{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T17:36:37Z","timestamp":1791048997264,"version":"4.1.0"},"reference-count":53,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2007,10,11]],"date-time":"2007-10-11T00:00:00Z","timestamp":1192060800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cancer"],"published-print":{"date-parts":[[2007,12]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>BACKGROUND.<\/jats:title><jats:p>An open\u2010label Phase II study of tipifarnib was conducted to evaluate its safety and efficacy in children with recurrent or refractory medulloblastoma (MB)\/primitive neuroectodermal tumor (PNET), high\u2010grade glioma (HGG), and diffuse intrinsic brainstem glioma (BSG).<\/jats:p><\/jats:sec><jats:sec><jats:title>METHODS.<\/jats:title><jats:p>Between January 2004 and July 2005, patients were enrolled and stratified as follows: Stratum 1, recurrent or refractory MB\/PNET; Stratum 2, recurrent or refractory HGG; and Stratum 3, recurrent or refractory BSG. Patients received tipifarnib 200 mg\/m<jats:sup>2<\/jats:sup> per dose twice daily for 21 days repeated every 28 days. Patients who received enzyme\u2010inducing anticonvulsants and other CYP3A4\/5 inducers or inhibitors were excluded. The primary objective was to estimate the sustained response rate in all strata.<\/jats:p><\/jats:sec><jats:sec><jats:title>RESULTS.<\/jats:title><jats:p>Ninety\u2010seven patients with a median age of 11.2 years (range, 3.2\u201321.9 years) were enrolled on the study, and 81 patients were evaluable for response. One of 35 patients with BSG and 1 of 31 patients with HGG had a sustained partial response. No responses were observed in 15 patients with MB\/PNET. Eight patients (3 HGG, 1 MB, and 4 BSG) remained stable for \u22654 courses (range, 4\u201325 courses). The median number of courses received was 2 (range, 1\u201325 courses). The most frequent grade 3 and 4 toxicities included neutropenia (18.7%), thrombocytopenia (14.3%), and leukopenia (14.3%). The 6\u2010month progression\u2010free survival rate (\u00b1standard deviation) was 14% \u00b1 6% for HGG, 6% \u00b1 6% for MB\/PNET and 3% \u00b1 3% for BSG.<\/jats:p><\/jats:sec><jats:sec><jats:title>CONCLUSIONS.<\/jats:title><jats:p>Tipifarnib tolerated well but had little activity as a single agent in children with recurrent central nervous system malignancies. Cancer 2007. \u00a9 2007 American Cancer Society.<\/jats:p><\/jats:sec>","DOI":"10.1002\/cncr.23078","type":"journal-article","created":{"date-parts":[[2007,10,11]],"date-time":"2007-10-11T13:09:39Z","timestamp":1192108179000},"page":"2535-2541","source":"Crossref","is-referenced-by-count":67,"title":["A phase II study of the farnesyl transferase inhibitor, tipifarnib, in children with recurrent or progressive high\u2010grade glioma, medulloblastoma\/primitive neuroectodermal tumor, or brainstem glioma: A children's oncology group study"],"prefix":"10.1002","volume":"110","author":[{"given":"Maryam","family":"Fouladi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. 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