{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,15]],"date-time":"2026-09-15T18:59:15Z","timestamp":1789498755154,"version":"build-2803163510"},"reference-count":17,"publisher":"Oxford University Press (OUP)","issue":"1","license":[{"start":{"date-parts":[[2024,10,29]],"date-time":"2024-10-29T00:00:00Z","timestamp":1730160000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2025,1,17]]},"abstract":"<jats:title>Abstract<\/jats:title>\n               <jats:p>Ceroid lipofuscinosis neuronal (CLN) encompasses rare inherited neurodegenerative disorders that present in childhood with clinical features including epilepsy, psychomotor delay, progressive vision loss, and premature death. Published experience utilizing umbilical cord blood transplant (UCBT) for these disorders is limited. This retrospective analysis includes patients with CLN (2, 3, and 5) who underwent UCBT from 2012 to 2020. All subjects (n\u2005=\u20058) received standard-of-care myeloablative conditioning. Four also enrolled in clinical trial NCT02254863 and received intrathecal DUOC-01 cells posttransplant. Median age at UCBT was 5.9 years. All subjects achieved neutrophil engraftment with &amp;gt;95% donor chimerism at a median of 28.5 days. Sinusoidal obstructive syndrome was not observed. Severe acute graft-versus-host disease occurred in 12.5%. Other complications included autoimmune hemolytic anemia (25%) and viral reactivation\/infection (62.5%). No transplant-related mortality was observed. Two CLN2 patients died, 1 from progressive disease and 1 from unknown cause at days +362 and +937, respectively. With median follow-up of 8 years, overall survival at 100 days and 24 months was 100% and 88%, respectively. Three of 4 CLN3 subjects stabilized Hamburg motor and language scores. While UCBT appears safe and feasible in these patients, given the variable expression and natural history, extended follow-up and further studies are needed to elucidate the potential impact of UCBT on clinical outcomes.<\/jats:p>","DOI":"10.1093\/stcltm\/szae080","type":"journal-article","created":{"date-parts":[[2024,10,29]],"date-time":"2024-10-29T21:57:30Z","timestamp":1730239050000},"source":"Crossref","is-referenced-by-count":2,"title":["Safety and feasibility of umbilical cord blood transplantation in children with neuronal ceroid lipofuscinosis: a retrospective study"],"prefix":"10.1093","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0009-0006-4220-3123","authenticated-orcid":false,"given":"Andrea","family":"Bauchat","sequence":"first","affiliation":[{"name":"Division of Pediatric Transplant and Cellular Therapy, Duke University , 2400 Pratt Street, Box 102502, Durham, NC 27705 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Veronika","family":"Polishchuk","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology, Oncology, and Stem\u00a0Cell\u00a0Transplant, Ann & Robert H. Lurie Children\u2019s Hospital of Chicago , 225 E Chicago Ave, Chicago, IL 60611 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vanessa A","family":"Fabrizio","sequence":"additional","affiliation":[{"name":"Colorado Children\u2019s Hospital, Anschutz Medical Campus, University of Colorado , 13123 E 16th Ave,\u00a0Aurora, CO 80045 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer E","family":"Brondon","sequence":"additional","affiliation":[{"name":"Fortrea Inc. , 8 Moore Dr,\u00a0Durham, NC 27703 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kristin M","family":"Page","sequence":"additional","affiliation":[{"name":"Department of Pediatrics and Medicine, Medical College of Wisconsin , 8915 W Connell Ct, Milwaukee, WI 53226 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy A","family":"Driscoll","sequence":"additional","affiliation":[{"name":"Division of Pediatric Transplant and Cellular Therapy, Duke University , 2400 Pratt Street, Box 102502, Durham, NC 27705 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul L","family":"Martin","sequence":"additional","affiliation":[{"name":"Division of Pediatric Transplant and Cellular Therapy, Duke University , 2400 Pratt Street, Box 102502, Durham, NC 27705 ,","place":["United States"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kris M","family":"Mahadeo","sequence":"additional","affiliation":[{"name":"Division of Pediatric Transplant and Cellular Therapy, Duke University , 2400 Pratt Street, Box 102502, Durham, NC 27705 ,","place":["United 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