Very long-term outcomes of pediatric patients treated for optic pathway gliomas: A longitudinal cohort study

Abstract Background Optic pathway gliomas (OPGs) represent 5% of childhood brain tumors. Successive relapses lead to multiple treatments exposing to late complications. Methods We included patients treated at Gustave Roussy (GR) between January 1980 and December 2015 for OPG, before 18 years old and...

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Veröffentlicht in:Neuro-oncology (Charlottesville, Va.) Va.), 2024-07, Vol.26 (7), p.1310-1324
Hauptverfasser: Morin, Alice, Allodji, Rodrigue, Kariyawasam, Dulanjalee, Touraine, Philippe, Puget, Stéphanie, Beccaria, Kevin, De Carli, Emilie, Kieffer, Virginie, Rivollet, Sophie, Abbou, Samuel, Fayech, Chiraz, Souchard, Vincent, Dufour, Christelle, De Vathaire, Florent, Bolle, Stéphanie, Grill, Jacques, Fresneau, Brice
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container_issue 7
container_start_page 1310
container_title Neuro-oncology (Charlottesville, Va.)
container_volume 26
creator Morin, Alice
Allodji, Rodrigue
Kariyawasam, Dulanjalee
Touraine, Philippe
Puget, Stéphanie
Beccaria, Kevin
De Carli, Emilie
Kieffer, Virginie
Rivollet, Sophie
Abbou, Samuel
Fayech, Chiraz
Souchard, Vincent
Dufour, Christelle
De Vathaire, Florent
Bolle, Stéphanie
Grill, Jacques
Fresneau, Brice
description Abstract Background Optic pathway gliomas (OPGs) represent 5% of childhood brain tumors. Successive relapses lead to multiple treatments exposing to late complications. Methods We included patients treated at Gustave Roussy (GR) between January 1980 and December 2015 for OPG, before 18 years old and alive at 5 years from diagnosis. Mortality and physical health conditions data were extracted from medical data files and updated, thanks to the GR long-term follow-up program and French national mortality registry for patients included in the French Childhood Cancer Survivor Study. Results We included 182 5-year OPG-childhood survivors in the analysis (sex ratio M/F 0.8, 35% with neurofibromatosis type 1 [NF1]). With a median follow-up of 17.2 years (range = 5–41), we registered 82 relapses, 9 second malignancies, and 15 deaths as first events after 5 years, resulting in 20-year conditional overall survival (C-OS) and late events-free survival of 79.9% (95% confidence interval [CI] = 71–86) and 43.5% (95% CI = 36–51), respectively. Radiotherapy exposure in NF1 patients (hazard ratio [HR] = 6, 95% CI = 1.7–21.2) and hypothalamic involvement (HR = 3.2, 95% CI = 1.4–7.3) were significantly associated with C-OS in multivariable analyses. Ninety-five percent of 5-year OPG survivors suffered from any health condition, especially visual acuity “
doi_str_mv 10.1093/neuonc/noae045
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Successive relapses lead to multiple treatments exposing to late complications. Methods We included patients treated at Gustave Roussy (GR) between January 1980 and December 2015 for OPG, before 18 years old and alive at 5 years from diagnosis. Mortality and physical health conditions data were extracted from medical data files and updated, thanks to the GR long-term follow-up program and French national mortality registry for patients included in the French Childhood Cancer Survivor Study. Results We included 182 5-year OPG-childhood survivors in the analysis (sex ratio M/F 0.8, 35% with neurofibromatosis type 1 [NF1]). With a median follow-up of 17.2 years (range = 5–41), we registered 82 relapses, 9 second malignancies, and 15 deaths as first events after 5 years, resulting in 20-year conditional overall survival (C-OS) and late events-free survival of 79.9% (95% confidence interval [CI] = 71–86) and 43.5% (95% CI = 36–51), respectively. Radiotherapy exposure in NF1 patients (hazard ratio [HR] = 6, 95% CI = 1.7–21.2) and hypothalamic involvement (HR = 3.2, 95% CI = 1.4–7.3) were significantly associated with C-OS in multivariable analyses. Ninety-five percent of 5-year OPG survivors suffered from any health condition, especially visual acuity “&lt;1/10” (n = 109), pituitary deficiency (n = 106), and neurocognitive impairment (n = 89). NF1 (HR 2.1) was associated with precocious puberty. With a median time post-diagnosis of 4.2 years, 33 cerebrovascular events were observed in 21 patients. Conclusions Late relapses, second malignancies, and cerebrovascular diseases are severe late events resulting in premature mortality. Morbidity is high and needs after-cancer care to improve quality of life. Risk factors could be considered to better stratify long-term follow-up. For the podcast associated with this article, please visit ‘https://soc-neuro-onc.libsyn.com/website/long-term-outcomes-of-pediatric-patients-with-optic-pathway-glioma’</description><identifier>ISSN: 1522-8517</identifier><identifier>ISSN: 1523-5866</identifier><identifier>EISSN: 1523-5866</identifier><identifier>DOI: 10.1093/neuonc/noae045</identifier><identifier>PMID: 38465768</identifier><language>eng</language><publisher>US: Oxford University Press</publisher><subject>Adolescent ; Adult ; Cancer Survivors - statistics &amp; numerical data ; Child ; Child, Preschool ; Female ; Follow-Up Studies ; Humans ; Infant ; Infant, Newborn ; Life Sciences ; Longitudinal Studies ; Male ; Neoplasm Recurrence, Local - epidemiology ; Neoplasm Recurrence, Local - pathology ; Neurofibromatosis 1 - complications ; Neurofibromatosis 1 - mortality ; Neurofibromatosis 1 - pathology ; Neurofibromatosis 1 - therapy ; Optic Nerve Glioma - pathology ; Optic Nerve Glioma - therapy ; Prognosis ; Survival Rate</subject><ispartof>Neuro-oncology (Charlottesville, Va.), 2024-07, Vol.26 (7), p.1310-1324</ispartof><rights>The Author(s) 2024. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com. 2024</rights><rights>The Author(s) 2024. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. All rights reserved. For commercial re-use, please contact reprints@oup.com for reprints and translation rights for reprints. All other permissions can be obtained through our RightsLink service via the Permissions link on the article page on our site—for further information please contact journals.permissions@oup.com.</rights><rights>Distributed under a Creative Commons Attribution 4.0 International License</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c293t-1a182eeae637a3249877a4a46d9eb49773719c15cb5c04c91dfc68070945eb2c3</citedby><cites>FETCH-LOGICAL-c293t-1a182eeae637a3249877a4a46d9eb49773719c15cb5c04c91dfc68070945eb2c3</cites><orcidid>0000-0001-5993-8077 ; 0000-0002-8462-7753 ; 0000-0001-7603-7828 ; 0000-0002-3870-202X ; 0000-0002-8374-9281 ; 0000-0002-3405-1944 ; 0000-0003-4773-3001 ; 0009-0009-5259-0181</orcidid></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,314,780,784,885,1583,27922,27923</link.rule.ids><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/38465768$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink><backlink>$$Uhttps://hal.science/hal-04724535$$DView record in HAL$$Hfree_for_read</backlink></links><search><creatorcontrib>Morin, Alice</creatorcontrib><creatorcontrib>Allodji, Rodrigue</creatorcontrib><creatorcontrib>Kariyawasam, Dulanjalee</creatorcontrib><creatorcontrib>Touraine, Philippe</creatorcontrib><creatorcontrib>Puget, Stéphanie</creatorcontrib><creatorcontrib>Beccaria, Kevin</creatorcontrib><creatorcontrib>De Carli, Emilie</creatorcontrib><creatorcontrib>Kieffer, Virginie</creatorcontrib><creatorcontrib>Rivollet, Sophie</creatorcontrib><creatorcontrib>Abbou, Samuel</creatorcontrib><creatorcontrib>Fayech, Chiraz</creatorcontrib><creatorcontrib>Souchard, Vincent</creatorcontrib><creatorcontrib>Dufour, Christelle</creatorcontrib><creatorcontrib>De Vathaire, Florent</creatorcontrib><creatorcontrib>Bolle, Stéphanie</creatorcontrib><creatorcontrib>Grill, Jacques</creatorcontrib><creatorcontrib>Fresneau, Brice</creatorcontrib><title>Very long-term outcomes of pediatric patients treated for optic pathway gliomas: A longitudinal cohort study</title><title>Neuro-oncology (Charlottesville, Va.)</title><addtitle>Neuro Oncol</addtitle><description>Abstract Background Optic pathway gliomas (OPGs) represent 5% of childhood brain tumors. Successive relapses lead to multiple treatments exposing to late complications. Methods We included patients treated at Gustave Roussy (GR) between January 1980 and December 2015 for OPG, before 18 years old and alive at 5 years from diagnosis. Mortality and physical health conditions data were extracted from medical data files and updated, thanks to the GR long-term follow-up program and French national mortality registry for patients included in the French Childhood Cancer Survivor Study. Results We included 182 5-year OPG-childhood survivors in the analysis (sex ratio M/F 0.8, 35% with neurofibromatosis type 1 [NF1]). With a median follow-up of 17.2 years (range = 5–41), we registered 82 relapses, 9 second malignancies, and 15 deaths as first events after 5 years, resulting in 20-year conditional overall survival (C-OS) and late events-free survival of 79.9% (95% confidence interval [CI] = 71–86) and 43.5% (95% CI = 36–51), respectively. Radiotherapy exposure in NF1 patients (hazard ratio [HR] = 6, 95% CI = 1.7–21.2) and hypothalamic involvement (HR = 3.2, 95% CI = 1.4–7.3) were significantly associated with C-OS in multivariable analyses. Ninety-five percent of 5-year OPG survivors suffered from any health condition, especially visual acuity “&lt;1/10” (n = 109), pituitary deficiency (n = 106), and neurocognitive impairment (n = 89). NF1 (HR 2.1) was associated with precocious puberty. With a median time post-diagnosis of 4.2 years, 33 cerebrovascular events were observed in 21 patients. Conclusions Late relapses, second malignancies, and cerebrovascular diseases are severe late events resulting in premature mortality. Morbidity is high and needs after-cancer care to improve quality of life. Risk factors could be considered to better stratify long-term follow-up. For the podcast associated with this article, please visit ‘https://soc-neuro-onc.libsyn.com/website/long-term-outcomes-of-pediatric-patients-with-optic-pathway-glioma’</description><subject>Adolescent</subject><subject>Adult</subject><subject>Cancer Survivors - statistics &amp; numerical data</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>Female</subject><subject>Follow-Up Studies</subject><subject>Humans</subject><subject>Infant</subject><subject>Infant, Newborn</subject><subject>Life Sciences</subject><subject>Longitudinal Studies</subject><subject>Male</subject><subject>Neoplasm Recurrence, Local - epidemiology</subject><subject>Neoplasm Recurrence, Local - pathology</subject><subject>Neurofibromatosis 1 - complications</subject><subject>Neurofibromatosis 1 - mortality</subject><subject>Neurofibromatosis 1 - pathology</subject><subject>Neurofibromatosis 1 - therapy</subject><subject>Optic Nerve Glioma - pathology</subject><subject>Optic Nerve Glioma - therapy</subject><subject>Prognosis</subject><subject>Survival Rate</subject><issn>1522-8517</issn><issn>1523-5866</issn><issn>1523-5866</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2024</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkU1v1DAQhi0EoqVw5Yh8hENafzvmtqpoi7QSF-BqzTqTrlESB9sB7b9n2yzlyGk8M48fWX4JecvZJWdOXk24pClcTQmQKf2MnHMtZKNbY54_nkXTam7PyKtSfjAmuDb8JTmTrTLamvacDN8xH-iQpvumYh5pWmpIIxaaejpjF6HmGOgMNeJUC60ZoWJH-5Rpmuu62v-GA70fYhqhfKSbR1usSxcnGGhI-5QrLcf-8Jq86GEo-OZUL8i3m09fr--a7Zfbz9ebbROEk7XhwFuBCGikBSmUa60FBcp0DnfKWSstd4HrsNOBqeB41wfTMsuc0rgTQV6QD6t3D4OfcxwhH3yC6O82W_8wY8oKpaX-xY_s-5Wdc_q5YKl-jCXgMMCEaSleOK2F0VLYI3q5oiGnUjL2T27O_EMafk3Dn9I4Xnh3ci-7Ebsn_O_3_3toWub_yf4AcReXFg</recordid><startdate>20240705</startdate><enddate>20240705</enddate><creator>Morin, Alice</creator><creator>Allodji, Rodrigue</creator><creator>Kariyawasam, Dulanjalee</creator><creator>Touraine, Philippe</creator><creator>Puget, Stéphanie</creator><creator>Beccaria, Kevin</creator><creator>De Carli, Emilie</creator><creator>Kieffer, Virginie</creator><creator>Rivollet, Sophie</creator><creator>Abbou, Samuel</creator><creator>Fayech, Chiraz</creator><creator>Souchard, Vincent</creator><creator>Dufour, Christelle</creator><creator>De Vathaire, Florent</creator><creator>Bolle, Stéphanie</creator><creator>Grill, Jacques</creator><creator>Fresneau, Brice</creator><general>Oxford University Press</general><general>Oxford University Press (OUP)</general><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>1XC</scope><orcidid>https://orcid.org/0000-0001-5993-8077</orcidid><orcidid>https://orcid.org/0000-0002-8462-7753</orcidid><orcidid>https://orcid.org/0000-0001-7603-7828</orcidid><orcidid>https://orcid.org/0000-0002-3870-202X</orcidid><orcidid>https://orcid.org/0000-0002-8374-9281</orcidid><orcidid>https://orcid.org/0000-0002-3405-1944</orcidid><orcidid>https://orcid.org/0000-0003-4773-3001</orcidid><orcidid>https://orcid.org/0009-0009-5259-0181</orcidid></search><sort><creationdate>20240705</creationdate><title>Very long-term outcomes of pediatric patients treated for optic pathway gliomas: A longitudinal cohort study</title><author>Morin, Alice ; Allodji, Rodrigue ; Kariyawasam, Dulanjalee ; Touraine, Philippe ; Puget, Stéphanie ; Beccaria, Kevin ; De Carli, Emilie ; Kieffer, Virginie ; Rivollet, Sophie ; Abbou, Samuel ; Fayech, Chiraz ; Souchard, Vincent ; Dufour, Christelle ; De Vathaire, Florent ; Bolle, Stéphanie ; Grill, Jacques ; Fresneau, Brice</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c293t-1a182eeae637a3249877a4a46d9eb49773719c15cb5c04c91dfc68070945eb2c3</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2024</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Cancer Survivors - statistics &amp; numerical data</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>Female</topic><topic>Follow-Up Studies</topic><topic>Humans</topic><topic>Infant</topic><topic>Infant, Newborn</topic><topic>Life Sciences</topic><topic>Longitudinal Studies</topic><topic>Male</topic><topic>Neoplasm Recurrence, Local - epidemiology</topic><topic>Neoplasm Recurrence, Local - pathology</topic><topic>Neurofibromatosis 1 - complications</topic><topic>Neurofibromatosis 1 - mortality</topic><topic>Neurofibromatosis 1 - pathology</topic><topic>Neurofibromatosis 1 - therapy</topic><topic>Optic Nerve Glioma - pathology</topic><topic>Optic Nerve Glioma - therapy</topic><topic>Prognosis</topic><topic>Survival Rate</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Morin, Alice</creatorcontrib><creatorcontrib>Allodji, Rodrigue</creatorcontrib><creatorcontrib>Kariyawasam, Dulanjalee</creatorcontrib><creatorcontrib>Touraine, Philippe</creatorcontrib><creatorcontrib>Puget, Stéphanie</creatorcontrib><creatorcontrib>Beccaria, Kevin</creatorcontrib><creatorcontrib>De Carli, Emilie</creatorcontrib><creatorcontrib>Kieffer, Virginie</creatorcontrib><creatorcontrib>Rivollet, Sophie</creatorcontrib><creatorcontrib>Abbou, Samuel</creatorcontrib><creatorcontrib>Fayech, Chiraz</creatorcontrib><creatorcontrib>Souchard, Vincent</creatorcontrib><creatorcontrib>Dufour, Christelle</creatorcontrib><creatorcontrib>De Vathaire, Florent</creatorcontrib><creatorcontrib>Bolle, Stéphanie</creatorcontrib><creatorcontrib>Grill, Jacques</creatorcontrib><creatorcontrib>Fresneau, Brice</creatorcontrib><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>Hyper Article en Ligne (HAL)</collection><jtitle>Neuro-oncology (Charlottesville, Va.)</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Morin, Alice</au><au>Allodji, Rodrigue</au><au>Kariyawasam, Dulanjalee</au><au>Touraine, Philippe</au><au>Puget, Stéphanie</au><au>Beccaria, Kevin</au><au>De Carli, Emilie</au><au>Kieffer, Virginie</au><au>Rivollet, Sophie</au><au>Abbou, Samuel</au><au>Fayech, Chiraz</au><au>Souchard, Vincent</au><au>Dufour, Christelle</au><au>De Vathaire, Florent</au><au>Bolle, Stéphanie</au><au>Grill, Jacques</au><au>Fresneau, Brice</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Very long-term outcomes of pediatric patients treated for optic pathway gliomas: A longitudinal cohort study</atitle><jtitle>Neuro-oncology (Charlottesville, Va.)</jtitle><addtitle>Neuro Oncol</addtitle><date>2024-07-05</date><risdate>2024</risdate><volume>26</volume><issue>7</issue><spage>1310</spage><epage>1324</epage><pages>1310-1324</pages><issn>1522-8517</issn><issn>1523-5866</issn><eissn>1523-5866</eissn><abstract>Abstract Background Optic pathway gliomas (OPGs) represent 5% of childhood brain tumors. Successive relapses lead to multiple treatments exposing to late complications. Methods We included patients treated at Gustave Roussy (GR) between January 1980 and December 2015 for OPG, before 18 years old and alive at 5 years from diagnosis. Mortality and physical health conditions data were extracted from medical data files and updated, thanks to the GR long-term follow-up program and French national mortality registry for patients included in the French Childhood Cancer Survivor Study. Results We included 182 5-year OPG-childhood survivors in the analysis (sex ratio M/F 0.8, 35% with neurofibromatosis type 1 [NF1]). With a median follow-up of 17.2 years (range = 5–41), we registered 82 relapses, 9 second malignancies, and 15 deaths as first events after 5 years, resulting in 20-year conditional overall survival (C-OS) and late events-free survival of 79.9% (95% confidence interval [CI] = 71–86) and 43.5% (95% CI = 36–51), respectively. Radiotherapy exposure in NF1 patients (hazard ratio [HR] = 6, 95% CI = 1.7–21.2) and hypothalamic involvement (HR = 3.2, 95% CI = 1.4–7.3) were significantly associated with C-OS in multivariable analyses. Ninety-five percent of 5-year OPG survivors suffered from any health condition, especially visual acuity “&lt;1/10” (n = 109), pituitary deficiency (n = 106), and neurocognitive impairment (n = 89). NF1 (HR 2.1) was associated with precocious puberty. With a median time post-diagnosis of 4.2 years, 33 cerebrovascular events were observed in 21 patients. Conclusions Late relapses, second malignancies, and cerebrovascular diseases are severe late events resulting in premature mortality. Morbidity is high and needs after-cancer care to improve quality of life. Risk factors could be considered to better stratify long-term follow-up. For the podcast associated with this article, please visit ‘https://soc-neuro-onc.libsyn.com/website/long-term-outcomes-of-pediatric-patients-with-optic-pathway-glioma’</abstract><cop>US</cop><pub>Oxford University Press</pub><pmid>38465768</pmid><doi>10.1093/neuonc/noae045</doi><tpages>15</tpages><orcidid>https://orcid.org/0000-0001-5993-8077</orcidid><orcidid>https://orcid.org/0000-0002-8462-7753</orcidid><orcidid>https://orcid.org/0000-0001-7603-7828</orcidid><orcidid>https://orcid.org/0000-0002-3870-202X</orcidid><orcidid>https://orcid.org/0000-0002-8374-9281</orcidid><orcidid>https://orcid.org/0000-0002-3405-1944</orcidid><orcidid>https://orcid.org/0000-0003-4773-3001</orcidid><orcidid>https://orcid.org/0009-0009-5259-0181</orcidid></addata></record>
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source MEDLINE; Oxford University Press Journals All Titles (1996-Current)
subjects Adolescent
Adult
Cancer Survivors - statistics & numerical data
Child
Child, Preschool
Female
Follow-Up Studies
Humans
Infant
Infant, Newborn
Life Sciences
Longitudinal Studies
Male
Neoplasm Recurrence, Local - epidemiology
Neoplasm Recurrence, Local - pathology
Neurofibromatosis 1 - complications
Neurofibromatosis 1 - mortality
Neurofibromatosis 1 - pathology
Neurofibromatosis 1 - therapy
Optic Nerve Glioma - pathology
Optic Nerve Glioma - therapy
Prognosis
Survival Rate
title Very long-term outcomes of pediatric patients treated for optic pathway gliomas: A longitudinal cohort study
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