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The MTD was defined as zero to one of six patients with dose-limiting toxicity (DLT), with at least two of three or two of six DLT at next higher dose.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifty-four patients, age 2 years to 20 years (median, 9 years), were treated: neuroblastoma (n = 39), Ewing sarcoma (n = 5), and other (n = 10). Prior therapy included autologous stem cell transplantation (n = 42), 13-cis-RA (n = 35), and 9-cis-RA (n = 1). One of four patients at 1,050 mg\/m<jats:sup>2<\/jats:sup>with prior liver transplant had grade 3 ALT\/abdominal pain\/nausea\/dehydration and grade 4 AST\/emesis. At 1,860 mg\/m<jats:sup>2<\/jats:sup>, one of seven patients had grade 3 hypoalbuminemia\/hypophosphatemia. At 2,475 mg\/m<jats:sup>2<\/jats:sup>, one of eight patients had grade 3 alkaline phosphatase; three of five patients had DLT at 3,300 mg\/m<jats:sup>2<\/jats:sup>: grade 3 AST\/ALT (n = 1), grade 4 bilirubin\/grade 3 AST\/ALT (n = 1), pseudotumor cerebri (n = 1). Pseudotumor cerebri also occurred at 600 mg\/m<jats:sup>2<\/jats:sup>and 800 mg\/m<jats:sup>2<\/jats:sup>. There was one complete response and 13 patients with stable disease (SD) for 8 or more courses in 30 assessable neuroblastoma patients. SD for 8 or more courses was seen in one of five Ewing sarcoma patients and one melanoma patient. Mean N-4-hydroxyphenyl retinamide plasma level (day 7, steady-state concentration) was 9.9 \u03bcmol\/L at MTD.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The pediatric MTD of oral capsular fenretinide was 2,475 mg\/m<jats:sup>2<\/jats:sup>per day, which achieved levels active against neuroblastoma in vitro with minimal toxicity. Response data support a phase II trial in neuroblastoma.<\/jats:p><\/jats:sec>","DOI":"10.1200\/jco.2005.03.9271","type":"journal-article","created":{"date-parts":[[2006,7,18]],"date-time":"2006-07-18T23:08:23Z","timestamp":1153264103000},"page":"3423-3430","source":"Crossref","is-referenced-by-count":128,"title":["Phase I Trial of Oral Fenretinide in Children With High-Risk Solid Tumors: A Report From the Children's Oncology Group (CCG 09709)"],"prefix":"10.1200","volume":"24","author":[{"given":"Judith G.","family":"Villablanca","sequence":"first","affiliation":[{"name":"From the Children's Hospital Los Angeles and the Department Pediatrics, University of Southern California Keck School of Medicine; Developmental Therapeutics Program, University of Southern California\u2013Children\u2019s Hospital Los Angeles Institute for Pediatric Clinical Research, Los Angeles; Children's Oncology Group, Arcadia, CA; Mayo Clinic, Rochester, MN; and the Children's National Medical Center and Department of Pediatrics, The George Washington University School of Medicine and Health Sciences,..."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark D.","family":"Krailo","sequence":"additional","affiliation":[{"name":"From the Children's Hospital Los Angeles and the Department Pediatrics, University of Southern California Keck School of Medicine; Developmental Therapeutics Program, University of Southern California\u2013Children\u2019s Hospital Los Angeles Institute for Pediatric Clinical Research, Los Angeles; Children's Oncology Group, Arcadia, CA; Mayo Clinic, Rochester, MN; and the Children's National Medical Center and Department of Pediatrics, The George Washington University School of Medicine and Health Sciences,..."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Matthew M.","family":"Ames","sequence":"additional","affiliation":[{"name":"From the Children's Hospital Los Angeles and the Department Pediatrics, University of Southern California Keck School of Medicine; Developmental Therapeutics Program, University of Southern California\u2013Children\u2019s Hospital Los Angeles Institute for Pediatric Clinical Research, Los Angeles; Children's Oncology Group, Arcadia, CA; Mayo Clinic, Rochester, MN; and the Children's National Medical Center and Department of Pediatrics, The George Washington University School of Medicine and Health Sciences,..."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Joel M.","family":"Reid","sequence":"additional","affiliation":[{"name":"From the Children's Hospital Los Angeles and the Department Pediatrics, University of Southern California Keck School of Medicine; Developmental Therapeutics Program, University of Southern California\u2013Children\u2019s Hospital Los Angeles Institute for Pediatric Clinical Research, Los Angeles; Children's Oncology Group, Arcadia, CA; Mayo Clinic, Rochester, MN; and the Children's National Medical Center and Department of Pediatrics, The George Washington University School of Medicine and Health Sciences,..."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Gregory H.","family":"Reaman","sequence":"additional","affiliation":[{"name":"From the Children's Hospital Los Angeles and the Department Pediatrics, University of Southern California Keck School of Medicine; Developmental Therapeutics Program, University of Southern California\u2013Children\u2019s Hospital Los Angeles Institute for Pediatric Clinical Research, Los Angeles; Children's Oncology Group, Arcadia, CA; Mayo Clinic, Rochester, MN; and the Children's National Medical Center and Department of Pediatrics, The George Washington University School of Medicine and Health Sciences,..."}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"C. 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