Safety updates in novel therapeutics for pediatric rheumatic disease.

Journal Article (Journal Article)

Purpose of review

Biologics and novel targeted therapeutics have transformed the management of pediatric rheumatic diseases over the past two decades; however, questions about short-term and long-term safety remain. Safety data gathered from recent clinical trials, long-term extensions of prior trials, registries, and other real-world evidence are summarized here for biologics and novel therapeutics commonly prescribed for pediatric rheumatic diseases.

Recent findings

With nearly 20 years of therapeutic experience, tumor necrosis inhibitors (TNFi) are generally well tolerated, although infections, malignancy, and development of new autoimmunity remain a concern. Risk of infections may be higher in IL-1 and IL-6 inhibitors, and lower in abatacept, compared with TNFi. Safety data for B-cell-targeted therapeutics and janus kinase inhibitors are emerging, but remain limited, especially in children.


Biologic and novel targeted therapeutics offer a promising future for children with pediatric rheumatic disease. However, long-term safety data in children remain limited for several agents. With any therapeutic option, both short-term and long-term safety concerns must be weighed against individual clinical needs when choosing the optimal treatment for each child.

Full Text

Duke Authors

Cited Authors

  • Randell, RL; Becker, ML

Published Date

  • September 2021

Published In

Volume / Issue

  • 33 / 5

Start / End Page

  • 403 - 408

PubMed ID

  • 34397604

Electronic International Standard Serial Number (EISSN)

  • 1531-6963

International Standard Serial Number (ISSN)

  • 1040-8711

Digital Object Identifier (DOI)

  • 10.1097/bor.0000000000000811


  • eng