Infant high grade gliomas (HGG) are the most frequent tumors of the central nervous system in children below 1 year of age. Standard therapy involves surgical resection and chemotherapy. Prognosis in infant HGG is better than in older patients, however, the absence of effective regimens of anti-recurrence therapy and the impossibility of radiation therapy implementation predetermine a negative prognosis in the group of infant gliomas in case of disease progression. In most patients with infant HGG of hemispheric localization, gene rearrangements of receptor tyrosine kinases – NTRK1/2/3 (24%), ALK (41%), ROS1 (28%), MET (7%) are described. The results of tyrosine kinase inhibitor administration show high efficacy and safety in the treatment of patients with NTRK-positive gliomas. The study was approved by the Independent Ethics Committee of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. The paper presents two examples of the successful use of targeted therapy in patients with infant HGG lacking the efficacy of the standard chemotherapy. In both cases, a persistent response was obtained: in the first case, a complete response to therapy was achieved, the duration of treatment is currently 1 year, in the second case – there is no progression of the disease during 20 weeks of therapy. Of the adverse events (AE) of targeted therapy in patients, only transitory neutropenia was noted in the first case, in the second case, AEs were not detected. In order to expand therapeutic options and prescribe targeted therapy drugs, a molecular genetic investigation of tumor tissue is mandatory for patients with hemispheric infant gliomas. The parents of the patients agreed to use the information, including photos of children, in scientific research and publications.
BACKGROUND/OBJECTIVES Relapse of medulloblastoma (MB) is highly lethal in previously irradiated patients. As one of therapeutic options for recurrence MB, high-dose chemotherapy with stem cell rescue (HDSCR) is suggested. The aim of our work was to evaluate the effectiveness of this therapy. DESIGN/METHODS We retrospectively analyzed the data of 8 pts with previously irradiated relapse MB using HDSCR. Initially, M0-stage was verified in 4 cases. Histological diagnoses were desmoplastic (2 pts), classic (2 pts), anaplastic (2 pts) and MB NOS (2 pts). Molecular genetic analyses was performed in 6 cases: Group 3 was verified in 2 cases (1-classic, 1-anaplastic), Group 4 – in 3 cases (1-classic, 1-anaplastic, 1-desmoplastic). Time to first PD was from 15 to 86 months (median=29,4 months). Local relapse was revealed in 1 pt, metastatic – in 5 pts, mixed – in 2 pts. RESULTS All pts were treated according HIT-REZ 2005 (3–5 cycles without/with intraventricular etoposide), with CR achieved in 3 pts and PR in 5 pts. HDCT regimens consisted of carboplatin, etoposide, thiotepa and temozolomide. 2 pts received re-irradiation – focal RT (1) and CSI (1). 7/8 patients died, 1 pt alive with PD. Time from HDCT to death was 5–15 months (median=9,6 months). CONCLUSIONS HDSCR for recurrent previously irradiated MB is ineffective. Use of other methods should be considered in these cases.
BACKGROUND/OBJECTIVES Treatment of children with CNS NGGCT remains challenge: 5y OS is 60 – 80%; relapses are very aggressive. DESIGN/METHODS Between 2003 and 2019, 14 children (median age 10.5, range 4 – 16 years) with localized intracranial NGGCT were treated with RT after induction chemotherapy (focal – 4, WVI+boost – 6, WBI+boost – 3, CSI+boost – 1). Tumor markers were elevated in 13 patients: 6 – AFP, 5 – HCG, 2 – both. One patient with level of HCG 72049 IU/l in serum and 121451 IU/l in CSF received 4 cycles of PEI + CSI 30 Gy with boost 54Gy. RESULTS At a median follow-up of 4,7 years (range 1 – 16,25 years), 12 patients are alive. 5-year PFS and OS are 77,1% and 85,7%, respectively. Two patients (both AFP and HCG) progressed during RT (1 – focal, 1 – WBI+boost), both died. Two patients with high level of HCG recurred after therapy (WVI+boost – 1, focal – 1), both are alive. The first of them at recurrence (mts of lateral ventricle) received 4 cycles of PEI and RT (WBI+boost). The second patient had level of HCG 620IU/l and initially received focal irradiation 54Gy. At recurrence with distant spinal mts he received HD-CHT with auto-SCT, surgical resection of residual tumor and CSI with boost. CONCLUSIONS Good results of treatment of localized CNS NGGCT with CSI, WBI or WVI in compare with focal RT show advantages of extended irradiation field. CSI should be considered for patients with extremely high levels of tumor markers and respectively poor prognostic histology.
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.
hi@scite.ai
10624 S. Eastern Ave., Ste. A-614
Henderson, NV 89052, USA
Copyright © 2024 scite LLC. All rights reserved.
Made with 💙 for researchers
Part of the Research Solutions Family.