Treatment Of Craniopharyngiomas Research Articles

Endocrine sequelae after pediatric craniopharyngioma treatment: a single-center retrospective cohort study.

Craniopharyngiomas (CP) are rare brain tumors with a low mortality rate, but with significant morbidity, in part due to the various long-term endocrine sequelae related to hypothalamic/pituitary deficiencies. Our objective was to assess the prevalence of endocrine dysfunction and outcome after treatment of CP at our institution and to apply the novel diagnostic criteria for hypothalamic syndrome (HS). In addition, we give an overview of treatments already attempted for hypothalamic obesity (HO). This retrospective cohort study included children treated and followed up for CP at the pediatric oncology and endocrinology department at University Hospitals Leuven between January 2000 and December 2023. Clinical and endocrine characteristics were collected during a five-year period following diagnosis of CP. The Müller radiological criteria and the novel diagnostic criteria for HS were applied. A brief literature review regarding treatments already attempted for HO was conducted. 15 patients with pediatric CP were included in the study, all of whom developed endocrine sequelae over time. Seven patients (47 %) presented with at least one hormonal deficit, and eight patients (53 %) developed panhypopituitarism over time. HO was clinically confirmed in nine patients (60 %). 10 patients (67 %) met the diagnostic criteria for HS. Currently, no overall effective treatment strategies are available for HO. Long-term endocrine sequelae and HO are highly prevalent in pediatric CP. Continuing multidisciplinary care to improve the quality of life of these patients is necessary. International cooperation and further long-term prospective trials for the treatment of HO are needed.

Semaglutide as a promising treatment for hypothalamic obesity: a six-month case series on four females with craniopharyngioma

PurposePatients with hypothalamic pathology often develop hypothalamic obesity, causing severe metabolic alterations resulting in increased morbidity and mortality. Treatments for hypothalamic obesity have not proven very effective, although the glucagon-like peptide-1 receptor agonist semaglutide has been shown to have positive effects. We examined semaglutide’s effect on weight loss in a sample of patients with hypothalamic obesity.MethodsFour female patients with hypothalamic obesity resulting from treatment of craniopharyngiomas were treated with semaglutide for six months. Whole Body Dual-energy x-ray absorptiometry scans were performed, and blood samples drawn at baseline and after six months. Semaglutide dosages were increased monthly along with tracking of body weight and eating behavior (Three Factor Eating Questionnaire, TFEQ-R18).ResultsBMI was reduced in all cases, with an average of 7.9 BMI (range: 6.7 to 10.1) corresponding to a weight loss of 17.0% (range: 11.3–22.4%) or 20.2 kg (range 16.2 kg to 23.4 kg). We found a comparable reduction in total fat mass (17.2%, p = 0.006) and lean mass (16.0%, p = 0.05), whereas bone mass was unchanged (2.6%, p = 0.12). All cases reported an increase in energy levels, improved mobility and physical activity. Unfavorable eating behaviors were reduced after 1 month of treatment (emotional eating − 41 points, p = 0.02, uncontrolled eating − 23 points, p = 0.11). HbA1c and total cholesterol were significantly reduced (p = 0.014 for both).ConclusionSemaglutide is a promising and safe treatment option for HO, that improves eating behavior, reduces weight, and improves metabolic markers.

Using a Monte Carlo simulation to analyze the ideal activity for a phosphorus-32 polymeric brachytherapy source for paraspinal tumors

A promising radioactive source for use in intracavitary brachytherapy is phosphorus-32. This source has been prominent as a minimally invasive treatment for craniopharyngiomas and in the treatment of metastatic bone diseases in general and has been developed at Nuclear and Energy Research Institute (IPEN). In this work, a Monte Carlo simulation was used to evaluate the ideal activity this source must have to deliver a 1 Gy/min dose rate. TOPAS was used to model the simulation, and its geometry was simulated with the source centered in the origin point, inserted into an isotropic volume of water of 17.42 cm3. The source has the decay properties of phosphorus-32. The total dose was calculated using volumetric scorers. The results were promising, showing that the initial activity must be 86.14 mCi or 2871 MBq to obtain the 1 Gy/min dose rate. With the initial activity measured in this work, it is possible to minimize the patient’s exposure to radiation, while ensuring the quality of the treatment.

Use of moderate hypofractionated radiotherapy in craniopharyngioma

External beam radiation therapy is used for inoperable, residual, or recurrent disease after surgery for craniopharyngioma. At present, conventional radiotherapy, hypofractionated radiotherapy (HFRT), and stereotactic radiotherapy (SRT) are available options. However, there are limited data on HFRT and SRT using modern radiotherapy (RT) devices. This paper presents the findings of a retrospective study conducted at a single institution based on our long-term experiences with eight craniopharyngioma patients who had underwent RT. The median time between the operation and RT was 8.5 months (ranging from 2.6 to 11 months). Six patients were treated with CyberKnife® (CK®), and two were treated with Tomotherapy® (HT®) using the helical intensity-modulated RT technique (H-IMRT). Patients treated with HT received a mean of 50 and 55 Gy RT in 25 and 30 fractions (conventional RT scheme), respectively. Out of the six patients treated with CK, three received a mean of 22 – 25 Gy in five fractions, and the other three received a mean of 40 – 42 Gy in 15 fractions. The median follow-up was 6 years (range: 2 – 9.1 years), and no progression was observed. The 3-year overall survival (OS) and progression-free survival (PFS) rates were 85.7% and 85.7%, respectively. OS and PFS at 5 years were likewise 85.7% and 85.7%, respectively. Of the two patients (25%) who were alive with a complete response, one received 50 Gy in 25 fractions with H-IMRT, and the other received RT through CyberKnife with five fractions. Five patients (62.5%) were alive with stable residual disease and one patient had died with a complete response. HFRT may be considered as a preferable option in the treatment of craniopharyngioma, in addition to conventional or SBRT treatment options.

Long-Term Outcomes of Surgery and Radiation Treatment for Adult Patients with Craniopharyngioma

Treatment of craniopharyngioma typically entails gross total resection (GTR) or subtotal resection with adjuvant radiation (STR-RT). We analyzed outcomes in adults with craniopharyngioma undergoing GTR versus STR-RT. This retrospective study enrolled 115 patients with craniopharyngioma in 5 institutions. Patients with STR received postoperative RT with stereotactic radiosurgery or fractionated radiation therapy per institutional preference and ability to spare optic structures. Median age was 44 years (range, 19-79 years). GTR was performed in 34 patients and STR-RT was performed in 81 patients with median follow-up of 78.9 months (range, 1-268 months). For GTR, local control was 90.5% at 2 years, 87.2% at 3 years, and 71.9% at 5 years. For STR-RT, local control was 93.6% at 2 years, 90.3% at 3 years, and 88.4% at 5 years. At 5 years following resection, there was no difference in local control (P= 0.08). Differences in rates of visual deterioration or panhypopituitarism were not observed between GTR and STR-RT groups. There was no difference in local control in adamantinomatous and papillary craniopharyngioma regardless of treatment. Additionally, worse local control was found in patients receiving STR-RT who were underdosed with fractionated radiation therapy (P= 0.03) or stereotactic radiosurgery (P= 0.04). Good long-term control was achieved in adults with craniopharyngioma who underwent STR-RT or GTR with no significant difference in local control. First-line treatment for craniopharyngioma should continue to be maximal safe resection followed by RT as needed to balance optimal local control with long-term morbidity.

Endoscopic Endonasal Approach for Residual and Recurrent Craniopharyngioma after Transcranial Approach: A Multi-institutional Experience

Abstract Objectives While the endoscopic endonasal approach (EEA) has become a well-established surgery for resection of craniopharyngiomas (CP), the utility of this procedure following subtotal resection from open transcranial approach (TCA) surgery has yet to be explored. Here we present a multi-institutional case series of patients who underwent EEA for treatment of recurrent CP originally treated by TCA, demonstrating the viability of this approach as salvage surgery. Design Retrospective cohort. Setting Loyola University Medical Center and University of Pittsburgh Medical Center. Participants Patients who underwent EEA for recurrent CP following an initial TCA between 2003 and 2018. Main Outcome Measures Gross total resection (GTR) rate, surgical complications, visual outcomes, and endocrine outcomes Results Patients who underwent EEA for recurrent CP following a prior TCA had a GTR rate of 77%. EEA reoperation was not associated with worsening of visual outcomes as compared with the primary TCA (0% visual worsening after EEA reoperation vs. 40% after primary TCA, p = 0.055). While primary TCA resulted in worsening endocrinopathies in 100% of patients (including seven patients with permanent diabetes insipidus), subsequent EEA for re-resection of CP resulted in stable (30.8%) or improved (69.2%) endocrine function in all patients (p < 0.001). Conclusions EEA achieves reasonable GTR rates and is an effective, safe surgical option for recurrent CP postinitial TCA. This approach demonstrated stable or improved visual and endocrinologic outcomes in all patients within our two-institution series. Thus, EEA should be considered as an efficacious form of retreatment in cases of progressive and recurrent CP.

Efficacy of the Craniopharyngioma Resection via Expanded Endoscopic Endonasal Transsphenoidal Approach in the Treatment of Complex Craniopharyngioma and Its Effect on Pituitary Function and Complications of Patients.

Surgical resection is the most effective method for craniopharyngioma, with complex operations and a high incidence of complications, especially for complex craniopharyngioma. The study focuses on selecting a proper surgical method to treat complex craniopharyngioma. A clinical study was conducted in this direction to explore the efficacy of expanded endoscopic endonasal transsphenoidal approach (EETS) and transcranial approach (TCA) in the treatment of complex craniopharyngioma and their effects on pituitary function and complications of patients. The clinical data of 73 patients with complex craniopharyngioma in Baoding No.2 Central Hospital from December 2017 to December 2022 were retrospectively analyzed. 13 patients who did not meet the admission criteria were excluded, and 60 patients were finally included. The included patients were divided into the TCA and EETS groups according to the treatment method. The surgical conditions, total tumor resection rate, clinical remission rate, and complications of patients in two groups were compared. The pituitary function of all patients, including thyroid stimulating hormone (TSH), adrenocorticotropic hormone (ACTH), and human growth hormone (hGH), was compared. A total of 60 patients were finally included in the study, including 30 cases in the EETS group and 30 cases in the TCA group. The two groups had no statistical difference in baseline demographic characteristics and pathological types (p > 0.05). Compared with the TCA group, the EETS group had less intraoperative blood loss, operation time, tumor resection time, and hospitalization time (p < 0.001) and had a significantly higher total tumor resection rate and clinical remission rate (p < 0.05). The EETS group had higher ACTH and hGH levels than the TCA group (p < 0.05). There was no statistical difference between the two groups incidence of complications and disease recurrence rate (p > 0.05). The craniopharyngioma resection via EETS has a significant therapeutic effect in complex craniopharyngioma with a high total tumor resection rate and clinical remission rate, which can protect the pituitary function of patients and provide more benefits for patients.

Identification of core genes of craniopharyngioma angiogenesis based on single-cell nuclear transcriptome sequencing.

This study aimed to explore the core genes of craniopharyngioma angiogenesis for targeted vascular therapy based on single-cell nuclear transcriptome sequencing. For single-cell nuclear transcriptome sequencing, we collected six samples from the tumor center and adjacent hypothalamic tumor tissues from three patients with craniopharyngioma, as well as four normal brain tissues based on Gene Expression Omnibus. We screened genes with differential up-regulation between vascular endothelial cells of craniopharyngioma and those of normal brain tissues, performed GO and KEGG analysis, constructed the protein-protein interaction network, and selected key genes verified using immunofluorescence. After data cleaning and quality control, 623 craniopharyngioma endothelial cells and 439 healthy brain endothelial cells were obtained. Compared with normal brain endothelial cells, craniopharyngioma endothelial cells were screened for 394 differentially up-expressed genes (DEGs). GO and KEGG results showed that DEGs probably modulated endothelial cells, adherens junction, focal adhesion, migration, actin cytoskeleton, and invasion via the PI3K-AKT, Rap1, Ras, Wnt, and Hippo pathways. The core genes screened were CTNNB1, PTK2, ITGB1, STAT3, FYN, HIF1A, VCL, SMAD3, PECAM1, FOS, and CDH5. This study obtained possible anti-angiogenic genes in craniopharyngioma. Our results shed novel insights into molecular mechanisms and craniopharyngioma treatment.

Endoscopic Endonasal total Removal of a Suprasellar, Preinfundibular Retro Chiasmatic Craniopharyngioma: A Surgical Case Report

Craniopharyngiomas are benign tumors (WHO Grade I), seen in children and adults. Because of their location, they can require challenging clinical and surgical management. In fact, often, because of the presence of calcifications, of a capsule very strongly adherent to neurovascular structures, of the relationship with hypophysis, pituitary stalk, chiasm, carotids, the circle of Willis, basilar artery, and third ventricle, risk of mortality and morbidity is still mandatory. Various surgical techniques have been proposed: transcranial, transsphenoidal, and supraorbital approaches for surgical resection and treatment of craniopharyngiomas. Still, there is no common consent, but often the endonasal transsphenoidal extended procedures are considered the gold standard in many cases. We present a surgical technique of a case of complete surgical removal of an infundibular and retro chiasmatic craniopharyngioma, via an endoscopic endonasal transphenoidal transplanum approach.

Vascular Morbidity and Mortality in Craniopharyngioma Patients-A Scoping Review.

Craniopharyngioma (CP) treatment, including surgery and radiotherapy, can have short- and long-term vascular side effects. Hypothalamic damage is related to morbid obesity and may increase the lifelong risk of experiencing vascular events in CP patients. This review summarized the available evidence regarding vascular complications in adamantinomatous or papillary CP patients, whatever their age at diagnosis. Three databases (Medline, CINAHL, Web of Science) were searched (06/2023) to retrieve eligible articles. The search was limited to peer-reviewed articles. Titles, abstracts, and full texts were screened by two independent reviewers, and data were extracted using a self-developed grid. Seventy-two studies were included in this review; the majority were case reports. Reported vascular sequela that occurred due to surgery were fusiform dilation of the carotid artery, stroke, vasospasm, hemorrhage, and aneurysm. Related conditions that emerged due to radiotherapy included Moyamoya syndrome and cavernoma. Cardiovascular morbidity and mortality often lead to hypothalamic obesity and metabolic syndrome in CP patients. Vascular damage is a rare complication of CP treatment. Surgical strategies should protect the surrounding hypothalamic and vascular structures. Patients receiving radiotherapy, particularly at a young age, should undergo magnetic resonance angiography monitoring to identify possible neurovascular sequela during post-treatment care.

Performance and symptom validity indicators among children undergoing cognitive surveillance following treatment for craniopharyngioma.

Performance validity tests (PVTs) and symptom validity tests (SVTs) are essential to neuropsychological evaluations, helping ensure findings reflect true abilities or concerns. It is unclear how PVTs and SVTs perform in children who received radiotherapy for brain tumors. Accordingly, we investigated the rate of noncredible performance on validity indicators as well as associations with fatigue and lower intellectual functioning. Embedded PVTs and SVTs were investigated in 98 patients with pediatric craniopharyngioma undergoing proton radiotherapy (PRT). The contribution of fatigue, sleepiness, and lower intellectual functioning to embedded PVT performance was examined. Further, we investigated PVTs and SVTs in relation to cognitive performance at pre-PRT baseline and change over time. SVTs on parent measures were not an area of concern. PVTs identified 0-31% of the cohort as demonstrating possible noncredible performance at baseline, with stable findings 1 year following PRT. Reliable digit span (RDS) noted the highest PVT failure rate; RDS has been criticized for false positives in pediatric populations, especially children with neurological impairment. Objective sleepiness was strongly associated with PVT failure, stressing need to consider arousal level when interpreting cognitive performance in children with craniopharyngioma. Lower intellectual functioning also needs to be considered when interpreting task engagement indices as it was strongly associated with PVT failure. Embedded PVTs should be used with caution in pediatric craniopharyngioma patients who have received PRT. Future research should investigate different cut-off scores and validity indicator combinations to best differentiate noncredible performance due to task engagement versus variable arousal and/or lower intellectual functioning.

Chinese expert consensus on diagnosis and treatment of craniopharyngioma (2024)

Craniopharyngioma is a sellar tumor with benign pathology and malignant clinical manifestations. Partial resection plus radiotherapy, internal radiation therapy and chemotherapy are the conventional treatment strategies forcraniopharyngioma in most hospitals, which cause many patients to lose the best time to cure and make reoperation more difficult after recurrence. Therefore, the Pediatric Group of Chinese Society of Neurosurgery, Chinese Medical Association organized domestic experts from Pediatrics, Pediatric Neurosurgery, Genetics and Endocrinology to reach a consensus on the following topics: pathological and molecular genetic classification of craniopharyngioma, basic principles of curing tumors, surgical classification of tumors, surgical approach, perioperative and long-term treatment plan. This is expected to improve the quality of life and prognosis of patients, and further standardize the diagnosis and treatment of craniopharyngioma.

Quality of life of pediatric patients with craniopharyngioma: A retrospective series from a low-middle-income country with more than 4 years follow-up.

Craniopharyngiomas (CPs) are rare, low-grade tumors characterized by a range of debilitating symptoms. Most of the existing literature reports postoperative outcomes of the different treatment modalities of childhood CP. However, few studies have reported the impact of these different treatment methods on the quality of life (QoL) of survivors of childhood CP. Therefore, we aim to assess the correlation between different surgical modalities on the QoL of patients with childhood CP from a lower-middle-income country. Twenty-nine survivors who underwent treatment for CP were included in the study. The selected patients had either been managed with complete resection, debulking, or placement of an Ommaya reservoir. QoL was assessed by the pediatric quality of life (PedsQL) questionnaire. The effect of the different treatment modalities on the QoL was assessed. Mean follow-up was 4.4 ± 2.19 years. The type of surgery was significantly related to the mean PedsQL scores for the total score as well as each of the individual domain scores (P < 0.001). Complete resection of the tumor resulted in the lowest mean (standard deviation) PedsQL total score of 56.6 ± 7.12 compared to the Ommaya reservoir with biopsy (83.3 ± 5.69) and debulking (93.8 ± 3.37) (P < 0.001). There was a significant effect of the type of surgical treatment on the QoL of the survivors of childhood CP. It is important to consider the long-term outcomes in addition to immediate postoperative outcomes when deciding on a treatment strategy while managing children with CP.

Patient with congenital factor VII deficiency undergoing brain tumor neurosurgery successfully treated with recombinant factor VIIa and fresh frozen plasma: A case report and literature review

Rationale: Congenital factor VII deficiency is the most common among rare bleeding disorders, characterized by spontaneous or traumatic bleeding. The clinical manifestation is heterogeneous, ranging from asymptomatic phenotype to life-threatening hemorrhages. Intracranial hemorrhage is a common complication of brain tumor neurosurgery, which significantly challenges the perioperative management of patients with hemostatic defects. Patient concerns: This report presented a 55-year-old man with congenital factor VII deficiency, who had no history of hemorrhage or family history. He underwent a craniotomy for the treatment of papillary craniopharyngioma. Diagnoses: The patient was diagnosed as papillary craniopharyngioma, factor VII deficiency, and atrial fibrillation. Interventions: To prevent bleeding, a total of 8 doses of recombinant activated factor VII and 1 dose of fresh frozen plasma were administered as the perioperative replacement therapy. This scheme was guided by a pharmacodynamic evaluation, laboratory tests, and imaging examinations. Outcomes: No excessive surgical bleeding was observed during the 22-day treatment. The patient was found to have compound heterozygous mutations, Ala304Thr (c.910G &gt; A) and IVS5-2A &gt; G (c.572-2A &gt; G), in the F7 gene. Lessons: This is the first reported case in which surgical hemorrhage secondary to brain tumor resection was successfully controlled in the presence of congenital factor VII deficiency. Perioperative coagulation state, hemostasis, and thrombosis events should be closely observed, and the interval and dosage of recombinant factor VIIa should be adjusted accordingly.

Endonasal endoscopic surgical treatment of craniopharyngiomas in adult patients. Our experience

Objective: to estimate the results of surgical treatment of adult patients with craniopharyngiomas (CP), who underwent endoscopic endonasal surgical treatment. Materials and methods. Treatment of 69 adult patients with CPs who underwent endonasal endoscopic surgery from 2014 to 2021 is described. Gender division: women 42% (60.9%), men 27 (39.1%). The minimum age of patients is 19 years (women 19, men 20), the maximum age is 73 years (women 73, men 66). The mean age of all patients in the group was 46.7±14.5 years, women 47.6±15.5 years, men 45.1±12.6 years. In 52 patients (75.4%) the surgical intervention was primary, and in 17 patients (24.6%) it was secondary. All patients were estimated for preoperative and postoperative endocrine and neuro-ophthalmological status in dynamics. Results. No hemorrhagic or access-associated complications have been reported. Major complications: postoperative CSF leak - 9 patients (13%), meningitis - 10 patients (14.5%), 7 out of 8 patients with CSF leak had meningitis. 7 out of 10 patients with meningitis also had CSF leak. Other complications: acute tension pneumocephalus (2 cases, 2.9%), electrolyte disturbances (45 cases, 65.2%), severe diencephalic dysfunction (SDD) (4 cases, 5.8%). The postoperative mortality rate was 3 cases (4.35%), of which 1 due to meningitis (33.3% of all deaths), due to SDD 2 cases (66.6%). The dynamic changes of visual function were as follows: improvement in 36 patients (66.7% of all patients with preoperative visual impairment), no significant change in 7 patients (13%), deterioration in 10 patients (18.5%), 4 of which were transient. 40 patients had hypopituitarism prior to surgery, 10 of whom also had diabetes insipidus. A worsening of hypopituitarism was observed in 13 patients (32.5% of all patients with preoperative hypopituitarism) and an onset of hypopituitarism in 18 patients (26.1%). The onset of diabetes insipidus in the postoperative period occurred in 23 patients (33.3%), 7 of whom with regression. Conclusions. Endonasal endoscopic approach is no longer an alternative for surgical treatment of certain forms of craniopharyngiomas, but it is a method of choice for most CP cases. The postoperative mortality is low. The quality of life of the operated patients is satisfactory due to a high percentage of improvement in visual function and preservation of endocrine function. The rates of CSF leak and purulent-septic complications correlate with the data of the world literature. Modern standards of treatment require the establishment of a multidisciplinary team of specialists in healthcare institutions to personalize the treatment process in patients with CPs.

Advances in the treatment of Adamantinomatous craniopharyngioma: How to balance tumor control and quality of life in the current environment: a narrative review.

Adamantinomatous craniopharyngioma (ACP) presents a significant challenge to neurosurgeons despite its benign histology due to its aggressive behavior and unique growth patterns. This narrative review explores the evolving landscape of ACP treatments and their efficacy, highlighting the continuous development in therapeutic approaches in recent years. Traditionally, complete resection was the primary treatment for ACP, but surgical -related morbidity have led to a shift. The invasive nature of the finger-like protrusions in the histological structure results in a higher recurrence rate for ACP compared to papillary craniopharyngioma (PCP), even after complete macroscopic resection. Given this, combining subtotal resection with adjuvant radiotherapy has shown potential for achieving similar tumor control rates and potentially positive endocrine effects. Simultaneously, adjuvant treatments (such as radiotherapy, intracystic treatment, and catheter implantation) following limited surgery offer alternative approaches for sustained disease control while minimizing morbidity and alleviating clinical symptoms. Additionally, advances in understanding the molecular pathways of ACP have paved the way for targeted drugs, showing promise for therapy. There is a diversity of treatment models for ACP, and determining the optimal approach remains a subject of ongoing debate in the present context. In order to achieve a good-term quality of life (QOL), the main goal of the cyst disappearance or reduction of surgical treatment is still the main. Additionally, there should be a greater emphasis on personalized treatment at this particular stage and the consideration of ACP as a potentially chronic neurosurgical condition. This review navigates the evolving landscape of ACP therapies, fostering ongoing discussions in this complex field.

Surgical outcomes and follow-up results of 53 pediatric craniopharyngioma cases: a single-center study.

Craniopharyngiomas are locally invasive neoplasms, and they cause potential lifelong morbidity because of their tendency for local recurrence. Despite advancements in endoscopic techniques, gross-total resection (GTR) of tumors with invasion or adhesion to important surrounding anatomical structures is extremely difficult. The authors present a single-center study that evaluated the impact of the endoscopic endonasal approach (EEA) on the surgical outcomes of pediatric craniopharyngiomas, the factors affecting the resection rate, and recurrence. A total of 44 pediatric patients (age ≤ 18 years) who were treated via the EEA for craniopharyngioma from August 1997 to June 2022, as well as their 53 operations, were included in this study. The preoperative radiological configuration and surgical data of these cases were assessed. Also, preoperative and postoperative clinical (endocrinologic, neurological, and ophthalmological), hypothalamic, physical and social development, and neurocognitive assessment data were described. In total, 37 cases (69.8%) had no history of operation beforehand. The most common symptoms at presentation were endocrine disturbances (98.1%), headache without vomiting (84.3%), and visual disturbance (51%). Cases were classified as infrasellar (1.9%), sellar (32.1%), sellar-suprasellar (52.8%), and suprasellar (13.2%) localization. GTR was achieved in 34/53 cases (64.1%). The rate of GTR was higher in infrasellar and sellar tumors compared with sellar-suprasellar and suprasellar tumors (p = 0.003), and preoperative hypothalamic involvement was associated with lower likelihood of GTR (p = 0.024). Moreover, with experience, the rate of GTR increased (p = 0.037). Postoperative complications, other than endocrine impairment, occurred in 10/53 cases (18.9%). The mean duration of follow-up was 53.57 months. At follow-up, 21/53 (39.6%) cases presented with tumor recurrence. The 5-year progression-free survival (PFS) rate was 48.5%. There was a statistically significant difference between the GTR and other-than-GTR groups in terms of PFS (p < 0.001). According to univariate analysis, smaller tumor (p = 0.017), infrasellar and sellar localization (p = 0.031), and GTR (p < 0.001) were significantly associated with decreased rate of recurrence. Also, there was a statistically significant association between the recurrence rate and adhesion strength of the tumor (p < 0.001). This retrospective cohort study revealed surgical indications for EEA, as well as factors affecting the resection rate, recurrence, and quality of life during the follow-up period of the included cases. The authors believe that GTR should be the goal for craniopharyngioma treatment, but the authors' treatment approach was to provide a balance between radical surgery with maximum safety and adjuvant treatment for long-term disease control.

The impact of facility type and volume on treatment and overall survival in craniopharyngioma.

Craniopharyngiomas are uncommon benign sellar and parasellar tumors with high overall survival (OS) and recurrence rates. Treatment is often surgical but may include adjuvant therapies. The impact of adjuvant therapy and surgical approach have been evaluated, however, facility volume and type have not. The purpose of this study is to analyze the influence of facility volume and type on treatment modalities, extent of surgery and survival of craniopharyngioma. The 2004-2016 National Cancer Database (NCDB) was queried for patients diagnosed with craniopharyngioma. Facilities were classified by type (academic vs. non-academic) and low-volume center (LVC) (Treating < 8 patients over the timeline) versus high-volume center (HVC), (Treating ≥ 8 patients over the timeline). Differences in treatment course, outcomes, and OS by facility type were assessed. 3730 patients (51.3% female) with mean age 41.2 ± 22.0 were included with a 5-year estimated OS of 94.8% (94.0-95.5%). 2564 (68.7%) patients were treated at HVC, of which 2142 (83.5%) were treated at academic facilities. Patients treated at HVC's were more likely to undergo both surgery and radiation. Surgical approach at HVC was more likely to be endoscopic. Patients treated at HVC demonstrated significantly higher 5-year OS compared to patients treated at LVC (96% [95% CI 95.6-97.1% versus 91.2% [95% CI 89-92.7%] with lower risk of mortality (Hazard ratio [95% CI] = 0.69 [0.56-0.84]). Treatment of craniopharyngioma at HVC compared to LVC is associated with improved OS, lower 30- and 90-day postoperative mortality risk, and more common use of both radiotherapy and endoscopic surgical approach.

FRI066 Treatment History And Comorbidities Reported By Patients With Hypothalamic Obesity Treated With Setmelanotide In A Phase 2 Trial

Abstract Disclosure: C.L. Roth: Other; Self; Study funding from Rhythm Pharmaceuticals, Inc. A.H. Shoemaker: Advisory Board Member; Self; Rhythm Pharmaceuticals, Inc., Saniona. Other; Self; Study funding from Rhythm Pharmaceuticals, Inc. M. Gottschalk: Advisory Board Member; Self; Rhythm Pharmaceuticals, Inc. Other; Self; Study funding from Rhythm Pharmaceuticals, Inc. J. Miller: Other; Self; Study funding from Rhythm Pharmaceuticals, Inc. G. Yuan: Employee; Self; Rhythm Pharmaceuticals, Inc. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. S. Malhotra: Employee; Self; Rhythm Pharmaceuticals, Inc. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. C. Scimia: Employee; Self; Rhythm Pharmaceuticals, Inc. Stock Owner; Self; Rhythm Pharmaceuticals, Inc. M.J. Abuzzahab: Consulting Fee; Self; Pfizer, Inc., Consynance, Rhythm Pharmaceuticals, Inc., Endo Pharmaceuticals. Background: Hypothalamic obesity (HO) is an acquired form of severe obesity that can occur following surgical resection or radiotherapy of brain tumors. Multiple factors associated with the development of HO—including degree of hypothalamic damage from tumor treatment, comorbidities (eg, pituitary deficiencies), polypharmacy used to treat comorbidities, and unique pathophysiology—confer treatment challenges; HO is often unresponsive to lifestyle modifications and traditional obesity pharmacotherapies. In a Phase 2, 16-week trial of setmelanotide in patients with HO, 16 of 18 patients (88.9% [90% confidence interval, 69.0%-98.0%]; P&amp;lt;0.0001) achieved ≥5% body mass index (BMI) reduction from baseline, with a mean percent BMI change of −15.4% in patients adhering to treatment (n=17). We assessed patient histories (ie, tumor treatments, prior attempts to lose weight, and comorbidities) reported in the trial to further characterize this patient population. Methods: Patients aged ≥6 to ≤40 years with documented evidence of HO were enrolled in an open-label Phase 2 trial (NCT04725240) and received setmelanotide once daily for 16 weeks. The primary endpoint was the proportion of patients with ≥5% BMI reduction from baseline at Week 16. Tumor treatments, prior attempts to lose weight, and comorbidities are summarized narratively. Results: Eighteen patients with HO (age range, 6-24 years) were treated with setmelanotide. HO was diagnosed in patients following treatment of craniopharyngioma (n=14), hamartoma (n=3), or juvenile pilocytic astrocytoma (n=1). Six patients (33.3%) had tumor recurrence and/or multiple tumor treatments. Thirteen patients (72.2%) reported prior lifestyle modifications (ie, calorie restriction [n=4], low-carbohydrate diet [n=6], dietary tracking/modification [n=4], food access restriction [n=1], personal training [n=1]) and/or pharmacotherapy use (ie, exenatide [n=3], semaglutide [n=1], liraglutide [n=2], metformin [n=2], methylphenidate [n=2], dextroamphetamine [n=1], lisdexamfetamine [n=2], naltrexone [n=3], oxytocin [n=3], phentermine [n=1]) without substantial benefit. Comorbidities occurring in ≥25% of patients were diabetes insipidus (n=14), hypothyroidism (n=14), growth hormone deficiency (n=11), adrenal insufficiency (n=10), hypogonadism (n=8), pan/hypopituitarism (n=8), psychiatric disorders (n=8), vision problems (n=6), gastrointestinal symptoms (n=6), obstructive sleep apnea (n=5), and hypothalamic-pituitary disorder (n=3). All patients adhering to treatment (n=17, including those with pituitary deficiencies and common comorbidities)) experienced BMI reductions with setmelanotide. Conclusions: Despite complex medical histories, hypothalamic damage, and various prior failed interventions, setmelanotide reduced BMI in patients with HO in a Phase 2 trial. Presentation: Friday, June 16, 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