182. The Use of Emerging Biological Treatments in Children

  1. Alan D. Irvine MD, FRCPI, FRCP2,3,
  2. Peter H. Hoeger MD4,5 and
  3. Albert C. Yan MD, FAAP, FAAD6,7
  1. Polly Livermore MSc, ANP V300 Nurse Prescriber, BSc, RN child and
  2. Clarissa Pilkington MBBS, BSc, MRCP(paeds)

Published Online: 24 MAY 2011

DOI: 10.1002/9781444345384.ch182

Harper's Textbook of Pediatric Dermatology, Volume 1, 2, Third Edition

Harper's Textbook of Pediatric Dermatology, Volume 1, 2, Third Edition

How to Cite

Livermore, P. and Pilkington, C. (2011) The Use of Emerging Biological Treatments in Children, in Harper's Textbook of Pediatric Dermatology, Volume 1, 2, Third Edition (eds A. D. Irvine, P. H. Hoeger and A. C. Yan), Wiley-Blackwell, Oxford, UK. doi: 10.1002/9781444345384.ch182

Editor Information

  1. 2

    Trinity College, Dublin, Ireland

  2. 3

    Our Lady's Children's Hospital, Dublin, Ireland

  3. 4

    University of Hamburg, Hamburg, Germany

  4. 5

    Catholic Children's Hospital Wilhelmstift, Hamburg, Germany

  5. 6

    University of Pennsylvania School of Medicine, Philadelphia, PA, USA

  6. 7

    The Children's Hospital of Philadelphia, Philadelphia, PA, USA

Author Information

  1. Paediatric Rheumatology, Great Ormond Street Hospital for Children, NHS Trust, London, UK

Publication History

  1. Published Online: 24 MAY 2011
  2. Published Print: 3 JUN 2011

ISBN Information

Print ISBN: 9781405176958

Online ISBN: 9781444345384

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Keywords:

  • Biological;
  • clinical trials;
  • etanercept;
  • infliximab;
  • adalimimab;
  • rituximab;
  • abatacept;
  • juvenile arthritis;
  • psoriasis;
  • alefacept;
  • ustekinumab;
  • efalizumab;
  • registries

Summary

Biological treatments are relatively new treatments that target specific molecules within the immune system. They can be powerful tools in treating chronic diseases that are resistant to conventional treatments. However, they need to be monitored for their side-effects as their long-term effects on the developing immune system are not yet known. Drug trials are few in children, and these need to be undertaken as the pharmacokinetics and side-effect profile is often different in children. The difficulties in conducting clinical trials in children are well known, including the much smaller number of patients available compared to adults. The clinical trials that have been undertaken have had to be multicentred and international in order to recruit the numbers needed. The published trials involving paediatric cases have been mainly in rheumatological diseases (e.g. etanercept, infliximab, adalimimab and abatacept in juvenile arthritis). These are reviewed, as well as relevant adult clinical trials for treatments that have been used in dermatological conditions (such as psoriasis) rather than rheumatological conditions: alefacept, ustekinumab and efalizumab. Their mechanisms of action are described, as well as the administration and side-effect profile.

As these are relatively new drugs that are costly, they are often seen by parents, patients and the health professionals as highly beneficial. Managing the expectations raised by these newer treatments is discussed, especially in the context of cases where treatment failures occur. This is only one aspect that highlights the need for these patients to be looked after by experienced multidisciplinary teams who can also ensure these patients are entered into registries for long-term follow-up to ensure that long-term sequelae can be documented and studied.