scholarly journals LGG-14. PRESENTATION CHARACTERISTICS AND TREATMENT OUTCOMES FOR PEDIATRIC OPTIC PATHWAY GLIOMAS IN QATAR

2021 ◽  
Vol 23 (Supplement_1) ◽  
pp. i34-i34
Author(s):  
Ata Maaz ◽  
Tayseer Yousif ◽  
Mohammed Abdulmajeed ◽  
Moegamad Ederies ◽  
Pedro Neri ◽  
...  
Keyword(s):  
Visual Impairment ◽  
Treatment Outcomes ◽  
Progression Free Survival ◽  
Neurofibromatosis Type ◽  
Visual Assessment ◽  
Optic Pathway ◽  
First Line ◽  
Visual Outcomes ◽  
First Line Therapy ◽  
Optic Pathway Gliomas

Abstract Objectives To review the presentation characteristics and treatment outcomes for pediatric optic pathway gliomas (OPG) in Qatar. Methods Retrospective review of data for children with OPG from January 2009 to February 2021. Presenting features, diagnostic imaging and indications for treatment were reviewed. Progression free survival (PFS) and overall survival(OS) were computed using standard statistical methods. Medical notes were also reviewed for visual outcomes. Results Nineteen patients were diagnosed with OPG during the study period. There were 10 (52%) females. Median age was 29 months (range 6–186) months. Eleven (57%) tumors were related to neurofibromatosis Type 1 (NF-1). Nine (47%) of OPG were located in optic nerves, 5 (26%) were chiasmatic/suprasellar, while the remaining 5(26%) involved a combination of structures. Seven(36%) children presented with oculo-visual symptoms. Another 7 were diagnosed on screening imaging for NF-1. Seven(36%) children had debulking surgery/biopsy, while the remaining patients were diagnosed on neuro-imaging alone. Thirteen (68%) patients were treated with chemotherapy and 2 received additional radiotherapy. Indications for non-surgical treatment included visual impairment (46%) and large/progressive tumor (54%). Carboplatin based regimes were used as first line chemotherapy for 76 % of patients. Five (38%) patients required more than one lines of treatment. OS and PFS at 36 months were 100% and 48%. Baseline visual assessment showed 5 children each (26%) had unilateral and bilateral visual impairment, while 9 (48%) had normal vision. Of the 6 children receiving chemotherapy for visual impairment, 2 (33%) showed improvement. Of the 7 children treated for large/progressive tumors, 3 (42%) showed partial response, 2(28%) had progressive disease and 1 had stable disease after the first line therapy. Conclusions Our results are in-keeping with international data for optic pathway gliomas. Early referral and diagnosis may improve visual outcomes for this group of tumors.

scholarly journals Neuroophthalmological management of optic pathway gliomas

2007 ◽  
Vol 23 (5) ◽  
pp. E1 ◽  
Author(s):  
Andrew G. Lee
Keyword(s):  
Growth Rate ◽  
Optic Nerve ◽  
Major Complication ◽  
Initial Step ◽  
Neurofibromatosis Type ◽  
Tumor Location ◽  
Optic Pathway ◽  
First Line ◽  
Younger Patients ◽  
Optic Pathway Gliomas

✓ The growth rate of optic pathway gliomas (OPGs) is unpredictable and quite variable, especially in children with neurofibromatosis Type 1 (NF1). Close neuroophthalmalogical clinical follow-up with serial imaging (magnetic resonance imaging of the brain with and without contrast enhancement) is the recommended initial step in management to establish the growth rate of the lesion in an individual patient. Typically, only symptomatic and/or radiographically growing tumors require treatment, and observation is the accepted first-line option. Although both chemotherapy and radiotherapy can stabilize growth or even decrease the size of tumors, chemotherapy, especially in younger patients, has fewer side effects than radiation therapy (such as secondary tumors, radiation necrosis, and Moyomoya disease) and is generally considered the first-line treatment for progressive lesions in younger patients. The tumor location defines prognosis in OPGs; optic nerve gliomas (ONG) have the lowest rate of complications and death, and optic chiasm and retrochiasmal gliomas the highest. Although the major complication of an OPG is visual loss, hypothalamic involvement can lead to death. Resection is an option for ONGs but is generally reserved for tumors confined to the optic nerve with poor or no vision, or for patients with severe, cosmetically unappealing proptosis, producing severe pain or exposure keratopathy in a blind eye. Resection is generally not an option for intrinsic chiasmal or retrochiasmal OPGs. Extrinsic (exophytic) components can be debulked surgically, and surgery can be performed for hydrocephalus (ventriculoperitoneal shunt placement). The approach to a patient with OPG must be individualized based on tumor location, radiographic or clinical progression, the presence of NF1, and a risk–benefit comparison for treatment.

Optic pathway gliomas: a review

2007 ◽  
Vol 23 (5) ◽  
pp. E2 ◽  
Author(s):  
Mandy J. Binning ◽  
James K. Liu ◽  
John R. W. Kestle ◽  
Douglas L. Brockmeyer ◽  
Marion L. Walker
Keyword(s):  
Progressive Disease ◽  
Clinical Course ◽  
Mass Effect ◽  
Neurofibromatosis Type ◽  
Low Grade ◽  
Optic Pathway ◽  
Intracranial Tumors ◽  
First Line ◽  
Optic Pathway Gliomas

✓Optic pathway gliomas represent approximately 3–5% of childhood intracranial tumors. They usually occur in children during the first decade of life and are seen in 11–30% of patients with neurofibromatosis Type 1 (NF1). Although these tumors are typically low-grade gliomas, the clinical course and natural history are highly variable, making treatment paradigms difficult. Overall, however, they are often indolent tumors that can be observed over time for progression without initial treatment, especially in patients with NF1. Chemotherapy is the first-line treatment for progressive tumors, and radiation therapy is reserved for patients with progressive disease who are older than 5–7 years. Surgery is reserved for large tumors causing mass effect or hydrocephalus and tumors confined to the orbit or unilateral optic nerve.

Outcome of neurofibromatosis type 1 patients treated with first line vinblastine for optic pathway gliomas: A Canadian multicenter study.

2015 ◽  
Vol 33 (15_suppl) ◽  
pp. 2019-2019 ◽  
Author(s):  
Alvaro Lassaletta ◽  
Katrin Scheinemann ◽  
Shayna M. Zelcer ◽  
Juliette Hukin ◽  
Beverley Wilson ◽  
...  
Keyword(s):  
Multicenter Study ◽  
Neurofibromatosis Type 1 ◽  
Neurofibromatosis Type ◽  
Optic Pathway ◽  
First Line ◽  
Optic Pathway Gliomas

Long-term visual outcomes of optic pathway gliomas in pediatric patients without neurofibromatosis type 1

2016 ◽  
Vol 129 (1) ◽  
pp. 173-178 ◽  
Author(s):  
Michael J. Wan ◽  
Nicole J. Ullrich ◽  
Peter E. Manley ◽  
Mark W. Kieran ◽  
Liliana C. Goumnerova ◽  
...  
Keyword(s):  
Neurofibromatosis Type 1 ◽  
Pediatric Patients ◽  
Neurofibromatosis Type ◽  
Optic Pathway ◽  
Visual Outcomes ◽  
Optic Pathway Gliomas

Follow-Up of Optic Pathway Gliomas in Children with Neurofibromatosis Type 1

1994 ◽  
Vol 25 (06) ◽  
pp. 295-300 ◽  
Author(s):  
Ch. Kuenzle ◽  
M. Weissert ◽  
E. Roulet ◽  
H. Bode ◽  
S. Schefer ◽  
...  
Keyword(s):  
Neurofibromatosis Type 1 ◽  
Neurofibromatosis Type ◽  
Optic Pathway ◽  
Optic Pathway Gliomas

Exploratory biomarker findings from cohort 2 of MODUL: An adaptable, phase 2, signal-seeking trial of fluoropyrimidine + bevacizumab ± atezolizumab maintenance therapy for BRAFwt metastatic colorectal cancer.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 3570-3570
Author(s):  
Josep Tabernero ◽  
Axel Grothey ◽  
Dirk Arnold ◽  
Michel Ducreux ◽  
Peter J. O'Dwyer ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Metastatic Colorectal Cancer ◽  
Maintenance Treatment ◽  
Predictive Biomarkers ◽  
Experimental Treatment ◽  
Progression Free Survival ◽  
Phase 2 ◽  
First Line ◽  
Tissue Samples ◽  
First Line Therapy

3570 Background: MODUL is an adaptable, phase 2, signal-seeking trial testing novel agents as first-line therapy for predefined subgroups of patients with metastatic colorectal cancer (mCRC). Previously reported findings demonstrated that adding atezolizumab to fluoropyrimidine (FP)/bevacizumab as first-line maintenance treatment after induction with FOLFOX + bevacizumab did not improve efficacy outcomes in BRAFwt mCRC. Given these efficacy results, exploratory assessments on tumour samples were conducted to provide insights into factors that might affect efficacy of maintenance treatment and provide guidance on appropriate therapeutic strategies for BRAFwt mCRC. Methods: In patients with BRAFwt tumours (Cohort 2), experimental treatment was FP/bevacizumab + atezolizumab. Primary efficacy endpoint: progression-free survival (PFS). Overall survival (OS) was a secondary endpoint. Archival tissue samples from 104 patients were analysed by immunohistochemistry (IHC) at HistoGeneX (PD-L1; CD8/GrB/FoxP3). SP142 antibody was used for PD-L1 IHC analysis, which evaluated PD-L1low (IC 0–1) vs PD-L1high (IC > 1) in correlation with PFS and OS in the control and experimental arms. CD8/GrB/FoxP3 triplex staining was also performed to evaluate potential correlations with efficacy. Results: 445 patients with BRAFwt mCRC were randomised (2:1 ratio) to maintenance treatment in Cohort 2. Archival samples from 104 patients were analysed: FP/bevacizumab + atezolizumab (n = 82); FP/bevacizumab (n = 22). The biomarker evaluable population (BEP) for PD-L1 was n = 81 for FP/bevacizumab + atezolizumab [PD-L1low n = 35 (43%); PD-L1high n = 46 (57%)] and n = 22 for FP/bevacizumab [PD-L1low n = 16 (72%); PD-L1high n = 6 (28%)]. The BEP for CD8/GrB was n = 50 for FP/bevacizumab + atezolizumab and n = 16 for FP/bevacizumab. No difference in PFS or OS was observed in the FP/bevacizumab + atezolizumab vs FP/bevacizumab arms for PD-L1high [PFS: HR = 1.5 (95% CI 0.45−4.8), p = 0.51; OS: HR = 1.3 (95% CI 0.38−4.1), p = 0.71] or PD-L1low [PFS: HR = 0.92 (95% CI 0.47−1.8), p = 0.81; OS: HR = 0.78 (95% CI 0.4−1.5), p = 0.48]. Similar results were observed with CD8/GrBhigh [PFS: HR = 0.73 (95% CI 0.27−2.0), p = 0.55; OS: HR = 0.66 (95% CI 0.24−1.8), p = 0.44], CD8/GrBlow [PFS: HR = 1.0 (95% CI 0.42–2.5), p = 0.96; OS: HR = 0.73 (95% CI 0.3–1.8), p = 0.5], FoxP3high [PFS: HR = 0.97 (95% CI 0.37−2.5), p = 0.95; OS: HR = 0.95 (95% CI 0.36−2.5), p = 0.91] and FoxP3low [PFS: HR = 0.73 (95% CI 0.29−1.9), p = 0.53; OS: HR = 0.5 (95% CI 0.19−1.3), p = 0.18]. Conclusions: These findings suggest that PD-L1, CD8/GrB and FoxP3 might not be predictive biomarkers in BRAFwt mCRC. Further analyses are needed to further evaluate potential predictive and prognostic factors of response in this setting. Clinical trial information: NCT02291289.

Pan-cancer evaluation of homologous repair deficiency somatic mutations and response to first-line anti-neoplastic therapy.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 10535-10535
Author(s):  
Jessica A Lavery ◽  
Samantha Brown ◽  
Gregory J. Riely ◽  
Philippe L. Bedard ◽  
Ben Ho Park ◽  
...  
Keyword(s):  
Somatic Mutations ◽  
De Novo ◽  
Stage Iv ◽  
Progression Free Survival ◽  
First Line ◽  
Major Mechanism ◽  
First Line Therapy ◽  
Oncogenic Mutation ◽  
Line Therapy ◽  
Response To Chemotherapy

10535 Background: Homologous recombination is a major mechanism of defective DNA repair, but it remains uncertain whether homologous repair deficient (HRD) tumors have favorable prognosis or are more/less likely to respond to treatment than tumors lacking such mutations. Objective: To determine whether lung (NSCLC) and colorectal (CRC) HRD+ tumors have better survival or response to chemotherapy than HRD- tumors. Methods: Patients with de novo stage IV NSCLC or CRC who had next generation sequencing (NGS) between 2015-2018 from one of four cancer centers were identified. Records were curated using the PRISSMM framework to ascertain treatment, overall survival (OS) and progression free survival based on imaging (PFS-I) and oncologists’ notes (PFS-M). Each NSCLC or CRC tumor was categorized as HRD+ if NGS revealed an oncogenic/likely oncogenic mutation in: ATM, BAP1, BARD1, BLM, BRCA1, BRCA2, BRIP1, CHEK2, FAM175A, FANCA, FANCC, NBN, PALB2, RAD50, RAD51, RAD51C, RTEL1, or MRE11A based on the OncoKB database. The tumor was categorized as HRD- if no oncogenic mutation in any of these genes was evident and HRD indeterminate (HRD?) if no mutation was identified but the panel did not include all genes. OS, PFS-I and PFS-M from start of first line therapy were reported by HRD status. The percentage with a good response to first line therapy (≥2x the median) and exceptional response (≥3x the median) was estimated for each endpoint. Results: For NSCLC 4% were HRD+, 59% HRD- and 37% HRD?. For CRC there were 5% HRD+, 60% HRD- and 35% HRD?. There were no significant differences for any survival endpoint between patients who were HRD+ vs HRD- in univariable analyses. The proportion of good and exceptional responders to first line systemic chemotherapy also did not vary by HRD status, though patients with HRD+ CRC were potentially more likely to be exceptional responders. Similarly, no differences between HRD+ and HRD- tumors were apparent for the subgroup receiving platinum containing therapy. Conclusions: NSCLC and CRC patients with somatic mutations in HRD oncogenic genes did not differ from patients lacking such a mutation with respect to OS or PFS. CRC patients with HRD+ tumors may be more likely to be exceptional responders, but sample sizes are limited. By May, the analysis will include breast and pancreatic cancer cases.[Table: see text]

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