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Original Article
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Volume 352:978-986 March 10, 2005 Number 10
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Treatment of Early Childhood Medulloblastoma by Postoperative Chemotherapy Alone
Stefan Rutkowski, M.D., Udo Bode, M.D., Frank Deinlein, M.D., Holger Ottensmeier, Ph.D., Monika Warmuth-Metz, M.D., Niels Soerensen, M.D., Norbert Graf, M.D., Angela Emser, Dipl.Math., Torsten Pietsch, M.D., Johannes E.A. Wolff, M.D., Rolf D. Kortmann, M.D., and Joachim Kuehl

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ABSTRACT

Background The prognosis for young children with medulloblastoma is poor, and survivors are at high risk for cognitive deficits. We conducted a trial of the treatment of this brain tumor by intensive postoperative chemotherapy alone.

Methods After surgery, children received three cycles of intravenous chemotherapy (cyclophosphamide, vincristine, methotrexate, carboplatin, and etoposide) and intraventricular methotrexate. Treatment was terminated if a complete remission was achieved. Leukoencephalopathy and cognitive deficits were evaluated.

Results Forty-three children were treated according to protocol. In children who had complete resection (17 patients), residual tumor (14), and macroscopic metastases (12), the five-year progression-free and overall survival rates (±SE) were 82±9 percent and 93±6 percent, 50±13 percent and 56±14 percent, and 33±14 percent and 38±15 percent, respectively. The rates in 31 patients without macroscopic metastases were 68±8 percent and 77±8 percent. Desmoplastic histology, metastatic disease, and an age younger than two years were 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 19 of 23 children, asymptomatic leukoencephalopathy was detected by magnetic resonance imaging. After treatment, the mean IQ was significantly lower than that of healthy controls within the same age group but higher than that of patients in a previous trial who had received radiotherapy.

Conclusions Postoperative chemotherapy alone is a promising treatment 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.

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Related Letters:

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.
Extract | Full Text | PDF  
N Engl J Med 2005; 352:2350-2353, Jun 2, 2005. Correspondence

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