Everolimus for subependymal giant cell astrocytoma: 5‐year final analysis
| Autor: | David Neal Franz, Karen Agricola, Darcy A. Krueger, Severine Peyrard, Katherine Holland‐Bouley, Maxwell Mays, Noah Berkowitz, Cindy Tudor, Sara Miao, Marguerite M. Care |
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| Rok vydání: | 2015 |
| Předmět: |
Adult
Male medicine.medical_specialty Adolescent Urology Phases of clinical research Antineoplastic Agents Astrocytoma Young Adult Tuberous sclerosis Tuberous Sclerosis Outcome Assessment Health Care Humans Medicine Everolimus Child Adverse effect Research Articles Subependymal giant cell astrocytoma Brain Neoplasms business.industry medicine.disease Surgery Clinical trial Upper respiratory tract infection Neurology Child Preschool Female Neurology (clinical) business Research Article Follow-Up Studies medicine.drug |
| Zdroj: | Annals of Neurology |
| ISSN: | 1531-8249 0364-5134 |
| DOI: | 10.1002/ana.24523 |
| Popis: | Objective To analyze the cumulative efficacy and safety of everolimus in treating subependymal giant cell astrocytomas (SEGA) associated with tuberous sclerosis complex (TSC) from an open-label phase II study (NCT00411619). Updated data became available from the conclusion of the extension phase and are presented in this ≥5-year analysis. Methods Patients aged ≥ 3 years with a definite diagnosis of TSC and increasing SEGA lesion size (≥2 magnetic resonance imaging scans) received everolimus starting at 3mg/m2/day (titrated to target blood trough levels of 5–15ng/ml). The primary efficacy endpoint was reduction from baseline in primary SEGA volume. Results As of the study completion date (January 28, 2014), 22 of 28 (78.6%) initially enrolled patients finished the study per protocol. Median (range) duration of exposure to everolimus was 67.8 (4.7–83.2) months; 12 (52.2%) and 14 (60.9%) of 23 patients experienced SEGA volume reductions of ≥50% and ≥30% relative to baseline, respectively, after 60 months of treatment. The proportion of patients experiencing daily seizures was reduced from 7 of 26 (26.9%) patients at baseline to 2 of 18 (11.1%) patients at month 60. Most commonly reported adverse events (AEs) were upper respiratory tract infection and stomatitis of mostly grade 1 or 2 severity. No patient discontinued treatment due to AEs. The frequency of emergence of most AEs decreased over the course of the study. Interpretation Everolimus continues to demonstrate a sustained effect on SEGA tumor reduction over ≥5 years of treatment. Everolimus remained well-tolerated, and no new safety concerns were noted. Ann Neurol 2015;78:929–938 |
| Databáze: | OpenAIRE |
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