Upadacitinib in pediatric IBD: 52-week results
Response in those with ulcerative colitis was especially strong.
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01/26/2026
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by Doug Brunk
Upadacitinib, a selective JAK1 inhibitor approved for adults with inflammatory bowel disease (IBD), may offer durable symptom control for children and adolescents with severe disease, according to a single-center study from Boston Children’s Hospital.
“This study matters because it provides long-term data for upadacitinib, a therapy already commonly used off-label in pediatric IBD but without established evidence for its safety and efficacy in the pediatric population,” lead study author Jennifer Bachand, MD, currently a GI fellow at Stanford University, told GI & Hepatology News.
Reporting at the 2026 Crohn’s & Colitis Congress®, a partnership between the AGA and the Crohn’s & Colitis Foundation, held in Las Vegas, Dr. Bachand, Lauren Collen, MD, and colleagues reviewed outcomes in 48 patients younger than 18 years with Crohn’s disease (CD) or ulcerative colitis (UC) who initiated upadacitinib and were followed for one year. Patients were diagnosed with CD or UC at a mean age of 10.9 years and began upadacitinib at a mean age of 14.1 years. Prior advanced therapy failures included anti–tumor necrosis factor agents (98%), ustekinumab (56%), vedolizumab (46%), tofacitinib (15%), and risankizumab (6%).
At 52 weeks, 81% of patients remained on upadacitinib, suggesting good treatment durability. Among those who continued therapy, 47% of patients with CD and 73% of those with UC achieved corticosteroid-free clinical remission. Inflammatory markers also improved, with 73% of patients demonstrating normalization of C-reactive protein levels. Endoscopic reassessment was available for just over half of the cohort, and 46% of those patients met criteria for endoscopic remission.
“We were surprised by the strong response to upadacitinib in patients who had previously failed tofacitinib, despite both drugs belonging to the same class,” Dr. Bachand said. “Six of the seven patients (86%) who had previously failed tofacitinib therapy remained on upadacitinib at 52 weeks, and four (57%) achieved corticosteroid-free remission. This suggests that failure with one JAK inhibitor does not necessarily preclude a response to upadacitinib, although this finding requires further investigation in larger cohorts.”
She added that the investigators were also surprised by the incidence of abnormal cholesterol levels, observed in 15% of patients. “More guidance is needed on the management of abnormal cholesterol in pediatric patients in remission on upadacitinib,” she said.
Only two patients discontinued treatment because of adverse events, and there were no reports of blood clots, malignancies, or deaths during follow-up. One serious infection was reported.
If larger studies confirm the safety and efficacy of upadacitinib for pediatric IBD, “it could change our treatment algorithm and ultimately lead to FDA approval for pediatric use,” Dr. Bachand concluded. “The particularly strong response in our ulcerative colitis cohort suggests it could become a preferred therapy for pediatric UC patients who have failed other advanced treatments. Larger studies are needed to confirm this difference and guide patient selection.”
The researchers reported no relevant financial disclosures.
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The guidelines emphasize four-hour gastric emptying studies over two-hour testing. How do you see this affecting diagnostic workflows in practice?
Dr. Staller: Moving to a four-hour solid-meal scintigraphy will actually simplify decision-making. The two-hour reads miss a meaningful proportion of delayed emptying; standardizing on four hours reduces false negatives and the “maybe gastroparesis” purgatory that leads to repeat testing. Practically, it means closer coordination with nuclear medicine (longer slots, consistent standardized meal), updating order sets to default to a four-hour protocol, and educating front-line teams so patients arrive appropriately prepped. The payoff is fewer equivocal studies and more confident treatment plans.
Metoclopramide and erythromycin are the only agents conditionally recommended for initial therapy. How does this align with what is being currently prescribed?
Dr. Staller: This largely mirrors real-world practice. Metoclopramide remains the only FDA-approved prokinetic for gastroparesis, and short “pulsed” erythromycin courses are familiar to many of us—recognizing tachyphylaxis limits durability. Our recommendation is “conditional” because the underlying evidence is modest and patient responses are heterogeneous, but it formalizes what many clinicians already do: start with metoclopramide (lowest effective dose, limited duration, counsel on neurologic adverse effects) and reserve erythromycin for targeted use (exacerbations, bridging).
Several agents, including domperidone and prucalopride, received recommendations against first-line use. How will that influence discussions with patients who ask about these therapies?
Dr. Staller: Two points I share with patients: evidence and access/safety. For domperidone, the data quality is mixed, and US access is through an FDA IND mechanism; you’re committing patients to EKG monitoring and a non-trivial administrative lift. For prucalopride, the gastroparesis-specific evidence isn’t strong enough yet to justify first-line use. So, our stance is not “never,” it’s just “not first.” If someone fails or cannot tolerate initial therapy, we can revisit these options through shared decision-making, setting expectations about benefit, monitoring, and off-label use. The guideline language helps clinicians have a transparent, evidence-based conversation at the first visit.
The guidelines suggest reserving procedures like G-POEM and gastric electrical stimulation for refractory cases. In your practice, how do you decide when a patient is “refractory” to medical therapy?
Dr. Staller: I define “refractory” with three anchors.
1. Adequate trials of foundational care: dietary optimization and glycemic control; an antiemetic; and at least one prokinetic at appropriate dose/duration (with intolerance documented if stopped early).
2. Persistent, function-limiting symptoms: ongoing nausea/vomiting, weight loss, dehydration, ER visits/hospitalizations, or malnutrition despite the above—ideally tracked with a validated instrument (e.g., GCSI) plus nutritional metrics.
3. Objective correlation: delayed emptying on a standardized 4-hour solid-meal study that aligns with the clinical picture (and medications that slow emptying addressed).
At that point, referral to a center with procedural expertise for G-POEM or consideration of gastric electrical stimulation becomes appropriate, with multidisciplinary evaluation (GI, nutrition, psychology, and, when needed, surgery).
What role do you see dietary modification and glycemic control playing alongside pharmacologic therapy in light of these recommendations?
Dr. Staller: They’re the bedrock. A small-particle, lower-fat, calorie-dense diet—often leaning on nutrient-rich liquids—can meaningfully reduce symptom burden. Partnering with dietitians early pays dividends. For diabetes, tighter glycemic control can improve gastric emptying and symptoms; I explicitly review medications that can slow emptying (e.g., opioids; consider timing/necessity of GLP-1 receptor agonists) and encourage continuous glucose monitor-informed adjustments. Pharmacotherapy sits on top of those pillars; without them, medications will likely underperform.
The guideline notes “considerable unmet need” in gastroparesis treatment. Where do you think future therapies or research are most urgently needed?
Dr. Staller: I see three major areas.
1. Truly durable prokinetics: agents that improve emptying and symptoms over months, with better safety than legacy options (e.g., next-gen motilin/ghrelin agonists, better-studied 5-HT4 strategies).
2. Endotyping and biomarkers: we need to stop treating all gastroparesis as one disease. Clinical, physiologic, and microbiome/omic signatures that predict who benefits from which therapy (drug vs G-POEM vs GES) would transform care.
3. Patient-centered trials: larger, longer RCTs that prioritize validated symptom and quality-of-life outcomes, include nutritional endpoints, and reflect real-world medication confounders.
Our guideline intentionally highlights these gaps to hopefully catalyze better trials and smarter referral pathways.
Dr. Staller is with the Division of Gastroenterology, Massachusetts General Hospital and Harvard Medical School, Boston.