Clinician’s Perspective
on Clinically
Meaningful Benefit in
Pediatric Patients
Ivor D. Hill, MB, ChB, MD.
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Disclosure
I serve on an Advisory Board to Abbvie Inc.
I do not intend to discuss an unapproved or
investigative use of a commercial product or
device in my presentation.
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Considerations in Pediatrics
Children are different!
Growth and Development
- puberty
- peer pressure
- omnipotence (non compliant)
Associated Functional Disorders
- affects about 25%
- pain, nausea, vomiting, constipation
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Considerations in Pediatrics
Children are different!
Symptoms of celiac disease
- age variations
Response to the GFD
- symptom resolution - usually good
- mucosal recovery - more complete
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Expectations for Treatment in Children
Symptom resolution
Mucosal recovery
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Clinical Assessment of Rx in Children
Scenarios
Symptoms + serology + biopsy
- symptom resolution
- progressive decline in serology
(Am J Gastroenterol 2014;109:1478-83)
Symptoms + serology + biopsy
- symptoms persist
- progressive decline in serology
- no other cause found
- consider biopsy
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Clinical Assessment of Rx in Children
Scenarios
Symptoms + serology + biopsy
- symptom resolution
- failure of serological decline
(no decline on 2 tests or still elevated after 24mth)
- consider repeat biopsy
Asymptomatic + serology + biopsy
- progressive decline in serology
- “presumptive resolution”
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Clinical Assessment of Rx in Children
Scenarios
Non- Biopsy Diagnosis
Symptoms + serology alone
- symptom resolution
- progressive decline in serology
- “presumptive resolution”.
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Evaluating Alternative Therapies
to the GFD
Clinically Meaningful Benefit in Children
Minimal Requirements:
- Symptom resolution
- Normalization of CD serology
Biopsy in some cases.
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Evaluating Alternative Therapies
to the GFD
Clinically Meaningful Benefit in Children
Study patient selection
Inclusion criteria:
• Symptomatic children
• Positive CD serology
• Biopsy confirmation
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Evaluating Alternative Therapies
to the GFD
Study patient selection.
*Review - 389 children
- ages 1-20 yr.
- biopsy proven
Symptoms in children
1. Common gastrointestinal symptoms
•
•
•
•
•
•
diarrhea
abdominal pain
bloating/abdominal distension
nausea/vomiting
weight loss/poor weight gain
decreased appetite
301/389
(77%)
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* Children seen in the Celiac
Center at Nationwide Children’s
Hospital - 2010-2014.
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Evaluating Alternative Therapies
to the GFD
Study patient selection.
Symptoms in children
2. Less common GI symptoms
22/389
• constipation
(6%)
• dysphagia
• aphthous ulcers
3. Non-GI symptoms
• growth failure
• fatigue
20/389
• anemia
(5%)
• dermatitis herpetiformis
• arthralgia
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Evaluating Alternative Therapies
to the GFD
Study patient selection.
Symptoms in children
4. Miscellaneous
• headache
• anxiety
• rash
• (non DH)
5. Asymptomatic
• type 1 DM, family member,
syndromes (Down, Williams, Turner)
4/389
(1%)
42/389
(11%)
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