Abstract | BACKGROUND: PROCEDURE: Patients ≤ 21 years of age at diagnosis with a recurrent, progressive, or refractory primary CNS malignancy and measurable disease, were eligible. Patients enrolled into four strata: ependymoma (N = 4), high-grade glioma (HGG) (N = 6), brainstem glioma (BSG) (N = 13), and primitive neuroectodermal tumor ( PNET) (N = 8). Oral topotecan was administered once daily at a dose of 0.8 mg/m(2)/day for 21 consecutive days repeated every 28 days. Response and toxicity profiles were evaluated. RESULTS: Twenty-six patients were evaluable (median age 9.2 years; 10 males). Two objective responses were observed in PNET patients with disseminated tumor at study entry. These two patients remain alive and in remission 7 and 9.5 years off study. Four other patients (two BSG, one PNET, and one HGG) had stable disease (median 4.6 months). The most common toxicities were hematologic. CONCLUSIONS: Daily oral topotecan at a dose of 0.8 mg/m(2)/day can be safely administered to children with recurrent or refractory brain tumors. This regimen identified activity in recurrent PNET. The prolonged progression free survival (PFS) in two PNET patients justifies consideration of this regimen in more advanced clinical trials.
|
Authors | Jane E Minturn, Anna J Janss, Paul G Fisher, Jeffrey C Allen, Ratnakar Patti, Peter C Phillips, Jean B Belasco |
Journal | Pediatric blood & cancer
(Pediatr Blood Cancer)
Vol. 56
Issue 1
Pg. 39-44
(Jan 2011)
ISSN: 1545-5017 [Electronic] United States |
PMID | 21108437
(Publication Type: Clinical Trial, Phase II, Journal Article, Research Support, Non-U.S. Gov't)
|
Chemical References |
- Topoisomerase I Inhibitors
- Topotecan
|
Topics |
- Brain Neoplasms
(drug therapy)
- Brain Stem Neoplasms
(drug therapy)
- Child
- Disease-Free Survival
- Ependymoma
(drug therapy)
- Female
- Glioma
(drug therapy)
- Humans
- Male
- Neuroectodermal Tumors, Primitive
(drug therapy)
- Topoisomerase I Inhibitors
(therapeutic use)
- Topotecan
(administration & dosage, toxicity)
- Treatment Outcome
|