Childhood-onset Craniopharyngioma Research Articles

Daytime sleepiness and health-related quality of life in patients with childhood-onset craniopharyngioma

Daytime sleepiness and health-related quality of life in patients with childhood-onset craniopharyngioma

Serum sodium level fluctuations following the resection of childhood-onset craniopharyngioma.

Craniopharyngiomas are low-grade malignancies (WHO I) in the sellar region. Most cases of childhood-onset craniopharyngioma are adamantinomatous craniopharyngioma, and neurosurgery is the treatment of choice. Affected patients have postoperative complications, including water and electrolyte disturbances, because these malignancies develop near the hypothalamus and pituitary gland. Determining postoperative serum sodium fluctuation patterns in these patients can reduce postoperative mortality and improve prognosis. To measure changes in serum sodium levels in pediatric patients who underwent craniopharyngioma surgery and identify influencing factors. This retrospective study measured the serum sodium levels of 202 patients aged 0-18 years who underwent craniopharyngioma resection in Beijing Tiantan Hospital and Beijing Children's Hospital and identified predictors of severe hyponatremia and hypernatremia. The mean age of the cohort was 8.35±4.35 years. The prevalence of hypernatremia, hyponatremia, and their severe forms (serum Na+ >150mmol/L and serum Na+ <130mmol/L) within 14 days after surgery was 66.3%, 72.8%, 37.1%, and 40.6%, respectively. The mean postoperative serum sodium level showed a triphasic pattern, characterized by two peaks separated by a nadir. Sodium levels peaked on days 2 (143.6±7.6mmol/L) and 14 (143.2±6.7mmol/L) and reached their lowest on day 6 (135.5±7.5mmol/L). A total of 31 (15.3%) patients met the diagnostic threshold for hyponatremia and hypernatremia of the triphase response, whereas 116 (57.4%) patients presented this pattern, regardless of met the diagnostic criteria or not. The prevalence of severe hyponatremia varied depending on preoperative endocrine hormone deficiency, tumor status (primary or recurrent), and surgical approach. Serum sodium levels after craniopharyngioma resection in children showed a triphasic pattern in most cases. The risk of postoperative hyponatremia varied depending on preoperative endocrine hormone deficiency, tumor status (primary or recurrent), and surgical approach.

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

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

Vision-related quality of life in patients with childhood-onset craniopharyngioma

Quality of life (QoL) is a critical component of aftercare in survivors of childhood-onset craniopharyngioma (CP). Visual impairment adversely affects QoL after CP. This study assessed the frequency of visual impairment in patients with CP and its association with QoL. This study analyzed vision-related QoL in patients recruited 2000–2019 in the prospective cohort studies KRANIOPHARYNGEOM 2000/2007. Ophthalmologic examinations were performed at diagnosis, three, 12, and 36 months, respectively after the diagnosis. The QoL (PEDQOL) scores, were also evaluated at three, 12, and 36 months, respectively after the CP diagnosis. Multivariable logistic regression was used to analyze factors associated with visual impairment during follow-up. One-hundred twenty patients were included in this study. On ophthalmological examination, visual impairment was observed in the majority of the patients (n = 84, 70%) at CP diagnosis. After surgery, vision was restored in 27 patients (32%) with visual impairment at diagnosis. In the first (p = 0.017) and third (p = 0.011) year after diagnosis, parents of patients with visual impairment reported lower social functioning (family). Reduced autonomy was found three years after diagnosis in self- (p = 0.029) and parental (p = 0.048) assessments. Next to visual impairment at diagnosis, no additional risk factors for visual impairment during follow-up could be identified. Visual impairment has a clinically relevant impact on QoL after CP. The visual status at CP diagnosis determines the visual outcome during follow-up. Early detection of visual impairment, regular QoL assessments, and risk-appropriate aftercare are recommended.Clinical Trial Registration KRANIOPHARYNGEOM 2000 (Clinical trial registration number: NCT00258453) and KRANIOPHARYNGEOM 2007 (Clinical trial registration number: NCT01272622).

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.

THU217 Vision-related Quality Of Life In Patients With Childhood-onset Craniopharyngioma - Results Of Hit-Endo And Kraniopharyngeom 2000/2007

Abstract Disclosure: P. Sowithayasakul: None. S. Boekhoff: None. J. Beckhaus: None. C. Friedrich: None. H.L. Muller: None. Quality of life (QoL) has become a critical component of therapeutic outcomes in the survivors of childhood-onset craniopharyngioma (CP). Visual deficiency adversely affects daily functioning and QoL in childhood CP. This cohort study aimed to report the vision-related QoL in CP patients. The primary outcome measure was pediatric QoL (PEDQOL score) in patients with and without visual impairment from presentation at the time of CP diagnosis to three-year follow-up. The risk factors associated with visual impairment were also analyzed. A total of 120 patients were included in this study, with a median age at diagnosis of 10 years (ranging from 1.3 to 16.8 years). The most common presenting symptoms at the time of CP diagnosis were headache (42%), visual impairment (20%), and growth retardation (12%). Visual impairment was found in 87 patients (70%). Ophthalmologic examination and PEDQOL scores, which were grouped into seven domains: physical functioning, cognition, social functioning friends, social functioning family, and body image, were evaluated at the third month, first-year, and third-year after diagnosis. Parents of children with visual impairment report worse social functioning in their families than parents of children without visual impairment, one (p=0.017) and three years (p=0.011) after diagnosis. After three years, children with visual impairment were found to have worse quality of life in the autonomy domain of self- (p=0.029) and parental assessment (p=0.048). High tumor volume (&amp;gt;21 cm³), radiation therapy and extrasellar tumor location were identified as potential risk factors for visual impairment. We conclude that visual impairment has a negative impact on QoL after CP. Early detection of visual impairment, regular QoL assessment, and risk-appropriate follow-up care are recommended. Presentation: Thursday, June 15, 2023

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.

Vision-related Quality of Life in Patients with Childhood-onset craniopharyngioma - Results of HIT-Endo and KRANIOPHARYNGEOM 2000/2007

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

Survival, obesity and quality of life after childhood-onset craniopharyngioma: The role of age at diagnosis and hypothalamic damage

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

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)

GH and Childhood-onset Craniopharyngioma: When to Initiate GH Replacement Therapy?

Craniopharyngioma is a benign brain tumor with frequent local recurrence or progression after treatment. GH replacement therapy (GHRT) is prescribed in children with GH deficiency resulting from childhood-onset craniopharyngioma. To evaluate whether a shorter delay of GHRT initiation after childhood-onset craniopharyngioma completion therapy increased the risk of a new event (progression or recurrence). Retrospective, observational, monocenter study. We compared a cohort of 71 childhood-onset patients with craniopharyngiomas treated with recombinant human GH (rhGH). Twenty-seven patients were treated with rhGH at least 12 months after craniopharyngioma treatment (>12-month group) and 44 patients before 12 months (<12-month group), among which 29 patients were treated between 6 and 12 months (6-12 month group). The main outcome was the risk of tumor new event (progression of residual tumor or tumor recurrence after complete resection) after primary treatment in the >12-month group and in the <12 month or in the 6- to 12-month group patients. In the >12-month group, the 2- and 5-year event-free survivals were respectively 81.5% (95% CI, 61.1-91.9) and 69.4% (95% CI, 47.9-83.4) compared with 72.2% (95% CI, 56.3-83.1) and 69.8% (95% CI, 53.8-81.2) in the <12-month group. The 2- and 5-year event-free survivals were the same in the 6- to 12-month group (72.4%; 95% CI, 52.4-85.1). By log-rank test, the event-free survival was not different between groups (P = .98 and P = .91).The median time for event was not statistically different.In univariate and multivariate analysis, the risk of craniopharyngioma new event was not associated with the GHRT time delay after craniopharyngioma treatment. No association was found between GHRT time delay after childhood-onset craniopharyngioma treatment and an increased risk of recurrence or tumor progression, suggesting GH replacement therapy can be initiated 6 months after last treatment for craniopharyngiomas.

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.

The effect of hypothalamic involvement and growth hormone treatment on cardiovascular risk factors during the transition period in patients with childhood-onset craniopharyngioma.

Hypothalamic damage may increase the risk of adulthood obesity and cardiovascular disease in patients with craniopharyngioma. We evaluated the effects of hypothalamic involvement (HI) and growth hormone (GH) discontinuation on cardiovascular risk factors during the transition period in patients with childhood-onset craniopharyngioma. Thirty-three patients (17 males, 16 females) underwent retesting for adult GH deficiency (GHD) between 2005 and 2020 at Seoul National University Children's Hospital. Postoperative HI was graded by Puget's criteria and data regarding GH replacement were collected. At retesting, body mass index (BMI), fasting blood glucose, insulin, high-density lipoprotein cholesterol (HDL-C), triglycerides, and blood pressure were assessed. The mean age of commencement and discontinuation of GH replacement for childhood GHD was 10.0±3.6 and 15.3±3.1 years, respectively. The mean age at retesting for adult GHD was 17.7±2.5 years. When patients were categorized by post-GH discontinuation duration, those with durations >6 months (n=27) showed lower HDL-C levels than those with <6 months (P=0.037). Patients with extensive HI (n=16) had higher BMI z-scores than did those with no HI or mild HI (P=0.020). Both the extent of HI and longer post-GH discontinuation duration were significantly predictive for decreased HDL-C levels (P<0.05, for both). The extent of HI and GH discontinuation duration during the transition period can increase cardiovascular risks in patients with childhood-onset craniopharyngioma.

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.

RARE-01. Cerebral infarction in childhood-onset craniopharyngioma patients: results of KRANIOPHARYNGEOM 2007

BACKGROUND: Cerebral infarction (CI) is a known vascular complication following treatment of suprasellar tumors. Risk factors for CI, incidence rate, and long-term prognosis are unknown for patients with childhood-onset craniopharyngioma (CP). METHODS: MRI of 244 CP patients, recruited between 2007 and 2019 in KRANIOPHA-RYNGEOM 2007, were reviewed for CI. Risk factors for CI and outcome after CI were analyzed. RESULTS: Twenty-eight of 244 patients (11%) presented with CI based on reference assessment of MRI. One CI occurred before initial surgery and one case of CI occurred after release of intracystic pressure by a cyst catheter. 26 of 28 CI were detected after surgical tumor resection at a median postoperative interval of one day (range: 0.5 - 53 days). Vascular lesions during surgical procedures were documented in 7 cases with CI. No relevant differences with regard to surgical approaches were found. In all 12 irradiated patients, CI occurred before irradiation. Multivariable analyses showed that hydrocephalus and gross-total resection at the time of primary diagnosis / surgery both were risk factors for CI. After CI, quality of life (PEDQOL) and functional capacity (FMH) were impaired. CONCLUSIONS: CI occurs in 11% of surgically-treated CP cases. Degree of resection and increased intracranial pressure are risk factors, which should be considered in the planning of surgical procedures for prevention of CI.

LTBK-02. Safety of growth hormone replacement therapy in childhood craniopharyngioma

Introduction: Treatment for childhood-onset craniopharyngioma (cCP) has shifted from complete to limited resection aiming to avoid additional hypothalamic morbidity. Up to 90% of cCP patients develop growth hormone deficiency (GHD). GH replacement therapy (GHRT) is of high importance for linear growth and metabolic state. Hardly any studies evaluated the optimal time to start GHRT in relation to tumor progression or recurrence. Our aim was to assess the effect of GHRT in cCP on tumor progression/recurrence. Methods: Patients with cCP diagnosed between 2001 and 2022, with at least one year of follow-up were included. Tumor progression/recurrence was defined as tumor progression/recurrence requiring intervention. Kaplan Meier and multivariable cox regression analyses were estimated for tumor progression/recurrence. Comparison was made between cCP patients with GHRT and without GHRT. Of the cCP patients receiving GHRT, those given GHRT ≤ 1 year of cCP diagnosis were compared to those given GHRT >1 year after cCP diagnosis. Results: Of 59 cCP patients, 52 were diagnosed with GHD and 51 (86.4%) received GHRT. Sixteen cCP patients (31.4%) developed tumor progression/recurrence during GHRT compared to four cCP patients (50.0%) without GHRT. Mean progression free survival (PFS) did not differ between cCP patients with or without GHRT (GHRT: 5.55 years 95% CI 3.74 - 7.36 vs. no GHRT: 3.69 years 95% CI 1.44 - 5.93). Of cCP patients who started GHRT ≤1 year after cCP diagnosis, 36.4% developed tumor progression/recurrence compared to 27.6% of cCP patients who received GHRT > 1 year after diagnosis (PFS: 8.45 years CI 95% 5.54 – 11.36 vs. 7.99 years CI 95% 6.03 – 9.94). Limited surgery was associated to tumor progression/recurrence (HR 6.99 CI 95% 2.10 – 23.25). Conclusion: GHRT does not seem to influence tumor progression/recurrence in cCP. These results support early initiation of GHRT in cCP patients to optimize linear growth and metabolic outcome.

RARE-09. Treatment of childhood-onset craniopharyngioma patients using proton beam therapy versus photon-based radiation therapy in the prospective KRANIOPHARYNGEOM 2007 trial

BACKGROUND: Proton beam therapy (PBT) compared to photon-based radiotherapy (XRT) offers the benefit to administer lower radiation doses to critical organs thereby possibly minimizing the risk of sequelae in patients with residual craniopharyngiomas (CP) after hypothalamus-sparing surgery. The validation in large CP patient cohorts is still pending. PATIENTS AND METHODS: Of 290 childhood-onset CP patients included 2007-2019 in the prospective multicenter trial KRANIOPHARYNGEOM 2007, 99 (34%) received external RT (65% PBT, 35% XRT). Outcome was compared between the different groups in terms of overall (OS) and event-free survival (EFS), quality of life (QoL using PEDQOL), functional capacity (FMH), and auxological data (BMI and height SDS) one, three and five years after irradiation/CP diagnosis. RESULTS: PBT became the predominant irradiation technique during the study period (used in 23% and 77% of all irradiated patients registered within the first and second half of the enrollment period, respectively). PBT as well as XRT were associated with high (p<0.001) EFS (PBT: 0.917 ± 0.040; XRT: 0.940 ± 0.041) compared to non-RT (EFS: 0.669 ± 0.044). OS was similar in all groups. No differences between PBT, XRT and non-RT CP patients concerning functional capacity and anthropometric parameters (height SDS, BMI SDS) have been obtained. Only in the PEDQOL domain “physical function”, proxy-assessed QoL was lower one year after PBT when compared to XRT treated and non-irradiated CP patients. CONCLUSION: PBT is similar efficient in preventing relapses and recurrences in childhood-onset CP patients. During follow-up, no clinically relevant differences between PBT and XRT in terms of QoL, functional capacity and degree of obesity as a marker of hypothalamic syndrome were detectable. While PBT is increasingly applied, studies on larger CP cohorts with longer follow-up after RT are warranted to analyze, whether it can prevent sequelae such as hypothalamic syndrome and severe obesity compared to XRT.

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 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.

Long-Term Outcomes of Paediatric-Onset Craniopharyngioma: A Retrospective Analysis from a Tertiary Care Centre in North India.

Craniopharyngiomas are associated with long-term morbidity in the form of hormone deficiencies, visual deficits, and hypothalamic obesity. To study the long-term outcomes, including cure rates, endocrine dysfunction, visual dysfunction, hypothalamic obesity, and mortality in pediatric-onset craniopharyngiomas. A retrospective data analysis of pediatric (onset <18 years) craniopharyngioma diagnosed between 2003 and 2018. Data were collected from electronic hospital records, case files, and direct patient interviews. The mean age at presentation was 10.4 ± 4.5 years (n = 62). The median duration of symptoms at diagnosis was 6 months (3-13 months). At presentation, central diabetes insipidus was present in four (6.5%), central hypothyroidism in 27 (43.5%), secondary adrenal insufficiency in 20 (32%) and delayed puberty in 15 (24%) patients. Hypothalamus was involved in 59/60 patients (98%). At last visit, 22.6% were obese in comparison to 4.6% at presentation, and anterior pituitary deficiency was present in 90% of the patients. Sixty-one percent patients (n = 62) had delayed puberty and 67% (n = 53) had short-stature. Out of 35 short children, nine (14%) children who received growth hormone had significant increase in height SD score (-3.8 (1.4) at start vs. -2.9 (1.2) at last follow-up; P = 0.008). Tumor progression was significantly less in the group that received RT compared to those who did not (8% vs. 39%; P = 0.002). Childhood-onset craniopharyngioma results in significant morbidity. The prevalence of pituitary hormones deficiency, visual deficits, and obesity are high at long-term follow-up. Incomplete tumor removal is also frequent. Thus, long-term monitoring is necessary for the timely management of the morbidities associated with craniopharyngioma.