Maria R. Pérez-Torres Lobato, Mariona Morell, Palma Solano-Páez, Marina Ortiz-Palacios, Ángela Menárgez, Melissa Panesso, Felisa Vázquez-Gómez, JLuis Moreno-Carrasco, Álvaro Lassaletta, Vicente Santa-María, Miriam Pavon-Mengual, Eduardo Quiroga, Miguel A. García-Ariza, Maria Mora-Matilla, Carmen Garrido-Colino, Jaime Verdú-Amoros, Cristina Nova-Lozano, María Vieito, Esteban X. Cordero-Asanza, Maria Clemente, Mónica Ramos-Albiat, Juan de las Morenas-Iglesias, Lucas Moreno, Anna Llort
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Cordero-Asanza, Maria Clemente, Mónica Ramos-Albiat, Juan de las Morenas-Iglesias, Lucas Moreno, Anna Llort","doi":"10.1002/pbc.31343","DOIUrl":null,"url":null,"abstract":"<div>\n \n \n <section>\n \n <h3> Purpose</h3>\n \n <p>We aim to describe the characteristics of patients with childhood-onset craniopharyngioma and to analyze factors that impair quality of life (QoL) in this population.</p>\n </section>\n \n <section>\n \n <h3> Methods</h3>\n \n <p>Multicenter national study including patients treated between 2008 and 2022, from 2 to 25 years of age diagnosed with craniopharyngioma. QoL was assessed once during patient's follow-up by age-adapted versions of Pediatric Quality of Life Inventory (PedsQL) questionnaire.</p>\n </section>\n \n <section>\n \n <h3> Results</h3>\n \n <p>Sixty-six patients were included. Median age at diagnosis was 5 years (interquartile range [IQR]: 3–8), while median follow-up was 7.4 years (IQR: 2.8–9.7). Most craniopharyngioma were suprasellar (93.9%), and 59.7% had hypothalamic involvement (HI). All patients underwent surgery, 44.4% received radiotherapy, and 23.6% intracystic therapy. Most frequent long-term complications were visual deficit (72.7%) and endocrine impairment (94.5%). Patients exhibited hypothyroidism requiring hormone replacement (92.4%), hypocortisolism (80.3%), diabetes insipidus (86.4%), and/or growth hormone therapy (50%). When parents evaluated QoL, PedsQL median score was 53.8 points out of 100 (IQR: 41–71.6). Higher scores were noted when patients assessed their own QoL (median score 64.8 [IQR: 57.3–81.8]), observing statistically significant differences (<i>p</i> = .019). QoL was impaired by repeated surgeries (<i>r</i> = −.44; <i>p</i> = .014), HI (median score 51.5 [IQR: 39–63.8] vs. 76.4 [59–84.8]; <i>p</i> = .001), radiotherapy (median score 51.9 [IQR: 38.1–61.3] vs. 63.8 [IQR: 49–82.5]; <i>p</i> = .02) and longer follow-up (<i>r</i> = −.3; <i>p</i> = .01).</p>\n </section>\n \n <section>\n \n <h3> Conclusion</h3>\n \n <p>In our study, most patients had significant comorbidities and low overall QoL scores, which was mainly affected by repeated surgery, radiation, and hypothalamic involvement. This reflects the need for further research and intensified studies of systemic therapy/alternate strategies to broaden the standard-of-care options, so that treatment-related sequalae can be avoided.</p>\n </section>\n </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"71 12","pages":""},"PeriodicalIF":2.4000,"publicationDate":"2024-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Long-term sequelae and quality of life after childhood-onset craniopharyngioma: Results of a Spanish multicenter study\",\"authors\":\"Maria R. Pérez-Torres Lobato, Mariona Morell, Palma Solano-Páez, Marina Ortiz-Palacios, Ángela Menárgez, Melissa Panesso, Felisa Vázquez-Gómez, JLuis Moreno-Carrasco, Álvaro Lassaletta, Vicente Santa-María, Miriam Pavon-Mengual, Eduardo Quiroga, Miguel A. 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引用次数: 0
摘要
目的：我们旨在描述儿童期颅咽管瘤患者的特征，并分析影响该人群生活质量（QoL）的因素：多中心全国性研究，包括2008年至2022年期间接受治疗的2至25岁颅咽管瘤患者。在患者随访期间，通过与年龄相适应的儿科生活质量量表（PedsQL）问卷对其生活质量进行一次评估：结果：共纳入66名患者。确诊时的中位年龄为5岁（四分位间距[IQR]：3-8岁），随访时间中位数为7.4年（IQR：2.8-9.7年）。大多数颅咽管瘤位于鞍上（93.9%），59.7%累及下丘脑（HI）。所有患者都接受了手术，44.4%接受了放射治疗，23.6%接受了囊内治疗。最常见的长期并发症是视力障碍（72.7%）和内分泌损伤（94.5%）。患者表现为甲状腺功能减退，需要补充激素（92.4%），皮质醇功能减退（80.3%），尿崩症（86.4%），和/或生长激素治疗（50%）。当家长评估 QoL 时，PedsQL 的中位数为 53.8 分（满分 100 分，IQR：41-71.6）。当患者评估自己的 QoL 时，得分更高（中位数为 64.8 分 [IQR：57.3-81.8]），观察到统计学上的显著差异 (p = .019)。重复手术（r = -.44；p = .014）、HI（中位数得分 51.5 [IQR：39-63.8] vs. 76.4 [59-84.8]；p = .001）、放疗（中位数得分 51.9 [IQR：38.1-61.3] vs. 63.8 [IQR：49-82.5]；p = .02）和较长的随访时间（r = -.3；p = .01）都会影响患者的 QoL：结论：在我们的研究中，大多数患者有明显的合并症，总体 QoL 评分较低，主要受反复手术、放射和下丘脑受累的影响。这反映出有必要进一步研究和加强系统治疗/替代策略的研究，以扩大标准治疗方案的选择范围，从而避免与治疗相关的后遗症。
Long-term sequelae and quality of life after childhood-onset craniopharyngioma: Results of a Spanish multicenter study
Purpose
We aim to describe the characteristics of patients with childhood-onset craniopharyngioma and to analyze factors that impair quality of life (QoL) in this population.
Methods
Multicenter national study including patients treated between 2008 and 2022, from 2 to 25 years of age diagnosed with craniopharyngioma. QoL was assessed once during patient's follow-up by age-adapted versions of Pediatric Quality of Life Inventory (PedsQL) questionnaire.
Results
Sixty-six patients were included. Median age at diagnosis was 5 years (interquartile range [IQR]: 3–8), while median follow-up was 7.4 years (IQR: 2.8–9.7). Most craniopharyngioma were suprasellar (93.9%), and 59.7% had hypothalamic involvement (HI). All patients underwent surgery, 44.4% received radiotherapy, and 23.6% intracystic therapy. Most frequent long-term complications were visual deficit (72.7%) and endocrine impairment (94.5%). Patients exhibited hypothyroidism requiring hormone replacement (92.4%), hypocortisolism (80.3%), diabetes insipidus (86.4%), and/or growth hormone therapy (50%). When parents evaluated QoL, PedsQL median score was 53.8 points out of 100 (IQR: 41–71.6). Higher scores were noted when patients assessed their own QoL (median score 64.8 [IQR: 57.3–81.8]), observing statistically significant differences (p = .019). QoL was impaired by repeated surgeries (r = −.44; p = .014), HI (median score 51.5 [IQR: 39–63.8] vs. 76.4 [59–84.8]; p = .001), radiotherapy (median score 51.9 [IQR: 38.1–61.3] vs. 63.8 [IQR: 49–82.5]; p = .02) and longer follow-up (r = −.3; p = .01).
Conclusion
In our study, most patients had significant comorbidities and low overall QoL scores, which was mainly affected by repeated surgery, radiation, and hypothalamic involvement. This reflects the need for further research and intensified studies of systemic therapy/alternate strategies to broaden the standard-of-care options, so that treatment-related sequalae can be avoided.
期刊介绍：
Pediatric Blood & Cancer publishes the highest quality manuscripts describing basic and clinical investigations of blood disorders and malignant diseases of childhood including diagnosis, treatment, epidemiology, etiology, biology, and molecular and clinical genetics of these diseases as they affect children, adolescents, and young adults. Pediatric Blood & Cancer will also include studies on such treatment options as hematopoietic stem cell transplantation, immunology, and gene therapy.
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