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Purpose: The study compares the surgical outcomes of very-early-onset ulcerative colitis (VEO-UC), which is a rare disease diagnosed in pediatric patients < 6 years, with those of older pediatric patients with ulcerative colitis (UC). Methods: A retrospective observational study of 57 pediatric patients with UC was conducted at a single center. The study compared surgical complications and postoperative growth between the two groups. Results: Out of the 57 patients, 6 had VEO-UC, and 5 of them underwent total colectomy. Compared with the surgical cases of older patients with UC (n = 6), the rate of postoperative complications in patients with VEO-UC (n = 5) was not significantly different, except for high-output ileostomy (80% vs. 0% at 3 weeks postoperatively, p = 0.02). The rate of postoperative central venous catheter (CVC) placement at > 90 days was higher in patients with VEO-UC (100% vs. 17%, p = 0.02). The median change in the Z-score of height before and 2 years after colectomy was not significantly different between VEO-UC and older patients (1.1 vs. 0.3, p = 0.13). Conclusion: With regard to complications and outcomes, total colectomy for VEO-UC patients and that for older pediatric UC patients is comparable. However, high-output ileostomy and the long duration of CVC placement may pose management challenges. [ABSTRACT FROM AUTHOR]

On February 24, 2021, the U.S. Food and Drug Administration (FDA) approved an efficacy supplement for HUMIRA® (adalimumab) injection to expand the indication of treatment of moderately to severely active ulcerative colitis (UC) to include pediatric patients 5 years of age and older. The effectiveness in pediatric patients with moderately to severely active UC was studied in a multicenter, randomized, double-blind trial (Study PUC-I, NCT02065557) in 93 pediatric patients 5 to 17 years of age. Adalimumab has been widely studied in multiple indications in adult and pediatric populations with a well-established safety profile; no apparent exposure-safety relationship has been identified in various pediatric populations treated with adalimumab across multiple indications. The approved dosing regimen in pediatric patients with UC differs from the regimen studied in the clinical trial and was determined based on a model-informed exposure bridging strategy, incorporating both efficacy and safety considerations. Specifically, the differences included switches from body weight-based (mg/kg) dosing regimens used in the pediatric trial to body weight-tiered, fixed-dose regimens, changes in dosing schedule, and the addition of an option of a less frequent dosing regimen for maintenance that was not studied in the clinical trial. This article provides a case example of successful model-informed drug development (MIDD), where modeling and simulation were utilized in combination with observed data from a clinical trial of limited size and scope to ultimately support the adalimumab approval in pediatric patients with UC. [ABSTRACT FROM AUTHOR]

5-Aminosalicylic acid preparations have been used as first-line drugs for treatment of ulcerative colitis (UC). However, some patients with UC present with exacerbation of symptoms because of allergy to mesalazine. Diagnosis of mesalazine allergy in active UC may be challenging because its symptoms mimic those of UC. Here we describe a 13-year-old boy with mesalazine allergy who achieved remission when his medication was changed from mesalazine to salazosulfapyridine. During his clinical course mesalazine was prescribed twice, and on each occasion exacerbation of the symptoms occurred. We considered a diagnosis of mesalazine allergy, and this was confirmed by a drug lymphocyte stimulation test; the result for salazosulfapyridine was negative. On the basis of criteria involving simple mucosal biopsy combined with endoscopy for predicting patients with UC who would ultimately require surgery, we considered that the UC in this case might be susceptible to steroid treatment, and we therefore treated the patient with salazosulfapyridine and prednisolone. Shortly afterwards, remission was achieved and the patient has remained in good condition on salazosulfapyridine alone. When treating patients with mesalazine, the possibility of allergy should always be borne in mind, especially when the clinical course is inconsistent with the results of biopsy. [ABSTRACT FROM AUTHOR]