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Clinical Trials (Clinician Perspective)

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Pediatric Inflammatory Bowel Disease

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Abstract

Pediatric patients with IBD are increasingly candidates for future clinical trials. Until now, very few RCTs have been conducted in pediatric populations with IBD. The main challenge will be the use of new therapies for treating active disease as well as for inducing and maintaining remission. However, accurate choice of the patient phenotype to be enrolled in the clinical trials is crucial for reaching both meaningful results and conclusions. Due to the heterogeneous nature of IBD, homogeneous populations of patients should be studied and compared even if this could prolong patient recruitment time and make the screening process more complex.

The principal variables that should be taken into account when patients with IBD are evaluated for clinical trial are: disease type; severity; topographic distribution; complications (i.e., presence of fistulas in CD); weight and linear growth pattern; serological markers for activity, nutrition, and absorption; and genotype. The latter will be increasingly included among variables capable to influence the outcome of different treatments as more insights into genotype–phenotype correlations will be gained and other genes of susceptibility for IBD will be described.

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Author information

Authors and Affiliations

  1. Pediatric Gastroenterology and Liver Unit, Pediatric Inflammatory Bowel Disease Center, University Hospital Umberto I, Sapienza University of Rome, Viale Regina Elena 324, 00161, Rome, Italy

    Salvatore Cucchiara MD, PhD, Giovanni Di Nardo MD & Marina Aloi MD

Authors
  1. Salvatore Cucchiara MD, PhD
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  2. Giovanni Di Nardo MD
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  3. Marina Aloi MD
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Corresponding author

Correspondence to Salvatore Cucchiara MD, PhD .

Editor information

Editors and Affiliations

  1. Perelman School of Medicine, The Children’s Hospital of Philadelphia, University of Pennsylvania, 34th St. & Civic Ctr. Blvd., Philadelphia, 19104, Pennsylvania, USA

    Petar Mamula

  2. School of Medicine--Greenville, Pediatric Gastroenterology & Nutrition, University of South Carolina, 200 Patewood Drive, Suite A-140, Greenville, 29615, South Carolina, USA

    Jonathan E. Markowitz

  3. Perelman School of Medicine, The Children's Hospital of Philadelphia, University of Pennsylvania, 34th St. & Civic Ctr. Blvd., Philadelphia, 19104, Pennsylvania, USA

    Robert N. Baldassano

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Cucchiara, S., Di Nardo, G., Aloi, M. (2013). Clinical Trials (Clinician Perspective). In: Mamula, P., Markowitz, J., Baldassano, R. (eds) Pediatric Inflammatory Bowel Disease. Springer, New York, NY. https://doi.org/10.1007/978-1-4614-5061-0_42

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  • DOI: https://doi.org/10.1007/978-1-4614-5061-0_42

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  • Publisher Name: Springer, New York, NY

  • Print ISBN: 978-1-4614-5060-3

  • Online ISBN: 978-1-4614-5061-0

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