Efficacy and Safety of Adalimumab for Paediatric Crohn's Disease: A Systematic Review
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Piotr Dziechciarz1, Andrea Horvath2, Jaroslaw Kierkuś3
1Department of Paediatrics Medical University of Warsaw, Warsaw, Poland piotrdz@hotmail.com.
Insights
Adalimumab therapy shows approximately 50% remission rates in children with Crohn's disease within the first year. While generally safe, further high-quality studies are needed to confirm effectiveness and safety in pediatric patients.
Area of Science:
- Pediatric Gastroenterology
- Immunology
- Pharmacology
Background:
- Adalimumab is an established therapy for adult Crohn's disease (CD).
- Evidence on its use in pediatric CD populations is less established.
- This study systematically reviews existing literature on adalimumab for pediatric CD.
Purpose of the Study:
- To systematically assess the published evidence on the efficacy and safety of adalimumab for Crohn's disease in children.
- Evaluate remission rates and adverse events in pediatric patients treated with adalimumab.
- Identify gaps in the current research for pediatric Crohn's disease treatment.
Main Methods:
- Systematic literature search of MEDLINE, EMBASE, and Cochrane Library up to July 2015.
- Included randomized controlled trials and observational studies on adalimumab for pediatric Crohn's disease.
- Analyzed 14 studies encompassing 664 patients aged 1.9 to 21 years.
Main Results:
- Pooled remission rates varied, with approximately 44% at 12 months.
- Primary non-response was observed in 6% of patients.
- Severe adverse events were reported in 12%, including 2 deaths and 1 medulloblastoma; withdrawal due to adverse events was 35% in one study.
Conclusions:
- Low-quality evidence suggests adalimumab achieves remission in about half of pediatric Crohn's disease patients within a year.
- The safety profile appears reasonable, but significant adverse events and withdrawals occurred.
- High-quality evidence is still needed to definitively establish the effectiveness and safety of adalimumab in pediatric Crohn's disease.
Background And Aim:
Adalimumab is well-established therapy for adults with Crohn's disease [CD]. The aim of the study was to systematically assess the published evidence on the efficacy and safety of adalimumab for Crohn's disease in children.
Methods:
MEDLINE, EMBASE, the Cochrane Library, and abstracts from the main gastroenterological meetings in the past 5 years were systematically searched up to July 2015 for randomised controlled trials and observational studies on the efficacy and safety of adalimumab for Crohn's treatment in children and adolescents.
Results:
A total of 14 studies [1 randomised controlled trial, 13 case series], altogether including 664 patients [age: 1.9 to 21 years] were available for analysis. The studies differed with respect to patients' characteristics, including percentage of infliximab-naïve patients, disease duration, site of the disease, adalimumab doses, treatment duration, and follow-up period. The pooled remission rates were: 30% [n = 93/309] at 4 weeks, 54% [n = 79/145] at 3 months, 45% [n = 18/40] at 4 months, 42% [n = 146/345] at 6 months, 57% [n = 20/35] at 8 months, and 44% [n = 169/383] at 12 months. Of the total patients, 6% [n = 13/207] were classified as primary non-responders and 12% [n = 69/599] had severe adverse events reported including 2 deaths and 1 medulloblastoma. Withdrawal rate due to adverse events reported in one study was 35% [n = 64/182].
Conclusion:
According to low-quality evidence based mainly on case series, approximately half of children with Crohn's disease on adalimumab therapy achieve remission during the first year of the therapy with reasonable safety profile. There is still a need for high-quality evidence on effectiveness and safety of adalimumab for paediatric Crohn's disease.
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