Additional file 3 Results from the Phase 1 questionnaires

Table 1 Outcomes suggested in Phase 1 by clinicians, for pre-school children

Domain / Outcome / Number (%) of clinicians suggesting the outcome (n=46)
Disease Activity / Daytime symptoms / 43 (93)
Nocturnal symptoms / 28 (61)
Symptoms affecting physical activity / 17 (37)
Need for bronchodilator / 23 (50)
Exacerbations / 29 (63)
Patient/parent overall view / 4 (8)
Ability to feed / 2 (4)
Wheezing in response to allergens / 3 (7)
Normal chest examination / 3 (7)
Disease Damage / Death / 1 (2)
Functional status / Activities of daily living / 9 (20)
School/nursery / 5 (11)
Quality of Life/family / Overall Quality of Life / 4 (9)
Family outcomes / 8 (17)
Adverse effects of therapy / Short term adverse effects / 10 (22)
Growth / 8 (17)
Other long term adverse effects / 1 (2)
Health resource utilisation / Unscheduled visit / 9 (18)
Hospital admission / 20 (45)
Admission to intensive care unit / 1 (2)
Other / Developmental milestones / 1 (2)

Table 2 Outcomes suggested in Phase 1 by clinicians for school-aged children

Domain / Outcome / Number (%) of clinicians suggesting the outcome (n=46)
Disease Activity / Daytime symptoms / 43 (92)
Nocturnal symptoms / 30 (63)
Symptoms affecting physical activity / 29 (63)
Need for bronchodilator / 27 (58)
Exacerbations / 31 (67)
Patient/parent overall view / 4 (8)
Lung function / 14 (30)
Inflammatory markers a / 2 (4)
Normal chest exam / 2 (4)
Change in FEV1 in response to Salbutamol / 2 (4)
Disease Damage / --No outcomes suggested--
Functional status / Activities of daily living / 14 (30)
School attendance / 27 (59)
Quality of Life/family / Overall Quality of Life / 7 (15)
Family outcomes / 1 (2)
Adverse effects of therapy / Short term adverse effects / 6 (13)
Growth / 11 (24)
Other long term adverse effects / 15 (30)
Health resource utilisation / Unscheduled visit / 7 (15)
Hospital admission / 13 (28)
Other / Achieving parent/ patient-defined goal / 1 (2)

Footnotes:

a: both clinicians suggested Exhaled Nitric Oxide (ENO)

Table 3 Frequency with which outcomes were suggested by parents of pre-school children (n=11)

Domain / Outcome / Number (%) of parents of preschool children suggesting outcome (n=11)
Disease activity / Daytime symptoms / 8 (73)
Nocturnal symptoms / 1 (9)
Symptoms affecting activity (eg exercise) / 3 (27)
Need for reliever medication (eg bronchodilator) / 2 (18)
Exacerbations / 4 (36)
Patient/parent overall view on asthma control / 1 (9)
Ability to feed / 1 (9)
Disease damage / --No responses categorised into this domain--
Functional status / ADL / 2 (18)
DOMAIN:
Quality of life/family / Overall QoL / 1 (9)
Emotional well being / 1 (9)
Adverse effects of therapy / Short term AE / 5 (45)
Growth / 1 (9)
Other long term AE / 4 (36)
Dependence / 1 (9)
Health resource utilisation / --No responses categorised into this domain--
Other / Health-related problems when child is older / 2 (18)

Table 4 Frequency with which outcomes were suggested by parents of school-aged children (n=27)

Domain / Outcome / Number (%) of parents of school-age children suggesting outcome (n=27)
Disease activity / Daytime symptoms / 9 (33)
Nocturnal symptoms / 20 (74)
Symptoms affecting physical activity / 11 (41)
Need for reliever medication (eg bronchodilator) / 3 (11)
Exacerbations / 13 (48)
Patient/parent overall view / 5 (19)
Ability to feed / 1 (4)
Disease damage / Death / 3 (11)
Functional status / Activities of daily living / 13 (48)
School/nursery attendance / 2 (7)
Quality of life/family / Overall Quality of Life / 4 (15)
Emotional well being / 1 (4)
Adverse effects of therapy / Short term adverse effects / 16 (60)
Growth / 2 (7)
Other long term adverse effects / 14 (52)
Development of dependence on medication / 2 (7)
Health resource utilisation / A+E/GP visit / 2 (7)
Hospital admission / 2 (7)
Other / Health-related problems when child is older / 5 (19)

Table 5 Frequency with which outcomes were suggested by young people (n=11)

Domain / Outcome / Number (%) of parents of young people suggesting outcome (n=11)
Disease activity / Daytime symptoms / 5 (45)
Nocturnal symptoms / 2 (18)
Symptoms affecting physical activity / 4 (36)
Exacerbations / 1 (9)
Disease damage / Death / 1 (9)
Functional status / Activities of daily living / 3 (27)
Quality of life/family / Overall quality of life / 1 (9)
Emotional well being / 1 (9)
Adverse effects of therapy / Short term adverse effects / 2 (18)
Growth / 1 (9)
Other long term adverse effects / 3 (27)
Health resource utilisation / Hospital admission / 1 (9)
Other / Health-related problems when child is older / 1 (9)

Additional file 3 Results from the Phase 1 questionnaires

Table 6: Whether outcomes were carried forward to Phase 2, including the frequency with which they were suggested by participants, and measured in RCTs in childhood asthma

Outcome / Clinicians School age children (n=46) / Clinicians
Pre school children (n=46) / Parents of school age children (n=27) / Young people (n=11) / Parents of preschool children (n=11) / RCTs including school age children (n=95) / RCTs including pre-school children (n=47) / Is outcome carried forward for school age children / Is outcome carried forward for pre-school age children / Notes
DISEASE ACTIVITY
Daytime symptoms / 43 (92) / 43 (93) / 9 (33) / 5 (45) / 8 (73) / 84 (88) / 43 (91) / Yes / Yes / 84/95 and 43/47 RCTs measured symptoms. Daytime, nocturnal and exercise-induced symptoms were included in these scores.
Nocturnal symptoms / 30 (63) / 28 (61) / 20 (74) / 2 (18) / 1 (9) / Yes / Yes
Symptoms affecting activity (eg exercise) / 29 (63) / 17 (37) / 11 (41) / 4 (36) / 3 (27) / Yes / Yes
Need for bronchodilator / 27 (58) / 23 (50) / 3 (11) / 0 / 2 (18) / 71 (75) / 39 (83) / Yes / Yes
Exacerbations / 31 (67) / 29 (63) / 13 (48) / 1 (9) / 4 (36) / 31 (33) / 17 (36) / Yes / Yes / This outcome includes suggestions by clinicians, that exacerbations, specifically in response to URTIs, are an important outcome
Patient/parent overall view on asthma control / 3 (6) / 3 (6) / 5 (19) / 0 / 1 (9) / 9 (9) / 6 (13) / Yes / Yes / Although parents did not specifically describe this outcome, 5 parents did describe terms relating to overall asthma control
Lung function / 14 (30) / 0 / 1 (4) / 0 / 0 / 89 (94) / 36 (77) / Yes / No / Not carried forward for pre-school children because it was suggested by no parents or clinicians
Bronchoconstriction induced by challenge / 0 / 0 / 0 / 0 / 0 / 28 (30) / 1 (2) / No / No / Not carried forward for school-age children because it was suggested by no parents or clinicians
Inflammatory markers / 2 (4) / 0 / 0 / 0 / 0 / 16 (17) / 3 (6) / No / No / Not carried forward (said by 2/46 clinicians; no parents)
Ability to feed / 0 / 2 (4) / 1 (4) / 0 / 1 (9) / * / * / No / No / *May have been measured as part of symptom score in RCTs, but not as an outcome in itself
Occurrence of wheeze in response to allergens / 0 / 3 (7) / 0 / 0 / 0 / * / * / No / No / *May have been measured as part of symptom score in RCTs, but not as an outcome in itself
Normal chest exam / 2 (4) / 3 (7) / 0 / 0 / 0 / 0 / 0 / No / No
Change in FEV1 in response to B2+ / 2 (4) / 3 (7) / 0 / 0 / 0 / 0 / 0 / No / No
Outcome / Clinicians School age children (n=46) / Clinicians
Pre school children (n=46) / Parents of school age children (n=27) / Young people (n=11) / Parents of preschool children (n=11) / RCTs including school age children / RCTs including pre-school children / Included for school age children / Included for pre-school age children / Notes
DISEASE DAMAGE
Death / 0 / 1 (2) / 3 (11) / 1 (9) / 0 / * / * / Yes / Yes / *Measured implicitly but not reported
Lung growth / 0 / 0 / 0 / 0 / 0 / 1 (1) / 0 / No / No
Development milestones / 1 (2) / 0 / 0 / 0 / 0 / 0 / 0 / No / No
FUNCTIONAL STATUS
ADL / 14 (30) / 9 (20) / 13 (48) / 3 (27) / 2 (18) / 6 (6) / 5 (10) / Yes / Yes
School/nursery / 27 (59) / 5 (11) / 2 (7) / 0 / 0 / 13 (14) / 4 (4) / Yes / Yes
QoL/ FAMILY OUTCOMES
Overall QoL / 7 (15) / 4 (9) / 4 (15) / 1 (9) / 1 (9) / 7 (7) / 5 (11) / Yes / Yes
Emotional well being / 0 / 0 / 1 (4) / 1 (9) / 1 (9) / * / * / No / No / Incorporated in QoL scores, but not measured as an outcome itself
Family outcomes / 1 (2) / 8 (17) / 0 / 0 / 0 / 7 (7) / 10 (21) / No / Yes
ADVERSE EFFECTS OF THERAPY
Short term AE / 6 (13) / 10 (22) / 16 (60) / 2 (18) / 5 (45) / 78 (82)* / 43 (96) * / Yes / Yes / * Did not distinguish between short and long term AEs
Growth / 11 (24) / 8 (17) / 2 (7) / 1 (9) / 1 (9) / 30 (32) / 16 (34) / Yes / Yes
Other long term AE / 15 (30) / 1 (2) / 14 (52) / 3 (27) / 4 (36) / * / * / Yes / Yes / * Did not distinguish between short and long term AEs
Dependence / 0 / 0 / 2 (7) / 0 / 1 (9) / 0 / 0 / No / No
HEALTH RESOURCE UTILISATION
A+E/GP visit / 7 (15) / 9 (18) / 2 (7) / 0 / 0 / 8 (8) / 7 (15) / Yes / Yes / * Either included in exacerbations or unscheduled HRU
Hospital admission / 13 (28) / 20 (45) / 2 (7) / 1 (9) / 0 / * / * / Yes / Yes / * Either included in exacerbations or unscheduled HRU
OTHER
Health-related problems when child is older / 0 / 0 / 7 (26) / 1 (9) / 2 (18) / 0 / 0 / Yes / Yes / Includes disease damage (ie progression of respiratory illness into older childhood or adulthood) and long term functional status (eg career, lifestyle when older)
Achieving parent/ patient-defined goal / 1 (2) / 0 / 0 / 0 / 0 / 0 / 0 / No / No

*: Issues relating to the reporting of outcomes in RCTs are described in the column entitled ‘notes’

Abbreviations: ADL=Activities of Daily Living; AE=Adverse effect; A+E=Accident and Emergency; B2+= Beta agonist; FEV1= Forced Expiratory volume in 1 second; ICS= Inhaled corticosteroids; PEFR=Peak Expiratory Flow Rate; QoL=Quality of life; URTI=upper respiratory tract infection