Browsing by Subject "pediatric patients"

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    Cytomegalovirus induced hemophagocytic lymphocytic histiocytosis in two pediatric patients with acute lymphoblastic leukemia
    (Elsevier, 2017-07-22) Waddell, Benjamin; Belcher, Chris; Willey, Emily; Medicine, School of Medicine
    Hemophagocytic lymphohistiocytosis (HLH) is a rare inflammatory condition with tissue destruction due to abnormal immune activation. We present a series of 2 cases of cytomegalovirus-induced HLH in children during maintenance chemotherapy for acute lymphoblastic leukemia. These cases emphasize the importance of considering secondary HLH in this high-risk subset of pediatric patients.
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    Radiation-Induced Large Vessel Cerebral Vasculopathy in Pediatric Patients with Brain Tumors Treated with Proton Radiotherapy
    (Elsevier, 2017) Kralik, Stephen F.; Shih, Chie-schin; Ho, Chang Y.; Finke, Whitney; Buchsbaum, Jeffrey; Watson, Gordon A.; Department of Radiology and Imaging Sciences, School of Medicine
    Purpose The purpose of this research is to evaluate the incidence, time to development, imaging patterns, risk factors, and clinical significance of large vessel cerebral vasculopathy in pediatric patients with brain tumors treated with proton radiotherapy. Materials and Methods A retrospective study was performed on 75 consecutive pediatric patients with primary brain tumors treated with proton radiotherapy. Radiation-induced large vessel cerebral vasculopathy (RLVCV) was defined as intracranial large vessel arterial stenosis or occlusion confirmed on MRA, CTA, and/or catheter angiography within an anatomic region with previous exposure to proton beam therapy and not present prior to radiotherapy. Clinical records were used to determine the incidence, timing, radiation dose to the large vessels, and clinical significance associated with the development of large vessel vasculopathy in these patients. Results RLVCV was present in 5/75 (6.7%) of patients and included tumor pathologies of craniopharyngioma (2), ATRT (1), medulloblastoma (1), and anaplastic astrocytoma (1). Median time from completion of radiotherapy to development was 1.5 years (mean 3.0 years; range 1.0-7.5 years). Neither mean age at time of radiotherapy (5.1 years) nor mean radiotherapy dose to the large vessels (54.5 Gy) were statistically significant risk factors. Four of the five patients with RLVCV presented with acute stroke, and demonstrated MRI evidence of acute infarcts in the expected vascular distributions. Angiography studies demonstrated collateral vessel formation in only two of the patients with RLVCV. No patients demonstrated acute hemorrhage or aneurysm. Two patients were treated with pial synangiomatosis surgery. Conclusion RLVCV can occur in pediatric patients with brain tumors treated with proton radiotherapy. Further studies are necessary to determine potential risk factors for large vessel vasculopathy with proton radiotherapy in comparison with conventional photon radiotherapy.
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    Safety Findings in Pediatric Patients During Long-Term Treatment With Teduglutide for Short-Bowel Syndrome–Associated Intestinal Failure: Pooled Analysis of 4 Clinical Studies
    (Wiley, 2021-09) Hill, Susan; Carter, Beth A.; Cohran, Valeria; Horslen, Simon; Kaufman, Stuart S.; Kocoshis, Samuel A.; Mercer, David F.; Merritt, Russell J.; Pakarinen, Mikko P.; Protheroe, Susan; Thompson, John F.; Vanderpool, Charles P. B.; Venick, Robert S.; Wales, Paul W.; Smith, Sharon E.; Yoon, MinJung; Grimm, Andrew A.; Pediatrics, School of Medicine
    Background This analysis assessed combined safety data from 4 clinical studies of teduglutide in pediatric patients with short-bowel syndrome–associated intestinal failure (SBS–IF). Methods Safety data from teduglutide-treated patients in 4 clinical trials were pooled. The completed 12-week and 24-week phase 3 core studies (NCT01952080/EudraCT 2013-004588-30 and NCT02682381/EudraCT 2015-002252-27) enrolled children aged 1–17 years with SBS–IF. Patients could elect to enroll in ongoing open-label extensions (NCT02949362/EudraCT 2016-000863-17 and NCT02954458/EudraCT 2016-000849-30). Interim data from ongoing studies were included. Results Safety data are reported for 89 pediatric patients treated with teduglutide for a median (range) of 51.7 (5.0–94.7) weeks. Adverse events (AEs) were reported in all patients; the most common were vomiting (51.7%), pyrexia (43.8%), upper respiratory tract infection (41.6%), and cough (33.7%). Thirty-five patients (39.3%) had AEs considered related to teduglutide treatment; abdominal pain and vomiting were most frequent (5.6% each). Three serious AEs in 3 patients (3.4%) were considered related to teduglutide treatment: ileus, d-lactic acidosis, and gastrointestinal obstruction due to hard stools. All 3 events resolved. One cecal polyp was detected, which was not biopsied or found on repeat colonoscopy. No cases of neoplasia occurred. Conclusion Based on integrated data from 4 clinical studies, including long-term follow-up for ≤161 weeks, teduglutide had a safety profile consistent with the individual core pediatric studies and as expected for pediatric patients with SBS–IF who never received teduglutide. The most frequent AEs reflected treatment with teduglutide, complications of the underlying disease, and typical childhood illnesses.