Ustekinumab in Pediatric Clinical Practice

A recent study (JR Dayan et al. JPGN 2019; 69: 61-67) provides some helpful insight into the use of ustekinumab.

Background: The authors conducted a retrospective review of 52 patients (73% younger than 18 years, 27% 18-21 years).

  • Median age at induction was 16.8 years.
  • 10 patients were biologic-naive; 42 had received at least one anti-TNF agent (18 had received two anti-TNFs).
  • 42 of the 52 patients had Crohn’s disease.
  • Of note, 64% of their patients had a normal baseline CRP and they defined “biomarker remission at 52 weeks” as having a normal CRP.  The high rate of normal baseline CRP likely indicates milder disease than many other refractory populations; though nearly half of the patients with Crohn’s disease were receiving steroids when ustekinumab was initiated.
  • Steroid-free remission was defined by Harvey Bradshaw Index ≤4 or partial Mayo Score <2 and off steroids for >4 weeks.

Dosing: 47 (90%) received induction with ustekinumab IV (260 mg if <55 kg, 390 mg if 55-85 kg, 520 mg if >85 kg) followed by 90 mg subcutaneous injections every 8 weeks

Key findings:

  • 75% of patients continued to receive ustekinumab at 52 weeks.
  • 50% of bio-exposed patients were in steroid-free remission
  • 90% of bio-naive were in steroid-free remission
  • 57% received a dose escalation (increased frequency due to inadequate clinical response); such that at 52 weeks, 12 were receiving q4 weeks, 9 were receiving q6-7 weeks, and 15 continued with q8 weeks.
  • With a median f/u of 18 months, the authors reported few serious adverse events: two patients had an anaphylactoid reaction with IV induction (Rx with steroids and epinephrine). One of these two went on to experience arthralgias, fatigue and headaches with maintenance injection and treatment was discontinued. One patient experienced “self-limited paresthesia of bilateral lower extremities at 16 months on therapy” (CHOP experience with 22 patients reported one case of transverse myelitis: #NASPGHAN17 More Abstracts)


  • The authors note low immunogeiecity of ustekinumab and “suggest that ustekinumab monotherapy is possible and preferable in children”
  • Limitations: Lack of better objective markers for response to treatment

My take: This data indicates that ustekinumab therapy was associated with clinical remission in 50% of patients who had received anti-TNF therapy and had higher response in a small sample of biologically-naive patients.  More experience is needed to confirm drug safety with long-term usage

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