Browsing by Author "Vanderpool, Charles"

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    Outcomes from a 12-Week, Open-Label, Multicenter Clinical Trial of Teduglutide in Pediatric Short Bowel Syndrome
    (Elsevier, 2017-02) Carter, Beth A.; Cohran, Valeria C.; Cole, Conrad R.; Corkins, Mark R.; Dimmitt, Reed A.; Duggan, Christopher; Hill, Susan; Horslen, Simon; Lim, Joel D.; Mercer, David F.; Merritt, Russell J.; Nichol, Peter F.; Sigurdsson, Luther; Teitelbaum, Daniel H.; Thompson, John; Vanderpool, Charles; Vaughan, Juliana F.; Li, Benjamin; Youssef, Nader N.; Venick, Robert S.; Venick, Robert S.; Kocoshis, Samuel A.; Department of Pediatrics, School of Medicine
    Objective To determine safety and pharmacodynamics/efficacy of teduglutide in children with intestinal failure associated with short bowel syndrome (SBS-IF). Study design This 12-week, open-label study enrolled patients aged 1-17 years with SBS-IF who required parenteral nutrition (PN) and showed minimal or no advance in enteral nutrition (EN) feeds. Patients enrolled sequentially into 3 teduglutide cohorts (0.0125 mg/kg/d [n = 8], 0.025 mg/kg/d [n = 14], 0.05 mg/kg/d [n = 15]) or received standard of care (SOC, n = 5). Descriptive summary statistics were used. Results All patients experienced ≥1 treatment-emergent adverse event; most were mild or moderate. No serious teduglutide-related treatment-emergent adverse events occurred. Between baseline and week 12, prescribed PN volume and calories (kcal/kg/d) changed by a median of −41% and −45%, respectively, with 0.025 mg/kg/d teduglutide and by −25% and −52% with 0.05 mg/kg/d teduglutide. In contrast, PN volume and calories changed by 0% and −6%, respectively, with 0.0125 mg/kg/d teduglutide and by 0% and −1% with SOC. Per patient diary data, EN volume increased by a median of 22%, 32%, and 40% in the 0.0125, 0.025, and 0.05 mg/kg/d cohorts, respectively, and by 11% with SOC. Four patients achieved independence from PN, 3 in the 0.05 mg/kg/d cohort and 1 in the 0.025 mg/kg/d cohort. Study limitations included its short-term, open-label design, and small sample size. Conclusions Teduglutide was well tolerated in pediatric patients with SBS-IF. Teduglutide 0.025 or 0.05 mg/kg/d was associated with trends toward reductions in PN requirements and advancements in EN feeding in children with SBS-IF.
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    Varied Presentations and Comorbidities in Pediatric Autoimmune Pancreatitis
    (Wolters Kluwer, 2021) Suter, Blair; Patel, Feenalie; Holland, Kathleen; Brown, Brandon P.; Bhatt, Heli; Puri, Kanika; McFerron, Brian; Vanderpool, Charles; Pediatrics, School of Medicine
    Autoimmune pancreatitis (AIP) is a chronic inflammatory condition rarely reported in children. In 2018, to standardize the approach to AIP, INternational Study Group of Pediatric Pancreatitis: In search for a cuRE (INSPPIRE) defined AIP, outlined the clinical course, and developed diagnostic and therapeutic recommendations. We performed a retrospective review of cases at our institution from January 1, 2016, to June 1, 2019, and compared their presentations with the INSPPIRE guidelines. Our patients showed variable laboratory, radiographic, and histologic findings, highlighting the difficulty in diagnosing AIP. Histologic samples were obtained in our patients due to diagnostic uncertainty, which ultimately confirmed the diagnosis. One patient was diagnosed with autoimmune hepatitis coexistent with AIP, which has not been previously described in the pediatric population. Exocrine and endocrine complications of AIP were also noted. In all cases, symptoms improved following treatment, and decompression of the common bile duct was seen on repeat imaging. Autoimmune pancreatitis (AIP) is a chronic inflammatory condition rarely reported in children. Cases of AIP at a single academic center were reviewed from January 01, 2016, to June 01, 2019. The aim of this report is to describe differences in presentation and diagnostic considerations of pediatric AIP while illustrating comorbid conditions that can occur.