Central Nervous System
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Jun 01 – Jun 08, 2026
SEZ6-targeting antibody-drug conjugate ABBV-706 in advanced small cell lung cancer and solid tumors: a phase 1 trial.
NAT MED · Q1 JOURNAL - RANK #1/195TOP-TIER
This open-label, phase 1 clinical trial assessed the safety, tolerability, pharmacokinetics, immunogenicity, and antitumor activity of ABBV-706, a SEZ6-targeting antibody-drug conjugate, in 288 patients with advanced solid tumors, with a focus on 124 relapsed/refractory (R/R) small cell lung cancer (SCLC) patients. ABBV-706 was administered intravenously every 3 weeks, with safety outcomes highlighting anemia (61%) and fatigue (38%) as the most common treatment-related adverse events in the monotherapy cohort, and grade 3 or higher adverse events in 61% of R/R SCLC patients. Efficacy data showed an objective response rate of 52% in R/R SCLC, with comparable ORRs (56% and 59%) between 1.8 mg/kg and 2.5 mg/kg doses and median overall survival of 12.4 months at 1.8 mg/kg. The study concluded 1.8 mg/kg as the recommended phase 2 dose based on safety and efficacy.
10.1038/s41591-026-04452-0
Tumor-targeted interferon-α gene therapy for glioblastoma: a phase 1 trial.
NAT MED · Q1 JOURNAL - RANK #1/195TOP-TIER
This phase 1/2a dose-escalation study evaluated Temferon, a genetically engineered autologous stem cell transplant delivering interferon-α2 to the tumor microenvironment in 24 newly diagnosed glioblastoma patients with unmethylated MGMT promoter. The primary endpoint was safety and tolerability within 90 days post-infusion; no dose-limiting toxicities occurred up to the highest dose tested. Median overall survival was 16.7 months and progression-free survival was 8.1 months from diagnosis, with most patients maintaining good performance status. The authors conclude Temferon is a safe and tolerable immunotherapeutic strategy for newly diagnosed glioblastoma.
10.1038/s41591-026-04419-1
May 25 – Jun 01, 2026
Long-Term Analysis of NRG Oncology RTOG 0539: A Phase II Trial of Observation for Low-Risk Meningioma and Radiotherapy for Intermediate- and High-Risk Meningioma.
J CLIN ONCOL · Q1 JOURNAL - RANK #6/326TOP-TIER
The study presents a prospective phase II trial (NRG Oncology RTOG 0539) on risk-adapted radiotherapy for WHO grade 1-3 meningioma patients. The primary objective was to assess the outcomes of observation for low-risk patients and radiotherapy for intermediate- and high-risk cohorts using a Kaplan-Meier analysis to estimate progression-free survival (PFS) and overall survival (OS) over a median follow-up of 11-12 years. Key results include 10-year PFS and OS rates of 85.2%/94.1% (low-risk), 72.2%/84.7% (intermediate-risk), and 42.5%/51.1% (high-risk), with grade 3+ radiotherapy toxicity rates of 9.6% and 15.1% in intermediate- and high-risk cohorts, respectively. The findings demonstrate the value of observation for low-risk patients and radiotherapy for higher-risk patients, while providing benchmarks for future trials.
10.1200/JCO-25-01441
Phase 1 evaluation of patients with newly diagnosed glioblastoma treated with radiation, nivolumab, and IDO1 enzyme inhibitor BMS-986205.
CLIN CANCER RES · Q1 JOURNAL - RANK #29/326TOP-TIER
This phase 1 trial evaluated the safety and tolerability of radiotherapy (RT) combined with nivolumab and the IDO1 enzyme inhibitor BMS-986205 in newly diagnosed glioblastoma (GBM) patients. The study included two cohorts: MGMT unmethylated patients (Cohort A) receiving RT, nivolumab, and escalating BMS-986205 doses, and MGMT methylated patients (Cohort B) receiving BMS-986205 at 25mg daily with RT, nivolumab, and temozolomide (TMZ). Treatment-related adverse events were mostly lower grade, with dose-limiting toxicities (grade 3 transaminase increases) observed in 2 patients at 50mg and 3 patients at 100mg BMS-986205, leading to a recommended phase 2 dose of 50mg daily. The study concluded that the combination therapy is safe and established a recommended dose for further evaluation in MGMT-unmethylated GBM patients.
10.1158/1078-0432.CCR-26-1124
May 18 – May 25, 2026
Phase 1/2 study of a WT1 peptide-dosing emulsion in pediatric patients with recurrent/refractory diffuse intrinsic pontine glioma, glioblastoma, or anaplastic astrocytoma.
EUR J CANCER · Q1 JOURNAL - RANK #45/326
This first-in-child phase 1/2 clinical trial prospectively evaluated a WT1 peptide vaccine in 18 pediatric patients with recurrent/refractory DIPG, glioblastoma, or anaplastic astrocytoma. Patients received intradermal injections, with 3.5 mg determined as the phase 2 dose after no dose-limiting toxicities in phase 1 (n=4). The primary endpoint, 9-month overall survival, was 44.4% (90% CI: 24.4-65.9) for all patients and 27.3% (90% CI: 7.9-56.4) for DIPG patients, with median OS of 5.4 months for DIPG; the vaccine was not statistically effective as the lower CI did not exceed the prespecified 20% threshold, though WT1-specific immune responders showed significantly longer median OS (9.9 vs. 4.9 months, p=0.024). The authors concluded the vaccine was well tolerated and induced immune responses warranting further development. (NCT02750891)
10.1016/j.ejca.2026.116808
Treatment Effect Reanalysis of the Randomized Individual Screening Trial of Innovative Glioblastoma Therapy in Newly Diagnosed Glioblastoma With External Control Data.
J CLIN ONCOL · Q1 JOURNAL - RANK #6/326TOP-TIER
The study reanalyzed three experimental arms of the phase II INSIGhT trial for newly diagnosed glioblastoma patients (ClinicalTrials.gov identifier: NCT02977780), using internal and external control data to estimate treatment effects. Experimental therapies—abemaciclib (n=72, HR 1.00 [95% CI 0.75–1.34]), neratinib (n=80, HR 0.93 [95% CI 0.70–1.24]), and CC-115 (n=12, HR 0.88 [95% CI 0.41–1.88])—did not demonstrate improved survival compared to internal controls receiving standard chemoradiation (n=70). External controls were constructed through propensity score matching with real-world and clinical trial datasets, and simulations assessed the validity and risks of such trial designs. The authors concluded that while external controls can provide valid estimates similar to internal controls, their reliability depends on the availability of comprehensive data to mitigate unmeasured confounding bias.
10.1200/JCO-25-01586
Precision-guided therapy in H3K27-altered diffuse midline glioma.
NAT COMMUN · Q1 JOURNAL - RANK #10/135TOP-TIER
This prospective clinical trial enrolled 68 children with H3K27-altered diffuse midline glioma (DMG) into the PRISM precision medicine study (NCT03336931). A total of 58 (85%) patients underwent whole-genome sequencing and RNAseq, with precision-guided therapy (PGT) recommended for 50 (74%). Eighteen patients receiving PGT were evaluable for survival, showing an overall response rate of 48% and median overall survival of 21.3 months versus 12.1 months for non-PGT recipients (p=0.34). The study concludes that PGT is feasible and may offer clinical benefit, particularly when administered after tumor progression.
10.1038/s41467-026-73304-9
Dose Escalation With Intensity-Modulated Proton Therapy for Patients with High-Risk Meningiomas - Results From a Phase 1 Trial.
CLIN CANCER RES · Q1 JOURNAL - RANK #29/326TOP-TIER
This prospective single-arm phase 1 trial evaluated the safety and efficacy of dose-escalated intensity-modulated proton therapy (IMPT) in 21 patients with high-risk meningiomas (16 grade 2, 5 grade 3). Patients received 66 Gy(RBE) for grade 2 gross disease or 63 Gy(RBE) for grade 3 tumor beds, with primary endpoints assessing acute dose-limiting toxicity (DLT) and secondary endpoints including progression-free survival (PFS) and overall survival (OS). No DLTs occurred, with 3-year PFS and OS rates of 88.2% and 95.0%, respectively, and five late grade 3 toxicities observed in four patients. The study concluded that dose-escalated IMPT demonstrated favorable early safety and efficacy, warranting further phase 2/3 trials.
10.1158/1078-0432.CCR-25-3856
Influence of FLAIR inclusion on patterns-of-failure and outcomes in glioblastoma: results from the UNITED prospective adaptive radiotherapy trial.
RADIOTHER ONCOL · Q1 JOURNAL - RANK #22/212
This post hoc analysis of a prospective phase 2 MR-guided adaptive radiotherapy trial (UNITED; NCT04726397) in newly diagnosed glioblastoma assessed whether including peritumoral FLAIR-hyperintense regions (FHR) in the clinical target volume affects patterns of failure and survival during standard chemo-radiotherapy. Patients underwent weekly online-adaptive MR-Linac planning with GTV as resection cavity plus enhancing tumor, CTV as GTV+5 mm, and optional inclusion of FHR beyond 5 mm, forming three groups: no FHR beyond 5 mm (n=24), FHR present not included (n=27), and FHR included (n=47). Recurrences were predominantly central (65–72%, p=0.717), median recurrent GTV coverage within CTV was 97.9% (p=0.105), and no significant differences in PFS (10.5/8.0/8.6 months, p=0.279) or OS (16.9/15.8/13.4 months, p=0.389) were observed. The study concludes FHR omission within a small-margin MR-guided paradigm does not adversely affect failure patterns or survival.
10.1016/j.radonc.2026.111579
Modern Molecular Profiling Recontextualizes the NRG/RTOG 0539 Trial and Reveals Hidden High-Risk and Radiotherapy Resistant Meningiomas.
NEURO-ONCOLOGY · Q1 JOURNAL - RANK #4/285TOP-TIER
This study reanalyzes data from the NRG/RTOG-0539, the first prospective phase 2 trial stratifying meningioma patients for adjuvant radiotherapy (RT) based on clinical risk, using modern molecular techniques. Tumor samples from 100 patients underwent multi-omic profiling, revealing that 10% of tumors would be reclassified and 7% would be reassigned based on updated WHO grading criteria. The study identified genomic and transcriptomic differences between RT responders and non-responders, indicating molecular predictors such as the Proliferative Molecular Group, CDKN2A/B deletion, and 1p/1q alterations outperformed traditional grading systems in predicting recurrence. These findings suggest molecular biomarkers should be integrated with modern grading frameworks to improve risk stratification and personalize treatment for meningioma patients.
10.1093/neuonc/noag081
May 11 – May 18, 2026
MRI-guided adaptive radiotherapy for high grade glioma (UNITED): a single-centre, single-arm, non-inferiority, phase 2 trial.
LANCET ONCOL · Q1 JOURNAL - RANK #8/326TOP-TIER
This single-arm, phase 2 trial evaluated the safety of MRI-guided adaptive radiotherapy with reduced margins for glioblastoma, involving 98 patients treated with either long-course (60 Gy/30 fractions) or short-course (40 Gy/15 fractions) chemoradiotherapy. The primary outcome, marginal failure risk, was 4% (95% CI 0-8), meeting non-inferiority criteria against a 10% historical margin. Grade 3-4 adverse events included lymphopenia (11%) and thrombocytopenia (4%), with one serious adverse event (1%) and no treatment-related deaths. The study concluded that MRI-guided adaptive radiotherapy allows margin reduction with low marginal failure rates, warranting further randomized trials for toxicity and quality-of-life comparisons.
10.1016/S1470-2045(26)00088-4
Personalized machine learning-guided radiation dose escalation in newly diagnosed glioblastoma: prospective pilot study.
NAT COMMUN · Q1 JOURNAL - RANK #10/135TOP-TIER
This prospective pilot trial (NCT03477513) assessed the feasibility and safety of machine-learning–guided personalized precision radiation therapy (PPRT) with concomitant and adjuvant temozolomide in 20 adults with newly diagnosed IDH-wildtype glioblastoma following gross total resection. Patients received ML-based tumor-infiltration–derived dose-escalated radiotherapy; primary endpoints were median progression-free survival (PFS) and safety, with secondary endpoints of recurrence patterns, toxicity, and overall survival (OS). PPRT caused no grade ≥3 acute toxicities (47 % grade 1, 53 % grade 2) but increased radiation necrosis versus 68 propensity-matched standard-of-care controls (47 % vs 12 %; p<0.001); nevertheless, PFS improved to 24.4 months versus 11.6 months (HR 0.28; p = 0.001) and OS to 35.4 months versus 17.7 months (HR 0.34; p = 0.003). Authors conclude PPRT is feasible and preliminarily prolongs survival, meriting randomized validation.
10.1038/s41467-026-72545-y
Adjuvant personalized multivalent neoantigen DNA vaccination for MGMT unmethylated glioblastoma: a phase 1 trial.
NAT CANCER · Q1 JOURNAL - RANK #11/326TOP-TIER
This open-label, single-arm phase 1 trial (GT-20, NCT04015700) evaluated the safety, feasibility, immunogenicity and preliminary efficacy of GNOS-PV01, a personalized multivalent neoantigen DNA vaccine, in nine adults with MGMT-unmethylated glioblastoma following surgical resection and chemoradiation. Patients received vaccines encoding 17–40 neoantigens each; safety was monitored and immune responses assessed in peripheral blood, while clinical outcomes included 6-month progression-free survival (PFS) and 12-month overall survival (OS). No serious adverse events, unexpected toxicities, or dose-limiting toxicities occurred; vaccine-induced T-cell activation/expansion was observed in all but one dexamethasone-treated patient. Six-month PFS and 12-month OS were each 66.7%, median PFS 8.5 months, median OS 16.3 months, and 24-month survival 33%, supporting further development of GNOS-PV01 for glioblastoma.
10.1038/s43018-026-01163-w
Biopsy with same-session MRI-guided laser interstitial thermal therapy versus biopsy alone in patients with primary unresectable glioblastoma: a multicentre randomised controlled trial.
LANCET REG HEALTH-EU · Q1 JOURNAL - RANK #3/185TOP-TIER
This multicentre, non-blinded randomised controlled trial aimed to evaluate the efficacy of LITT plus biopsy versus biopsy alone in adults with unresectable glioblastoma. Patients were randomized 1:1, treated across seven Dutch neurosurgical centers, and followed for at least 18 months or until death. Among 26 patients, median survival was 4.8 months (95%CI 3.4–NE) for controls and 7.8 months (95%CI 2.58–NE) for LITT, with mean QLQ-C30 summary scores at 5 months of 77 (SD 11) vs 81 (SD 11). Although the study was terminated early and lacked power, it supports further investigation into LITT as a potentially useful minimally invasive cytoreductive approach for these patients.
10.1016/j.lanepe.2026.101696
Apr 27 – May 04, 2026
A phase 2 study of niraparib concomitant with tumor treating fields in patients with recurrent grade 4 glioma.
NEURO-ONCOL ADV · Q1 JOURNAL - RANK #50/285
This phase 2 trial evaluated niraparib combined with tumor treating fields (TTFields) in recurrent grade 4 glioma patients (n=9). The single-arm primary efficacy cohort (Cohort A) used a Simon’s 2-stage design, with a disease control rate (objective response or stable disease ≥16 weeks) as the primary endpoint. No objective responses occurred; only 1 of 8 patients (12.5%) achieved stable disease lasting over 16 weeks, failing to meet the efficacy benchmark for Stage 2, leading to termination for futility. The combination was safe with grade 1-2 scalp toxicity and nausea (67% each), but showed no efficacy signal, and the surgical cohort (Cohort B, n=1) was closed due to slow accrual.
10.1093/noajnl/vdag076
Phase 1 LITESPARK-001 study of belzutifan in participants with advanced solid tumors: Results of the glioblastoma expansion cohort.
NEURO-ONCOL ADV · Q1 JOURNAL - RANK #50/285
This study evaluated belzutifan, a HIF-2α inhibitor, in recurrent IDH wild-type glioblastoma patients within the phase 1 LITESPARK-001 trial. The prospective trial enrolled participants who had received prior radiotherapy and temozolomide. Key results showed single-agent belzutifan did not demonstrate antitumor activity, indicating no efficacy in this cohort. The authors concluded that while targeting HIF-2α may have a role, belzutifan alone was ineffective for this patient population.
10.1093/noajnl/vdaf242
Phase I trial of Ipatasertib plus Atezolizumab enhances PI3K/AKT pathway immune responses in solid tumors and refractory glioblastoma.
CLIN CANCER RES · Q1 JOURNAL - RANK #29/326TOP-TIER
This Phase 1b, open-label, investigator-initiated clinical trial (NCT03673787) evaluated ipatasertib plus atezolizumab in adult patients with treatment-refractory advanced solid tumors and recurrent glioblastoma, using a 3+3 dose escalation design and six exploratory cohorts. The primary objectives were to assess safety, immune-modulatory effects, and preliminary efficacy, reporting that the combination was well tolerated with no dose-limiting toxicities at ipatasertib 400 mg daily and atezolizumab 1200 mg every three weeks. Pharmacodynamic analyses showed depletion of FOXP3+ regulatory T cells and increased CD8+ effector T-cell infiltration, with durable exceptional responses observed in treatment-refractory glioblastoma patients. Results demonstrated preliminary efficacy and significant immunomodulatory activity in clinical samples.
10.1158/1078-0432.CCR-25-4364
Apr 20 – Apr 27, 2026
Combination of metronomic oral pazopanib and topotecan for recurrent glioblastoma with and without previous bevacizumab: Results from a phase 2 clinical trial.
NEURO-ONCOL ADV · Q1 JOURNAL - RANK #50/285
This prospective phase 2 clinical trial evaluated daily oral pazopanib (600 mg) plus metronomic topotecan (0.25 mg) in 31 adults with recurrent glioblastoma, split into bevacizumab-naïve (Group A, n=9) and bevacizumab-pretreated (Group B, n=22) cohorts. The primary endpoints were 6-month progression-free survival (PFS-6) for Group A and 3-month PFS (PFS-3) for Group B, with secondary analyses of median PFS, overall survival (OS), safety, and patient-reported outcomes. Group A failed to meet its endpoint (PFS-6 = 11%), whereas Group B narrowly achieved its target (PFS-3 = 18%); median PFS and OS showed only marginal improvement over historical controls. The combination was generally tolerable—hypertension was the most frequent toxicity—and patient-reported outcomes paralleled disease status, leading investigators to conclude that this regimen is safe but lacks sufficient efficacy to warrant further study in GBM.
10.1093/noajnl/vdaf261
Resection and permanent intracranial brachytherapy using modular, biocompatible cesium-131 implants for recurrent aggressive meningiomas: 5-year results from a prospective phase 2 trial.
J NEUROSURG · Q1 JOURNAL - RANK #35/312
This prospective phase 2 trial evaluated resection plus Cs-131 collagen tile brachytherapy (R+CTBT) for recurrent aggressive meningiomas. Twenty-nine tumors in 27 patients (median age 66) were treated; 90% were WHO grade 2. Local control at 48 and 60 months was 73% and 48% vs 21% and 17% with prior treatment (HR 0.145, p<0.001). Surgery-related complications occurred in 14% and radiation brain injury in 14%, all resolved with medical therapy, concluding R+CTBT significantly improved local control with acceptable complications.
10.3171/2025.12.JNS25868
Targeted therapies plus radiotherapy for diffuse intrinsic pontine glioma: the randomized phase 2 BIOMEDE trial.
NAT MED · Q1 JOURNAL - RANK #1/195TOP-TIER
The BIOMEDE randomized, biomarker-guided, phase 2 clinical trial assessed whether adding the EGFR inhibitor erlotinib, the mTOR inhibitor everolimus, or the multikinase inhibitor dasatinib to standard radiotherapy improves survival in 233 children with biopsy-proven diffuse intrinsic pontine glioma. Patients were randomized to erlotinib (n = 36), dasatinib (n = 102) or everolimus (n = 95); outcomes were compared with a prior temozolomide-treated cohort (n = 66), and tumors underwent central immunohistochemistry, whole-exome, and RNA sequencing. Median overall survival was 9.7 months (95% CI 7.8–14.6) for erlotinib, 9.9 months (8.8–11.2) for dasatinib, and 11.9 months (10.7–14.2) for everolimus versus 10.8 months (9.5–13.0) in controls, leading to early trial termination for futility; everolimus showed fewer ocular, renal, skin, and gastrointestinal adverse events. TP53 mutations predicted poorer survival (HR 2.8, 95% CI 1.9–4.2), while mTOR pathway alterations associated with better everolimus response, with four patients surviving >6 years.
10.1038/s41591-026-04354-1
CERN 09-02: A phase 2 trial of carboplatin and bevacizumab for recurrent adult ependymomas.
NEURO-ONCOL ADV · Q1 JOURNAL - RANK #50/285
This prospective single-arm Phase 2 trial evaluated carboplatin and bevacizumab in 22 adults with recurrent ependymoma. The primary endpoint was 12-month progression-free survival (PFS-12) >50%, which was met with a rate of 76.4% (95% CI: 52.2-89.4) and median PFS of 18.0 months. Two patients achieved partial responses (9.1%) with no grade ≥4 toxicities, though spine tumor patients reported worsening symptoms despite radiographic stability. The study concluded the regimen met its efficacy endpoint but noted potential bevacizumab pseudo-response effects in brain tumors.
10.1093/noajnl/vdag016
Apr 13 – Apr 20, 2026
Evaluation of Regorafenib in Newly Diagnosed and Recurrent Glioblastoma: GBM AGILE Phase II/III Bayesian Randomized Platform Trial.
J CLIN ONCOL · Q1 JOURNAL - RANK #6/326TOP-TIER
The primary objective was to evaluate the efficacy of regorafenib versus standard treatments in newly diagnosed unmethylated (NDU) and recurrent (RD) glioblastoma patients, using a phase II/III prospective, Bayesian adaptive randomized platform trial (GBM AGILE). Patients were randomized to regorafenib or control arms (temozolomide + radiotherapy for NDU and lomustine for RD), and overall survival (OS) was the primary endpoint, with Bayesian analysis performed monthly until limited efficacy criteria were met. Regorafenib did not improve OS: median HRs were 1.05 (NDU), 1.07 (RD), and 1.07 (all), with final probabilities of benefit (HR <1.00) of 0.421, 0.312, and 0.296 respectively, and was linked to increased toxicity compared to control. The study concluded that regorafenib showed no OS advantage and led to its removal from NCCN guidelines for recurrent glioblastoma.
10.1200/JCO-25-01137
Apr 06 – Apr 13, 2026
L-RNA aptamer-based CXCL12 inhibition combined with radiotherapy and bevacizumab in newly-diagnosed glioblastoma: expansion of the phase I/II GLORIA trial.
NAT COMMUN · Q1 JOURNAL - RANK #10/135TOP-TIER
This phase I/II expansion trial evaluated the safety and efficacy of combining radiotherapy with dual inhibition of CXCL12 (using L-RNA aptamer olaptesed pegol) and VEGF (using bevacizumab) in six patients with newly-diagnosed, incompletely resected glioblastoma lacking MGMT promoter methylation. The primary endpoint was safety, with secondary endpoints including maximum tolerable dose, pharmacokinetics, tumor vascularization, progression-free survival (PFS), and overall survival (OS). The combination was well-tolerated with no treatment-related deaths, resulting in abrogated tumor perfusion and median PFS and OS of 9.1 and 19.9 months respectively, which significantly outperformed radiotherapy plus CXCL12 inhibition alone in post-hoc analysis (p=0.009 for PFS; p=0.021 for OS). These findings establish proof-of-principle for dual inhibition of CXCL12 and VEGF following radiotherapy in this patient population.
10.1038/s41467-026-71362-7
Cabozantinib in patients with non-locally pretreated brain metastases from renal cell carcinoma: Results of the multicenter phase II trial CABRAMET.
EUR J CANCER · Q1 JOURNAL - RANK #45/326
The CABRAMET trial was a multicenter, prospective, open-label phase II study designed to evaluate the efficacy of cabozantinib, a VEGFR tyrosine kinase inhibitor, in patients with non-locally pretreated brain metastases (BM) from renal cell carcinoma (RCC). It included 26 RCC patients with BM and fewer than three prior systemic treatments (excluding cabozantinib), who received an oral dosage of 60 mg/day. The study found a 6-month progression-free rate (6m-BM-PFR) of 56%, an overall BM objective response rate (ORR) of 61.5%, and a median BM progression-free survival of 10.7 months, with median overall survival of 15.0 months. These results demonstrated cabozantinib’s efficacy and tolerability in this patient cohort, positioning it as a promising treatment for this challenging population.
10.1016/j.ejca.2026.116712
Mar 30 – Apr 06, 2026
Hearing Preservation After Upfront Gamma Knife Radiosurgery Versus Initial Conservative Management in Patients With Newly Diagnosed Vestibular Schwannoma: Results From a Prospective Randomized Study.
NEUROSURGERY · Q1 JOURNAL - RANK #26/312
This single-center, open-label, randomized clinical trial with parallel group design (1:1) compared upfront Gamma Knife Radiosurgery (12 Gy) to initial conservative management in 52 patients with newly diagnosed vestibular schwannoma (max diameter ≤20 mm) to assess tumor control and serviceable hearing preservation at 5-year follow-up. At 5 years, 100% (24/24) of patients in the upfront GKRS group achieved tumor control, while 50% (14/28) in the conservative group required active treatment; serviceable hearing (Gardner-Robertson class 1-2) was preserved in 65% of the GKRS group versus 50% of the conservative group, though this difference was not statistically significant (P = .388). The study concluded that upfront GKRS provides excellent tumor control in vestibular schwannoma but does not significantly improve hearing preservation compared to initial conservative management. No major adverse events were reported in either group during the study period.
10.1227/neu.0000000000004016