High-grade glioma in infants and very young children: characteristics, treatment, and outcomes.

PURPOSE: High-grade gliomas in infants and very young children (less than 3 to 5 years old) pose significant challenges due to the limited scientific literature available and high risks associated with treatments. This study aims to investigate their characteristics, treatment, and outcomes. METHODS: A cohort study was conducted at Children's Cancer Hospital, Egypt. Cases included children aged < 5 years old with confirmed CNS high-grade glioma. Baseline clinical and radiological characteristics, besides potential prognostic factors were assessed. RESULTS: In total, 76 cases were identified, 7 of them were < 1 year old. Gross- or near-total resection (GTR/NTR) was achieved in 32.9% of all cases. Of the tested cases, H3K27M-alteration was present in 5 subjects only. The 3-year OS and EFS for all cases were 26.9% and 15.4%, respectively. Extent of resection was the most important prognostic factor, as those achieving GTR/NTR experienced more than double the survival compared to those who do not (p = 0.05). Age had a "bimodal" effect on EFS, with those aged 1 to 3 years old faring better than younger and older age groups. Subjects with midline tumors had worse survival compared to non-midline tumors (1-year EFS = 18.5% vs 35%, respectively, p = 0.02). CONCLUSION: This study in a large cohort of HGG in infants and very young children offers insights into the characteristics and treatment challenges. Extent of resection, age group, and tumor localization are important prognostic factors. Further research with larger sample size is warranted to refine treatment approaches and improve outcomes.

F18-FET PET in pediatric brain tumors: integrative analysis of image derived parameters and clinico-pathological data.

BACKGROUND: F18-FET PET has an established diagnostic role in adult brain gliomas. In this study we analyzed image derived static and dynamic parameters with available conventional MRI, histological, clinical and follow-up data in assessment of pediatric brain tumor patients at different stages of the disease. METHODS: Forty-four pediatric patients with median age 7 years, diagnosed with brain tumors and underwent forty-seven 18F-FET PET scans either initially (20 scans) or post-therapy (27 scans) were enrolled. Standardized analysis of summed FET PET images early from 10-20 min and late from 30-40 min post-injection were used for static (mean and maximum tumor to brain ratio [TBR] and biological tumor volume [BTV]) parameters evaluation as well as the time activity curve [TAC]. RESULTS: Nineteen out of 20 initially assessed patients had pathologically and/or clinico-radiologically proven neoplastic lesions and one patient had pathologically proven abscess. Receiver operator curve (ROC) marked early TBR max 2.95, early TBR mean 1.76, late TBR max 2.5 and late TBR mean 1.74 as discriminator points with diagnostic accuracy reaching 90% when TBR max was combined with dynamic parameters. Significant association was found between initial FET scans, early and late BTV and event free survival (EFS) (P value=0.042 and 0.005 respectively). In post-therapy assessment, the diagnostic accuracy of conventional MRI was 81.48% when used alone and 96.30% when combined with F18-FET PET scan findings. A cutoff point of 3.2 cm3 for late BTV, in post-therapy scans, was successfully marked as a predictor for therapy response (P value 0.042) and was significantly associated with EFS (P value 0.002). In FET-avid / MRI non-enhancing lesions, early TBR max was able to detect highly malignant processes (high-grade tumors in initial scans and residue/recurrence in post-therapy scans) with 80% sensitivity and 100% specificity when cutoff value of 2.25 was used (P value=0.024). In patients with FET-avid brainstem lesions, whether enhancing or non-enhancing in MRI scans, 81.8% were associated with high risk diagnoses and 68.2% of them were associated with poor therapy outcome. The degree of FET uptake matched tumor-grading, but did not show significant association with OS or EFS (P value>0.05). CONCLUSIONS: F18-FET PET seems to be an evolving pediatric neuro-imaging technique with valuable diagnostic and prognostic information at initial and post-therapy evaluation.

Prognostic factors affecting infantile medulloblastoma outcomes in the molecular era: a 12-year single-center experience from Egypt.

Aims: To assess the clinical, pathological and molecular characteristics (Sonic hedgehog and group 3/4 molecular subtypes expression) and treatment modalities for infantile medulloblastoma in correlation with outcomes. Materials & methods: A retrospective study of 86 medulloblastoma patients (≤3 years) was conducted. M0 patients <2.5 years received four cycles of chemotherapy followed by focal radiotherapy (FRT) and chemotherapy. Between 2007 and 2015, Metastatic patients <2.5 years received craniospinal irradiation (CSI) after the end of chemotherapy. After 2015, metastatic patients <2.5 years received CSI postinduction. Results: The hazard ratio for death was significantly higher in the FRT (HR = 2.8) group compared with the CSI group (hazard ratio = 1). Metastatic disease significantly affected the overall survival of the Sonic hedgehog group and the overall survival and event-free survival of group 3/4. Conclusion: Metastatic disease had a significant impact on outcomes. FRT is not effective in treating infantile medulloblastoma.

This study aimed to analyze the management of and prognostic factors affecting the outcomes of 86 young children (<3 years of age at presentation) diagnosed with medulloblastoma, an aggressive brain tumor that is commonly seen in this age group. All children had surgical operations aiming at resecting their tumors, followed by chemotherapy and irradiation. Study results showed that disease disseminated into the nervous system was associated with poorer outcomes compared with localized disease. Administration of local irradiation to the primary tumor site in the brain only, without exposing the spinal cord to radiotherapy, was associated with a higher risk of death.

Neuroblastoma-associated Opsoclonous Myoclonous Ataxia Syndrome: Profile and Outcome Report on 15 Egyptian Patients.

Opsoclonous myoclonous ataxia syndrome (OMAS) is a rare primarily immune-mediated disease in children. The current study aim was to find out the patterns and outcome of OMAS associated with neuroblastoma (NBL) among Children's Cancer Hospital-Egypt patients. Data was reviewed for 15 eligible patients enrolled between 2007 and 2016. OMAS treatment included prednisolone and cyclophosphamide with/without intravenous immunoglobulin; NBL treatment was given according to risk-corresponding protocol. Patients' age ranged from 0.75 to 12 years at presentation with male/female: 1.1/1. Concurrent diagnosis of OMAS and NBL occurred in 6 patients (40%). OMAS preceded NBL within 0.25 to 2 years in 33%, while NBL preceded OMAS within 0.5 to 1.5 years in 27%. Full OMAS picture was present in 10/15 patients, while 20% presented with truncal ataxia and myoclonus, 1 with truncal ataxia and opsoclonus, and 1 had opsoclonus and myoclonus. Median time till improvement of manifestations was 5 months. The 5-year OMAS progression-free survival was 33.3%, where 10 patients needed second-line therapy due to relapse/progression of OMAS. The median time to progression was 28 months measured from OMAS diagnosis. All patients remained alive with NBL 5-year overall survival of 100% and event-free survival of 85.7% for. However, 73% of the patients showed late sequelae ranging from ocular to cognitive, behavioral and motor disorders; rarely seizures and hemolytic anemia.

Palliative and end-of-life symptoms management for children with diffuse intrinsic pontine glioma.

Background: This study aimed to investigate diffuse intrinsic pontine glioma-specific symptoms in the last 12 weeks before death and to describe current palliative care. Materials & methods: A retrospective study included 80 pediatric diffuse intrinsic pontine glioma patients diagnosed between January 2018 and December 2019. Results: The most frequently encountered symptoms were headache, gait disturbance, vomiting, dysphagia, sensory loss, seizures and constipation. Steroids were used in 96% of patients with a high success rate, as well as analgesics (67.5%), antiemetics (59%), neuropathic medication (42.5%) and anticonvulsants (37.5%). Re-irradiation improved symptoms in 50% of patients. Conclusion: Steroids were efficient in managing many symptoms, with tolerated side effects. The symptomatic treatment succeeded in relieving end-of-life symptoms. Re-irradiation should be considered a good palliative tool in addition to regular symptomatic treatment.

This study aimed to describe symptoms in 80 patients with aggressive brain tumors in the brain stem, as well as proper management of the symptoms at the end of life. The symptoms encountered were headache, imbalance during walking, vomiting and fits. They were managed successfully with steroids, painkillers, nausea and vomiting medications and anti-fit medication. Giving radiotherapy for a second time improved symptoms in 50% of patients, and steroids can be used efficiently to improve the misery of these patients. These data can help to put a strategy for managing these devastating symptoms in the end-of-life period.

Management and outcome of pediatric brainstem and cerebellar peduncular low-grade gliomas: a retrospective analysis of 62 cases.

PURPOSE: This study was designed to present our experience and recommendations regarding the management of pediatric brainstem and peduncular low-grade gliomas (LGGs). METHODS: Retrospective analysis was performed for pathologically proven brainstem or cerebellar peduncular LGGs in patients admitted between 2014 and 2019. These lesions were classified into the dorsal exophytic, focal brainstem, cervicomedullary, lower peduncular, and upper peduncular groups, and this classification was the basis for the surgical approach for the lesions. RESULTS: Sixty-two pediatric patients were included, and their distribution among the aforementioned groups were as follows: 12, 12, 3, 16, and 19 cases in the dorsal exophytic, focal brainstem, cervicomedullary, upper peduncular, and lower peduncular groups, respectively. Stereotactic biopsy was performed for all cases in the focal brainstem group, whereas other groups underwent open excision. Gross total resection (GTR) was achieved in 20 cases (40%), near-total resection (NTR) was achieved in 17 cases (34%), and subtotal resection (STR) was achieved in 13 cases (26%). The extent of GTR and NTR for the upper peduncular, lower peduncular, dorsal exophytic, and cervicomedullary groups were 81.2%, 68.4%,75%, and 66.6%, respectively. Then, 32 cases received chemotherapy. The 3- and 5-year progression-free survival rates were 95% (95% confidence interval (CI) 89.5-100%) and 90.3% (95% CI 79.9-100%), respectively. A significant difference in the 3-year progression-free survival rate was observed between the GTR and NTR groups (p = 0.06; 100% vs. 88.2% (95% CI 72.9-100%)). CONCLUSION: Surgery plays a definitive curative role in grossly resected cases. Additionally, the role of surgical debulking should be considered, even if GTR is impossible. Meanwhile, chemotherapy showed a beneficial role in patients with focal brainstem lesions and progressive lesions, those with STR, and some patients with NTR.

Management of pediatric craniopharyngioma: 10-year experience from high-flow center.

PURPOSE: To report our experience and management strategies during 10 years for 137 childhood craniopharyngiomas treated at a single institution. METHODS: Medical records of children with craniopharyngioma treated at Children's Cancer Hospital Egypt (CCHE-57357) from July 2007 to December 2017 were retrospectively reviewed. Beta-catenin as an immunohistochemical marker was assessed also in available specimens. RESULTS: Our registry included 137 patients. Headache (n = 122), visual failure (n = 118), and hypothyroidism(n = 78) were the most common findings on presentation. Three management protocols were identified; 65 patients were primarily followed up after surgery, 71 patients had radiotherapy after surgery, and one patient underwent surgery for Ommaya insertion with intracystic interferon injection. Overall, gross total resection/near total resection was achieved in 48 cases (35.04%), subtotal resection was achieved in 58 patients (42.33%), 29 (21.16%) had biopsy and Ommaya reservoir, and two patients with calcified lesions had no operations. Fifty-four patients showed recurrence/progression of their lesions. Allover, 5-year progression-free survival (PFS) was 52.3%, while it was 34.49% and 72.25% for the follow-up group and the radiotherapy group, respectively. Beta-catenin mutations were positive in 61/95 patients; 5-year PFS for beta-catenin negative and positive cases was 65.5% and 39.4% respectively (p = 0.087). Mortality was reported in eight patients. Intraoperative endoscopy-assisted assessment was the cornerstone of tailored decision-making. CONCLUSION: The concepts of conservative surgery and multimodal management should be applied to reach the perfect balance between the quality of life and the best tumor control rates. Beta-catenin mutations more than 5% are associated with statistically trending aggressive clinical behavior. The CCHE-57357 algorithm of individualized management protocol was presented.

Optimizing diagnostic imaging data using LI-RADS and the Likert scale in patients with hepatocellular carcinoma.

PURPOSE: The study aimed to compare the diagnostic performance of the Liver Imaging Reporting and Data System (LI-RADS), which incorporates fixed criteria, and the Likert scale (LS), which mainly depends on an overall impression in liver lesion diagnosis. MATERIAL AND METHODS: Diagnostic data of 110 hepatic nodules in 103 high-risk patients for hepatocellular carcinoma (HCC) were included. Data including diameter, arterial hyperenhancement, washout, and capsule were reviewed by 2 readers using LI-RADS and LS (range, score 1-5). Inter-reader agreement (IRA), intraclass agreement (ICA), and diagnostic performance were determined by Fleiss, Cohen's k, and logistic regression, respectively. RESULTS: There were 53 triphasic enhanced computed tomography (CT) and 50 dynamic magnetic resonance (MR) examinations. Overall, IRA was excellent (k = 0.898). IRA was good for arterial hyperenhancement (k = 0.705), washout (k = 0.763), and capsule (k = 0.771) and excellent for diameter (k = 0.981) and tumour embolus (k = 0.927). Overall, ICA between LI-RADS and LS was fair 0.32; ICA was good for scores of 1 (k = 0.682), fair for scores of 2 (k = 0.36), moderate for scores of 5 (k = 0.52), but no agreement was found for scores of 3 (k = -0.059) and 4 (k = -0.022). LIRADS produced relatively high accuracy (87.3% vs. 80%), relatively low sensitivity (84.3% vs. 98%), and significantly higher specificity (89.83% vs. 64.4%) and positive likelihood ratio (+LR: 8.29 vs. 2.75) compared to LS approach. CONCLUSIONS: LI-RADS revealed higher diagnostic accuracy as compared to LS with statistical proof higher specificity and +LR showing its ability to foretell malignancy in high-risk patients. We recommend the practical application of the LI-RADS system in the detection and treatment response monitoring of patients with HCC.

Pediatric low grade focal brainstem glioma: outcomes of different treatment strategies and prognostic factors.

Background: This study explores the prognostic factors and outcomes of different treatment modalities in focal brain stem glioma (FBSG). Materials & methods: Pediatric FBSG patients diagnosed during 2010-2017 were retrospectively reviewed for clinical and therapeutic data. Results: A total of 71 cases were identified and the median age was 6.4 years. The 5-year overall- and progression-free survival were 74.5 and 70.6%, respectively. Radiotherapy was the main line of treatment (66.2%) and there were no survival differences between radiotherapy, chemotherapy and surveillance groups. Two independent poor prognostic factors were identified on multivariate analysis: age <8 years and cervicomedullary tumor site (p = 0.02 for both). Conclusion: Surveillance, radiotherapy and chemotherapy have comparable clinical outcomes in pediatric FBSG.

Management and outcome of pediatric Wilms tumor with malignant inferior Vena cava thrombus: largest cohort of single-center experience.

BACKGROUND: Wilms tumor (WT) with an inferior Vena cava (IVC) malignant thrombus comprises 4-10% of all WT cases. METHODS: This retrospective analysis included 51 pediatric patients presenting at Children Cancer Hospital Egypt-57357 from July 2007 to December 2016 with the diagnosis of WT with malignant IVC thrombus. RESULTS: Median age at presentation = 4.4 years and 28 cases (55%) were females. Twenty-five patients (49%) were metastatic and 4 patients (7.8%) had bilateral disease. Forty-seven cases (92.2%) had favorable histology with no evidence of anaplasia. Level of thrombus extension at presentation was classified as infra-hepatic, retro-hepatic, supra-hepatic and intra-cardiac in 33, 9, 6 and 3 patients, respectively. Fifty patients started neoadjuvant chemotherapy (CTH) with 16 patients showing complete resolution of thrombus after 6 weeks of CTH. None of the patients developed thrombus progression after neoadjuvant CTH; one patient had stationary intra-cardiac thrombus, while remaining patients showed partial regression of their thrombus and had nephrectomy with en-bloc thrombectomy. The mean cranio-caudal dimension of IVC thrombi at initial presentation was 6.5 cm, and 3.6 cm post 6th week of CTH. The 5-year OS and EFS were 75.9% and 71.1%, respectively. There was no significant correlation of initial levels of thrombus extension with survival. CONCLUSION: Neoadjuvant chemotherapy followed by radical nephrectomy with en-bloc thrombectomy and radiotherapy seems a successful approach for management of patients with WT and IVC tumor thrombus. Measurement of the cranio-caudal dimension of thrombus and its response to treatment should be considered in the surgical planning.

Correction to: Risk stratification of pediatric high-grade glioma: a newly proposed prognostic score.

The original version of this article unfortunately contained an error. The author apologizes for having provided an incorrect name: "Mohamed S. Zaghluol" should be "Mohamed S. Zaghloul". Given in this article is the correct author name.

The outcome of childhood adrenocortical carcinoma in Egypt: A model from developing countries.

Adrenocortical carcinoma (ACC) is a rare, aggressive endocrine neoplasm. Complete surgical resection is the single most important treatment. Most available information has been learned from experience with its more frequent adult counterpart. In this study, we assessed the features and survival outcome of patients with ACC at Children's Cancer Hospital Egypt (CCHE). Patients diagnosed at CCHE between July 2007 and November 2016 were followed up on until November 2018. Patients with stages I and II were operated upon, while stages III and IV had received combinations of doxorubicin, etoposide, platinol, and mitotane (DEPM) beside the attempt to conduct surgery when feasible. Data belonging to 18 patients (7 men and 11 women) were analyzed; median age at diagnosis was 48.5 months. Sixteen patients had presented with secreting tumors. Six patients were diagnosed with stage I disease; four with stage II; three with stage III; and five with stage IV carcinoma. By the end of this study, 10 patients have survived; five-year overall survival of 66.3%. Surviving patients were all of stage I or II diseases and were all in remission. Seven patients who did not survive died due to tumor progression, while one patient died after chemotherapy. The prognosis of ACC is essentially dependent on a successful complete resection of the tumor and thus on the initial tumor stage. The mitotane and DEP protocols may help control tumor growth in the advanced stages for only short periods. Key pointsInitial stage and resectability are the main indicators of outcomes in adrenocortical carcinoma.Chemotherapeutic agents used in developed countries did not achieve the same outcomes.Further molecular-pharmacology differentiation is needed for various ethnic populations.

Watch and See Strategy in Selected Neuroblastoma Case Scenarios: Success and Limitations.

Neuroblastoma (NBL) in infants has the potential to regress/mature spontaneously. The literature showed some cases, subjected to initial observation, with reasonable outcome. Deferring/avoiding active treatment was investigated in selected favorable NBL cases. Patients enrolled on the watch and see strategy (W&S) had small primary tumor, localized stages 1 to 2, uncomplicated stage 4s, or stage 3. Tissue biopsy was not mandatory for infants below 6 months with localized mass. On progression, active intervention was indicated according to disease stage and risk after biological characterization. In total, 32 patients were enrolled on W&S strategy; male/female:2.6/1. Twelve had stages 1 to 2, 16 had stage 4s, and 4 were stage 3. Primary adrenal site was reported in 85% patients, and 65% patients had small mass (≤5 cm). Five-year overall and event-free survival were 100% and 80.9±7%, respectively, with a 43-month median follow-up duration. Spontaneous total/near total resolution of mass occurred in 50% patients. Median time to regression was 1.7 months, and 20.7 months until resolution. Only 19% patients witnessed progression; median time to progression was 4.8 months. W&S is a reasonable approach for localized and uncomplicated stages 3 and 4S NBL. Extended tumor size is a newly investigated entity in the present study. All progressive cases were safely rescued with 100% survival outcome.

Management of pediatric CNS meningiomas: CCHE-57357 experience in 39 cases.

PURPOSE: Our aim is to present our experience in the management of pediatric meningiomas in the largest referral center in Egypt for pediatric tumors focusing on variables including clinical picture, anatomic location, histopathology, treatment strategies, and outcome together with their possible correlation to prognosis. METHODS: We retrospectively reviewed the medical records of 39 pediatric patients who were treated for CNS meningiomas in Children's Cancer Hospital-Egypt (CCHE-57357) 2007-2017. RESULTS: The prevalence of pediatric meningioma was 1.42%. Four cases had type 2 neurofibromatosis (NFII). The mean age was 8.19 years. The presence of NFII was associated with challenging multiple lesions, older age of presentation and poorer prognosis and functional outcome. Convexity was the commonest location. Gross total resection (GTR) was achieved in 28 cases, subtotal resection (STR) in 8 cases, and biopsy was decided in 3 patients. Histopathological examination revealed WHO grade I in 16 patients and higher grades in 23 patients (59%). The 5-year overall survival (OS) rate was 87.8% while the 5-years event-free survival (EFS) rate was 85.6%. Tumor location, histopathology, and clinical presentation were not statistically correlated to prognosis. CONCLUSIONS: Pediatric CNS meningiomas are uncommon pediatric tumors but of an aggressive clinical and pathological behaviors as compared to adult meningiomas. The presence of NFII is associated with a poorer prognosis and functional outcomes. Although being challenging, the maximum and safe surgical excision should be exercised even in recurrent cases in order to achieve the best outcome. Adjuvant radiotherapy provides good tumor control for inoperable residual atypical or anaplastic meningiomas.

Risk stratification of pediatric high-grade glioma: a newly proposed prognostic score.

OBJECTIVES: High-grade glioma (HGG) is a clinical challenge. Radiation Therapy Oncology Group Recursive Partitioning Analysis (RTOG-RPA) for HGG remains the standard for assessing the prognosis of adult HGG. This study assesses the validity of the RTOG-RPA to be applied to pediatric HGG. METHODS: A retrospective study was conducted on 59 pediatric HGG treated in the Children's Cancer Hospital, Egypt (CCHE) between 2007 and 2016. Several factors were studied as predictors for the disease survival, including age, gender, increased intracranial hypertension, tumor characteristics and pathology, CSF seeding, performance status, post-surgical residual, and radiation dose. The statistically significant results were integrated into a Cox-regression model to develop a prognostic risk score. RESULTS: Kaplan-Meier statistics identified 13 factors that impacted the overall survival. However, Cox model showed that the histological grade IV [HR 14.2, 95%CI; (3.5-57), P < 0.0001], thalamic infiltration [HR 8.7; 95%CI; (2.9-25.9), P < 0.0001], PS ≥ 60 [HR 0.317; 95%CI; (0.13-0.776); P = 0.012], and maximum tumor dimension > 3.3 cm [HR 10.2; 95%CI; (1.58-65.89); P = 0.015] were the independent variables that predicted the overall survival. A risk score was proposed based on the presence of one or more of these factors. The median OS for the low risk (score 0-1), the intermediate-low risk (score 2), the intermediate-high risk (score 3), and the high risk (score 4) were 40, 18.5, 9.5, and 2.5 months, respectively, (P < 0.0001). CONCLUSION: The proposed model and risk score could stratify pediatric patients as the RTOG-RPA do for the adults.

Pediatric brain tumors in a low/middle income country: does it differ from that in developed world?

Central nervous system (CNS) tumors are the most frequent solid tumors in children and adolescents. The epidemiology of these tumors differs in areas of the world. However, very little data is available in the low/middle income countries (LMIC). The aim of this study is to describe the characteristics of primary childhood brain tumors treated at a leading LMIC pediatric cancer hospital and its difference from that in other countries. One thousand one hundred fourteen children and adolescent having CNS tumors were treated in the largest pediatric cancer hospital in the Middle East during a period of 5½ years. They were diagnosed histopathologically in 80.2 %, through medical imaging in 19.4 % and via both tumor markers and imaging in the remaining 0.4 % of cases. Through epidemiological analysis was performed using all available patients' data revealed that 96 % of the patients had primary brain tumors, while only 4 % the primary lesion was in the spinal cord. The most common histological type was astrocytic tumor (30.0 %, pilocytic (GI) = 13.2 %, GII = 10.5 % and GIII + IV (high grade) = 6.3 %) followed by embryonal tumor (23.2 %, medulloblastoma = 18.7 %, PNET = 2.8 %, ATRT = 1.5 % and ependymoblastoma = 0.2 %) then ependymoma in 8.7 %, craniopharyngeoma in 5.3 %. The mean age at diagnosis was 7.1 ± 4.2 years which did not differ significantly by gender nor residency but it differed by the pathological subtype. The frequency of each pathological type was different among different age groups. Though the present study was a hospital-based analysis in a low/middle income country, yet it did not differ from the well-established population-based study reports in the high income countries.

Clear cell sarcoma of the kidney: patients' characteristics and improved outcome in developing countries.

BACKGROUND: Clear cell sarcoma of the kidney (CCSK) is a rare and aggressive tumor accounting for 5% of pediatric renal tumors with an incidence of 20 patients per year in the USA. It is bone metastasizing with poor prognosis. Our aim was to show characteristics of patients in relation to improved outcome in one of the developing countries. PROCEDURE: We included all patients diagnosed as CCSK in the period between July 2007 and March 2012 at Children's Cancer Hospital, Egypt. Patients' demographics, clinical presentation, pathology, and management were reviewed. Follow up was continued until April 2013. RESULTS: Twenty-five patients were identified in the defined time interval, accounting for 7% all renal tumors diagnosed at the hospital. Mean age was 36 months. Abdominal swelling and hematuria were the most common presentations. Stages I, II, III, IV, and V represented 9 (36%), 3 (12%), 8 (32%), 3 (12%), and 2 (8%), respectively. Twenty-four patients had radical nephrectomy either upfront or after neo-adjuvant chemotherapy. Surgery was followed by adjuvant chemotherapy. Abdominal radiotherapy was given for local stages II and III. Twenty-two patients reached complete remission, while one patient had stationary disease and two patients died due to progression and relapse. Overall survival was 88.5% and event-free survival was 87.8% at 45 months. CONCLUSION: Although previous studies indicate poor prognosis of CCSK, our experience shows that those patients can be treated using extensive chemotherapy combined with proper local control.

Bilateral adrenal neuroblastoma: peculiar pattern of a rare pediatric presentation.

BACKGROUND: Bilateral suprarenal neuroblastoma (BSN) is a rare presentation. Few previously published literature showed BSN patients to have favorable pattern and prognosis. This study aim was to evaluate clinical and biological features in relation to outcome of Egyptian patients with BSN. METHODS: Included patients were diagnosed from 2007 to 2017, retrospectively. Tissue biopsy, imaging and bone marrow were evaluated at presentation. Clinical, demographic, biological variables and risk group were determined and analyzed in relation to overall (OS) and event-free-survival (EFS). RESULTS: BSN patients (n = 33) represented 2% of hospital patients with neuroblastoma during the 10-year study period, 17 were males and 16 were females. Twenty-four patients (72.7%) were infants, and 9 patients (27.3%) were above 1 year of age (range: 1 month to 3 years). Metachronous disease was present in only one patient. Amplified MYCN was found in 10 patients. Initially, most patients (n = 25) had distant metastasis, 6 had stage 3 versus 2 stage 2. Fifteen were high risk (HR), 15 intermediate (IR), 1 low risk (LR) and 2 were undetermined due to inadequate tissue biopsy. Three-year OS for HR and IR patients were 40.5% and 83.9% versus 23.2% and 56.6% EFS; respectively. CONCLUSION: BSN treatment is similar to unilateral disease. A more conservative surgical approach with adrenal tissue preservation on less extensive side should be considered. Biological variables and extent of disease are amongst the most important prognostic determinants. Future studies are warranted to further address the biologic profiling of BSN and highlight prognostic significance of size difference between both adrenal sides.

Atypical teratoid rhabdoid tumor in a lower middle­income country: Challenges to cure.

Atypical teratoid rhabdoid tumor (ATRT) is a rare type of potentially fatal childhood brain tumor. The present study aimed to examine the overall survival (OS) and event-free survival (EFS) outcomes of pediatric patients with ATRT and to analyze the impact of different prognostic factors, including age, sex, tumor site and size, metastatic disease, the extent of resection, radiotherapy, and chemotherapy, on survival. The present study included 47 patients with ATRT treated at the Children's Cancer Hospital of Egypt (Cairo, Egypt) between July 2007 and December 2017. These patients were treated according to the Dana-Farber Cancer Institute protocol 02-294 for 51 weeks. Various prognostic factors, including age, sex, tumor size and initial metastatic status, exhibited no impact on the radiological response measured at 6 weeks and at the end of treatment. The primary tumor site significantly affected the response to treatment at 6 weeks (P=0.008). Toxicity-related mortality occurred in 29.8% of patients. The median duration of the treatment protocol was 66.9 weeks. The duration of treatment was in the present cohort was longer than the actual 51 weeks of the protocol due to prolonged supportive care of the included patients. Patients who encountered toxicity received reduced dose of chemotherapy in the subsequent cycles in the protocol. Age, initial metastatic status, tumor site and resection extent did not significantly affect the patient outcomes. Preoperative tumor size significantly affected the EFS (P=0.03) and OS (P=0.04). Radiotherapy administration significantly affected the OS (P<0.001) and EFS (P<0.001). The median EFS and OS of patients were 9.3 and 10.3 months, respectively. A total of 24 (51.1%) patients exhibited disease progression or recurrence. The progression sites were local (n=6), metastatic (n=9) or both local and metastatic (n=9). The results of the present study demonstrated that the therapeutic regimen should be patient-adjusted to maintain the treatment intensity and avoid toxicity-related mortality. In lower middle-income countries, short and intensified induction followed by consolidation of treatment, either by single or tandem autologous stem cell transplant, is needed to avoid prolonged exposure to myelosuppression and toxicity-related mortality.

Plasma miRNA expression profile in pediatric pineal pure germinomas.

Background: Pure germinomas account for 40% of pineal tumors and are characterized by the lack of appreciable tumor markers, thus requiring a tumor biopsy for diagnosis. MicroRNAs (miRNA) have emerged as potential non-invasive biomarkers for germ cell tumors and may facilitate the non-invasive diagnosis of pure pineal germinomas. Material and methods: A retrospective chart review was performed on all patients treated at the Children's Cancer Hospital Egypt diagnosed with a pineal region tumor between June 2013 and March 2021 for whom a research blood sample was available. Plasma samples were profiled for miRNA expression, and DESeq2 was used to compare between pure germinoma and other tumor types. Differentially expressed miRNAs were identified. The area under the curve of the receive;r operating characteristic curve was constructed to evaluate diagnostic performance. Results: Samples from 39 pediatric patients were available consisting of 12 pure germinomas and 27 pineal region tumors of other pathologies, including pineal origin tumors [n = 17; pineoblastoma (n = 13) and pineal parenchymal tumors of intermediate differentiation (n = 4)] and others [n = 10; low-grade glioma (n = 6) and atypical teratoid rhabdoid tumor (n = 4)]. Using an adjusted p-value <0.05, three miRNAs showed differential expression (miR-143-3p, miR-320c, miR-320d; adjusted p = 0.0058, p = 0.0478, and p = 0.0366, respectively) and good discriminatory power between the two groups (AUC 90.7%, p < 0.001) with a sensitivity of 25% and a specificity of 100%. Conclusion: Our results suggest that a three-plasma miRNA signature has the potential to non-invasively identify pineal body pure germinomas which may allow selected patients to avoid the potential surgical complications.