Cargando…

RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective

Craniopharyngiomas are rare, histologically benign, sellar/parasellar tumors with significant tumor and therapy related morbidity and impairment in quality of life (QOL). We report survey results from patients/families affected by childhood-onset craniopharyngioma to identify opportunities for impro...

Descripción completa

Detalles Bibliográficos
Autores principales: Marshall, Emily, Cheng, Sylvia, Crowley, Julia, McCormack, Shana, Rood, Brian, Hankinson, Todd, DeCuypere, Michael, Lam, Sandy, Goldman, Stewart, Boekhoff, Svenja, Muller, Hermann L, Velasco, Ryan, Storm, Phillip B, Resnick, Adam, Prados, Michael, Mueller, Sabine, Kline, Cassie, Malbari, Fatema
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9165153/
http://dx.doi.org/10.1093/neuonc/noac079.038
_version_ 1784720322022342656
author Marshall, Emily
Cheng, Sylvia
Crowley, Julia
McCormack, Shana
Rood, Brian
Hankinson, Todd
DeCuypere, Michael
Lam, Sandy
Goldman, Stewart
Boekhoff, Svenja
Muller, Hermann L
Velasco, Ryan
Storm, Phillip B
Resnick, Adam
Prados, Michael
Mueller, Sabine
Kline, Cassie
Malbari, Fatema
author_facet Marshall, Emily
Cheng, Sylvia
Crowley, Julia
McCormack, Shana
Rood, Brian
Hankinson, Todd
DeCuypere, Michael
Lam, Sandy
Goldman, Stewart
Boekhoff, Svenja
Muller, Hermann L
Velasco, Ryan
Storm, Phillip B
Resnick, Adam
Prados, Michael
Mueller, Sabine
Kline, Cassie
Malbari, Fatema
author_sort Marshall, Emily
collection PubMed
description Craniopharyngiomas are rare, histologically benign, sellar/parasellar tumors with significant tumor and therapy related morbidity and impairment in quality of life (QOL). We report survey results from patients/families affected by childhood-onset craniopharyngioma to identify opportunities for improvement in management. An anonymous REDCap survey was distributed via social media and clinic visits to patients/families of craniopharyngioma survivors. Survey questions investigated perspectives on clinical management and functional and survival outcomes at initial diagnosis and recurrence. A total of 159 patients/families completed the survey, 40% (n=64) reported craniopharyngioma recurrence. For primary craniopharyngioma, maximal safe resection was the most frequent treatment reported (n=84), followed by partial resection (n=40), radiation (n=8), biopsy (n=5), and chemotherapy (n=3). Most patients (n=120) decided on a treatment plan within one week, 63 (40%) decided in one day. For recurrent craniopharyngioma, maximal safe resection and radiation were the most frequent interventions (n=33 each), followed by partial resection (n=13), chemotherapy (n=4) and biopsy (n=2). Multiple treatment options and/or participation in a clinical trial were offered to similar numbers of patients across primary and recurrent diagnoses (~21% for each). Most recurrent craniopharyngioma patients decided on management within one week (n=43). Long term effects related to tumor and treatment were identified as the primary concern in all respondents. The most common deficits for all patients were neuro-endocrine followed by vision and neurocognition problems. Neuro-endocrine complications were self-reported as the biggest impact on QOL. Families reported that they would prefer treatment options with the potential for improved QOL, even if these options also carried an increased risk of recurrence. Craniopharyngioma continues to be predominantly treated with surgery and radiation initially and with recurrence. Survivors have multiple comorbidities, with an interest in targeted therapies that preserve QOL. Novel therapies to prevent co-morbidities and provide long term benefits are necessary and upcoming.
format Online
Article
Text
id pubmed-9165153
institution National Center for Biotechnology Information
language English
publishDate 2022
publisher Oxford University Press
record_format MEDLINE/PubMed
spelling pubmed-91651532022-06-05 RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective Marshall, Emily Cheng, Sylvia Crowley, Julia McCormack, Shana Rood, Brian Hankinson, Todd DeCuypere, Michael Lam, Sandy Goldman, Stewart Boekhoff, Svenja Muller, Hermann L Velasco, Ryan Storm, Phillip B Resnick, Adam Prados, Michael Mueller, Sabine Kline, Cassie Malbari, Fatema Neuro Oncol Craniopharyngioma and Rare Tumors Craniopharyngiomas are rare, histologically benign, sellar/parasellar tumors with significant tumor and therapy related morbidity and impairment in quality of life (QOL). We report survey results from patients/families affected by childhood-onset craniopharyngioma to identify opportunities for improvement in management. An anonymous REDCap survey was distributed via social media and clinic visits to patients/families of craniopharyngioma survivors. Survey questions investigated perspectives on clinical management and functional and survival outcomes at initial diagnosis and recurrence. A total of 159 patients/families completed the survey, 40% (n=64) reported craniopharyngioma recurrence. For primary craniopharyngioma, maximal safe resection was the most frequent treatment reported (n=84), followed by partial resection (n=40), radiation (n=8), biopsy (n=5), and chemotherapy (n=3). Most patients (n=120) decided on a treatment plan within one week, 63 (40%) decided in one day. For recurrent craniopharyngioma, maximal safe resection and radiation were the most frequent interventions (n=33 each), followed by partial resection (n=13), chemotherapy (n=4) and biopsy (n=2). Multiple treatment options and/or participation in a clinical trial were offered to similar numbers of patients across primary and recurrent diagnoses (~21% for each). Most recurrent craniopharyngioma patients decided on management within one week (n=43). Long term effects related to tumor and treatment were identified as the primary concern in all respondents. The most common deficits for all patients were neuro-endocrine followed by vision and neurocognition problems. Neuro-endocrine complications were self-reported as the biggest impact on QOL. Families reported that they would prefer treatment options with the potential for improved QOL, even if these options also carried an increased risk of recurrence. Craniopharyngioma continues to be predominantly treated with surgery and radiation initially and with recurrence. Survivors have multiple comorbidities, with an interest in targeted therapies that preserve QOL. Novel therapies to prevent co-morbidities and provide long term benefits are necessary and upcoming. Oxford University Press 2022-06-03 /pmc/articles/PMC9165153/ http://dx.doi.org/10.1093/neuonc/noac079.038 Text en © The Author(s) 2022. Published by Oxford University Press on behalf of the Society for Neuro-Oncology. https://creativecommons.org/licenses/by-nc/4.0/This is an Open Access article distributed under the terms of the Creative Commons Attribution-NonCommercial License (https://creativecommons.org/licenses/by-nc/4.0/), which permits non-commercial re-use, distribution, and reproduction in any medium, provided the original work is properly cited. For commercial re-use, please contact journals.permissions@oup.com
spellingShingle Craniopharyngioma and Rare Tumors
Marshall, Emily
Cheng, Sylvia
Crowley, Julia
McCormack, Shana
Rood, Brian
Hankinson, Todd
DeCuypere, Michael
Lam, Sandy
Goldman, Stewart
Boekhoff, Svenja
Muller, Hermann L
Velasco, Ryan
Storm, Phillip B
Resnick, Adam
Prados, Michael
Mueller, Sabine
Kline, Cassie
Malbari, Fatema
RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
title RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
title_full RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
title_fullStr RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
title_full_unstemmed RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
title_short RARE-13. Clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
title_sort rare-13. clinical management and functional and survival outcomes in pediatric craniopharyngioma, a patient and family perspective
topic Craniopharyngioma and Rare Tumors
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9165153/
http://dx.doi.org/10.1093/neuonc/noac079.038
work_keys_str_mv AT marshallemily rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT chengsylvia rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT crowleyjulia rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT mccormackshana rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT roodbrian rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT hankinsontodd rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT decuyperemichael rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT lamsandy rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT goldmanstewart rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT boekhoffsvenja rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT mullerhermannl rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT velascoryan rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT stormphillipb rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT resnickadam rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT pradosmichael rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT muellersabine rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT klinecassie rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective
AT malbarifatema rare13clinicalmanagementandfunctionalandsurvivaloutcomesinpediatriccraniopharyngiomaapatientandfamilyperspective