Tofacitinib in Pediatric Ulcerative Colitis: A Retrospective Multicenter Experience.

BACKGROUND: Tofacitinib has recently been approved for treatment of moderate-to-severe ulcerative colitis (UC) in adults, yet pediatric data are limited. This international multicenter study describes the effectiveness and safety of tofacitinib in pediatric UC. METHODS: This is a retrospective review of children diagnosed with UC treated with tofacitinib from 16 pediatric centers internationally. The primary outcome was week 8 corticosteroid-free clinical remission (Pediatric Ulcerative Colitis Activity Index <10). Secondary outcomes were clinical response (≥20-point decrease in Pediatric Ulcerative Colitis Activity Index) at week 8, corticosteroid-free clinical remission at week 24, and colectomy rate and adverse safety events through to last follow-up. The primary outcome was calculated by the intention-to-treat principle. RESULTS: We included 101 children with a mean age at diagnosis of 12.8 ±â€…2.8 years and a median disease duration of 20 months (interquartile range [IQR], 10-39 months). All had treatment failure with at least 1 biologic agent, and 36 (36%) had treatment failure with 3 agents. Median follow-up was 24 weeks (IQR, 16-54 weeks). Sixteen (16%) children achieved corticosteroid-free clinical remission at week 8, and an additional 30 (30%) demonstrated clinical response. Twenty (23%) of 88 children achieved corticosteroid-free clinical remission at week 24. A total of 25 (25%) children underwent colectomy by median 86 days (IQR, 36-130 days). No serious drug-related adverse events were reported; there was 1 case of herpes zoster and 2 cases of minor blood test perturbations. CONCLUSIONS: In this largest real-life pediatric cohort to date, tofacitinib was effective in at least 16% of patients with highly refractory UC by week 8. Adverse events were minor and largely consistent with adult data.

Tofacitinib, widely reported in adult ulcerative colitis, has very limited pediatric data. This international collaboration is the largest pediatric study on the efficacy and safety of tofacitinib to date, providing important supportive data to clinicians and regulators.

Growth hormone use in pediatric inflammatory bowel disease.

OBJECTIVES: Impaired linear growth is a known complication of pediatric inflammatory bowel disease (IBD), but the use of growth hormone (GH) in this population is not well-described. The primary aim of this study is to determine whether growth hormone use in pediatric IBD leads to improved height outcomes. METHODS: This was a retrospective chart review of patients with IBD aged 0-21 years followed at a single center between 2018 and 2021 treated with at least 1 year of GH. Records collected included demographics, IBD phenotype, IBD disease activity scores, medications, weight z-score, height z-score, bone age, and details of GH therapy including testing for GH deficiency. The primary outcome measure was change in height z-score after 1 year of GH treatment. RESULTS: Forty-six patients were identified and 18 were excluded. Of the 28 patients included (7 female; 25.0 %), 26 (92.9 %) had a diagnosis of Crohn's disease (CD) and 2 (7.1 %) had ulcerative colitis (UC). The mean (SD) age at GH initiation was 9.6 (3.4) years. Among all participants, there was a significant mean difference in height z-score from baseline to 1 year on therapy (-2.25 vs. -1.50, respectively; difference, 0.75; 95 % CI, 0.56 to 0.94; p<0.001). Among the 19 subjects that completed GH therapy there was a significant mean difference between baseline and final height z-scores (-2.41 vs. -0.77, respectively; difference, 1.64; 95 % CI, 1.30 to 1.98; p<0.001). CONCLUSIONS: GH use was associated with improved height outcomes. The pediatric IBD patients in this cohort had significant improvements in height z-scores both after one year on therapy and at completion of GH therapy.

Righting the Autonomy-Supervision Pendulum: Understanding the Impact of Independent Rounds on Medical Students, Residents, and Faculty.

PURPOSE: To explore trainee and faculty perspectives on an independent rounding intervention on general pediatrics wards at 2 institutions. METHOD: In July 2018, the authors introduced independent rounds 1 to 2 times a week at 2 training sites. In this qualitative study, the authors conducted semistructured focus groups with a purposive sample of junior trainees (clerkship medical students and postgraduate year [PGY] 1 residents), senior trainees (PGY-2 and PGY 3-5 residents), and hospital medicine faculty between October 2018 and May 2019. Focus groups were audio-recorded, transcribed verbatim, and analyzed for themes using the constant comparative approach associated with grounded theory. RESULTS: Focus groups included 27 junior trainees, 20 senior trainees, and 18 faculty. Six themes emerged: (1) Independent rounds contributed to all trainees' development; (2) Senior residents described increased motivation to take full ownership of their patients and educational needs of the team; (3) Faculty expressed concerns about decreased opportunities for teaching and feedback; however, all trainees reported unique learning from having faculty both present and absent from rounds; (4) No significant patient safety events were reported; (5) All participants identified communication and patient progression concerns; and (6) A tension emerged between decreased faculty and enhanced trainee career satisfaction. Participants identified solutions to identified barriers to further improve this educational intervention. CONCLUSIONS: As a result of independent rounding, trainees described increased motivation to take ownership of their patients and team. Both rounding experiences contributed to their development as physicians in different ways. Further studies should explore patient and caregiver perspectives and concerns about communication and patient care progression when designing future interventions to promote resident autonomy.

Yoga as adjunct therapy for adolescents with inflammatory bowel disease: A pilot clinical trial.

OBJECTIVES: To determine whether utilizing yoga as an adjunctive therapy to the medical standard of care for adolescents with inflammatory bowel disease (IBD) is: (1) feasible and acceptable, (2) effective in reducing disease severity, intestinal inflammation and improving wellness. DESIGN: Prospective, non-randomized, 8-week pilot study for adolescents with a diagnosis of IBD. Feasibility and acceptability of the intervention were assessed weekly and post-intervention via surveys and a focus group (week 8). Disease severity, intestinal inflammation, and wellness measures were assessed at baseline and post-intervention (week 8). INTERVENTION: Over the 8-week study period, patients were assigned three 60-minute, in-person yoga classes at weeks 1, 3 and 8, and three 30-minute, online yoga videos per week. MAIN OUTCOME MEASURES: Primary outcome measures were feasibility and acceptability. Secondary outcome measures assessed preliminary clinical efficacy by examining pre- and post-intervention change in disease severity (PUCAI), intestinal inflammation (fecal calprotectin), and six wellness measures (PROMIS-37). RESULTS: Nine adolescents with IBD participated. Eight participated in one or more yoga videos per week and all nine attended at least two in-person yoga classes. Focus group themes revealed that the intervention was well liked, with all participants reporting reduced stress, improved emotional self-awareness, and increased ability to identify and manage the physical symptoms of IBD. Participants had difficulty, however, completing the yoga videos due to time limitations and competing priorities. We lacked power to detect any statistically significant changes in PUCAI, calprotectin, or any of the six PROMIS-37 domains. CONCLUSIONS: A combination of in-person instructor led yoga with video-based yoga is a feasible and acceptable adjunct therapy for adolescents with IBD. Participants reported reduced stress and improved ability to identify and manage physical symptoms. A larger, randomized controlled trial is necessary to determine if the yoga protocol results in clinically and statistically significant improvements in inflammatory biomarkers and patient reported outcomes.