Nicolas U. Gerber
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View article: IDH mutations are rare events in SHH medulloblastoma
IDH mutations are rare events in SHH medulloblastoma Open
View article: Molecular characterization and clinical features of diffuse midline glioma in the pediatric precision oncology registry INFORM
Molecular characterization and clinical features of diffuse midline glioma in the pediatric precision oncology registry INFORM Open
Diffuse midline glioma (DMG; a subtype of pediatric high-grade glioma) is a fatal disease in children, due to the localization in critical structures of the central nervous system, its invasive nature, and limited treatment options. Molecu…
View article: The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science
The children's brain tumor network (CBTN) - Accelerating research in pediatric central nervous system tumors through collaboration and open science Open
View article: Temozolomide-based radio-chemotherapy for newly diagnosed pediatric high-grade gliomas (HIT-HGG-2007): A prospective, multicenter, single-arm, phase II trial
Temozolomide-based radio-chemotherapy for newly diagnosed pediatric high-grade gliomas (HIT-HGG-2007): A prospective, multicenter, single-arm, phase II trial Open
Background: The HIT-HGG-2007 trial investigated temozolomide (TMZ) radio-chemotherapy for pediatric patients with high-grade gliomas (pHGG) to demonstrate therapeutic non-inferiority compared to previous intensive radio-chemotherapy regime…
View article: Advancing CNS tumor diagnostics with expanded DNA methylation-based classification
Advancing CNS tumor diagnostics with expanded DNA methylation-based classification Open
DNA methylation-based classification is integral to contemporary neuro-oncological diagnostics, as highlighted by the current World Health Organization (WHO) classification of central nervous system (CNS) tumors. We introduce the Heidelber…
View article: Author Correction: The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial
Author Correction: The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial Open
View article: Salvage therapies for first relapse of SHH medulloblastoma in early childhood
Salvage therapies for first relapse of SHH medulloblastoma in early childhood Open
Background Sonic hedgehog (SHH) medulloblastoma is the most common molecular group of infant and early childhood medulloblastoma (iMB) and has no standard of care at relapse. This work aimed to evaluate the post-relapse survival (PRS) and …
View article: Supplementary Data S1 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Data S1 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Data S1. Definition of response, Description of response criteria used by sites to evaluate tumor response, Definition of extend of resection, Definition of extend of resection used by sites.
View article: Supplementary Table S5 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Table S5 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Table S5. Method of NTRK fusion detection as reported by participating centers.
View article: Supplementary Table S4 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Table S4 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Table S4. Other molecular alterations reported.
View article: Supplementary Figure S1 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Figure S1 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Figure S1. Diagram of population patients.
View article: Supplementary Table S1 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Table S1 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Table S1. NTRK fusion type and histology.
View article: Supplementary Table S2 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Table S2 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Table S2. Responses based on clinical assessment for each line of therapy. Non-targeted therapy and TRKi given concurrently in a specific line were excluded.
View article: Data from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Data from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Purpose:Tropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients.Experimental Design:We conducted an international retrospec…
View article: Supplementary Figure S2 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Figure S2 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Figure S2. A) Hazard ratio for progression or death according to clinical characteristics. B) Hazard ratio for death according to clinical characteristics. LGG: Low-grade glioma, HGG: High-grade glioma
View article: Supplementary Table S3 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Supplementary Table S3 from Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Supplementary Table S3. Survival outcome.
View article: MSH2, MSH6, MLH1, and PMS2 immunohistochemistry as highly sensitive screening method for DNA mismatch repair deficiency syndromes in pediatric high-grade glioma
MSH2, MSH6, MLH1, and PMS2 immunohistochemistry as highly sensitive screening method for DNA mismatch repair deficiency syndromes in pediatric high-grade glioma Open
Pediatric high-grade glioma (pedHGG) can occur as first manifestation of cancer predisposition syndromes resulting from pathogenic germline variants in the DNA mismatch repair (MMR) genes MSH2 , MSH6 , MLH1 , and PMS2 . The aim of this stu…
View article: Case Report: Clinical awareness about the effect of laser interstitial thermal therapy on pediatric high-grade brain tumors after radiotherapy
Case Report: Clinical awareness about the effect of laser interstitial thermal therapy on pediatric high-grade brain tumors after radiotherapy Open
The use of magnetic resonance-guided laser interstitial thermal therapy (LITT) for the treatment of brain tumors and epileptic lesions has increased in the field of pediatric neurosurgery. However, very little is known about the effect of …
View article: Predicting Anatomical Brain Tumor Growth by Guided Denoising Diffusion Models
Predicting Anatomical Brain Tumor Growth by Guided Denoising Diffusion Models Open
View article: Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions
Clinical Characteristics and Outcomes of Central Nervous System Tumors Harboring NTRK Gene Fusions Open
Purpose: Tropomyosin receptor kinase (TRK) fusions are detected in less than 2% of central nervous system tumors. There are limited data on the clinical course of affected patients. Experimental Design: We conducted an international retros…
View article: Lenvatinib Plus Ifosfamide and Etoposide in Children and Young Adults With Relapsed Osteosarcoma
Lenvatinib Plus Ifosfamide and Etoposide in Children and Young Adults With Relapsed Osteosarcoma Open
Importance The combination of ifosfamide and etoposide (IE) is commonly used to treat relapsed or refractory osteosarcoma; however, second-line treatment recommendations vary across guidelines. Objective To evaluate whether the addition of…
View article: Acute toxicity of chemotherapy in central nervous system germ cell tumour patients according to age
Acute toxicity of chemotherapy in central nervous system germ cell tumour patients according to age Open
Background SIOP-CNS-GCT-II European trial was opened for the treatment of patients of any age with central nervous system germ cell tumour (CNS-GCT). Four courses of pre-irradiation chemotherapy were delivered. The influence of patient age…
View article: DIPG-77. SOMATIC<i>TP53</i>,<i>PIK3R1, NF1</i> OR<i>TERT</i> MUTATIONS ARE ASSOCIATED WITH INFERIOR CLINICAL OUTCOME IN H3K27M-ALTERED DIFFUSE MIDLINE GLIOMA
DIPG-77. SOMATIC<i>TP53</i>,<i>PIK3R1, NF1</i> OR<i>TERT</i> MUTATIONS ARE ASSOCIATED WITH INFERIOR CLINICAL OUTCOME IN H3K27M-ALTERED DIFFUSE MIDLINE GLIOMA Open
BACKGROUND H3K27-altered diffuse midline gliomas (DMGs) have poor prognosis with no standard of care therapy beyond radiation (RT). While RT prologues survival, not all patients respond. Prior work has shown somatic TP53 mutations predict …
View article: IMG-25. PREDICTING ANATOMICAL TUMOR GROWTH IN PEDIATRIC HIGH-GRADE GLIOMAS VIA DENOISING DIFFUSION MODELS
IMG-25. PREDICTING ANATOMICAL TUMOR GROWTH IN PEDIATRIC HIGH-GRADE GLIOMAS VIA DENOISING DIFFUSION MODELS Open
BACKGROUND Pediatric diffuse midline glioma (DMG) has a poor prognosis with radiotherapy as the standard of palliative care. Radiation strategies using individual geometric doseshaping have the potential to maximize patient benefit. This s…
View article: TRLS-14. PNOC022 REPORT: A COMBINATION THERAPY TRIAL USING AN ADAPTIVE PLATFORM DESIGN FOR PATIENTS WITH DIFFUSE MIDLINE GLIOMA AT INITIAL DIAGNOSIS, POST-RADIATION THERAPY, OR PROGRESSION
TRLS-14. PNOC022 REPORT: A COMBINATION THERAPY TRIAL USING AN ADAPTIVE PLATFORM DESIGN FOR PATIENTS WITH DIFFUSE MIDLINE GLIOMA AT INITIAL DIAGNOSIS, POST-RADIATION THERAPY, OR PROGRESSION Open
BACKGROUND Children with diffuse midline glioma (DMG) face dismal prognoses. PNOC DMG-ACT (DMG-Adaptive Combination Trial, PNOC022) is an open-label, international trial investigating the efficacy of combination therapies for patients with…
View article: TRLS-07. PNOC016: INTRATUMORAL PHARMACOKINETICS AND PHARMACODYNAMIC (PK/PD) RESULTS FROM A TARGET VALIDATION STUDY OF A NOVEL PAN-HDAC AND PI3K INHIBITOR, FIMEPINOSTAT, IN CHILDREN AND YOUNG ADULTS WITH NEWLY DIAGNOSED DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG), RECURRENT MEDULLOBLASTOMA (MB), AND RECURRENT HIGH-GRADE GLIOMA (HGG)
TRLS-07. PNOC016: INTRATUMORAL PHARMACOKINETICS AND PHARMACODYNAMIC (PK/PD) RESULTS FROM A TARGET VALIDATION STUDY OF A NOVEL PAN-HDAC AND PI3K INHIBITOR, FIMEPINOSTAT, IN CHILDREN AND YOUNG ADULTS WITH NEWLY DIAGNOSED DIFFUSE INTRINSIC PONTINE GLIOMA (DIPG), RECURRENT MEDULLOBLASTOMA (MB), AND RECURRENT HIGH-GRADE GLIOMA (HGG) Open
BACKGROUND An intact blood-brain barrier is a limitation to effective therapies for pediatric brain tumors. PNOC016 investigated the tumor penetration of an orally available, promising pan-HDAC and PI3K-inhibitor, fimepinostat, in high-ris…
View article: HGG-26. MLH1, MSH2, MSH6, AND PMS2 IMMUNOHISTOCHEMISTRY REPRESENTS A HIGHLY SENSITIVE SCREENING METHOD FOR MISMATCH REPAIR DEFICIENCY SYNDROMES IN PEDIATRIC HIGH-GRADE GLIOMA
HGG-26. MLH1, MSH2, MSH6, AND PMS2 IMMUNOHISTOCHEMISTRY REPRESENTS A HIGHLY SENSITIVE SCREENING METHOD FOR MISMATCH REPAIR DEFICIENCY SYNDROMES IN PEDIATRIC HIGH-GRADE GLIOMA Open
BACKGROUND Pediatric central nervous system (CNS) tumors can occur as first manifestations of cancer predisposition syndromes (CPS) resulting from pathogenic variants in DNA mismatch repair (MMR) genes MLH1, MSH2, MSH6, and PMS2. Early det…
View article: LGG-40. TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY PEDIATRIC LOW-GRADE GLIOMA (PLGG): REVERSIBLE DECREASES IN GROWTH VELOCITY IN THE PHASE 2 FIREFLY-1 TRIAL
LGG-40. TYPE II RAF INHIBITOR TOVORAFENIB IN RELAPSED/REFRACTORY PEDIATRIC LOW-GRADE GLIOMA (PLGG): REVERSIBLE DECREASES IN GROWTH VELOCITY IN THE PHASE 2 FIREFLY-1 TRIAL Open
BACKGROUND: Tovorafenib is an investigational, selective, CNS-penetrant, type II RAF inhibitor. The ongoing FIREFLY-1 (NCT04775485) phase 2 study (Kilburn LK, et al. Nat Med. 2023) of tovorafenib in BRAF-altered pLGG resulted in antitumor …
View article: GCT-19. SIOP CNS GCT II: PRESENTATION AND OUTCOME OF BIFOCAL NON-GERMINOMATOUS GERM CELL TUMOURS (NGGCTS)
GCT-19. SIOP CNS GCT II: PRESENTATION AND OUTCOME OF BIFOCAL NON-GERMINOMATOUS GERM CELL TUMOURS (NGGCTS) Open
BACKGROUND Between 2/2012 and 7/2018, 112 patients with non-germinoma were registered as intend-to-treat patients within the SIOP CNS GCT II trial: 88 patients were male and 24 female. Median age was 13 years (range: 0,1-25,1 years). 95 pa…
View article: GCT-20. PRESENTATION AND OUTCOME OF BIFOCAL GERMINOMA TREATED ACCORDING TO SIOP CNS GCT II
GCT-20. PRESENTATION AND OUTCOME OF BIFOCAL GERMINOMA TREATED ACCORDING TO SIOP CNS GCT II Open
BACKGROUND Between 2/2012 and 7/2018, 261 patients with germinoma were registered as intent-to-treat patients within the SIOP CNS GCT II trial. 209 patients were male/ 52 female. The median age was 14.1 years (range 5,0-41,0 years). 194 pa…