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Publication Details
AFRICAN RESEARCH NEXUS
SHINING A SPOTLIGHT ON AFRICAN RESEARCH
medicine
High-dose busulfan-thiotepa with autologous stem cell transplantation followed by posterior fossa irradiation in young children with classical or incompletely resected medulloblastoma
Pediatric Blood and Cancer, Volume 61, No. 5, Year 2014
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Description
Background: 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-dose busulfan-thiotepa with autologous stem cell rescue (ASCT) followed by focal radiation therapy (RT). Procedure: 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 600mg/m2 and thiotepa at a dose of 900mg/m2 followed by ASCT. Focal RT was delivered at least 70 days after ASCT. Results: The median follow-up was 40.5 months (range, 14.5-191.2 months). The 3-year event-free survival (EFS) and OS were 68% (95% CI 45-84%) and 84% (95% CI 61-94%), respectively. Acute toxicity consisted mainly in hepatic veno-occlusive 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. Conclusions: This intensive treatment resulted in a high overall survival rate in young children with newly diagnosed non-metastatic classic or incompletely resected MB. In spite of a high incidence of hepatic veno-occlusive disease (32%), the acute toxicity was manageable. Delayed neuropsychological side effects remain main concerns. These results should to be confirmed in a larger cohort. © 2014 Wiley Periodicals, Inc.
Authors & Co-Authors
Bergthold, Guillaume
France, Villejuif
Institut de Cancerologie Gustave Roussy
France, Reims
American Memorial Hospital
Kababri, Maria El
Morocco, Agdal Rabat
Children Hospital
Varlet, Pascale
France, Paris
Centre Hospitalier Sainte-anne
Dhermain, Frédéric G.
France, Villejuif
Institut de Cancerologie Gustave Roussy
Sainte-Rosé, Christian
France, Paris
Hôpital Necker Enfants Malades
Raquin, Marie Anne
France, Villejuif
Institut de Cancerologie Gustave Roussy
Kieffer, Virginie
France, Villejuif
Institut de Cancerologie Gustave Roussy
France, Saint-maurice
Hôpital National de Saint-maurice
Goma, Gisele
France, Villejuif
Institut de Cancerologie Gustave Roussy
Grill, Jacques
France, Villejuif
Institut de Cancerologie Gustave Roussy
Valteau-Couanet, Dominique
France, Villejuif
Institut de Cancerologie Gustave Roussy
Dufour, Christelle
France, Villejuif
Institut de Cancerologie Gustave Roussy
Statistics
Citations: 11
Authors: 11
Affiliations: 6
Identifiers
Doi:
10.1002/pbc.24954
ISSN:
15455009
e-ISSN:
15455017
Research Areas
Cancer
Environmental
Maternal And Child Health
Study Design
Cohort Study