Background The prognosis for young children with medulloblastomais poor, and survivors are at high risk for cognitive deficits.We conducted a trial of the treatment of this brain tumor byintensive postoperative chemotherapy alone.
Methods After surgery, children received three cycles of intravenouschemotherapy (cyclophosphamide, vincristine, methotrexate, carboplatin,and etoposide) and intraventricular methotrexate. Treatmentwas terminated if a complete remission was achieved. Leukoencephalopathyand cognitive deficits were evaluated.
Results Forty-three children were treated according to protocol.In children who had complete resection (17 patients), residualtumor (14), and macroscopic metastases (12), the five-year progression-freeand overall survival rates (±SE) were 82±9 percentand 93±6 percent, 50±13 percent and 56±14percent, and 33±14 percent and 38±15 percent,respectively. The rates in 31 patients without macroscopic metastaseswere 68±8 percent and 77±8 percent. Desmoplastichistology, metastatic disease, and an age younger than two yearswere independent prognostic factors for tumor relapse and survival.Treatment strategies at relapse were successful in 8 of 16 patients.There were no major instances of unexpected toxicity. In 19of 23 children, asymptomatic leukoencephalopathy was detectedby magnetic resonance imaging. After treatment, the mean IQwas significantly lower than that of healthy controls withinthe same age group but higher than that of patients in a previoustrial who had received radiotherapy.
Conclusions Postoperative chemotherapy alone is a promisingtreatment for medulloblastoma in young children without metastases.
Source Information
From the Department of Pediatric Oncology, Children's Hospital (S.R., F.D., H.O., J.K.), and the Departments of Neuroradiology (M.W.-M.) and Pediatric Neurosurgery (N.S.), University of Würzburg, Würzburg; the Department of Pediatric Oncology, Children's Hospital (U.B.) and National Reference Center for Brain Tumors, Department of Neuropathology (T.P.), University of Bonn, Bonn; the Department of Pediatric Oncology, Children's Hospital, University of Homburg/Saar, Homburg/Saar (N.G.); Institute for Medical Statistics, Epidemiology, and Informatics, University of Mainz, Mainz (A.E.); the Department of Pediatric Oncology, Children's Hospital, University of Regensburg, Regensburg (J.E.A.W.); and the Department of Radiotherapy, University of Leipzig, Leipzig (R.D.K.) all in Germany. Dr. Kuehl is deceased.
Address reprint requests to Dr. Rutkowski at the Department of Pediatric Oncology, Children's Hospital, University of Würzburg, Josef-Schneider-Str. 2, D-97080 Würzburg, Germany, or at rutkowski{at}mail.uni-wuerzburg.de.
Treatment of Brain Tumors
Paulino A. C., Teh B. S., Sadeh M., Seiter K., Ashby L., LaRocca R., Ryken T., Aiken R. D., Rutkowski S., Ottensmeier H., Pietsch T., Stupp R., Hegi M. E., DeAngelis L. M.
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N Engl J Med 2005;
352:2350-2353, Jun 2, 2005.
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