{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,19]],"date-time":"2026-08-19T19:59:42Z","timestamp":1787169582396,"version":"build-2736575974"},"reference-count":20,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2024,6,20]],"date-time":"2024-06-20T00:00:00Z","timestamp":1718841600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J. pediatr. gastroenterol. nutr."],"published-print":{"date-parts":[[2024,10]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:p>\n                    This study analyzed qualitative and quantitative survey responses from 51 pediatric primary sclerosing cholangitis (PSC) patients and caregivers using the PSC Partners Patient Registry\u2013\n                    <jats:italic>Our Voices<\/jats:italic>\n                    survey. The most common symptoms reported by children\/caregivers include: fatigue (71%), abdominal pain (69%), anxiety (59%), appetite loss (51%), insomnia (49%), and pruritus (45%). When experiencing symptoms at their worst, over half of patients\/caregivers reported limitations in physically demanding activities (67%), work\/school duties (63%), social life activities (55%), and activities for fun or exercise (53%). Over half of patients\/caregivers expressed willingness to participate in clinical trials, however none reported ever participating in trials for new or investigational PSC drugs. This study revealed a substantial patient\/caregiver\u2010reported symptom burden for children with PSC that impacts quality of life and limits access to clinical trials. Future efforts should focus on developing patient\u2010centered clinical endpoints for PSC trials, increasing trial availability for pediatric PSC patients, and reducing logistical barriers to trial involvement.\n                  <\/jats:p>","DOI":"10.1002\/jpn3.12287","type":"journal-article","created":{"date-parts":[[2024,6,20]],"date-time":"2024-06-20T06:29:59Z","timestamp":1718864999000},"page":"835-840","source":"Crossref","is-referenced-by-count":1,"title":["Caregiver\u2010reported symptom\u00a0burden and preferences for therapeutic goals in pediatric primary sclerosing cholangitis"],"prefix":"10.1002","volume":"79","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4101-7055","authenticated-orcid":false,"given":"Holly Payton","family":"Shifman","sequence":"first","affiliation":[{"name":"Department of Pediatrics Boston Children's Hospital, Harvard Medical School Boston Massachusetts USA"},{"name":"Department of Pediatrics, Boston Medical Center Boston University School of Medicine Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joanne","family":"Hatchett","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure Greenwood Village Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruth\u2010Anne","family":"Pai","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure Greenwood Village Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ricky","family":"Safer","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure Greenwood Village Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel","family":"Gomel","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure Greenwood Village Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mary","family":"Vyas","sequence":"additional","affiliation":[{"name":"PSC Partners Seeking a Cure Greenwood Village Colorado USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Medicine University of California, San Francisco San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer C.","family":"Lai","sequence":"additional","affiliation":[{"name":"Department of Medicine University of California, San Francisco San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5565-6670","authenticated-orcid":false,"given":"Sharad I.","family":"Wadhwani","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, University of California San Francisco San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,6,20]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.livres.2017.12.002"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cld.2022.03.009"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1572-0241.2007.01103.x"},{"key":"e_1_2_8_5_1","doi-asserted-by":"publisher","DOI":"10.1186\/s12955-021-01739-3"},{"key":"e_1_2_8_6_1","doi-asserted-by":"publisher","DOI":"10.1186\/s41687-022-00484-5"},{"key":"e_1_2_8_7_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.cgh.2018.04.047"},{"key":"e_1_2_8_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMra1506330"},{"key":"e_1_2_8_9_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.pcl.2021.07.006"},{"key":"e_1_2_8_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/apt.13894"},{"key":"e_1_2_8_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/hep.23678"},{"key":"e_1_2_8_12_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.gastro.2017.02.038"},{"key":"e_1_2_8_13_1","unstructured":"PSC Partners Seeking a Cure. Patient registry.2023. Accessed January 20 2023.https:\/\/pscpartners.org\/about\/participate\/patient-registry.html"},{"key":"e_1_2_8_14_1","unstructured":"U.S. Food and Drug Administration. Condition\u2010specific meeting reports and other information related to patients' experience. 2023. Accessed September 1 2023.https:\/\/www.fda.gov\/industry\/prescription-drug-user-fee-amendments\/condition-specific-meeting-reports-and-other-information-related-patients-experience"},{"key":"e_1_2_8_15_1","doi-asserted-by":"publisher","DOI":"10.1177\/2050312118822927"},{"key":"e_1_2_8_16_1","unstructured":"ClinicalTrials.gov. 2024. 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