Brodlie M, McKean MC, Moss S, et al. Arch Dis Child (2012). doi:10.1136/archdischild-2011-301570
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1Institute of Cellular Medicine,
Newcastle upon Tyne, UK
2Department of Respiratory
Paediatrics, Great North
Children’s Hospital, Newcastle
upon Tyne, UK
David Anthony Spencer,
Department of Paediatric
Respiratory Medicine, Great
North Children’s Hospital,
Queen Victoria Rd, Newcastle
upon Tyne NE1 4LP, UK; david.
Accepted 22 March 2012
Objective To report the oral corticosteroid-sparing
effect of omalizumab in children with severe asthma.
Design 16-week therapeutic trial.
Setting Tertiary paediatric asthma clinic.
Patients 34 children with severe asthma maintained
on oral prednisolone (median age 12 years; 15 children
<12 years and 19 children ≥12 years).
Interventions Fortnightly or monthly subcutaneous
injections of omalizumab; the dose was calculated as per
manufacturer’s instructions based on body weight and
serum immunoglobulin E concentration.
Main outcome measures Reduction in prednisolone
dose; mini-Asthma Quality of Life Questionnaire (AQLQ);
Childhood Asthma Control Test (ACT); forced expiratory
volume in 1 s (FEV1).
Results Median daily prednisolone dose reduced
from 20 mg to 5 mg (n=34, p<0.0001), including
seven children who stopped prednisolone completely.
Mini-AQLQ score increased from 3.5 to 5.9 (n=24,
p<0.0001). Childhood ACT score increased from 12
to 20 (n=23, p=0.0001). FEV1 increased from 2.10 to
2.25 litres (n=31, non-signifi cant). The reduction in
prednisolone dose and improvements in mini-AQLQ and
childhood ACT were signifi cant in children both under
and over 12 years of age, with no differences in outcome
detected between these two groups.
Conclusions A 16-week therapeutic trial of
omalizumab allowed a signifi cant reduction in
daily prednisolone dose and was associated with
improvements in asthma control and quality of life in
34 children with severe asthma. Similar benefi ts were
seen in children both above and below 12 years of age.
These uncontrolled data are very encouraging. There
is an urgent requirement for a multicentre randomised
placebo-controlled trial of omalizumab in children with
severe asthma, with reduction in oral corticosteroid
dose as the primary outcome measure.
Asthma is the most common chronic disease of
childhood in developed countries.1 The majority
of asthmatic children respond well to appropri-
ate guideline-based treatment such as that out-
lined by the British Thoracic Society and Scottish
Intercollegiate Guidelines Network (BTS/SIGN).2
Children with symptoms that do not respond
adequately to treatment should be referred to spe-
cialist paediatric respiratory services for further
investigation and management.3 4 Following criti-
cal re-examination of the diagnosis and addressing
of the basics of asthma management, including a
The oral corticosteroid-sparing effect of omalizumab
in children with severe asthma
Malcolm Brodlie,1,2 Michael C McKean,2 Samantha Moss,2
David A Spencer2
nurse-led home visit, a group of children remain
who have genuinely severe disease that does not
respond to standard approaches.5–7 Many such
children require maintenance treatment with oral
corticosteroids, which is associated with consid-
erable adverse effects.8 9
Deaths in children from asthma are now thank-
fully unusual in most countries, but the condition
is still associated with considerable morbidity.1 3 10
In severe asthma this is refl ected in hospital admis-
sions, frequent outpatient attendances, time off
school and impaired quality of life for both the
patient and close family members.1 11 In the longer
term severe asthma in childhood may lead to the
development of irreversible airways obstruction,
impact on educational attainment and infl uence
Omalizumab is a monoclonal anti-immu-
noglobulin E (IgE) antibody that has emerged
as a therapeutic option in IgE-mediated asth-
ma.13 14 The majority of patients included in
published studies have been adults.14 But there
What is already known on this topic
Despite many impressive anecdotal success
stories and increasing clinical use, evidence
is sparse for omalizumab in children with
genuinely severe asthma maintained on oral
Studies to date have largely included children
with mild to moderate disease in North
American healthcare settings.
NICE guidance issued in October 2010 does
not recommend the use of omalizumab in
children under 12 years of age.
What this study adds
A 16-week therapeutic trial of omalizumab
was associated with a signifi cant reduction
in prednisolone dose and improvements in
asthma control and quality of life in children
with severe asthma.
Benefi ts were found in children both above
and below 12 years of age.
ADC Online First, published on June 9, 2012 as 10.1136/archdischild-2011-301570
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published online June 9, 2012 Arch Dis Child
Malcolm Brodlie, Michael C McKean, Samantha Moss, et al.
omalizumab in children with severe asthma
The oral corticosteroid-sparing effect of
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