{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T02:23:13Z","timestamp":1772158993572,"version":"3.50.1"},"reference-count":41,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2014,1,28]],"date-time":"2014-01-28T00:00:00Z","timestamp":1390867200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2014,5]]},"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>The aim of the study is to evaluate the outcome of young children with high risk localized medulloblastomas (newly diagnosed classical or incompletely resected) treated by high\u2010dose busulfan\u2013thiotepa with autologous stem cell rescue (ASCT) followed by focal radiation therapy (RT).<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>Between September 1994 and January 2010, 19 children younger than 5 years old at diagnosis fulfilling the above inclusion criteria were treated at the Institute Gustave Roussy. After conventional chemotherapy, they received busulfan at a dose of 600\u2009mg\/m<jats:sup>2<\/jats:sup> and thiotepa at a dose of 900\u2009mg\/m<jats:sup>2<\/jats:sup> followed by ASCT. Focal RT was delivered at least 70 days after ASCT.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The median follow\u2010up was 40.5 months (range, 14.5\u2013191.2 months). The 3\u2010year event\u2010free survival (EFS) and OS were 68% (95% CI 45\u201384%) and 84% (95% CI 61\u201394%), respectively. Acute toxicity consisted mainly in hepatic veno\u2010occlusive disease (6\/19 patients) and bone marrow aplasia (all patients). No toxic death occurred. The Full Scale Intellectual Quotient tended to decrease over time at a mean rate of 0.9 point per year from the date of diagnosis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>This intensive treatment resulted in a high overall survival rate in young children with newly diagnosed non\u2010metastatic classic or incompletely resected MB. In spite of a high incidence of hepatic veno\u2010occlusive disease (32%), the acute toxicity was manageable. Delayed neuropsychological side effects remain main concerns. These results should to be confirmed in a larger cohort. Pediatr Blood Cancer 2014;61:907\u2013912. \u00a9 2014 Wiley Periodicals, Inc.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.24954","type":"journal-article","created":{"date-parts":[[2014,1,28]],"date-time":"2014-01-28T08:02:44Z","timestamp":1390896164000},"page":"907-912","source":"Crossref","is-referenced-by-count":22,"title":["High\u2010dose busulfan\u2013thiotepa with autologous stem cell transplantation followed by posterior fossa irradiation in young children with classical or incompletely resected medulloblastoma"],"prefix":"10.1002","volume":"61","author":[{"given":"Guillaume","family":"Bergthold","sequence":"first","affiliation":[{"name":"Department of Paediatric and Adolescent Oncology Institut Gustave Roussy Villejuif France"},{"name":"American Memorial Hospital, CHU de Reims Reims France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria El","family":"Kababri","sequence":"additional","affiliation":[{"name":"Unit of Pediatric Hematology and Oncology Rue Lamfadel Cherkaoui Rabat Institut Children Hospital, Rabat Rabat Morocco"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pascale","family":"Varlet","sequence":"additional","affiliation":[{"name":"Department of Neuropathology Sainte\u2010Anne Hospital Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Frederic","family":"Dhermain","sequence":"additional","affiliation":[{"name":"Department of Radiation Oncology Institut Gustave Roussy Villejuif France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christian","family":"Sainte\u2010Rose","sequence":"additional","affiliation":[{"name":"Department of Neurosurgery Necker\u2010Enfants Malades Hospital Paris France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marie\u2010Anne","family":"Raquin","sequence":"additional","affiliation":[{"name":"Department of Paediatric and Adolescent Oncology Institut Gustave Roussy Villejuif 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