Data Lacking on Biologic and JAK Inhibitor Therapies for Pediatric Rheumatology Patients

itch on child
Male doctor examining girl’s arm.
Randomized, controlled trials in pediatric inflammatory rheumatic diseases (PiRDs) of patients who underwent bDMARDs and JAK inhibitors are reviewed.

There is a compelling need for data on biologic therapies for pediatric inflammatory rheumatic diseases (PiRDs), according to a systemic review of randomized controlled trials (RCTs) of PiRDs published in Pediatric Rheumatology. The study authors noted that although adult and pediatric rheumatology diseases have several pathological and therapeutic discrepancies, pediatric treatment is generally based on existing adult data. This lack of data has also lead to numerous therapies approved for use in adults but not in children.

Investigators conduced a systemic literature search that included only RCTs of patients aged 20 years and younger treated with predefined biologic disease modifying antirheumatic drugs (bDMARDs) or Janus kinase (JAK) inhibitors, with a confirmed PiRD diagnosis, and with a sample size of at least 5 patients. Investigators analyzed baseline demographic and clinical characteristics for each RCT.

Investigators identified 35 unique RCTs that matched their inclusion criteria. Most of the RCTs (60%) were performed for TNF inhibitors, and 17% and 9% were for IL-1 inhibitors and IL-6 inhibitors, respectively. There was sparse or no data on several cytokine and JAK inhibitors for PiRDs. There were 25 RCTs conducted in juvenile idiopathic arthritis (JIA) patients and 10 RCTs in non-JIA patients, including Kawasaki disease, plaque psoriasis, systemic lupus erythematosus, and noninfectious uveitis. For the JIA RCTs, the mean age of the children ranged from 8 to 15.3 years. For the non-JIA RCTs, the mean age ranged from 2.2 to 15.2 years. The main conclusion inthe majority of the RCTS was that bDMARDs were more effective than placebo or standard of care for PiRDs.

The study was limited by not including observational studies, single-arm studies or RCTs that included adults and children, and some RCTs for PiRDs may have been missed.

“More efficacy and safety data, ideally combined with [pharmacokinetic] data from PiRD patients, will optimize bDMARDs and JAK inhibitor use in pediatric rheumatology,” the study authors wrote, which ultimately decreases disease burden and optimizes patient care in pediatric patients. They strongly advocated increasing the number of international multicenter RCTs in PiRD patients using support from research networks such as the Pediatric Rheumatology Collaborative Study Group (PRCSG), among others.


Welzel T, Winskill C, Zhang N, Woerner A, Pfister M. Biologic disease modifying antirheumatic drugs and Janus kinase inhibitors in paediatric rheumatology – what we know and what we do not know from randomized controlled trials. Pediatr Rheumatol Online J. Published online March 25, 2021. doi:10.1186/s12969-021-00514-4