Treatment of early childhood medulloblastoma by postoperative chemotherapy alone.
By S. Rutkowski, U. Bode, F. Deinlein, H. Ottensmeier, M. Warmuth-Metz, N. Soerensen, N. Graf, A. Emser, T. Pietsch, J. Wolff and 2 more. New England Journal of Medicine, 2005
623 citations recorded by the source corpus.
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.
Subfields: Genetics, Pediatrics, Perinatology and Child Health, Pulmonary and Respiratory Medicine
View Paper: https://www.semanticscholar.org/paper/8ef93001d7bf36d7085b2c1ced5f4c691d5e8a26
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In Genetics
Citations in the corpus
- Molecular subgroups of medulloblastoma: an international meta-analysis of transcriptome, genetic aberrations, and clinical data of WNT, SHH, Group 3, and Group 4 medulloblastomas (2012, background): “This is likely associated with the high frequency of desmoplastic histology among SHH tumors in infants, as it has been demonstrated that desmoplastic/extensive nodular histology in this age group is a marker of favorable prognosis [21].”
- Outcomes for children and adolescents with cancer: challenges for the twenty-first century. (2010, background): “An apparent improvement in outcome for younger children with medulloblastoma for the most recent reporting period may reflect increasing use of intensive chemotherapy, with or without autologous stem cell rescue, to delay or eliminate use of radiation therapy.(29,30) Bone sarcomas (osteosarcoma and ”
- Integrated Genomics Identifies Five Medulloblastoma Subtypes with Distinct Genetic Profiles, Pathway Signatures and Clinicopathological Features (2008, background): “Recent reports suggest that desmoplastic tumors in infants have a very good prognosis [48–50].”