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Bakhsheshian J, Jin DL, Chang KE, et al.
Risk factors associated with the surgical management of craniopharyngiomas in pediatric patients: analysis of 1961 patients from a national registry database.
Neurosurg Focus. 2016; 41(6):E8 [PubMed] Related Publications

OBJECTIVE Patient demographic characteristics, hospital volume, and admission status have been shown to impact surgical outcomes of sellar region tumors in adults; however, the data available following the resection of craniopharyngiomas in the pediatric population remain limited. The authors sought to identify potential risk factors associated with outcomes following surgical management of pediatric craniopharyngiomas. METHODS The Nationwide Inpatient Sample database and Kids' Inpatient Database were analyzed to include admissions for pediatric patients (≤ 18 years) who underwent a transcranial or transsphenoidal craniotomy for resection of a craniopharyngioma. Patient-level factors, including age, race, comorbidities, and insurance type, as well as hospital factors were collected. Outcomes analyzed included mortality rate, endocrine and nonendocrine complications, hospital charges, and length of stay. A multivariate model controlling for variables analyzed was constructed to examine significant independent risk factors. RESULTS Between 2000 and 2011, 1961 pediatric patients were identified who underwent a transcranial (71.2%) or a transsphenoidal (28.8%) craniotomy for resection of a craniopharyngioma. A major predilection for age was observed with the selection of a transcranial (23.4% in < 7-year-olds, 28.1% in 7- to 12-year-olds, and 19.7% in 13- to 18-year-olds) versus transphenoidal (2.9% in < 7-year-olds, 7.4% in 7- to 12-year-olds, and 18.4% in 13- to 18-year-olds) approach. No significant outcomes were associated with a particular surgical approach, except that 7- to 12-year-old patients had a higher risk of nonendocrine complications (relative risk [RR] 2.42, 95% CI 1.04-5.65, p = 0.04) with the transsphenoidal approach when compared with 13- to 18-year-old patients. The overall inpatient mortality rate was 0.5% and the most common postoperative complication was diabetes insipidus (64.2%). There were no independent factors associated with inpatient mortality rates and no significant differences in outcomes among groups based on sex and race. The average length of stay was 11.8 days, and the mean hospital charge was $116,5 22. Hospitals with medium and large bed capacity were protective against nonendocrine complications (RR 0.53, 95% CI 0.3-0.93, p = 0.03 [medium]; RR 0.45, 95% CI 0.25-0.8, p < 0.01 [large]) and total complications (RR 0.73, 95% CI 0.55-0.97, p = 0.03 [medium]; RR 0.68, 95% CI 0.51-0.9, p < 0.01 [large]) when compared with hospitals with small bed capacity (< 200 beds). Patients admitted to rural hospitals had an increased risk for nonendocrine complications (RR 2.56, 95% CI 1.11-5.9, p = 0.03). The presence of one or more medical comorbidities increased the risk of higher total complications (RR 1.38, 95% CI 1.14-1.68), p < 0.01 [1 comorbidity]; RR 2.37, 95% CI 1.98-2.84, p < 0.01 [≥ 2 comorbidities]) and higher total hospital charges (RR 2.9, 95% CI 1.08-7.81, p = 0.04 [1 comorbidity]; RR 9.1, 95% CI 3.74-22.12, p < 0.01 [≥ 2 comorbidities]). CONCLUSIONS This analysis identified patient age, comorbidities, insurance type, hospital bed capacity, and rural or nonteaching hospital status as independent risk factors for postoperative complications and/or increased hospital charges in pediatric patients with craniopharyngioma. Transsphenoidal surgery in younger patients with craniopharyngioma was a risk factor for nonendocrine complications.

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Zhang S, Fang Y, Cai BW, et al.
Intracystic bleomycin for cystic craniopharyngiomas in children.
Cochrane Database Syst Rev. 2016; 7:CD008890 [PubMed] Related Publications

BACKGROUND: Craniopharyngiomas are the most common benign histological tumours to involve the hypothalamo-pituitary region in childhood. Cystic craniopharyngiomas account for more than 90% of the tumours. The optimal treatment of cystic craniopharyngioma remains controversial. Radical resection is the treatment of choice in patients with favourable tumour localisation. When the tumour localisation is unfavourable, a gross-total or partial resection followed by radiotherapy is the main treatment option in adults. However, it presents a risk of morbidity, especially for children. Intracystic bleomycin has been utilised potentially to delay the use of radiotherapy or radical resection, to decrease morbidity. This review is the second update of a previously published Cochrane review.
OBJECTIVES: To assess the benefits and harmful effects of intracystic bleomycin in children from birth to 18 years with cystic craniopharyngioma when compared to placebo (no treatment), surgical treatment (with or without adjuvant radiotherapy) or other intracystic treatments.
SEARCH METHODS: We searched the electronic databases CENTRAL (2016, Issue 1), MEDLINE/PubMed (from 1966 to February 2016) and EMBASE/Ovid (from 1980 to February 2016) with pre-specified terms. In addition, we searched the reference lists of relevant articles and reviews, conference proceedings (International Society for Paediatric Oncology 2005-2015) and ongoing trial databases (Register of the National Institute of Health and International Standard Randomised Controlled Trial Number (ISRCTN) register) in February 2016.
SELECTION CRITERIA: Randomised controlled trials (RCTs), quasi-randomised trials or controlled clinical trials (CCTs) comparing intracystic bleomycin and other treatments for cystic craniopharyngiomas in children (from birth to 18 years).
DATA COLLECTION AND ANALYSIS: Two review authors independently performed the study selection, data extraction and 'Risk of bias' assessment. We used risk ratio (RR) for binary data and mean difference (MD) for continuous data. If one of the treatment groups experienced no events and there was only one study available for the outcome, we used the Fischer's exact test. We performed analysis according to the guidelines in the Cochrane Handbook for Systematic reviews of Interventions.
MAIN RESULTS: We could not identify any studies in which the only difference between the treatment groups was the use of intracystic bleomycin. We did identify a RCT comparing intracystic bleomycin with intracystic phosphorus(32) ((32)P) (seven children). In this update we identified no additional studies. The included study had a high risk of bias. Survival could not be evaluated. There was no clear evidence of a difference between the treatment groups in cyst reduction (MD -0.15, 95% confidence interval (CI) -0.69 to 0.39, P value = 0.59, very low quality of evidence), neurological status (Fisher's exact P value = 0.429, very low quality of evidence), third nerve paralysis (Fischer's exact P value = 1.00, very low quality of evidence), fever (RR 2.92, 95% CI 0.73 to 11.70, P value = 0.13, very low quality of evidence) or total adverse effects (RR 1.75, 95% CI 0.68 to 4.53, P value = 0.25, very low quality of evidence). There was a significant difference in favour of the (32)P group for the occurrence of headache and vomiting (Fischer's exact P value = 0.029, very low quality of evidence for both outcomes).
AUTHORS' CONCLUSIONS: Since we identified no RCTs, quasi-randomised trials or CCTs of the treatment of cystic craniopharyngiomas in children in which only the use of intracystic bleomycin differed between the treatment groups, no definitive conclusions could be made about the effects of intracystic bleomycin in these patients. Only one low-power RCT comparing intracystic bleomycin with intracystic (32)P treatment was available, but no definitive conclusions can be made about the effectiveness of these agents in children with cystic craniopharyngiomas. Based on the currently available evidence, we are not able to give recommendations for the use of intracystic bleomycin in the treatment of cystic craniopharyngiomas in children. High-quality RCTs are needed.

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Bonfrate A, Farah J, De Marzi L, et al.
Influence of beam incidence and irradiation parameters on stray neutron doses to healthy organs of pediatric patients treated for an intracranial tumor with passive scattering proton therapy.
Phys Med. 2016; 32(4):590-9 [PubMed] Related Publications

PURPOSE: In scattering proton therapy, the beam incidence, i.e. the patient's orientation with respect to the beam axis, can significantly influence stray neutron doses although it is almost not documented in the literature.
METHODS: MCNPX calculations were carried out to estimate stray neutron doses to 25 healthy organs of a 10-year-old female phantom treated for an intracranial tumor. Two beam incidences were considered in this article, namely a superior (SUP) field and a right lateral (RLAT) field. For both fields, a parametric study was performed varying proton beam energy, modulation width, collimator aperture and thickness, compensator thickness and air gap size.
RESULTS: Using a standard beam line configuration for a craniopharyngioma treatment, neutron absorbed doses per therapeutic dose of 63μGyGy(-1) and 149μGyGy(-1) were found at the heart for the SUP and the RLAT fields, respectively. This dose discrepancy was explained by the different patient's orientations leading to changes in the distance between organs and the final collimator where external neutrons are mainly produced. Moreover, investigations on neutron spectral fluence at the heart showed that the number of neutrons was 2.5times higher for the RLAT field compared against the SUP field. Finally, the influence of some irradiation parameters on neutron doses was found to be different according to the beam incidence.
CONCLUSION: Beam incidence was thus found to induce large variations in stray neutron doses, proving that this parameter could be optimized to enhance the radiation protection of the patient.

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Du C, Feng CY, Yuan J, Yuan X
Ectopic recurrence of pediatric craniopharyngiomas after gross total resection: a report of two cases and a review of the literature.
Childs Nerv Syst. 2016; 32(8):1523-9 [PubMed] Related Publications

INTRODUCTION: Ectopic recurrent craniopharyngioma is rare. We reported two pediatric cases and reviewed the related literature.
METHOD: We retrospectively studied 177 craniopharyngioma cases treated by the senior author (Yuan X) between years 2003 and 2013. Two ectopic recurrent craniopharyngiomas were identified. One was discovered under the right frontal lobe and the other was found in the fourth ventricle. Both patients underwent a second radical resection without complications. Then we conducted an extensive review of peer-reviewed, English-language literatures in the US National Library of Medicine, focusing on the treatment modalities, recurrent sites, and clinical outcomes.
RESULTS: Sixty ectopic recurrent tumors have been reported so far (including this study). Thirty-three tumors were located in the previous surgical corridors and 27 were disseminated along the cerebrospinal fluid pathway. All recurrent tumors were surgically removed. The gross total resection (GTR) rates were 87 and 63 %, respectively.
CONCLUSION: The natural course of recurrent ectopic craniopharyngiomas is progressive. GTR is the treatment of choice. Regular follow-ups are strongly recommended to detect any further recurrence.

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Bao Y, Qiu B, Qi S, et al.
Influence of previous treatments on repeat surgery for recurrent craniopharyngiomas in children.
Childs Nerv Syst. 2016; 32(3):485-91 [PubMed] Related Publications

PURPOSE: Craniopharyngioma is a common pediatric brain tumor, with a high rate of recurrence after primary treatment. This retrospective study investigated the effect of various primary treatments on surgical strategies and outcomes for recurrent craniopharyngiomas.
METHODS: The study population comprised 35 children (mean age 8.77 years, range 1-16 years) with recurrent craniopharyngioma re-operated from January 1990 to January 2009. The recurrent craniopharyngiomas were excised whenever possible. For analysis, the patients were divided into four groups according to the primary treatment: radical tumor resection (A), incomplete tumor resection (B), radiotherapy + incomplete tumor resection (C), and Ommaya reservoir placement + incomplete tumor resection (D).
RESULTS: Group B had a significantly shorter recurrence-free interval than groups A, C, or D. Outcomes were significantly different among the four groups. The hypothalamic status scores of groups A (2.38 ± 0.27) and C (2.28 ± 0.42) were significantly higher than that of group B (1.64 ± 0.20). There were no statistical differences between any two other groups.
CONCLUSIONS: In children, the primary treatments for craniopharyngioma should be considered when choosing the surgical strategy for recurrence. Radiotherapy before repeated surgery can result in a worse functional outcome and hypothalamic-pituitary function.

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Yosef L, Ekkehard KM, Shalom M
Giant craniopharyngiomas in children: short- and long-term implications.
Childs Nerv Syst. 2016; 32(1):79-88 [PubMed] Related Publications

OBJECT: Craniopharyngiomas (CPs) are histologically benign tumors, yet they may carry an unfavorable prognosis. "Giant" tumors are associated with worse prognosis. Our aim was to evaluate the impact of tumor's size on different short- and long-term clinical factors.
METHODS: Between 2002 and 2012, a total of 36 consecutive CP patients less than 18 years of age and with at least 12 months of post-operative follow-up (FU) underwent a total of 54 operations for excision of CPs. Gross total resection (GTR) was the goal for all the first surgical resections. Twenty-seven patients were identified as eligible for inclusion in this study. Data were retrospectively collected by reviewing pertinent clinic/office notes and inpatient records as well as pre- and post-operative imaging. Long-term neurosurgical, ophthalmological, and endocrinological outcomes were obtained from records of the most recent FU office visit. Statistical analysis was performed to compare data from patients with tumors greater than or equal to 4.5 cm (nine patients) to those with smaller ones (<4.5 cm; 18 patients).
RESULTS: Mean age at the time of surgery was 5.4 years (median 5 years, range 1.3-15.3 years) for patients in the large-tumor group (LTG) and 8.9 years (median 9.6 years, range 2.1-17.1 years) for the small-tumor group (STG). Average duration of follow-up was 82.1 and 105.4 months for LTG and STG patients, respectively. There was a noticeable difference in the rate of emergent surgeries between the two groups (33.3 vs. 5.5 % in the LTG and STG, respectively; p = 0.055) as well as in recurrent surgeries (RR = 3.76; CI = 95 %, 1.793-7.877) and radiotherapy (RR = 2, 95 % CI 0.775-5.154). Rates of residual tumor on both initial post-operative imaging and last FU imaging were significantly increased in LTG patients (44.5 vs. 27.7 % and 66.6 vs. 16.6 %; respectively). Progression-free survivals (PFS) assessed at 2 and 5 years were 33.3 vs. 73.3 % (RR = 2.2, 95 % CI = 0.171-1.202) and 33.3 vs. 53.3 % (RR = 1.6, 95 % CI 0.221-1.765) in favor of smaller tumors. No difference in 2-, 5-, and 10-year overall survival was found. We found no significant difference in mean BMI at last follow-up between the two groups (23.83 ± 4.86 and 27.33 ± 8.09, respectively; p = 0.27), although significantly more patients in the LTG had shorter stature (mean height SDS -1.72 ± 1.88 and -0.17 ± 1.08 in LTG and STG patients, respectively; p = 0.027).
CONCLUSION: Tumor's size has significant impact on the management of CP in children. It affects both short-term factors (initial symptoms, urgency of surgical resection, extent of resection, and perioperative morbidity) as well as long-term parameters (PFS, rate of adjuvant treatments/recurrent surgeries, and metabolic/endocrinal/ophthalmological and functional outcomes). We think that a proper, world-wide accepted definition of what is "large," "giant," or even "monstrous" CP should be established. This will enable carrying multi-institutional studies on a larger group of patients, allowing further determining the importance of tumor's size in the management and outcome of craniopharyngiomas in children.

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Maarouf M, El Majdoub F, Fuetsch M, et al.
Stereotactic intracavitary brachytherapy with P-32 for cystic craniopharyngiomas in children.
Strahlenther Onkol. 2016; 192(3):157-65 [PubMed] Related Publications

PURPOSE: Although microsurgery remains the first-line treatment, gross total resection of cystic craniopharyngeomas (CP) is associated with significant morbidity and mortality and the addition of external irradiation to subtotal resection proves to achieve similar tumor control. However, concern regarding long-term morbidity associated with external irradiation in children still remains. With this retrospective analysis, the authors emphasize intracavitary brachytherapy using phosphorus-32 (P-32) as a treatment option for children with cystic CP.
PATIENTS AND METHODS: Between 1992 and 2009, 17 children (median age 15.4 years; range 7-18 years) with cystic CP underwent intracavitary brachytherapy using P-32. Eleven patients were treated for recurrent tumor cysts; 6 patients were treated primarily. MR imaging revealed solitary cysts in 7 patients; 10 patients had mixed solid-cystic lesions (median tumor volume 11.1 ml; range 0.5-78.9 ml). The median follow-up time was 61.9 months (range 16.9-196.6 months).
RESULTS: Local cyst control could be achieved in 14 patients (82 %). Three patients showed progression of the treated cystic formation (in-field progression) after a median time of 8.3 months (range 5.3-10.3 months), which led to subsequent interventions. The development of new, defined cysts and progression of solid tumor parts (out-of-field progression) occurred in 5 patients and led to additional interventions in 4 cases. There was neither surgery-related permanent morbidity nor mortality in this study. The overall progression-free survival was 75, 63, and 52 % after 1, 3, and 5 years, respectively.
CONCLUSION: Intracavitary brachytherapy using P-32 represents a safe and effective treatment option for children harboring cystic CP, even as primary treatment. However, P-32 does not clearly affect growth of solid tumor parts or the development of new cystic formations.

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Hoffmann A, Boekhoff S, Gebhardt U, et al.
History before diagnosis in childhood craniopharyngioma: associations with initial presentation and long-term prognosis.
Eur J Endocrinol. 2015; 173(6):853-62 [PubMed] Related Publications

OBJECTIVE: Childhood craniopharyngiomas (CP) are often diagnosed after a long duration of history (DOH). Tumor size, hypothalamic involvement (HI), and obesity are associated with reduced overall survival (OS) and functional capacity (FC). The effect of DOH and specific symptoms in history on presentation at initial diagnosis and long-term prognosis are unknown.
DESIGN: Retrospective analysis of patients' records and prospective longitudinal follow-up.
METHODS: Histories of 411 CP patients recruited in HIT Endo, KRANIOPHARYNGEOM 2000 were retrospectively evaluated for DOH, symptoms, and characteristics. The effect of specific manifestations and DOH on clinical presentation and tumor characteristics at time of initial CP diagnosis and long-term outcome were analyzed. Main outcome measures were 10-year OS and progression-free survival (PFS), FC, and BMI during longitudinal follow-up.
RESULTS: Median DOH was 6 months (range: 0.1-108 months) and correlated with age at diagnosis. Tumor size, HI, degree of resection, and BMI at diagnosis were not related to DOH. In multivariate analysis adjusted for age at diagnosis, only hydrocephalus was found to have a relevant influence on DOH. Visual and neurological deficits were associated with larger initial tumor size and impaired 10-year OS. Weight gain and growth failure were observed with longest DOH. PFS and FC were not related to any specific symptom. Endocrine deficits at diagnosis were associated with long DOH.
CONCLUSIONS: CP is frequently diagnosed after long DOH, especially in older children. However, DOH was not associated with tumor size, HI, survival, or FC. Visual and neurological deficits necessitate rapid diagnostic workup.

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Özyurt J, Müller HL, Thiel CM
A systematic review of cognitive performance in patients with childhood craniopharyngioma.
J Neurooncol. 2015; 125(1):9-21 [PubMed] Related Publications

Craniopharyngiomas are rare brain tumors of the sellar/suprasellar region, often adversely affecting patients' physical and psychosocial functioning. Until a few years ago, knowledge on cognitive deficits in craniopharyngioma patients was based on little valid evidence, with considerable inconsistencies across studies. Findings from recent research, with partly larger sample sizes, add to existing evidence to provide a more clear and reliable picture. The current review aims to summarize and systemize current findings on cognitive deficits in childhood craniopharyngioma, taking account of patient- and treatment-related variables where possible. Those studies were included that reported results of childhood craniopharyngioma patients tested with formalized neuropsychological tests (irrespective of their age at study, group size ≥10). A systematic assignment of test results to subcomponents of broader cognitive domains (e.g. to specific memory systems and processes) allows for a first comprehensive overview of patterns of spared and impaired cognitive functions. We show that episodic memory recall in particular is impaired, largely sparing other memory components. In accordance with recent knowledge on mammillary function, patients with hypothalamic involvement appear to be at particular risk. Deficits in higher cognitive processes, relying on the integrity of the prefrontal cortex and its subcortical pathways, may also occur, but results are still inconsistent. To gain deeper insight into the pattern of deficits and their association with patient- and treatment-related variables, further multi-site research with larger cohorts is needed.

Yano S, Kudo M, Hide T, et al.
Quality of Life and Clinical Features of Long-Term Survivors Surgically Treated for Pediatric Craniopharyngioma.
World Neurosurg. 2016; 85:153-62 [PubMed] Related Publications

OBJECTIVE: Several studies have reported treatment methods and results for pediatric craniopharyngiomas; however, few have evaluated patients' quality of life (QOL) after long-term follow-up. To evaluate treatment options, we assessed the QOL of patients with pediatric craniopharyngioma approximately 19 years after surgery and analyzed factors affecting QOL.
METHODS: Twenty-six survivors who underwent resection of craniopharyngiomas at <15 years of age enrolled in this study and their physical condition was assessed. QOL was assessed by a short-form health survey (SF-36 version 2) for patients older than 19 years of age or by Child Health Questionnaire Parent Form-50 for patients 18 years of age and younger. Patients were divided into good and fair QOL groups according to their physical and mental summary scores. Factors affecting the QOL of both groups were evaluated.
RESULTS: Median follow-up time was 19.1 years (range, 2.8-44.1 years). Twenty-two (84.6%) patients were employed or in school; 14 (53.8%) had visual deficits. Panhypopituitarism was diagnosed in 22 of 26 (84.6%) subjects. SF-36 analysis indicated that patients had significantly lower scores for general and mental health. Visual deficits, obesity, and complications during follow-up significantly affected the fair QOL group long-term. Patients' basic characteristics, initial resection rates, times of operation or irradiation did not significantly affect long-term QOL.
CONCLUSION: Long-term survivors lived independently but had a lower overall QOL. Not only monitor short-term results based on estimation of the initial resection or recurrence rate, it is important to preserve visual and hypothalamic function and monitor arising complications for extended periods to improve patients' long-term QOL.

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Bomer I, Saure C, Caminiti C, et al.
Comparison of energy expenditure, body composition, metabolic disorders, and energy intake between obese children with a history of craniopharyngioma and children with multifactorial obesity.
J Pediatr Endocrinol Metab. 2015; 28(11-12):1305-12 [PubMed] Related Publications

UNLABELLED: Craniopharyngioma is a histologically benign brain malformation with a fundamental role in satiety modulation, causing obesity in up to 52% of patients.
AIM: To evaluate cardiovascular risk factors, body composition, resting energy expenditure (REE), and energy intake in craniopharyngioma patients and to compare the data with those from children with multifactorial obesity.
POPULATION: All obese children and adolescents who underwent craniopharyngioma resection and a control group of children with multifactorial obesity in follow-up between May 2012 and April 2013.
MATERIALS AND METHODS: Anthropometric measurements, bioelectrical impedance, indirect calorimetry, energy intake, homeostatic model assessment insulin resistance (HOMA-IR), and dyslipidemia were evaluated.
RESULTS: Twenty-three patients with craniopharyngioma and 43 controls were included. Children with craniopharyngioma-related obesity had a lower fat-free mass percentage (62.4 vs. 67.5; p=0.01) and a higher fat mass percentage (37.5 vs. 32.5; p=0.01) compared to those with multifactorial obesity. A positive association was found between %REE and %fat-free mass in subjects with multifactorial obesity (68±1% in normal REE vs. 62.6±1% in low REE; p=0.04), but not in craniopharyngioma patients (62±2.7 in normal REE vs. 61.2±1.8% in low REE; p=0.8). No differences were found in metabolic involvement or energy intake.
CONCLUSIONS: REE was lower in craniopharyngioma patients compared to children with multifactorial obesity regardless of the amount of fat-free mass, suggesting that other factors may be responsible for the lower REE.

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Hoffmann A, Bootsveld K, Gebhardt U, et al.
Nonalcoholic fatty liver disease and fatigue in long-term survivors of childhood-onset craniopharyngioma.
Eur J Endocrinol. 2015; 173(3):389-97 [PubMed] Related Publications

OBJECTIVE: Hypothalamic obesity in childhood craniopharyngioma (CP) patients carries a high risk for development of metabolic syndrome. In metabolic syndrome, the development of nonalcoholic fatty liver disease (NAFLD) is known. The aim of this study is to detect the risk for NAFLD in childhood-onset CP.
DESIGN: This cross-sectional study included liver computed tomography (CT); ultrasound analysis of abdomen; measurements of serum parameters, height, weight and body composition; and daily medication of patients with childhood-onset CP.
METHODS: A total of 384 patients recruited in trials HIT Endo and KRANIOPHARYNGEOM 2000 were analyzed. Ninety-four survivors were included by fulfilling the criteria of proven hypothalamic involvement (HI), a minimum time interval of 5 years between diagnosis and study, and a minimum age of 18 years at the time of evaluation. A total of 19 patients agreed to participate. To quantify the degree of steatosis hepatis, analyses of liver density were performed once by non-contrasted CT of liver sections.
RESULTS: NAFLD occurs in about 50% of CP patients with HI and is associated with elevated liver enzymes and homeostasis model assessment index. BMI is not an effective predictive factor but body fat mass measured by near-infrared spectroscopy (NIRS) is. Over half of CP patients (60%) with NAFLD are treated with stimulating agents, with risk of hepatic side effects.
CONCLUSIONS: NAFLD is a major adverse late effect in childhood-onset CP. NIRS rather than BMI should be used to measure body composition and predict NAFLD. Stimulating agents for treatment of fatigue and daytime sleepiness in CP should be prescribed judiciously.

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Gump JM, Donson AM, Birks DK, et al.
Identification of targets for rational pharmacological therapy in childhood craniopharyngioma.
Acta Neuropathol Commun. 2015; 3:30 [PubMed] Free Access to Full Article Related Publications

INTRODUCTION: Pediatric adamantinomatous craniopharyngioma (ACP) is a histologically benign but clinically aggressive brain tumor that arises from the sellar/suprasellar region. Despite a high survival rate with current surgical and radiation therapy (75-95 % at 10 years), ACP is associated with debilitating visual, endocrine, neurocognitive and psychological morbidity, resulting in excheptionally poor quality of life for survivors. Identification of an effective pharmacological therapy could drastically decrease morbidity and improve long term outcomes for children with ACP.
RESULTS: Using mRNA microarray gene expression analysis of 15 ACP patient samples, we have found several pharmaceutical targets that are significantly and consistently overexpressed in our panel of ACP relative to other pediatric brain tumors, pituitary tumors, normal pituitary and normal brain tissue. Among the most highly expressed are several targets of the kinase inhibitor dasatinib - LCK, EPHA2 and SRC; EGFR pathway targets - AREG, EGFR and ERBB3; and other potentially actionable cancer targets - SHH, MMP9 and MMP12. We confirm by western blot that a subset of these targets is highly expressed in ACP primary tumor samples.
CONCLUSIONS: We report here the first published transcriptome for ACP and the identification of targets for rational therapy. Experimental drugs targeting each of these gene products are currently being tested clinically and pre-clinically for the treatment of other tumor types. This study provides a rationale for further pre-clinical and clinical studies of novel pharmacological treatments for ACP. Development of mouse and cell culture models for ACP will further enable the translation of these targets from the lab to the clinic, potentially ushering in a new era in the treatment of ACP.

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Sterkenburg AS, Hoffmann A, Gebhardt U, et al.
Survival, hypothalamic obesity, and neuropsychological/psychosocial status after childhood-onset craniopharyngioma: newly reported long-term outcomes.
Neuro Oncol. 2015; 17(7):1029-38 [PubMed] Related Publications

BACKGROUND: Quality of life (QoL) and long-term prognosis are frequently, and often severely, impaired in craniopharyngioma (CP) patients. Knowledge of risk factors for long-term outcome is important for optimization of treatment.
METHODS: Overall survival (OS) and progression-free survival (PFS), body mass index (BMI), neuropsychological status (EORTCQLQ-C30, MFI-20), and psychosocial status were analyzed in 261 patients with childhood-onset CP diagnosed before 2000 and longitudinally observed in HIT-Endo.
RESULTS: Twenty-year OS was lower (P = .006) in CP with hypothalamic involvement (HI) (n = 132; 0.84 ± 0.04) when compared with CP without HI (n = 82; 0.95 ± 0.04). OS was not related to degree of resection, sex, age at diagnosis, or year of diagnosis (before/after 1990). PFS (n = 168; 0.58 ± 0.05) was lower in younger patients (<5 y at diagnosis) (n = 30; 0.39 ± 0.10) compared with patients aged 5-10 years (n = 66; 0.52 ± 0.08) and older than 10 years (n = 72; 0.77 ± 0.06) at diagnosis. PFS was not associated with HI, degree of resection, or sex. HI led to severe weight gain during the first 8-12 years of follow-up (median BMI increase: +4.59SD) compared with no HI (median increase: +1.20SD) (P = .00). During >12 years of follow-up, patients with HI presented no further increase in BMI. QoL in CP patients with HI was impaired by obesity, physical fatigue, reduced motivation, dyspnea, diarrhea, and nonoptimal psychosocial development.
CONCLUSIONS: OS and QoL are impaired by HI in long-term survivors of CP. HI is associated with severe obesity, which plateaus after 12 years. OS/PFS are not related to degree of resection, but gross-total resection should be avoided in cases of HI to prevent further hypothalamic damage, which exacerbates sequelae.

Purcell PL, Shinn JR, Otto RK, et al.
Nasoseptal flap reconstruction of pediatric sellar defects: a radiographic feasibility study and case series.
Otolaryngol Head Neck Surg. 2015; 152(4):746-51 [PubMed] Related Publications

OBJECTIVES: In this study, we used computed tomography measurements to investigate the feasibility of nasoseptal flap reconstruction of sellar defects in children, and we reviewed our institutional experience with the procedure.
STUDY DESIGN: Cross-sectional and case series.
SETTING: Pediatric tertiary care facility.
METHODS: We obtained 10 normal maxillofacial scans for each year of age from birth to 18. Computer-assisted nasal and skull-base measurements were performed. Patients with incomplete pneumatization were excluded from analysis. Reconstruction was presumed feasible if the ratio of nasoseptal flap length to associated sellar defect length was greater than 1. Chart review identified surgical patients.
RESULTS: Of 190 scans, 125 had complete pneumatization. Of these, 120 (96%) displayed a ratio of nasoseptal flap length to sellar defect length greater than 1, suggesting that reconstruction would be feasible. Mean ratio of flap length to defect length for all subjects was 1.47 (SD 0.33; 95% CI, 1.41-1.53). Only 5 of 125 patients (4%) had a ratio less than 1; the median age for these patients was 15 years, which is older than the median age of 12 years for subjects with a ratio greater than 1 (P = .02). An inverse relationship was identified between age and ratio of flap length to defect length (r = -0.49, P < .001). Case series identified 6 children, ages 5 to 17; flap length was never described as a limitation.
CONCLUSIONS: Nasoseptal flap length is not a limiting factor for reconstruction of pediatric sellar defects. When compared with older patients, younger patients tend to have greater nasoseptal flap length relative to sellar defect length.

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Klimo P, Venable GT, Boop FA, Merchant TE
Recurrent craniopharyngioma after conformal radiation in children and the burden of treatment.
J Neurosurg Pediatr. 2015; 15(5):499-505 [PubMed] Related Publications

OBJECT: In this paper the authors present their experience treating children with recurrent craniopharyngioma who were initially managed with surgery followed by conformal radiation therapy (CRT).
METHODS: A departmental oncology information system was queried to identify all children (< 18 years old) who received CRT for a craniopharyngioma between 1998 and 2010 (inclusive) and specifically those who experienced tumor progression. For each patient, the authors recorded the type of recurrence (solid, cystic, or both), the time interval to first progression and each subsequent progression, the associated treatment complications, and disease status at last follow-up evaluation.
RESULTS: Among the 97 patients that met criteria for entry into this study, 18 (18.6%) experienced tumor progression (9 cystic, 3 solid, 6 cystic and solid). The median time to first recurrence was 4.62 years (range 1.81-9.11 years). The subgroup included 6 female and 12 male patients with a median age of 7.54 years (range 3.61-13.83 years). Ten patients experienced first progression within 5 years of CRT. The 5- and 10-year treatment-free survival rates for the entire cohort were 89.0% (95% confidence interval [CI] 80.5%-93.9%) and 76.2% (95% CI 64%-85%), respectively. Seven patients had a single episode of progression and 11 had more than 1. The time interval between each subsequent progression was progressively shorter. The 18 patients underwent 38 procedures. The median follow-up duration for this group was 9.32 years (range 4.04-19.0 years). Three patients died, including 1 from perioperative complications.
CONCLUSIONS: Craniopharyngioma progression after prior irradiation is exceedingly difficult to treat and local control is challenging despite repeated surgical procedures. Given our results, gross-total resection may need to be the surgical goal at the time of first recurrence, if possible. Decompressing new cyst formation alone has a low rate of long-term success.

Related: Pituitary Tumors USA

Daubenbüchel AM, Hoffmann A, Gebhardt U, et al.
Hydrocephalus and hypothalamic involvement in pediatric patients with craniopharyngioma or cysts of Rathke's pouch: impact on long-term prognosis.
Eur J Endocrinol. 2015; 172(5):561-9 [PubMed] Related Publications

OBJECTIVE: Pediatric patients with sellar masses such as craniopharyngioma (CP) or cyst of Rathke's pouch (CRP) frequently suffer disease- and treatment-related sequelae. We analyzed the impact and prognostic relevance of initial hydrocephalus (HY) and hypothalamic involvement (HI) on long-term survival and functional capacity (FC) in children with CP or CRP.
SUBJECTS AND METHODS: Using retrospective analysis of patient records, presence of initial HY or HI was assessed in 177 pediatric patients (163 CP and 14 CRP). Twenty-year overall survival (OS) and progression-free survival (PFS), FC, and BMI were analyzed with regard to initial HY, degree of resection, or HI.
RESULTS: Of the 177 patients, 105 patients (103/163 CP and 2/14 CRP) presented with initial HY and 96 presented with HI. HY at diagnosis was associated (P=0.000) with papilledema, neurological deficits, and higher BMI at diagnosis and during follow-up. OS, PFS, and FC were not affected by HY at initial diagnosis. HI at diagnosis (96/177) had major negative impact on long-term prognosis. Sellar masses with HI were associated with lower OS (0.84±0.04; P=0.021), lower FC (P=0.003), and higher BMI at diagnosis and last follow-up (P=0.000) when compared with sellar masses without HI (OS: 0.94±0.05). PFS was not affected by HI or degree of resection.
CONCLUSIONS: Initial HY has no impact on outcome in patients with sellar masses. OS and FC are impaired in survivors presenting with initial HI. PFS is not affected by HY, HI, or degree of resection. Accordingly, gross-total resection is not recommended in sellar masses with initial HI to prevent further hypothalamic damage.

Related: Pituitary Tumors

Greenfield BJ, Okcu MF, Baxter PA, et al.
Long-term disease control and toxicity outcomes following surgery and intensity modulated radiation therapy (IMRT) in pediatric craniopharyngioma.
Radiother Oncol. 2015; 114(2):224-9 [PubMed] Related Publications

PURPOSE: To report long-term progression-free survival (PFS) and late-toxicity outcomes in pediatric craniopharyngioma patients treated with IMRT.
PATIENTS AND METHODS: Twenty-four children were treated with IMRT to a median dose of 50.4Gy (range, 49.8-54Gy). The clinical target volume (CTV) was the gross tumor volume (GTV) with a 1cm margin. The planning target volume (PTV) was the CTV with a 3-5mm margin. Median follow-up was 107.3months.
RESULTS: The 5- and 10-year PFS rates were 65.8% and 60.7%. The 5- and 10-year cystic PFS rates were 70.2% and 65.2% while the 5- and 10-year solid PFS were the same at 90.7%. Endocrinopathy was seen in 42% at initial diagnosis and in 74% after surgical intervention, prior to IMRT. Hypothalamic dysfunction and visual deficits were associated with increasing PTV and number of surgical interventions.
CONCLUSIONS: IMRT is a viable treatment option for pediatric craniopharyngioma. Despite the use of IMRT, majority of the craniopharyngioma patients experienced long-term toxicity, many of which present prior to radiotherapy. Limitations of retrospective analyses on small patient cohort elicit the need for a prospective multi-institutional study to determine the absolute benefit of IMRT in pediatric craniopharyngioma.

Related: Pituitary Tumors

Kalina MA, Wilczek M, Kalina-Faska B, et al.
Carbohydrate-lipid profile and use of metformin with micronized fenofibrate in reducing metabolic consequences of craniopharyngioma treatment in children: single institution experience.
J Pediatr Endocrinol Metab. 2015; 28(1-2):45-51 [PubMed] Related Publications

AIM: To evaluate auxology and metabolic disturbances in children with craniopharyngioma, and to present observational results of treatment of metabolic sequels with metformin and micronized fenofibrate.
METHODS: The studied group comprised 22 children [median age at diagnosis 10.5 (0.17-16.75) years; median follow-up 5.1 years]. Assessment included height standard deviations (SDS), body mass index (BMI) SDS, concentrations of lipids, glucose and insulin (fasting or oral glucose tolerance test) and homeostatic model assessment of insulin resistance (HOMA-IR) index. Ten adolescents with hyperinsulinemia and dyslipidemia received therapy with metformin (500-1500 mg/daily) and micronized fenofibrate (160 mg/daily).
RESULTS: At diagnosis, median hSDS was -1.66 (range: -4.08; +0.1). Nine (40.9%) children were growth hormone-treated. There was gradual increase of BMI SDS, 18 (81.8%) patients being overweight at the final assessment. Dyslipidaemia was found in 19 patients (86.4%), hyperinsulinaemia in 11 patients (50%) and elevated HOMA-IR in 15 patients (68.2%). Decrease of triglycerides [median 263.5 (171-362) mg/dL vs. 154 (102-183) mg/dL] and HOMA-IR [8.64 (5.08-12.65) vs. 4.68 (0.7-7.9)] was significant in the group treated with metformin and fenofibrate for 6 months.
CONCLUSIONS: Significant auxologic changes and metabolic abnormalities were found in children treated for craniopharyngioma. The use of metformin and fenofibrate seemed to attenuate these disturbances in a short-term observation.

Related: Pituitary Tumors

Flitsch J, Aberle J, Burkhardt T
Surgery for pediatric craniopharyngiomas: is less more?
J Pediatr Endocrinol Metab. 2015; 28(1-2):27-33 [PubMed] Related Publications

Surgery for craniopharyngiomas, especially in childhood and adolescence, has evolved from an era of aggressive strategies - with the primary goal of gross total removal and accepting an impaired functional outcome - to a more individually tailored therapy that avoids immediate treatment-related and long-term morbidity. Modern imaging techniques and a wider understanding of hypothalamic risk factors have led to surgical strategies adapted to the localization of these inhomogenously grown pathologies. Whereas purely infradiaphragmatic as well as supradiaphragmatic/infrachiasmatic tumors have a favorably surgical outcome with higher gross total resection rates in experienced hands, lesions within the third ventricle extending beyond the mammillary bodies remain a problem. The same is valid for lesions beyond 3 cm in diameter, more or less independent of their localization. Aside from the traditional microscopic approach via the subfrontal or pterional craniotomy, transsphenoidal approaches and other minimal invasive surgical methods, e.g., catheter implantation into cystic formations of the tumor have become popular. Radiotherapy, with its risks and limitations, can effectively be added to avoid recurrences. Nowadays, surgery as part of an interdisciplinary treatment strategy is still the typical first choice. However, taking the patient's long-term prognosis into considertaion, the surgical complication rates have to be minimized.

Related: Pituitary Tumors

Hoffmann A, Postma FP, Sterkenburg AS, et al.
Eating behavior, weight problems and eating disorders in 101 long-term survivors of childhood-onset craniopharyngioma.
J Pediatr Endocrinol Metab. 2015; 28(1-2):35-43 [PubMed] Related Publications

BACKGROUND: As a result of hypothalamic involvement and/or treatment-related hypothalamic damage, up to 75% of childhood craniopharyngioma patients develop hypothalamic obesity.
METHODS: Eating behavior was analyzed in 101 survivors of childhood craniopharyngioma, recruited from 1980 to 2001 in the HIT-Endo multicenter study, and in 85 body mass index (BMI)-matched healthy controls using the Inventory for Eating Behavior and Weight Problems (IEG) and the Inventory for Eating Disorders (ESI).
RESULTS: Severely obese patients (BMI>8 SD; n=9) presented with pathological eating behavior, more weight problems, and eating disorders, as compared to obese (BMI 3-8 SD; n=44) and normal or overweight patients (BMI<3 SD; n=48). Craniopharyngioma patients with different degrees of obesity showed similar or even less pathological findings as compared to BMI-matched normal controls.
CONCLUSION: Severe obesity is associated with pathological eating behavior/disorders in craniopharyngioma patients. As these disorders are not disease-specific, risk factors for hypothalamic obesity should be the focus of further craniopharyngioma research.

Related: Pituitary Tumors

Conroy R, Gomes L, Owen C, et al.
Clinical equipoise: Protons and the child with craniopharyngioma.
J Med Imaging Radiat Oncol. 2015; 59(3):379-85 [PubMed] Related Publications

INTRODUCTION: Childhood craniopharyngioma is a complex condition to manage. Survival figures are high but the potential for long-term morbidity is great. There is much debate regarding the best management for these tumours with increasing interest in the use of proton beam therapy. We have therefore reviewed our radiotherapy (RT) practice at Westmead Hospital and the literature regarding the use of protons for these children.
METHODS: Three children have received fractionated stereotactic RT for craniopharyngioma at Westmead Hospital since 2007. Each RT plan was reviewed and additional organs at risk were contoured to enable comparison with published proton data.
RESULTS: Planning target volume coverage was similar with all modalities: with the conformity index ranging from 0.70 to 0.78 in our patients compared with 0.50-0.84 in the published data. RT dose to temporal lobes, hippocampi and whole brain was also similar with protons and photons. Proton beam therapy may give lower dose to the Circle of Willis than stereotactic RT.
CONCLUSION: Currently there is no clear evidence that proton beam therapy will improve survival or reduce morbidity for children with craniopharyngioma. However, proton therapy has the potential to reduce RT dose to the Circle of Willis, which may reduce the risk of future cerebrovascular complications. We propose that more resources should be allocated to ensuring these patients are managed by experienced multidisciplinary teams through the continuum from diagnosis to long-term follow-up.

Related: Pituitary Tumors

Olsson DS, Andersson E, Bryngelsson IL, et al.
Excess mortality and morbidity in patients with craniopharyngioma, especially in patients with childhood onset: a population-based study in Sweden.
J Clin Endocrinol Metab. 2015; 100(2):467-74 [PubMed] Related Publications

CONTEXT: Craniopharyngiomas (CPs) in adults have been associated with excess mortality.
OBJECTIVE: The aim of the study was to investigate mortality and morbidity in patients with childhood-onset and adult-onset CP.
METHODS: PATIENTS with CP were identified and followed in Swedish national health registries, 1987 through 2011. The inclusion criteria for the CP diagnosis were internally validated against patient records in 28% of the study population.
SETTINGS: This was a nationwide population-based study.
PATIENTS: A total of 307 patients (151 men and 156 women) were identified and included (mean follow-up, 9 years; range, 0-25 years). The inclusion criteria had a positive predictive value of 97% and a sensitivity of 92%.
INTERVENTION: There were no interventions.
MAIN OUTCOME MEASURES: Standardized mortality ratios (SMRs) and standardized incidence ratios (SIRs) with 95% confidence intervals were calculated using the Swedish population as the reference.
RESULTS: During the study, 54 patients died compared with the expected number of 14.1, resulting in an SMR of 3.2 (2.2-4.7) for men and 4.9 (3.2-7.2) for women. PATIENTS with childhood-onset (n = 106) and adult-onset (n = 201) CP had SMRs of 17 (6.3-37) and 3.5 (2.6-4.6), respectively. PATIENTS with hypopituitarism (n = 250), diabetes insipidus (n = 110), and neither of these (n = 54) had SMRs of 4.3 (3.1-5.8), 6.1 (3.5-9.7), and 2.7 (1.4-4.6), respectively. The SMR due to cerebrovascular diseases was 5.1 (1.7-12). SIRs were 5.6 (3.8-8.0) for type 2 diabetes mellitus, 7.1 (5.0-9.9) for cerebral infarction, 0.7 (0.2-1.7) for myocardial infarction, 2.1 (1.4-3.0) for fracture, and 5.9 (3.4-9.4) for severe infection. The SIR for all malignant tumors was 1.3 (0.8-2.1).
CONCLUSIONS: This first nationwide population-based study of patients with CP demonstrated excess mortality that was especially marked in patients with childhood-onset disease and among women. Death due to cerebrovascular diseases was increased 5-fold. Hypopituitarism and diabetes insipidus were negative prognostic factors for mortality and morbidity. PATIENTS with CP had increased disease burden related to type 2 diabetes mellitus, cerebral infarction, fracture, and severe infection.

Related: Pituitary Tumors

Khan MJ, Humayun KN, Donaldson M, et al.
Longitudinal changes in body mass index in children with craniopharyngioma.
Horm Res Paediatr. 2014; 82(6):372-9 [PubMed] Related Publications

OBJECTIVE: We investigated the relationship of body mass index at presentation and pituitary status with long-term changes in BMI over a period of 5 years.
STUDY DESIGN: Craniopharyngioma patients (n = 25) attending a tertiary pediatric endocrine center were divided into three groups based on their BMI at presentation [BMI ≥ 2 standard deviation scores (SDS), 0-1.99 SDS, and <0 SDS) and then analyzed for trends of BMI over a period of up to 5 years.
RESULTS: Median (interquartile range) BMI SDS and hypopituitarism at presentation versus at the 5-year follow-up were as follows: BMI SDS ≥ 2 group (n = 10): 3.55 (0.68), 6/10 versus 3.76 (1.13), 8/10; BMI SDS 0-1.99 group (n = 11): 1.68 (1.05), 3/11 versus 1.64 (2.04), 7/11, and BMI SDS <0 group (n = 4): -0.23 (0.93), 2/4 versus 0.61, 4/4. At the 5-year follow-up, 10/10, 7/11, and 1/4 subjects when divided in groups according to BMI at presentation were obese.
CONCLUSIONS: Our data indicate that obesity at presentation, rather than panhypopituitarism either at or after presentation, predicts obesity 5 years after diagnosis. However, obesity at presentation is not always associated with the subsequent development of panhypopituitarism. Pediatric craniopharyngioma subjects who have BMI SDS ≥ 2 at presentation require early and aggressive intervention to help prevent the complications of obesity.

Related: Pituitary Tumors

Zheng J, Fang Y, Cai BW, et al.
Intracystic bleomycin for cystic craniopharyngiomas in children.
Cochrane Database Syst Rev. 2014; (9):CD008890 [PubMed] Related Publications

BACKGROUND: Craniopharyngiomas are the commonest benign histological tumours to involve the hypothalamo-pituitary region in childhood. Cystic craniopharyngiomas comprise more than 90% of the tumours. The optimal treatment of cystic craniopharyngioma remains controversial. Radical resection is the treatment of choice in patients with favourable tumour localisation. When the tumour localisation is unfavourable, a gross-total or partial resection followed by radiotherapy is the main treatment option in adults. However, it presents a risk of morbidity, especially for children. Intracystic bleomycin has been utilised potentially to delay the use of radiotherapy or radical resection, to decrease morbidity. This review is an update of a previously published Cochrane review.
OBJECTIVES: To assess the benefits and harmful effects of intracystic bleomycin in children from birth to 18 years with cystic craniopharyngioma when compared to placebo (no treatment), surgical treatment (with or without adjuvant radiotherapy) or some other intracyctic treatments.
SEARCH METHODS: We searched the electronic databases CENTRAL (2014, Issue 1), MEDLINE/PubMed (from 1966 to March 2014) and EMBASE/Ovid (from 1980 to March 2014) with pre-specified terms. In addition, we searched the reference lists of relevant articles and reviews, conference proceedings (International Society for Paediatric Oncology 2005-2013) and ongoing trial databases (Register of the National Institute of Health and International Standard Randomised Controlled Trial Number (ISRCTN) register) in May 2014.
SELECTION CRITERIA: Randomised controlled trials (RCTs), quasi-randomised trials or controlled clinical trials (CCTs) comparing intracystic bleomycin and other treatments for cystic craniopharyngiomas in children (from birth to 18 years).
DATA COLLECTION AND ANALYSIS: Two review authors independently performed the data extraction and 'Risk of bias' assessment. We used risk ratio (RR) for binary data and mean difference (MD) for continuous data. We planned that if one of the treatment groups experienced no events and there was only one study available for the outcome, we would use the Fischer's exact test.
MAIN RESULTS: We could not identify any studies in which the only difference between the treatment groups was the use of intracystic bleomycin. We did identify a RCT comparing intracystic bleomycin with intracystic phosphorus(32) ((32)P) (n = 7 children). The trial had a high risk of bias. Survival could not be evaluated. There was no evidence of a significant difference between the treatment groups in cyst reduction (MD -0.15, 95% confidence interval (CI) -0.69 to 0.39, P value = 0.59), neurological status (Fisher's exact P value = 0.429), 3rd nerve paralysis (Fischer's exact P value = 1.00), fever (RR 2.92, 95% CI 0.73 to 11.70, P value = 0.13) or total adverse effects (RR 1.75, 95% CI 0.68 to 4.53, P value = 0.25). There was a significant difference in favour of the (32)P group for the occurrence of headache and vomiting (Fischer's exact P value = 0.029 for both outcomes).
AUTHORS' CONCLUSIONS: Since we identified no RCTs, quasi-randomised trials or CCTs of the treatment of cystic craniopharyngiomas in children in which only the use of intracystic bleomycin differed between the treatment groups, no definitive conclusions could be made about the effects of intracystic bleomycin in these patients. Only one low-power RCT comparing intracystic bleomycin with intracystic (32)P treatment was available, but no definitive conclusions can be made about the effectiveness of these agents in children with cystic craniopharyngiomas. Based on the currently available evidence, we are not able to give recommendations for the use of intracystic bleomycin in the treatment of cystic craniopharyngiomas in children. High-quality RCTs are needed.

Related: Bleomycin Pituitary Tumors

Raghunathan V, Dhaliwal MS, Gupta A, Jevalikar G
From cerebral salt wasting to diabetes insipidus with adipsia: case report of a child with craniopharyngioma.
J Pediatr Endocrinol Metab. 2015; 28(3-4):323-6 [PubMed] Related Publications

UNLABELLED: Craniopharyngioma is associated with a wide and interesting variety of sodium states both by itself and following surgical resection. These are often challenging to diagnose, especially given their dynamic nature during the perioperative course. We present the case of a boy with craniopharyngioma who had hyponatremia due to cerebral salt wasting preoperatively, developed diabetes insipidus (DI) intraoperatively and proceeded to develop hypernatremia with adipsic DI.
CONCLUSION: Cerebral salt wasting is a rare presenting feature of craniopharyngioma. Postoperative DI can be associated with thirst abnormalities including adipsia due to hypothalamic damage; careful monitoring and a high index of suspicion are required for its detection. Adipsic DI is a difficult condition to manage; hence a conservative surgical approach is suggested.

Related: Pituitary Tumors

Barakat LP, Li Y, Hobbie WL, et al.
Health-related quality of life of adolescent and young adult survivors of childhood brain tumors.
Psychooncology. 2015; 24(7):804-11 [PubMed] Free Access to Full Article Related Publications

OBJECTIVE: Our aim was to expand research on predictors of health-related quality of life (HRQOL) for adolescent and young adult survivors of childhood brain tumors who are not living independently by evaluating the mediating role of family functioning in the association of disease severity/treatment late effects with survivor self-report and caregiver-proxy report of physical and emotional HRQOL.
METHODS: Mothers (N = 186) and their survivors living at home (N = 126) completed self-report and caregiver-proxy report of physical and emotional HRQOL. Mothers completed family functioning measures of general family functioning, caregiving demands, and caregiver distress. Medical file review and caregiver report were used to evaluate disease severity/treatment late effects.
RESULTS: Using structural equation models, family functioning was adjusted for sociodemographic factors. Disease severity/treatment late effects had significant direct effects on self-report and caregiver-proxy report of physical and emotional HRQOL. Family functioning had a significant direct effect on caregiver-proxy report of physical and emotional HRQOL, but these findings were not confirmed for self-report HRQOL. Model-fit indices suggested good fit of the models, but the mediation effect of family functioning was not supported.
CONCLUSIONS: Disease severity/treatment late effects explained self-report and caregiver-proxy report of physical and emotional HRQOL for these adolescent and young adult survivors of childhood brain tumors. Family functioning was implicated as an important factor for caregiver-proxy report only. To enhance physical and emotional HRQOL, findings underscore the importance of coordinated, multidisciplinary follow-up care for the survivors who are not living independently and their families to address treatment late effects and support family management.

Related: Childhood Medulloblastoma / PNET Medulloblastoma

Hoffmann A, Gebhardt U, Sterkenburg AS, et al.
Diencephalic syndrome in childhood craniopharyngioma--results of German multicenter studies on 485 long-term survivors of childhood craniopharyngioma.
J Clin Endocrinol Metab. 2014; 99(11):3972-7 [PubMed] Related Publications

CONTEXT: Childhood craniopharyngiomas (CPs) are known to be associated with an increased risk of excessive weight gain and hypothalamic obesity. Atypical clinical manifestations include the development of a diencephalic syndrome (DS) with a failure to thrive or maintain weight at appropriate body mass index.
CASES AND METHODS: In a retrospective study, we analyzed 21 of 485 childhood CP patients (4.3%) who presented with a low weight (< -2 body mass index SD) at the time of diagnosis. Eleven of the 21 patients were identified with a DS due to proven hypothalamic involvement. We show the clinical manifestations of DS and weight development before and after diagnosis in these 11 patients. The first significant differences between patients with low weight at diagnosis and normal-weight patients at diagnosis are observed at 5 years of age. Within the first 2 years after diagnosis, the weight of DS patients and normal-weight patients converge to a similar level. Tumor size does not play a role with respect to DS development. Finally, tumor characteristics of DS patients were compared with magnetic resonance imaging scans of obese CP patients at the time of diagnosis.
CONCLUSIONS: DS is a rare clinical manifestation in childhood CP and should be considered as a discrete diagnosis in failure to thrive. DS at the time of diagnosis does not preclude weight gain after diagnosis of a CP with hypothalamic involvement.

Related: Pituitary Tumors

Bailey S, Parkes J
Intracystic interferon therapy in childhood craniopharyngioma: who, when and how?
Clin Endocrinol (Oxf). 2015; 82(1):29-34 [PubMed] Related Publications

A 13-year-old male presents with fluctuating visual disturbance and headaches. Imaging reveals a significant predominantly cystic suprasellar tumour, typical of a craniopharyngioma. The patient has growth hormone deficiency but the rest of the hypothalamic/pituitary axis is intact. What are the options for therapy in 2014? Specifically, is there a role for local treatment with interferon injected into the cyst cavity? The aim of management in children with craniopharyngiomas is to reduce the impact of the tumour as much as possible, while minimising the morbidity associated with treatment. There are a number of therapeutic options available: surgery, radiotherapy and the insertion of therapeutic agents directly into the tumour cyst. The role of intracystic therapy in the form of interferon is discussed; including when to use this therapeutic option and practical details of its use.

Related: Pituitary Tumors

Bishop AJ, Greenfield B, Mahajan A, et al.
Proton beam therapy versus conformal photon radiation therapy for childhood craniopharyngioma: multi-institutional analysis of outcomes, cyst dynamics, and toxicity.
Int J Radiat Oncol Biol Phys. 2014; 90(2):354-61 [PubMed] Free Access to Full Article Related Publications

PURPOSE: We compared proton beam therapy (PBT) with intensity modulated radiation therapy (IMRT) for pediatric craniopharyngioma in terms of disease control, cyst dynamics, and toxicity.
METHODS AND MATERIALS: We reviewed records from 52 children treated with PBT (n=21) or IMRT (n=31) at 2 institutions from 1996-2012. Endpoints were overall survival (OS), disease control, cyst dynamics, and toxicity.
RESULTS: At 59.6 months' median follow-up (PBT 33 mo vs IMRT 106 mo; P<.001), the 3-year outcomes were 96% for OS, 95% for nodular failure-free survival and 76% for cystic failure-free survival. Neither OS nor disease control differed between treatment groups (OS P=.742; nodular failure-free survival P=.546; cystic failure-free survival P=.994). During therapy, 40% of patients had cyst growth (20% requiring intervention); immediately after therapy, 17 patients (33%) had cyst growth (transient in 14), more commonly in the IMRT group (42% vs 19% PBT; P=.082); and 27% experienced late cyst growth (32% IMRT, 19% PBT; P=.353), with intervention required in 40%. Toxicity did not differ between groups. On multivariate analysis, cyst growth was related to visual and hypothalamic toxicity (P=.009 and .04, respectively). Patients given radiation as salvage therapy (for recurrence) rather than adjuvant therapy had higher rates of visual and endocrine (P=.017 and .024, respectively) dysfunction.
CONCLUSIONS: Survival and disease-control outcomes were equivalent for PBT and IMRT. Cyst growth is common, unpredictable, and should be followed during and after therapy, because it contributes to late toxicity. Delaying radiation therapy until recurrence may result in worse visual and endocrine function.

Related: Pituitary Tumors

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