Joanna Trelinska1, Iwona Dachowska1, Dobromila Baranska2, Konrad Stawiski1, Katarzyna Kotulska3, Wojciech Fendler1,4, Sergiusz Jozwiak5, Wojciech Mlynarski1. 1. Department of Pediatrics, Oncology, Hematology and Diabetology, Medical University of Lodz, Lodz, Poland. 2. Department of Pediatric Radiology, Medical University Hospital, Lodz, Poland. 3. Department of Neurology & Epileptology and Pediatric Rehabilitation, The Children's Memorial Health Institute, Warsaw, Poland. 4. Department of Biostatistics and Translational Medicine, Medical University of Lodz, Lodz, Poland. 5. Department of Child Neurology, Medical University of Warsaw, Warsaw, Poland.
Abstract
OBJECTIVE: One of the therapeutic options for patients with tuberous sclerosis (TCS) and subependymal giant cell astrocytoma (SEGA) is everolimus treatment once daily, every day, to attain trough concentrations of 5-15 ng/ml (standard treatment). The aim of this study was to evaluate the efficacy and safety of a reduced dose of everolimus (three times a week with a daily dose as in standard treatment-maintenance therapy) in a group of patients who were previously treated with standard dose for at least 12 months. MATERIALS AND METHODS: Ten patients (six males, four females; median age 14.23 years) with TSC-related SEGA who met inclusion criteria were included into a single-arm, prospective trial. All the patients were followed over at least 12 months (median 12 and range 12-24 months). Tumor volumes from day 0, 90, 180, and 360 were evaluated by an experienced radiologist and an objective computer-based method and compared. Adverse events (AEs) noted during maintenance therapy were compared to the AEs observed during standard everolimus therapy. RESULTS: The differences in SEGA volume between subsequent time points (day 0, 90, 120, and 360) were not statistically significant. No clinical symptoms of tumor regrowth were observed. AEs were significantly less severe and less frequent during maintenance compared with standard therapy. CONCLUSIONS: Maintenance therapy with reduced-dose everolimus is an effective therapeutic option for patients with TSC and SEGA after the completion of standard therapy and may moderate the rates of adverse effects.
OBJECTIVE: One of the therapeutic options for patients with tuberous sclerosis (TCS) and subependymal giant cell astrocytoma (SEGA) is everolimus treatment once daily, every day, to attain trough concentrations of 5-15 ng/ml (standard treatment). The aim of this study was to evaluate the efficacy and safety of a reduced dose of everolimus (three times a week with a daily dose as in standard treatment-maintenance therapy) in a group of patients who were previously treated with standard dose for at least 12 months. MATERIALS AND METHODS: Ten patients (six males, four females; median age 14.23 years) with TSC-related SEGA who met inclusion criteria were included into a single-arm, prospective trial. All the patients were followed over at least 12 months (median 12 and range 12-24 months). Tumor volumes from day 0, 90, 180, and 360 were evaluated by an experienced radiologist and an objective computer-based method and compared. Adverse events (AEs) noted during maintenance therapy were compared to the AEs observed during standard everolimus therapy. RESULTS: The differences in SEGA volume between subsequent time points (day 0, 90, 120, and 360) were not statistically significant. No clinical symptoms of tumor regrowth were observed. AEs were significantly less severe and less frequent during maintenance compared with standard therapy. CONCLUSIONS: Maintenance therapy with reduced-dose everolimus is an effective therapeutic option for patients with TSC and SEGA after the completion of standard therapy and may moderate the rates of adverse effects.
Authors: Konrad Stawiski; Joanna Trelińska; Dobromiła Baranska; Iwona Dachowska; Katarzyna Kotulska; Sergiusz Jóźwiak; Wojciech Fendler; Wojciech Młynarski Journal: MAGMA Date: 2017-03-20 Impact factor: 2.310
Authors: Elena Cristina De Sautu De Borbón; Juan Manuel Guerra Vales; Carlos Lumbreras Bermejo; Felix Guerrero Ramos; María José Buj Padilla; Jesús González de la Aleja; Montserrat Morales Conejo Journal: Orphanet J Rare Dis Date: 2021-05-31 Impact factor: 4.123