohn’s disease
(Griffiths)[2]
Appropriate
biomarkers (as
secondary or
pharmaco-dynamic
endpoints)
Organ failure-free days;
Mortality (not as the
primary outcome)
Acute:
Primary – CDAI
Secondary- PCDAI
Growth
Remission:
CDAI;
Physician
assessment;
Parent/patient
assessment
Height velocity
Change in Pediatric
Overall Performance
Category score
between admission
and discharge; overall
functioning at 3 or 6
months
HRQoL
continued
Disease Activity
Haematology:
Signs-organ specific
measures
Symptoms-clinican
reported
Symptoms – patient
reported
Global ratingphysician
Global rating-patient
reported
GVHD
(Pavletic)[3]
Neonatology:
Neonatal apnoea
(Finer)[4]
Neonatology:
Neonatal cardiovascular instability
(Short)[5]
Physical consequences
of disease – damage/
complications
Adverse
effects of
therapy
Functional status
Clinician assessed:
Grip strength;
2-minute walk
time;
Karnofsky/
Lansky scale
Social outcome/ family
outcome, including
Quality of Life
SF-36
Resource utilisation
CHRIS
Patient assessed:
HAP;
ASK;
Frequency/
duration/severity of
apnoea
Associated postnatal
morbidity;
Measures of organ
perfusion
Primary outcome:
Combined endpoint of
mortality or severe
neurological outcome
Secondary outcomes:
NEC;ROP;BPD;
Neurodevelopment at
18 months
Adverse effects
of drugsarrhythmia,
hypertension,
seizures,
hormonal
effects
Development at 2
years
Duration of hospital stay;
Duration of assisted
ventilation
Number of days
hospitalized for apnoea
continued
Disease Activity
Neonatology:
Acute pain
measurements:
Behavioural/
Haemodynamic/
Metabolic/
Respiratory/
Renal;
Need for
supplemental opiates;
Frequency of
postnatal
complications of
prematurity
Pain
(Anand)[6,7]
Neonatology
Neonatal postoperative cardiac
dysfunction
(Roth) [8]
Endpoint specific to
underlying cardiac
diagnosis. Secondary
measures: blood
lactate;oxygenation;c
reatinine clearance,
BNP, Need for
inotropes; cerebral
perfusion
Physical consequences
of disease – damage/
complications
Neurobehavioural
assessment
Adverse
effects of
therapy
Global
developmental
delay
Interventionspecific
adverse effects
Duration of delayed
sternal closure;
Mortality beyond 30th
postoperative day;
Functional status
Sleeping pattern;
Feeding/Growth;
Mother-infant
interaction;
Cry characteristics;
Pain response;
Development of
addiction;
Substance abuse;
Anxiety disorders;
Neurological/
cognitive
development at 18 or
36 months
Neurodevelopment at
1 to 2 years
Social outcome/ family
outcome, including
Quality of Life
Resource utilisation
Length of hospital stay/
ventilation/ NICU stay as
represented by
physiological definition
of criteria to account for
inter-centre variations in
practice
Duration of assisted
ventilator;
Duration of NICU stay;
Duration of hospital stay
after cardiac surgery;
Disease Activity
continued
Neonatology:
Seizures
(Clancy)[9]
Neurology:
Infantile spasms
(West Delphi
Group) [10,11]
Psychiatry:
Bipolar affective
disorder
(Carlson)[12]
Physical consequences
of disease – damage/
complications
Primary: Cessation of
EEG-detected
seizures
Clinical outcome –
spasm cessation
Electroclinical
outcome- resolution
of hypsarrhythmia
Relapse-free
response;
Continued subtle
spasm; Time to
relapse;Presence of
other seizures
Primary: Mania
(YMS); Depression
(CDRS).Secondary:A
DHD symptomology;
Aggressive
behaviour;
Global improvement
Adverse
effects of
therapy
Functional status
Social outcome/ family
outcome, including
Quality of Life
Neurodevelopment at
8 years
Development at 2
years;Death and other
serious adverse effects
associated with the
illness;
Nonserious adverse
events associated with
the illness;
Presence of and
progression to other
seizure types
Academic outcome
and cognitive
function
Family outcomeCHQ;
Other social effects
Resource utilisation
Disease Activity
continued
Psychiatry:
Seizure frequency
Non- epileptic
seizures
(LaFrance)[13]
Respiratory:
CF
(Ramsey)[14]
< 6 years of age
Chest x-ray score;
Oxygenation;
Inflammatory
markers;
Illness severity;
Bronchoscopy/
BAL/brushings
>6 years of age
As above, and
Spirometry;
Sputum:
microbiology/ DNA
Physical consequences
of disease – damage/
complications
Depression;
Personality
characteristics;
Arousal
Growth;
Frequency of
pulmonary
exacerbations
Adverse
effects of
therapy
Functional status
Social outcome/ family
outcome, including
Quality of Life
HRQoL, including illness
perceptions and individual
concerns;
Employment;
Family functioning
QWB
Resource utilisation
ER visits;
Hospital admission
Disease Activity
continued
Respiratory:
Asthma-
Disease damage/
complications
Adverse
effects of
therapy
Functional status
Social outcome/ family
outcome/Quality of Life
Symptoms
FEV1/FVC
PEFR
Admission to hospital
(Smith)[15]
acute
chronic
Rheumatology:
IIM
(IMACS)[16–20]
Symptoms
FEV1/FVC
PEFR
Frequency of
medication use;
Bronchial hyperresponsiveness
Global activity
(physician/parent/
patient assessment);
Muscle strenghth
(Manual testing);
Enzymes:
CK, aldose, LD,
AST, ALT;
CMAS;
Extra-skeletal muscle
disease
Resource utilisation
Core set:
Global damage
assessment;
Assessment of different
organ systems
(VAS/SDI)
Extended core set:
MRI: muscle fibrosis/
scarring/ atrophy;
serum creatinine;
cutaneous assessment
Functional status
HRQoL
HAQ/CHAQ
HRQoL (SF36)
Disease Activity
Disease damage/
complications
Global assessmentphysician; parent/
child; activity tool;
AntiDNA antibody;
24 hr proteinuria
Serum creatinine
Global assessmentphysician; parent/
child; activity tool;
Muscle strength;
Laboratory: Muscle
enzymes
Global damage toolSDI; physician;
Height and weight
Pubertal stage:
Tanners; menses
Global assessmentphysician; MDI
Height and weight;
Pubertal stage:
Tanners; menses
Muscle strength-CMAS
CHAQ
Physician global
assessment;
Number of active
joints; ESR
Number of joints with
limited range of
movement
Functional ability
continued
Rheumatology:
SLE
(PRINTO)[21–23]
DM
(PRINTO)
Rheumatology:
Juvenile arthritis
(Giannini) [24]
Adverse
effects of
therapy
Functional status
Social outcome/ family
outcome/Quality of Life
Resource utilisation
Overall well being (Parent/
child reported)
Abbreviations: ADHD= Attention Deficit Hyperactivity Disorder; ASK= Activity Scale for Kids; AST= aspartate aminotransferase; ALT=
Alanine aminotransferase; BAL= Broncheoalveolar lavage; BNP= Brain Natriuretic Peptide; BPD=Bronchopulmonary Dysplasia; CDAI=
Crohns Disease Activity Index; CDRS= Childrens Depression Rating Scale; CHAQ= Childhood Health Assessment Questionnaire; CHQ=Child
Health Questionnaire; CHRIS= Child Health Rating Inventories; CK= Creatinine Kinase; CMAS=Childhood Myositis Assessment Scale; ESR=
Erytherocyte Sedimentation Rate; FEV1= Forced Expiratory Volume in 1 second; FVC= Forced Vital capacity; HAP= Human Activity Profile;
HAQ= Health Assessment Questionnaire; HRQoL=Health Related Quality of Life; LD= Lactate Dehydrogenase; MDI=Myositis Disease Index;
NEC=Necrotising Enterocolitis; NICU= Neonatal Intensive Care Unit; PCDAI= Paediatric Crohn’s Disease Index; PEFR= Peak Expiratory
Flow Rate; QWB: Quality of Well Being Scale; ROP=Retinopathy of Prematurity; SDI=SLE Collaborating clinic/ACR damage index; VAS=
Visual Analogue Scale; YMS=Young mania rating scale
Condition
Study
Dentistry:
Dental restoration
(De Rouen) [25]
Specific clinical trial
assessing the safety
profile of amalgam
in terms of side
effects of mercury
exposure ( a
component of
amalgam)
Disease Activity
Studies selecting outcomes for use in specific clinical trials
Consequences of
Adverse effects of
Functional status
Social outcome, family
disease – damage/ therapy
outcome, including
complications
Quality of Life
Memory;
Attention;
Motor/ visual skills;
IQ;
NCV;
Clinical signs;
Urinary GST/
albumin/ porphyrin
Resource utilisation
Abbreviations: NCV= Nerve conduction velocity; GST= Glutathione S-Transferase
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