The safe and effective use of tofacitinib and ustekinumab combination therapy in infantile onset inflammatory bowel disease.

Abstract

Infantile-onset inflammatory bowel disease (IOIBD) is a rare and severe subset of very-early-onset IBD, often associated with immune dysregulation and poor response to conventional therapies. Data regarding the use of Janus kinase inhibitors (JAKI) in this population is limited. We conducted a retrospective chart review of four patients treated with tofacitinib in combination with ustekinumab after failing multiple agents at a tertiary referral center. All four patients demonstrated clinical, laboratory, and endoscopic remission. No adverse events were observed. Genetic evaluation did not reveal monogenic causes in any of these patients. These findings suggest that JAKI may represent a safe and effective therapeutic option for carefully selected patients with difficult-to-treat IOIBD, either as a bridge to or in combination with ustekinumab. Larger prospective studies are warranted to better define the role of JAKI in this challenging patient population.