Craniopharyngioma Patients Research Articles

Postoperative hypothalamic-pituitary dysfunction and long-term hormone replacement in patients with childhood-onset craniopharyngioma.

Hypothalamic-pituitary axis dysfunction is a common complication in post-operative craniopharyngioma(CP) patients, and it greatly impacts the long-term quality of life of such patients. To better understand the effects of postoperative hypothalamic-pituitary dysfunction and long-term hormone replacement therapy in patients with childhood CP, we assessed approximately 200 patients with childhood-onset CP postoperatively. Clinical details of patients with childhood-onset CP who underwent sellar tumor resection in Beijing Children's Hospital and Beijing Tiantan Hospital from 2018 to 2019 were retrieved retrospectively. The participants were followed up to assess the effects of post-operative long-term hormone replacement therapy and assess the tumor recurrence rate. The median age of admission was 8.1 (1.8, 14.3) years. Headache (45.5%), visual impairment (39.5%), and nausea (33.0%) were the most common clinical manifestations. ACP accounted for 95% of all CP cases. The incidence of central adrenal insufficiency and central hypothyroidism within the first week after surgery was 56.2% and 70.3%, respectively. At the same time 85.5% of the patients required at least one dose of desmopressin to control urine output. Total survival and tumor recurrence rates were 98.6% and 26.1%, respectively, with a median follow-up time of 29.7 (19.0, 40.3) months. During the follow-up period, 28.1% patients met the diagnostic criteria for short stature, while 54.4% fit the criteria for obesity. In addition, 94.4% of the patients were taking at least one kind of hormone substitution, and 74.7% were taking three or more. The prevalence of levothyroxine, glucocorticoid, desmopressin, and growth hormone replacement therapy was 87.3%, 77.5%, 78.9% and 31.0%, respectively. The proportion of patients treated with the substitutive combination of levothyroxine, hydrocortisone, and desmopressin was 54.9%. This study is a large-sample systematic postoperative endocrine function evaluation of patients with childhood-onset CP. Due to the high prevalence of post-operative hypothalamic-pituitary dysfunction, patients with CP usually require long-term multiple hormone substitution therapy. Individualized management and accurate hormone replacement dosage for postoperative childhood-onset CP patients are of great importance.

Outcome after proton beam therapy versus photon-based radiation therapy in childhood-onset craniopharyngioma patients-results of KRANIOPHARYNGEOM 2007.

Proton beam therapy (PBT) is being increas16ingly used to treat residual craniopharyngioma (CP) after hypothalamus-sparing surgery. Compared to photon-based radiation therapy (XRT) with PBT, less irradiation in the penumbra reduces the scattered dose to critical organs neighboring but outside the area of treatment, minimizing the risk of sequelae. Between 2007 and 2019, 99 of 290 (34%) childhood-onset CP patients recruited in KRANIOPHARYNGEOM 2007 received external radiation therapy (RT) (65% PBT, 35% XRT). Outcome was analyzed in terms of survival, endocrinological and anthropometric parameters (BMI and height SDS), quality of life (QoL using PEDQOL), and functional capacity (FMH) with special regard to irradiation technique. PBT became predominant (used in 43% and 72% of all irradiated patients registered within the first and second halves of the recruitment period, between 2008 and 2013 and 2013 and 2018, respectively). Five-year event-free survival rates after PBT or XRT were comparable (92% ± 4% vs. 91% ± 4%, p = 0.42) and higher than for the whole cohort since diagnosis, including non-RT patients (37% ± 4%). Radiation doses to the hypothalamus and pituitary did not differ between PBT and XRT. Endocrine deficits due to disturbances of the hypothalamic-pituitary axis (HPA) were already common before irradiation. During the first 5 years after CP diagnosis/RT, no differences between PBT, XRT, and non-RT CP patients concerning functional capacity and anthropometric parameters have been obtained. Only for the PEDQOL domain "physical function", parental-assessed QoL was lower 12 months after PBT versus XRT or non-RT patients. QoL, functional capacity, degree of obesity, and endocrinopathy varied over time from diagnosis, but by 5 years, there was no significant difference between PBT and XRT upfront or delayed, nor was there any compromise in historic survival rates, which remained high >90%. RT of any type is extremely effective at stabilizing disease after hypothalamic-sparing surgery. The purported specific benefits of PBT-reducing sequelae are not proven in this study where the organ of critical interest is itself diseased, increasing an urgent need to better address and treat the tumor-induced endocrine harm from diagnosis in dedicated pituitary services. Other hypothesized benefits of PBT versus XRT on vascular events and secondary cancers await longer comparison. https://clinicaltrials.gov/study/, identifier NCT01272622.

THU216 The Influence Of Age At Diagnosis On Clinical Presentation And Prognosis Of Childhood-onset Craniopharyngioma

Abstract Disclosure: J. Beckhaus: None. C. Friedrich: None. S. Boekhoff: None. G. Calaminus: None. B. Bison: None. M. Eveslage: None. B. Timmermann: None. J. Flitsch: None. H.L. Muller: None. The association between age at diagnosis (AaD) and clinical presentation and prognosis of pediatric patients with craniopharyngioma (CP) is not clear. Seven hundred and nine patients diagnosed with adamantinomatous CP have been recruited between 1999 and 2021 in HIT-ENDO, KRANIOPHARYNGEOM 2000/2007, KRANIOPHARYNGEOM Registry 2019 and prospectively observed. In our cohort study, we analyzed clinical presentation and outcome in these CP patients with regard to different AaD: Infants and toddlers (under 2 years), early childhood (2-6 years), middle childhood (6-12 years), and early adolescence (12-18 years). Following a median follow-up of 8.4 years, functional capacity (FMH), overall and event-free survival (EFS), and quality of life (QoL) (PEDQOL) were evaluated. 45.4% of the patients presented with severe obesity (BMI > 3SDS) at last visit. Children with AaD <6y showed lower EFS but higher QoL than children with AaD >6y. Reduced functional capacity (FMH) percentiles were linked to higher BMI-SDS at last visit (rho=-0.125, 95% CI [-0.21; -0.04]). Posterior hypothalamic involvement and surgical lesion were independent risk factors for both, severe obesity at last visit (OR=2.94, 95% CI [1.73;5.08]) and events (HR=1.59, 95% CI [1.12;2.26]). In the PEDQOL body image domain, patients with posterior hypothalamic involvement and lesion report lower scores. Nearly half of all patients suffer from sequelae of severe obesity and the hypothalamic syndrome. A diagnosis of CP before the age of 6 years may enable patients to adjust to their limitations earlier, but it may also increase the risk of relapse. The cause of severe obesity and a lower QoL is not AaD but posterior hypothalamic involvement of CP. Presentation: Thursday, June 15, 2023

THU219 Vascular Damage After Craniopharyngioma

Abstract Disclosure: J. Beckhaus: None. S. Boekhoff: None. B. Bison: None. C. Friedrich: None. H.L. Muller: None. Craniopharyngiomas are rare, embryonic, malformational tumors of the (supra-)sellar region. Due to tumor and/or treatment-related hypothalamic lesions, patients develop morbid obesity. Obesity is a major vascular risk factor, leading to reduced 20-year overall survival and a 3- to 19-fold higher cardiovascular mortality after craniopharyngioma compared to the general population. An analysis within the Childhood Cancer Survivor Study (CCSS) revelaed a 10-fold higher relative risk for stroke in adult survivors of childhood/young adult cancers compared to their siblings. To study craniopharyngioma-specific risk factors for vascular damages we searched three databases (Pubmed, CINAHL and Web of Science) and included 49 articles after title and abstract screening. Eligible studies were case reports, cohort and cross-sectional studies. Neurovascular damages due to surgical interventions included cerebral infarcts, stroke, aneurysms, anterior/middle cerebral artery syndrome (ACA/MCA), cerebral venous sinus thrombosis, and seizures. Radiation-induced vascular disease described Moyamoya syndrome, radionecrosis and thrombosis. Tumor-induced inflammation is suspected to play a causal role in the etiology of vascular complications in proximity of the tumor. Therefore, early diagnostics such as echocradiography or cardial MRI and risk-adapted prevention and treatment options for vascular disease are needed to improve long-term prognosis after craniopharyngioma treatment. This review summarizes the available evidence for vascular disease after pediatric- and adult-onset craniopharyngioma to derive possible treatment pathways. Individualized, tertiary prevention programs are required for craniopharyngioma patients and survivors to overcome or reduce the risk of vascular complications and elevate their quality of survival. Presentation: Thursday, June 15, 2023

THU220 Legal Degree Of Disability In Childhood-Craniopharyngioma Survivors After Follow-up - Results Of The Hit-Endo Study

Abstract Disclosure: S. Boekhoff: None. M. Eveslage: None. J. Beckhaus: None. C. Friedrich: None. H.L. Muller: None. Craniopharyngiomas are rare low-grade embryonic malformational tumors of the sellar/parasellar area. After a diagnosis during childhood and adolescence, endocrine and hypothalamic long-term sequalae affect the prognosis. Financial resources for psychological rehabilitation and involvement of craniopharyngioma survivors are made available by a legal status of the degree of disability (GdB), according to the German Social Code Book V. This study analyzed the relationship between clinical/psychosocial traits and quality of life (QoL) measures and the resulting GdB. The HIT-Endo study included pediatric craniopharyngioma patients (%lt;18 years of age at the time of diagnosis) and diagnosed before the year 2000. The GdB was evaluated in a cohort of 108 patients following a median follow-up time of 16 years. The GdB at follow-up was evaluated in relation to endocrine, ophthalmological, neuropsychological, and psychosocial factors. 44 patients (41%) did not receive a GdB, three patients (3%) received a GdB of 30-40, 43 patients (40%) a GdB of 50-90 and 18 patients (17%) the maximal GdB of 100. Higher GdB was associated with lower levels education, higher body mass index (BMI SDS), more severe visual impairment, and hypothalamic involvement of the tumor. Patients with a GdB of 100 reported impairments in social and occupational settings, as well as loss of physical and cognitive function, fatigue, dyspnea, and pain problems. In addition, they performed worse on the German Daily Life Ability Scale (FMH) than individuals with lower GdBs. The GdB reflects the long-term effects of craniopharyngioma during childhood and adolescence and is linked to physical and psychosocial problems. A low functional capacity, as measured by FMH scale, may indicate the eligibility for a higher GdB of craniopharyngioma survivors. Presentation: Thursday, June 15, 2023

Blood Lipid Disorders in Post-Operative Craniopharyngioma Children and Adolescents and the Improvement with Recombinant Human Growth Hormone Replacement.

The high prevalence of hypothalamic obesity (HO) and dyslipidemia in individuals with craniopharyngioma (CP) following surgery is a cause for increasing concern. However, few studies have explored the lipid profile in pediatric CP patients, with inconsistent findings. In addition, the role of recombinant human growth hormone (rhGH) replacement remains unclear in these patients. To compare the blood lipid profile among post-operative craniopharyngioma children and adolescents with that among healthy controls and to reveal the effects of rhGH replacement. Data of 79 post-operative craniopharyngioma children and adolescents in our center were retrospectively collected. Sixty patients underwent rhGH replacement during the follow-ups. We selected 36 patients who received rhGH replacement therapy, while 20 patients received rhGH replacement for at least 1 year and had complete lipid data before and after treatment and compared them with 19 patients who did not receive rhGH replacement therapy. Craniopharyngioma patients had higher total cholesterol (TC) (5.17 vs 3.77 mmol/L), triglyceride (TG) (1.51 vs 0.73 mmol/L), and low-density lipoprotein cholesterol (LDL-C) (3.14 vs 2.10 mmol/L), and lower high-density lipoprotein cholesterol (HDL-C) (1.06 vs 1.39 mmol/L) than controls (all p < 0.001). The lipid profile of obese and non-obese patients was not significantly different. After rhGH replacement, TC was 0.90 mmol/L lower (p = 0.002) and LDL-C was 0.73 mmol/L lower (p = 0.010) than baseline. Although the baseline LDL-C was higher, patients with rhGH replacement had lower LDL-C (-0.73 mmol/L adjusted for age and sex, p = 0.045) after the initiation of replacement compared with patients without rhGH replacement. The lipid profile of obese and non-obese children and adolescents with craniopharyngioma was unfavorable, and rhGH replacement could improve their lipid profile.

Factors predicting outcomes of endoscopic endonasal approach in craniopharyngioma patients.

Endoscopic endonasal approach (EEA) is commonly used for resection of craniopharyngioma (CP). Treatment outcomes of EEA for CP were related to numerous factors; however, they have been evaluated in few studies. The objective of this study is to investigate factors associated with the outcomes of CP following this operation. The records of patients with CP, who underwent EEA at our institution from January 2014 to June 2022, were retrospectively reviewed. Surgical outcomes, including the extent of resection, visual recovery, and endocrinological outcomes, were reported. Clinical and radiographic factors were analyzed for their associations with treatment outcomes using logistic regression analyzes. This study cohort consisted of 28 patients with CP. Gross total resection (GTR) was achieved in 12 patients (43%). Post-operative visual status improved, stabilized, and deteriorated in 89%, 6%, and 6% of the patients, respectively. There were no patients recovered from pre-operative pituitary dysfunctions, while post-operative hypoadrenalism, hypothyroidism, and hypogonadism were found in 9 (36%), 11 (42%), and 4 (22%) patients, respectively. Post-operative permanent diabetic insipidus was found in 13 patients (50%). Greater suprasellar extension of the tumor was associated with a lower rate of GTR (P = 0.011). Diabetes mellitus (DM) was associated with poor visual recovery (P = 0.022). Larger tumor size and Puget grade 2 were associated with postoperative hypoadrenalism (P = 0.01 and 0.023, respectively). In addition, Puget grade 2 was associated with post-operative hypothyroidism (P = 0.017). For EEA in CP, the extent of resection could be determined by suprasellar extension of the tumor. DM was a poor predicting factor for visual recovery, while larger tumors and Puget grade 2 had a higher risk of post-operative hypopituitarism.

The Price of Success-The Long-Term Outcomes of Children with Craniopharyngioma-Two Institutions' Experience.

An analysis of patients below 21 years old treated due to craniopharyngioma in the years 1979-2022 was performed with the aim of evaluating the long-term outcome and treatment side-effects. The standard statistical tests were used, and 56 patients with a median age of 11 years were evaluated. Surgery was the primary treatment in 55 patients; however, in only 29 it was the only neurosurgical intervention. Eighteen children were treated with radiotherapy (RTH) in primary treatment. The most common neurosurgical side effects observed were visual and endocrine deficits and obesity, which were diagnosed in 27 (49%), 50 (91%), and 25 (52%) patients, respectively. Complications after RTH were diagnosed in 14 cases (32%). During the median follow-up of 8.4 years (range: 0.4-39.8 years), six patients died and the 5- and 10-year overall survival was 97% and 93%, respectively. Five-year progression-free survival for gross total resection, resection with adjuvant RTH, and non-radical resection alone was 83%, 68%, and 23%, respectively (p = 0.0006). Surgery combined with RTH provides comparable results to gross tumor resection in terms of oncologic outcome in craniopharyngioma patients. Adjuvant irradiation applied in primary or salvage treatment improves disease control. The rate of complications is high irrespective of improved surgical and radiotherapeutic management.

Development of multifocal glioblastoma after radiotherapy for craniopharyngioma: illustrative case.

Radiation-induced glioblastoma (GBM) in patients previously treated for craniopharyngioma is a rare phenomenon. To the authors' knowledge, only seven cases have previously been documented in the literature. Herein, the authors report a case of a patient presenting with a new diagnosis of multifocal GBM 15 years after having received adjuvant radiotherapy for a craniopharyngioma. Magnetic resonance imaging revealed an extensive enhancing infiltrative lesion in the right frontal lobe as well as two satellite lesions in the contralateral frontal lobe. Histopathology on biopsy was consistent with GBM. Even though this case is rare, it is nevertheless important to recognize GBM as a potential side effect of radiation. Long-term follow-up in postradiation craniopharyngioma patients is crucial for early detection.

IMG-01. LONGITUDINAL FMRI OF VISUAL, AUDITORY AND MOTOR FUNCTION IN PEDIATRIC CRANIOPHARYNGIOMA AFTER TREATMENT WITH PROTON RADIOTHERAPY

Abstract INTRODUCTION Craniopharyngioma accounts for approximately 4% of childhood central nervous system tumors and has a 5-year overall survival rate exceeding 90%. Survivors of childhood craniopharyngioma may experience neurologic sequalae, including visual and fine motor deficits, and subclinical hearing loss associated with tumor and treatment. Contemporary treatment includes radical surgery or more limited surgery and proton radiotherapy (PRT). Here we report on a longitudinal fMRI study probing visual, auditory, and motor function in pediatric craniopharyngioma patients. METHOD: The study (NCT01419067) was IRB approved and informed consent was obtained for each patient. Functional MR images were acquired at 4 time points: after surgery and before PRT (TP1), and 18-months (TP2), 36-months (TP3), and 60-months (TP4) after PRT. Data from a sensory survey fMRI task (visual: flashing checkerboard; auditory: dissonant ascending/descending pure tones; and sensory/motor: button pushing when stimuli were on) for 67 patients were analyzed at the 4 time points. The median age was 11.7 years (range 8.3-20.2 years) and the female/male ratio was 37/30. RESULTS Significant brain activation was seen in visual cortices, auditory cortices, and supplementary motor area (SMA) and motor cortices for all 4 time points. Over the time, visual and motor cortices showed minimum changes in activation; however, there was a small increase in SMA. For the auditory cortices, the activation volume in Heschl’s gyri increased bilaterally, and the lateralization index decreased (TP1: 70%; TP2: 46%; TP3: 39%; and TP4: 26%) due to larger increases in the right Heschl’s gyrus. CONCLUSION Increasing activation in Heschl’s gyri, especially on the right side, after PRT suggests decreasing efficiency in distinguishing tones in these patients. These findings may be associated with late effects involving the specific gyri or neuropathways affected by surgery or irradiation.

Evaluating Eating Behaviour, Energy Homeostasis, and Obesity in Childhood-Onset Craniopharyngioma: A Feasibility Study

Introduction: Craniopharyngiomas are rare brain tumours (incidence 1.1–1.7 cases/million/year). Although non-malignant, craniopharyngioma causes major endocrine and visual morbidities including hypothalamic obesity, yet mechanisms leading to obesity are poorly understood. This study investigated the feasibility and acceptability of eating behaviour measures in patients with craniopharyngioma to inform the design of future trials. Methods: Patients with childhood-onset craniopharyngioma and controls matched for sex, pubertal stage, and age were recruited. After an overnight fast, participants received the following measures: body composition, resting metabolic rate, oral glucose tolerance test including magnetic resonance imaging (patients only), appetite ratings, eating behaviour, and quality of life questionnaires, ad libitum lunch, and an acceptability questionnaire. Data are reported as median ± IQR, with effect size measure (Cliff’s delta) and Kendall’s tau for correlations, due to the small sample size. Results: Eleven patients (median age = 14 years; 5 F/6 M) and matched controls (median age = 12 years; 5 F/6 M) were recruited. All patients had received surgery, and 9/11 also received radiotherapy. Hypothalamic damage post-surgery was graded (Paris grading): grade 2 n = 6; grade 1 n = 1; grade 0 n = 2. The included measures were deemed highly tolerable by participants and their parent/carers. Preliminary data suggest a difference in hyperphagia between patients and controls (d = 0.5), and a relationship between hyperphagia with body mass index standard deviation score (BMISDS) in patients (τ = 0.46). Discussion: These findings demonstrate that eating behaviour research is feasible and acceptable to craniopharyngioma patients and there is an association between BMISDS and hyperphagia in patients. Thus, food approach and avoidance behaviours may be useful targets for interventions to manage obesity in this patient group.

Treatment of childhood-onset craniopharyngioma patients using proton beam therapy vs photon-based radiation therapy in the prospective Kraniopharyngeom 2007 trial

Searchable abstracts of presentations at key conferences in endocrinology ISSN 1470-3947 (print) | ISSN 1479-6848 (online)

Marital Status Independently Predicts Mortality in Adult Patients with Craniopharyngioma: A SEER-Based Study

The relationship between marital status and overall survival (OS) in adult patients with craniopharyngioma has not been explored in depth. We aimed to elucidate the impact of marital status on the prognosis of craniopharyngioma patients excluding bias from baseline demographics and treatment. We extracted 1539 patients diagnosed with craniopharyngioma between 2000 and 2019 from the Surveillance, Epidemiology, and End Results database and divided patients into 4 marital subgroups: married, single, divorced/separated, and widowed. Kaplan-Meier curves with a log-rank test were used to discern differences in OS between marital subgroups. Univariate and multivariate Cox regression were used to identify independent prognostic factors of mortality. There were 1539 eligible patients: 863 (56.1%) were married, 466 (30.3%) were single, 135 (8.8%) were divorced/separated, and 75 (4.9%) were widowed. Widowed patients had the worst mean OS, 5-year OS and 10-year OS at 84.2months, 58.0% and 26.9%, respectively. After stratifying patients by age, our multivariate analysis showed that marital status was an independent predictor of mortality in middle-aged craniopharyngioma patients (40-60years, P < 0.001), but not in young adults (18-39years, P=0.646) or elderly patients (>60years, P= 0.076). Among middle-aged patients, single (hazard ratio 1.72, confidence interval 1.19-2.47, P= 0.004) and divorced/separated patients (hazard ratio=2.29, confidence interval=1.49-3.54, P < 0.001) showed a higher risk of mortality compared to married patients (reference). Marital status is an independent prognostic factor predicting OS for middle-aged patients with craniopharyngioma. Providing additional social and psychological support for single and divorced/separated patients may improve outcomes for this vulnerable population.

Prognostic Factors and Treatment Impact on Overall Survival in Adult Craniopharyngioma

To examine the demographics, tumor characteristics, treatments, and clinical outcomes of a large adult craniopharyngioma population. The 2004-2018 National Cancer Database was queried to investigate adult patients with craniopharyngioma. Univariable and multivariable Cox hazard ratio analysis was conducted to analyze the overall survival (OS) impact of demographic and clinical variables. A total of 666 adult patients with craniopharyngioma were identified with a mean age of 51 years (standard deviation 16 years). On multivariable analysis, independent of demographic and clinical variables, increased age, uninsured status, Medicaid, Medicare, Charlson-Deyo Comorbidity Index of 2, and tumor size greater than 40 mm were independently associated with worse OS. There was no significant difference in survival between histologic subtypes. Gross total resection (GTR) (hazard ratio [HR] 0.602, 95% confidence interval [CI] 0.384-0.942, P= 0.026) and subtotal resection (STR) with adjuvant radiotherapy (HR 0.316, 95% CI 0.140-0.710, P= 0.005) were independently associated with improved OS. GTR with radiotherapy trended towards improved OS (HR 0.601, 95% CI 0.334-1.083, P=0.090), but STR alone and radiotherapy alone demonstrated no significant difference in survival compared with no treatment on multivariable analysis. Kaplan-Meier survival models demonstrated improved survival with GTR, GTR+ radiation therapy, and STR+ radiation therapy. Patients undergoing endoscopic resection had significantly lower GTR rates and greater rates of adjuvant radiotherapy compared with open approaches but no difference in OS. Adult patients with craniopharyngioma who underwent GTR or STR with adjuvant radiotherapy had significantly improved overall survival. Endoscopic approaches had lower rates of GTR but no difference in OS.

Comparison of neurocognitive and quality-of-life outcomes in pediatric craniopharyngioma patients treated with partial resection and radiotherapy versus gross-total resection only.

The optimal management of pediatric craniopharyngioma patients remains controversial, shifting from radical resection (gross-total resection [GTR]) to a more conservative approach with partial resection/biopsy followed by radiotherapy (PR+RT). To the authors' knowledge, no previous studies have compared neurocognitive and quality-of-life (QOL) outcomes between the two main treatments. In this study, the authors compared changes in intellectual, adaptive, and QOL scores in children treated for craniopharyngioma with GTR and those treated with PR+RT. Patients underwent annual neurocognitive and QOL evaluations for up to 10 years posttreatment, including the Full-Scale IQ, Verbal Comprehension Index (VCI), Perceptual Reasoning Index (PRI), Working Memory Index (WMI), and Processing Speed Index (PSI). Child- and parent-reported QOL scores and adaptive behavior in different domains were assessed. General linear mixed models were used to examine change in scores over time by treatment group with adjustment for significant covariates. Scores from 43 patients treated between 2009 and 2019 (21 GTR, 22 PR+RT) were examined. Within the PR+RT group, 9 patients had intensity-modulated RT and 13 had proton beam therapy. The treatment groups were similar in sex (44% male) and age (median 7.3 years). There were no significant differences in the trajectory of intellectual functioning or QOL scale scores between the two groups. However, patients who underwent GTR exhibited significant improvement over time in overall adaptive behavior (p = 0.04) and conceptual skills (p = 0.01), which was not observed in patients treated with PR+RT. Long-term pediatric craniopharyngioma survivors treated with GTR and PR+RT have similar intellectual function and QOL. Larger studies are needed to explore small but clinically significant differences between the two groups.

Legal Degree of Disability in Childhood-Craniopharyngioma Survivors During Long-Term Follow-Up

Cranioparyngiomas are rare low-grade embryonic malformational tumors of the sellar/parasellar region. The prognosis after diagnosis during childood and adolescence is influenced by (neuro)endocrine long-term sequelae. A legal status of the degree of disability (GdB), according to the German Social Code Book V that is worthy of support provides financial means for psychosocial integration and participation of craniopharyngioma survivors. HIT-Endo is a German registry study on craniopharyngioma patients aged≤18 years at diagnosis . In a sample of 108 patients, the degree of disability and the association with endocrine, ophthalmological, neuropsychological (QLQ-C30; MFI-20; FMH-scale) and psychosocial parameters was analyzed after a mean follow-up period of 16 years. 47 patients (43%) did not receive a GdB or received a GdB of 30-40, 43 patients (40%) a GdB of 50-90 and 18 patients (17%) the maximal GdB of 100. Higher GdB were associated with lower education, higher body mass index standard deviation and a higher degree of visual impairment and hypothalamic involvement of the craniopharyngioma. Patients with a GdB of 100 reported loss in physical and cognitive function, dyspnea, and pain (QLQ-C30), as well as fatigue (MFI-20), and limitations in social and occupational contexts. They further had a lower functional capacity (German daily life ability scale (FMH)) compared to those with a smaller GdB. The GdB is associated with psychosocial and physical impairments and reflects the long-term consequences of craniopharyngioma. A low functional capacity may indicate a high GdB in later life of craniopharyngioma survivors.

Risk Factors Associated with Postoperative Obesity in Childhood-Onset Craniopharyngioma Patients: A Case-Control Study

Identifying risk factors associated with obesity after craniopharyngioma (CP) resection is pivotal for the prediction and prevention of postoperative obesity. Although multiple elegant studies have investigated this issue, studies focusing on Asian pediatric patients are missing. Herein, we retrospectively analyzed the risk factors associated with obesity after childhood-onset CP surgery in our center, aiming to provide insights into approaches reducing the occurrence of postoperative obesity. The clinical data of 53 children with CP who met the inclusion criteria from July 2011 to August 2020 in our center were collected for retrospective analysis. Univariate and multivariate logistic retrospective analyses were used to identify independent risk factors contributing to postoperative obesity. A review of the available literature reporting the risk factors associated with obesity after CP surgery over the past twodecades was performed for comparison. The median age at diagnosis of this cohort was 11.0years, with a median follow-up of 44.0months (range= 8-119months). Eighteen (34.0%) experienced obesity at the last follow-up. Multivariate logistic regression analysis showed preoperative body mass index standard deviation score (odds ratio [OR], 1.71; 95% confidence interval [CI]: 1.01-2.90; P=0.046), preoperative hypothalamic involvement (OR, 29.38; 95% CI: 1.76-490.66; P=0.019), and age at diagnosis (OR, 0.76; 95% CI: 0.61-0.95; P=0.016) were independent risk factors for obesity after childhood-onset CP resection. Our results combined with previous literature support preoperative body mass index standard deviation score, preoperative hypothalamic involvement, and age at diagnosis are the independent risk factors associated with obesity after childhood-onset CP resection.

Endoscopic versus Nonendoscopic Surgery for Resection of Craniopharyngiomas

While surgery is a critical treatment option for craniopharyngiomas, the optimal surgical approach remains under debate. Herein, we studied a large cohort of craniopharyngioma patients to identify predictors of endoscopic surgery (ES) and to compare survival outcomes between patients undergoing ES versus nonendoscopic surgery (NES). The National Cancer Database was queried for patients receiving definitive surgical treatment in 2010-2016. Cox proportional hazards and propensity score-adjusted Kaplan-Meier analyses assessed mortality risk and overall survival, respectively. Predictors of surgical approach were evaluated via logistic regression. Of 1721 patients, 508 (29.5%) underwent ES, 877 (50.9%) were female, and the average age was 41.8± 21.3years. Matched ES and NES cohorts exhibited 5-year overall survival rates of 88.0% and 79.8%, respectively (P= 0.004). ES was associated with reduced mortality (Hazard Ratio= 0.634; 95% confidence interval [CI], 0.439-0.914; P= 0.015). Patients treated at academic facilities (Odds Ratio [OR]= 2.095; 95% CI, 1.529-2.904; P < 0.001) or diagnosed recently (OR= 1.132; 95% CI, 1.058-1.211; P < 0.001) were more likely to undergo ES, while those with tumor sizes >3cm (OR= 0.604; 95% CI, 0.451-0.804; P < 0.001) or receiving adjuvant radiotherapy (OR= 0.641; 95% CI, 0.454-0.894; P= 0.010) were more likely to receive NES. Surgical inpatient stays were significantly shorter with ES compared to NES (8.0 vs. 10.5days, P < 0.001). On linear regression, ES usage increased by 82.4% and NES usage decreased by 23.4% between 2010 and 2016 (R2= 0.575, P= 0.031). ES of craniopharyngioma was associated with reduced mortality and shorter inpatient stays compared to NES. Factors including tumor size, extent of resection, facility type, and year of diagnosis may predict receiving ES. There is a trend towards increased usage of ES for surgical management of craniopharyngiomas.

Risk factors for and predictive nomogram of overall survival in adult patients with craniopharyngiomas: A SEER population-based study.

Studies with relatively large sample size as well as long-term follow-up focusing on adult craniopharyngioma (CP) patients are still lacking. We attempted to identify independent prognostic factors and establish a nomogram model to estimate survival rates for adult CP patients.The Surveillance, Epidemiology, and End Results database was used to obtain data on patients with CP. Univariable and multivariable Cox analyses were utilized to identify the prognostic factors of adult CP patients. A survival prediction model was constructed and its predictive performance was also assessed.A total of 991 patients (695 in training group and 296 in validation group) were eligible for final inclusion. Multivariate Cox analysis presented that age at diagnosis, marital status, race, tumor size, and surgery type were statistically significant prognostic factors for overall survival (all P < .05). A graphical predicting nomogram model was developed to calculate the predicted patients’ survival probabilities at 1, 2, 5, and 10 years. The concordance indexes were 0.708 ± 0.019 and 0.750 ± 0.025 for the training and validation samples, respectively, demonstrating favorable discrimination abilities. Similarly, the time-dependent area under curve also showed overall satisfactory discrimination ability. Favorable consistencies between the predicted and actual survival were presented according to the calibration curves.An easy-to-use nomogram, being proven to be with reliable discrimination ability and accuracy, was established to help predict overall survival for adult patients with CP using the identified significant prognostic factors.

RARE-03. Vision-related quality of life in patients with childhood-onset craniopharyngioma – results of KRANIOPHARYNGEOM 2000 / 2007

Abstract BACKGROUND: Quality of life (QoL) has become a critical component of therapeutic outcomes in the survivors of childhood-onset adamantinomatous craniopharyngioma (CP) patients. Visual deficiency adversely affects daily functioning and QoL. This study aimed to report the vision-related QoL and associated factors in CP patients. PATIENTS AND METHODS: 120 CP patients recruited between 2001 and 2019 in KRANIOPHARYNGEOM 2000/2007 were included in this study. Mean age at CP diagnosis was 10 years (range: 1.3-16.8 years); age at study was 18 years (range: 6-33 years). The primary outcome measures were QoL assessed by PEDQOL, functional capacity measured by Fertigkeitenskala Münster-Heidelberg ability scale and risk factors associated with decreased QoL during 3 years follow-up in CP patients with and without visual impairment (VI). RESULTS: The most common presenting symptoms were headache (42%), VI (20%), and growth retardation (12%). After diagnosis, VI defined as visual acuity less than 20/40, was found in 87 (70%) patients. Ophthalmologic examination and PEDQOL score were evaluated at three months, one year, and 3 years after CP diagnosis. A difference in the parental assessment of CP patient social functioning within the family was found between patients with and without VI in the first year (48 vs. 39, p=0.017) and third year (43 vs. 37, p=0.011). For the PEDQOL domain autonomy, a difference in self- (51 vs. 45, p=0.029) and parental (47 vs. 42, p=0.048) assessment was observed 3 years after diagnosis. In terms of risk factors, tumor volume more than 21 mm3, incomplete tumor resection and optic atrophy at initial presentation were associated with VI during follow-up. CONCLUSIONS: VI has an impact on QoL after CP. Accordingly, early detection of VI, together with the assessment of the patient’s QoL and the provision of adequate support during follow-up plays an important role in minimizing adverse late effects after CP.