Background: The treatment effect of ustekinumab for Crohn disease (CD) that is refractory to tumor necrosis factor (TNF)-α blockers has been approved in adults, but the indication for pediatric cases remains elusive. The present study aimed to assess the applicability of ustekinumab therapy for biologic naïve pediatric patients.
Methods: Four patients who developed CD until 15 years of age and then came to receive ustekinumab as the first-line biologic therapy were enrolled. Clinical and laboratory findings, treatment history, outcomes and adverse events of the biologics were retrospectively studied by using the medical records and interviews to attending doctors. To evaluate the inflammatory profiles at the time of starting ustekinumab, serum concentrations of interleukin (IL)-12/IL-23p40, TNF-α, IL-1β, IL-6, IL-8 and IL-10 were measured by the enzyme-linked immunosorbent or immunobead assays.
Results: All patients underwent ustekinumab therapy because of the refractory or relapsing disease on conventional therapy and the individual reasons including optic neuritis. Three out of four patients obtained complete remission followed by successful maintenance, although one failed. No adverse events occurred at all. Pretreatment IL-12/IL-23p40 levels of all three responders, but not one non-responder or healthy controls, exceeded 80 pg/ml. On the other hand, the other cytokine levels of all patients were indifferently negligible.
Conclusions: Ustekinumab was effectively used as one of the first-line biologics for the treatment of pediatric-onset CD. The limited indication to anti-TNF therapies or the elevated levels of serum IL-12/IL-23p40 may be a preferable choice of the treatment in childhood.