Reirradiation for diffuse intrinsic pontine glioma: prognostic radiomic factors at progression.

PURPOSE: Diffuse intrinsic pontine glioma (DIPG) is a lethal pediatric brain tumor. Radiation therapy (RT) is the standard treatment, with reirradiation considered in case of progression. However, the prognostic factors for reirradiation are not well understood. This study aims to investigate the outcomes of DIPG patients undergoing reirradiation and identify clinical and radiomic prognostic factors. METHODS: We conducted a retrospective analysis of patients with DIPG who underwent reirradiation at our institution between January 2016 and December 2023. Using PyRadiomics, we extracted radiomic features of tumors at the time of progression from FLAIR MRI images and collected clinical data. We used the least absolute shrinkage and selection operator (lasso) for Cox's proportional hazard model with leave-one-out cross-validation to select optimal prognostic factors for survival after reirradiation. RESULTS: The study included 18 patients who underwent reirradiation at first progression, receiving a total dose of 20 Gy or 24 Gy in 2­Gy fractions. Reirradiation was well tolerated, with no severe toxicity. Most patients (78%) showed neurological improvement after treatment. Median survival after progression was 29.2 weeks. The Cox model demonstrated a concordance of 0.81 (95% CI: 0.75-0.88), revealing that tumor sphericity and structural gray-level heterogeneity in FLAIR MRI images were associated with longer survival of reirradiated patients. CONCLUSION: Reirradiation is a safe and effective approach for patients with DIPG. MRI-based radiomic models could be helpful in predicting survival after reirradiation.

Anti-PD-1 Therapy in Advanced Pediatric Malignancies in Nationwide Study: Good Outcome in Skin Melanoma and Hodgkin Lymphoma.

BACKGROUND/AIM: The role of immune checkpoint inhibitors (ICIs; anti-PD1) in the treatment of childhood cancers is still evolving. The aim of this nationwide retrospective study was to assess the safety and effectiveness of ICIs used in a group of 42 patients, with a median age of 13.6 years, with various types of advanced malignancies treated in pediatric oncology centers in Poland between 2015 and 2023. RESULTS: The indications for treatment with anti-PD1 were as follows: Hodgkin lymphoma (11); malignant skin melanoma (9); neuroblastoma (8); and other malignancies (14). At the end of follow-up, complete remission (CR) was observed in 37.7% (15/42) of children and disease stabilization in 9.5% (4/42), with a mean survival 3.6 (95% CI = 2.6-4.6) years. The best survival (OS = 1.0) was observed in the group of patients with Hodgkin lymphoma. For malignant melanoma of the skin, neuroblastoma, and other rare malignancies, the estimated 3-year OS values were, respectively, 0.78, 0.33, and 0.25 (p = 0.002). The best progression-free survival value (0.78) was observed in the group with malignant melanoma. Significantly better effects of immunotherapy were confirmed in patients ≥ 14 years of age and good overall performance ECOG status. Severe adverse events were observed in 30.9% (13/42) patients.

Risk-Adapted Treatment Strategies with Pre-Irradiation Chemotherapy in Pediatric Medulloblastoma: Outcomes from the Polish Pediatric Neuro-Oncology Group.

Craniospinal irradiation (CSI) has been a major component of the standard of care treatment backbone for childhood medulloblastoma. However, chemotherapy regimens have varied based on protocol, patient age, and molecular subtyping. In one of the largest studies to date, we analyzed treatment outcomes in children with newly-diagnosed medulloblastoma treated with pre-irradiation chemotherapy followed by risk-adapted radiotherapy and maintenance chemotherapy. A total of 153 patients from the Polish Pediatric Neuro-Oncology Group were included in the analysis. The median age at diagnosis was 8.0 years, and median follow-up time was 6.4 years. Sixty-seven patients were classified as standard-risk and eighty-six as high-risk. Overall survival (OS) and event-free survival (EFS) for standard-risk patients at 5 years (±standard error) were 87 ± 4.3% and 84 ± 4.6%, respectively, while 5-year OS and EFS for high-risk patients were 81 ± 4.3% and 79 ± 4.5%, respectively. Only one patient had disease progression prior to radiotherapy. This study demonstrates promising survival outcomes in patients treated with pre-irradiation chemotherapy followed by risk-adapted CSI and adjuvant chemotherapy. Such an approach may be useful in cases where the initiation of radiotherapy may need to be delayed, a common occurrence in many institutions globally.

Infrared Spectroscopy as a Potential Diagnostic Tool for Medulloblastoma.

INTRODUCTION: Medulloblastoma (MB) is the most common malignant tumor of the central nervous system in childhood. FTIR spectroscopy provides a holistic view of the chemical composition of biological samples, including the detection of molecules such as nucleic acids, proteins, and lipids. This study evaluated the applicability of FTIR spectroscopy as a potential diagnostic tool for MB. MATERIALS AND METHODS: FTIR spectra of MB samples from 40 children (boys/girls: 31/9; age: median 7.8 years, range 1.5-21.5 years) treated in the Oncology Department of the Children's Memorial Health Institute in Warsaw between 2010 and 2019 were analyzed. The control group consisted of normal brain tissue taken from four children diagnosed with causes other than cancer. Formalin-fixed and paraffin-embedded tissues were sectioned and used for FTIR spectroscopic analysis. The sections were examined in the mid-infrared range (800-3500 cm-1) by ATR-FTIR. Spectra were analysed using a combination of principal component analysis, hierarchical cluster analysis, and absorbance dynamics. RESULTS: FTIR spectra in MB were significantly different from those of normal brain tissue. The most significant differences related to the range of nucleic acids and proteins in the region 800-1800 cm-1. Some major differences were also revealed in the quantification of protein conformations (α-helices, ß-sheets, and others) in the amide I band, as well as in the absorbance dynamics in the 1714-1716 cm-1 range (nucleic acids). It was not, however, possible to clearly distinguish between the various histological subtypes of MB using FTIR spectroscopy. CONCLUSIONS: MB and normal brain tissue can be distinguished from one another to some extent using FTIR spectroscopy. As a result, it may be used as a further tool to hasten and enhance histological diagnosis.

Nivolumab for the Treatment of Advanced Pediatric Malignancies.

BACKGROUND/AIM: Nivolumab is an immune checkpoint inhibitor with high antitumor activity in selected neoplasms. The aim of the study was to evaluate the efficacy and safety of nivolumab in pediatric patients with various types of highly malignant advanced tumors. PATIENTS AND METHODS: Ten patients with a median age of 15.1 years were included in the study. The indications for treatment were: malignant skin melanoma (n=5), brain tumor (n=2), malignant melanoma of the brain (n=1), Hodgkin lymphoma (n=1) and soft tissue sarcoma (n=1). RESULTS: Complete disease remission was observed in 4 patients. Overall survival at 24 months from diagnosis for the entire group was 0.36. Two patients receiving combination therapy of nivolumab and ipilimumab did not achieve a remission. Adverse events of immunotherapy were observed in 4/10 patients. CONCLUSION: Nivolumab is a promising option in pediatric advanced malignancies. Treatment with immunotherapy was relatively well tolerated, and emerging side-effects were manageable.

Diagnostics and treatment of diffuse intrinsic pontine glioma: where do we stand?

INTRODUCTION: Diffuse intrinsic pontine glioma (DIPG) is a rare clinically, neuro-radiologically, and molecularly defined malignancy of the brainstem with a median overall survival of approximately 11 months. Our aim is to evaluate the current tendency for its treatment in Europe in order to develop (inter)national consensus guidelines. METHODS: Healthcare professionals specialized in DIPG were asked to fill in an online survey with questions regarding usual treatment strategies at diagnosis and at disease progression in their countries and/or their centers, respectively. RESULTS: Seventy-four healthcare professionals responded to the survey, of which 87.8% were pediatric oncologists. Only 13.5% of the respondents biopsy all of their patients, 41.9% biopsy their patients infrequently. More than half of the respondents (54.1%) treated their patients with radiotherapy only at diagnosis, whereas 44.6% preferred radiotherapy combined with chemotherapy. When the disease progresses, treatment strategies became even more diverse, and the tendency for no treatment increased from 1.4% at diagnosis to 77.0% after second progression. 36.5% of the healthcare professionals treat children younger than 3 years differently than older children at diagnosis. This percentage decreased, when the disease progresses. Most of the participants (51.4%) included less than 25% of their patients in clinical trials. CONCLUSION: This survey demonstrates a large heterogeneity of treatment regimens, especially at disease progression. We emphasize the need for international consensus guidelines for the treatment of DIPG, possible by more collaborative clinical trials.

Medulloblastoma with transitional features between Group 3 and Group 4 is associated with good prognosis.

Medulloblastoma, the most common malignant pediatric brain tumor, is a heterogeneous disease, with the existence of at least four molecular types: Wingless (WNT), Sonic Hedgehog (SHH), Group 3 and Group 4 tumors. The latter two groups, which can be identified by an application of multi-gene expression or methylation profiling, show sometimes ambiguous categorization and are still classified for diagnostic reason as non-SHH/non-WNT medulloblastomas in updated WHO 2016 classification. In order to better characterize non-SHH/non-WNT tumors, we applied the method based on the Nanostring nCounter Technology, using the 26 genes codeset in 68 uniformly treated medulloblastoma patients. This allowed for identification of tumors, which shared common Group 3 and Group 4 gene signatures. We recognized three transcriptional groups within non-WNT/non-SHH tumors: Group 3, Group 4 and the Intermediate 3/4 Group. Group 3, in line with previously published results, showed poor prognosis with survival rate < 40%, frequent metastases, large cell/anaplastic pathology and presence of tumors with MYCC amplification. This is in contrast to patients from the Intermediate 3/4 Group who showed the best survival rate (100%). Overall and progression free survival were better for this group than for Group 3 (p = 0.001, for both) and Group 4 (p = 0.064 and p = 0.066, respectively). Our work supports the view that within the non-WNT/non-SHH tumors different risk groups exist and that the current two groups classifier may be not sufficient for proper clinical categorization of individual patients.

The germline variants in DNA repair genes in pediatric medulloblastoma: a challenge for current therapeutic strategies.

BACKGROUND: The defects in DNA repair genes are potentially linked to development and response to therapy in medulloblastoma. Therefore the purpose of this study was to establish the spectrum and frequency of germline variants in selected DNA repair genes and their impact on response to chemotherapy in medulloblastoma patients. METHODS: The following genes were investigated in 102 paediatric patients: MSH2 and RAD50 using targeted gene panel sequencing and NBN variants (p.I171V and p.K219fs*19) by Sanger sequencing. In three patients with presence of rare life-threatening adverse events (AE) and no detected variants in the analyzed genes, whole exome sequencing was performed. Based on combination of molecular and immunohistochemical evaluations tumors were divided into molecular subgroups. Presence of variants was tested for potential association with the occurrence of rare life-threatening AE and other clinical features. RESULTS: We have identified altogether six new potentially pathogenic variants in MSH2 (p.A733T and p.V606I), RAD50 (p.R1093*), FANCM (p.L694*), ERCC2 (p.R695C) and EXO1 (p.V738L), in addition to two known NBN variants. Five out of twelve patients with defects in either of MSH2, RAD50 and NBN genes suffered from rare life-threatening AE, more frequently than in control group (p = 0.0005). When all detected variants were taken into account, the majority of patients (8 out of 15) suffered from life-threatening toxicity during chemotherapy. CONCLUSION: Our results, based on the largest systematic study performed in a clinical setting, provide preliminary evidence for a link between defects in DNA repair genes and treatment related toxicity in children with medulloblastoma. The data suggest that patients with DNA repair gene variants could need special vigilance during and after courses of chemotherapy.

ALK Expression Is a Novel Marker for the WNT-activated Type of Pediatric Medulloblastoma and an Indicator of Good Prognosis for Patients.

ALK gene rearrangements were identified in a variety of cancers, including neuroblastoma, where the presence of ALK expression is associated with adverse prognosis. ALK mutations have recently been found in the pediatric brain tumor medulloblastoma, and microarray data indicate that ALK is highly expressed in a subset of these tumors. Therefore, we investigated whether ALK expression correlates with transcriptional profiles and clinical features of medulloblastoma. Tumors from 116 medulloblastoma patients were studied at diagnosis for the detection of ALK expression at the RNA level by an application of NanoString technology and at the protein level by immunohistochemistry using antibody ALK clone D5F3. The results indicate that ALK expression, at both the RNA and the protein levels, is strongly associated with the WNT-activated type of tumors and therefore may serve as a useful marker for the detection of this type of medulloblastoma. Importantly, ALK protein expression alone is also an indicator of good prognosis for medulloblastoma patients.

Contrast enhancement pattern predicts poor survival for patients with non-WNT/SHH medulloblastoma tumours.

Recent studies revealed the biological heterogeneity of medulloblastoma, with the existence of at least four groups which are associated with several clinical and morphological features. We investigated for further correlations between molecular types, location of tumours, their contrast enhancement pattern and survival of patients. Altogether 76 tumours were analyzed and molecular subtypes were identified by immunohistochemistry using representative antibodies, detection of chromosome 6 monosomy and CTNNB1 mutation. The site of the tumour was assessed on diagnosis using Magnetic Resonance images and intra-operative surgical reports. In addition, the gadolinium enhancement pattern was also investigated in pre-treatment tumours. Cerebellar hemispheric location was associated with SHH tumours (p < 0.001), as opposed to midline location being typical for WNT and non-WNT/SHH tumours. Remarkably, for patients with non-WNT/SHH tumours, the extensive gadolinium enhancement pattern (present in >75% of tumour volume) predicted worse OS and EFS than for those with none/weak or heterogeneous enhancement (>10-75% of tumour volume), (both p < 0.001). Our analysis indicates that distribution of the medulloblastoma tumours location is related to the biological characteristics of tumour. Importantly, the enhancement pattern of the tumour may be a clinically useful prognostic marker for patients with non-WNT/SHH medulloblastomas.

Papillary ependymoma with unique superficial cortical location: immunohistochemical and ultrastructural studies. A case report.

Ependymomas are relatively rare neoplasms of the central nervous system that typically develop along cerebral ventricles and central canal of spinal cord. Occasionally, the tumours of ependymal origin arise supratentorially in brain parenchyma as ectopic cortical mass without any connection to the ventricular system. Ependymomas are heterogeneous group of tumours including cellular, papillary, clear cell and tanacytic histology. The papillary ependymoma is an unusual variant of ependymoma characterized by distinct morphology resembling other papillary tumours and corresponding to WHO grade II malignancy. We present an unique case of ependymoma with distinctive papillary morphology at ectopic superficial cortical localization. The tumour occurred in eleven-years-old girl as a large, well-circumscribed mass in the left parietal lobe without continuity with the ventricular system. The patient presented with severe headache, vomiting and sudden-onset right hemiparesis. Histopathologically, the tumour revealed distinct papillary pattern with numerous pseudorosettes. Immunohistochemically, the neoplastic cells of both papillary structures and pseudorosettes were positive for glial fibrillary acidic protein and vimentin, whereas they were only slightly immunoreactive for epithelial membrane antigen and negative for cytokeratins. Ultrastructural findings revealed the presence of cilia usually located in the neoplastic cell bodies and intermediate glial-like filaments. The final diagnosis of papillary ependymoma at ectopic superficial localization was based on both, immunophenotypic profile and ultrastructural features that confirmed ependymal nature of neoplastic cells.

Papillary pineocytoma in child: a case report.

BACKGROUND: Papillary pineocytoma is an extremely rare tumor usually with a poor outcome. CASE REPORT: We report a case of a 10-year-old-girl with pineal gland tumor and obstructive hydrocephalus diagnosed using MRI. The child was successful treated by insertion of a ventriculoperitoneal shunt and consecutive tumor resection by supracerebellar-infratentorial approach. Histopathological examination showed a papillary structure of the pineocytoma. As such, tumors are considered to be aggressive the child was subjected to radio- and chemotherapy. CONCLUSION: At six year follow-up after surgery, the patient is symptom-free and the MRI shows no tumor recurrence.

[Children with neurofibroma type 1 treated in the Children's Memorial Health Institute]. / Dzieci z neurofibromatoza typu 1 (NF1--choroba Recklinghausena) w materiale Instytutu "Pomnik--Centrum Zdrowia Dziecka".

UNLABELLED: Neurofibromatosis type I (NF1) is a relatively frequent autosomal dominant disorder with different manifestations from mild cosmetic problems to severe disease requiring multidisciplinary treatment. THE AIM of our study was lo analyze types of disorders in paediatric population of NF1 patients treated in the Children's Memorial Health Institute and to present the schedule of care adopted in our institution. MATERIAL AND METHODS: Medical records of 130 children, 70 girls and 60 boys aged from 1 year to 20 years 3 ms treated in the Children's Memorial Health Institute were analyzed. Type of mutation (familial or sporadic), age at diagnosis, type and frequency of disorders in the whole group of patients and in the familial and sporadic types were assessed. RESULTS: The familial type of NF1 was found in 58 patients, among whom there were, 23 patients from 10 families. It was sporadic in 40 pts and in 32 the type of mutation is unknown. The age at diagnosis ranged from 3 months to 17 years, median 6 years. The most frequent disorders were: T2-weighted hyperintensities of the white matter in 77 pts (59%), schooling problems in 60 pts (46%), skeletal deformations in 49 pts (37%), dysmorphia in 39 pts (30%), hyperactivity in 34 pts (26%), epilepsy in 29 pts (22%). There was a correlation between white matter hyperintensities and the presence of optic nerves gliomas (71%), schooling problems (58%), hyperactivity (77%) and dysarthria (85%). In 65 pts (53%) optic nerve gliomas were found, 45 patients required treatment. Plexiform neurofibroma requiring treatment was found in 25 pts (19%,), 6 had intraspinal penetration and in 4 spine compression was observed. Two patients were diagnosed with ganglioneuroma. Malignant tumours were found in 7 pts (5%), with a prevalence of soft tissues sarcomas. Skeletal disorders occurred more frequently in tNFl sporadic type than in familial cases (p=0.05). Based on our experience a management protocol was formulated. At the time of diagnosis this includes examination by the oncologist, neurologist, ophthalmologist, geneticist and additional investigations: abdominal ultrasound, chest X-rays, MR of the brain; found disorders indicate further consultations and studies. The check-up should be repeated annually and include all previous studies that revealed any abnormalities. CONCLUSIONS: 1. Diagnosis of NF1 is delayed until complications of the disease occur. 2. In the studied group skeletal disorders were more frequent in the sporadic than in the familial type. 3. The paediatric population with NF1 varies from the adult NF1 patients in type of disorders, diagnostic and treatment requirements. Therefore there is a need for different care standards in the two groups. 4. The knowledge about NF1 and the standards of care should be transferred to general practitioners in order to diagnose these patients early and to refer them to specialized centres.

[Cancer among adolescents 15-19 years of age. Own experience]. / Nowotwory u mlodziezy w wieku 15-19 lat. Badania wlasne.

BACKGROUND: Adolescents aged 15-19 years are variably included in analyses of childhood cancer. They should be considered separately because tumours that occur in adolescents differ from those in younger children. AIM: To describe the distribution of tumour types and treatment results in this group of patients treated in the Department of Pediatric Oncology at The Children's Memorial Health Institute. METHODS: Retrospective analysis of medical records of patients aged 15-19 years treated in our institution was performed. It included demographic data, tumour types and treatment results. RESULTS: Between 1998-2004, 207 pts, 110 boys and 97 girls aged 15-19 yrs (median--16.5 yrs) were treated. Distribution of tumours was as follows: CNS tumours--74 pts (35.7%), HD -18 pts (8.7%), NHL--13 pts (6.3%), bone tumours--31 pts (15%), STS--23 pts (11.1%), gonadal tumours--12 pts (5.8%), carcinomas--16 pts (7.7%), hepatomas--5 pts (2.4%), neuroblastoma--3pts (1.5%) and other 12 pts (5.8%). Out of 207 pts 130 are alive (62.8%). Seventy seven (37.2%) pts died--64 (83.1%) from disease, 9 (11.6%) from chemotherapy complications, 4 due to other reasons. 111 pts completed treatment and are disease free for 11 months to 7 yrs (median 3 yrs 11 mos) from diagnosis. Nineteen patients are still treated. Treatment results are as follows: CNS tumours--58.1%, HD - 88.8%, NHL--69.2%, bone tumours--51.6%, STS--65.2%, gonadal tumours--83.3%, carcinomas-- 56.25%. CONCLUSIONS: Spectrum of malignancies that occur in adolescents 15-19 years of age differs from younger children. Unlike younger patients epithelial carcinomas of adults are observed in this age group. Outcome of treatment is inferior to younger patients. Adolescents should be offered optimal treatment. This specific group should be studied in many aspects.

[Central venous catheters in children with cancer. Risk of complications. One centre experience]. / Centralne dostepy dozylne u dzieci z choroba nowotworowa. Ryzyko powiklan. Doswiadczenia jednego osrodka.

OBJECTIVE: To describe the incidence and type of central venous catheters (CVC) complications in children treated for solid tumours. MATERIAL AND METHODS: Between 1997-2005, 500 paediatric patients were treated for cancer. The CVC complications were analyzed according to the CVC type, blood product transfusion (BT) and parenteral nutrition (TPN). Chi-square test was used for statistics. RESULTS: For 566 surgically inserted CVCs: 147 (25.8%) were ports, 413 (73,6%) tunnelled catheters: Broviacs--227 (39.9%), Groshongs--186 (32.7%) and other--6 (1%). total number of CVC days was 288 944, (median: 422, range: 2-2583). 297 complications (rate of 1.02/1000 CVC days) were observed: 81 catheter infections (0.28), 77 mechanical complications (0.266), 59 no aspiration events (0.204), 52 thrombotic occlusions (0.179) and 28 tunnel infections (0.096). At the end of the study period 121 (28%) CVCs were prematurely removed due to: infection (52), mechanical cause (49), thrombotic occlusion (14), no aspiration (6). Mechanical complications in catheters comparing to ports were more frequent (p= 0.007). There were more infections in Broviacs than Groshongs catheters (p=0.022) and in children receiving BT and TPN (p=0.046 and 0.003). CONCLUSIONS: CVC's related complications were relatively rare. Most common were infections and concerned catheters and these complications were most frequent in patients receiving BP and TPN. Risk of mechanical complications was higher in catheters than ports.

[Treatment results of children and adolescents with brain stem tumours. Own experiences]. / Wyniki leczenia dzieci i mlodziezy z guzami pnia mózgu--doswiadczenia wlasne.

INTRODUCTION: Despite progress in neuro-oncology, treatment results of children and adolescents with brain stem tumours remain poor. There are also controversies concerning the role of chemotherapy in the treatment of these tumours. THE AIM of our study was to analyze treatment results of patients with brain stem tumours treated with radiotherapy alone and with the addition of chemotherapy and to assess which chemotherapy protocol is the most efficient. MATERIAL, METHODS: Between 1981-2004, 126 patients were treated in our department. The patients were divided into 2 groups according to treatment methods applied. The first group consisted of 49 patients treated with radiotherapy alone. In the second group 77 patients were irradiated and received chemotherapy. Overall survival (OS) at 1 and 5 years was analyzed in both groups. The efficacy of treatment of patients from the second group was assessed by evaluating reaction to radiotherapy, to different chemotherapy protocols and duration of tumour regression. Additionally OS was assessed separately for patients with low grade gliomas treated in the second group. RESULTS: Seven out of 49 patients from the first group are alive with a follow up from 8 years 9 months to 24 years, median 6 years 7 months. In the second group, 25 out of 77 patients are alive with the follow up from 7 months to 8 years 2 months, median--l year 7 months. OS at l and 5 years for patients treated with radiotherapy alone is 48 and 14% and for those receiving additionally irradiation 59 and 18% respectively (statistically insignificant). Best tumour response was achieved in 42% of patients with radiotherapy but in 75% of cases regression lasted from 2 weeks to l year 3 months, median 4 months. Addition of chemotherapy consisting of cisplatinum and temozolamide resulted in tumour regression in 27% of patients lasting from 4 weeks to 4 years 5 months, median 4 months. OS for patients with LGG was 34.6% at 5 years. CONCLUSIONS: 1. Radiotherapy in brain stem tumours allows to achieve tumour regression but short duration of remission and poor final outcome forces to search for other, effective treatment. 2. Patients with brain stem tumours may benefit from chemotherapy in terms of prolonging survival. 3. Chemotherapy consisting of temozolamide and cisplatinum seems to be promising and might add to improvement of treatment results, but further clinical studies are needed. 4. Patients with low grade gliomas of the brain stem constitute a separate group with a better prognosis.

[Conservative treatment in patients with unilateral retinoblastoma]. / Próba leczenia zachowawczego u pacjentów z jednooczna postacia retinoblastoma.

UNLABELLED: THE AIM of our study was to evaluate results of conservative treatment of patients with unilateral retinoblastoma. MATERIAL AND METHODS: Twenty one patients, 11 boys and 10 girls aged 2 months to 4, 5 years (median age 1 year) were studied. Local disease advancement according to Reese-Elsworth was defined in all patients. Neoadjuvant chemotherapy consisting of Vincristine, Etoposide and Carboplatin was administered. After every 2 courses tumour response was evaluated. Sixteen patients were treated with chemotherapy alone. Local treatment including brachytherapy, thermochemotherapy and cryotherapy was implemented and the choice of the method depended on the tumour's localization, size and response to chemotherapy. Statistical analysis using demographic data and survival curves were performed. RESULTS: On completion of treatment all patients achieved tumour regression. Eleven patients are progression free with a follow-up from 10 months to 6 years 4 months (median--2 yrs 5m). In 10 patients relapse was observed. A total of seven enucleations were performed in the examined group. In histopathological examination viable tumour cells were present in all removed eye balls. Distant metastases were not observed in any of these patients. All patients are alive with a follow-up from 10 months to 9 yrs 6 months (median--4 yrs 7 months) from diagnosis. Disease free survival and ocular survival is 44% and 54% respectively. CONCLUSION: Neoadjuvant chemotherapy in unilateral retinoblastoma allows to avoid enucleation in some patients.

[Rare tumours--are they really rare in the Polish children population?]. / Nowotwory rzadkie--czy rzeczywiscie rzadkie w populacji polskich dzieci?

Clear statement that pediatric neoplasms are really rare is not easy. Thus the incidence of rare tumours in children has not been defined so far. The paper efforts to assess the topic of rare tumours of childhood in the Polish population. Following two categories are proposed: tumours typical for adults, but possible in children (neoplasms of epithelial origin--mainly carcinomas, melanomas, carcinoids) and paediatric tumours consisting less than 10% of cases in corresponding clinical groups according to the ICCC classification. Data on 317 patients aged 0-18 years treated in centres associated in the Polish Paediatric Group for Solid Tumours (PPGST) were analysed. Classical adult malignancies were registered in 130 patients: carcinomas in 90 (mean age 12.6 +/- 4.5 years), melanomas in 25 (mean age 9.4 +/- 4.9) and carcinoids in 9 (mean age 14.5 +/- 1.2 years). Non epithelial neoplasms were registered in 187 patients (mean age 10.4 +/- 5.5). That group included rare tumours of soft tissue, CNS, bones and other organs. Treatments of certain groups were specified by separate therapeutic protocols within PPGST. Rare malignancies of adult-type among children under 18 years of age in Poland comprised 1.5% of all pediatric neoplasms. The incidence of adult-type neoplasms increased with age until 14 years. In patients over 15 years of age the number of registered cases decreased. It may suggest a first peak of incidence in early adolescence or an underestimation of number of patients with carcinoma aged over 15 years. In the analyzed group, the mean age of patients with carcinomas and other epithelial and unspecified tumours significantly exceeded the age of children with rare neoplasms of non-epithelial origin (12.1 +/- 4.7 vs 10.4 +/- 5.5 years; p<0.05). A very young age at diagnosis of malignant melanomas (mean 9.4 years) and numerous cases of carcinomas affecting the digestive tract (n=24; 27% of all carcinomas), especially those located in colorectal region (n=10), seem surprising. The preliminary analysis of the collected data on rare neoplasms in Poland encourage to undertake a prospective study, meant to link the epidemiology and characteristics of rare epithelial tumours in childhood with diagnostic and therapeutic suggestions for these types that are not coordinated within Polish Paediatric Group of Solid Tumours.

[Can proton magnetic resonance spectroscopy be of any value as a prognostic factor in medulloblastoma?]. / Czy protonowa spektroskopia magnetyczna moze miec wartosc prognostyczna w medulloblastoma? Doniesienie wstepne.

UNLABELLED: Proton MR spectroscopy (MRS) is a noninvasive chemical analysis of metabolites in brain tissue. Metabolite ratios are useful because age dependant normal values are known and differ significantly from the values obtained in various diseases of brain tissues. AIM: The aim of our study was to evaluate whether MRS correlates with magnetic resonance imaging (MRI), course of disease, pathology of resected tumours after preoperative chemotherapy and can be of prognostic value in patients with medulloblastoma (MB). MATERIALS AND METHODS: Eight patients with histologically proven MB were studied. All patients underwent MRS and MRI at diagnosis and after neoadjuvant chemotherapy consisting of VCR,VP,CTX,CBCA. Preoperative chemotherapy was followed by surgery, radiotherapy and maintenance chemotherapy. Assessment whether MRI correlates with MRS after preoperative chemotherapy was performed. Additionally MRS was correlated with the course of disease and pathology results. RESULTS: Out of 8 patients 5 had partial remission of their tumours on MRI. Two of them had favorable reaction on MRS defined as elevation of NAA and Cr decrease in Cho,Lac/Lip. Both are alive and disease free. In 2 patients no changes, compared with MRS at diagnosis were found. Both died of the disease. One had an indistinct positive change and died. Stabilization on MRI was observed in 1 patient, who also had a good reaction on MRS. He is alive, disease free. Two patients had disease progression on MRI. Both had poor reaction on MRS, one died, one is alive with disease. It was noticed that good response on MRS correlated with chemotherapy induced tumour changes observed in pathology specimens from resected tumours. Small number of patients does not allow to draw conclusions but our results signal that MRS might be of prognostic value in brain tumours.

[Treatment results of patients over 3 years of age with medulloblastoma]. / Wyniki leczenia pacjentów powyzej trzeciego roku zycia z medulloblastoma/PNET leczonych wedlug wlasnego protokolu w Instytucie "Pomnik - Centrum Zdrowia Dziecka".

UNLABELLED: THE AIM of the study was to assess the survival of patients with medulloblastoma (MB)/PNET treated according to own protocol and to compare it with the earlier treatment protocol group. MATERIALS AND METHODS: Analysis of 77 patients was undertaken. For the purpose of this study we divided our patients into 2 groups. First group consisted of 34 patients (21 high risk, 12 standard risk, 1 unknown) treated with surgery and radiotherapy and surgery followed by chemo- and radiotherapy. Among the second group including 43 patients (23 HRMB, 6 SRMB, 11 supratentorial PNET's, 3 unknown) treated according to own protocol since 1997. Surgery was performed in all patients. 4 courses of chemotherapy were administered (VCR, VP, CBDCA, CTX, IF, CDDP) and craniospinal irradiation was implemented. Maintenance chemotherapy consisting of 8 courses of VCR, CDDP, CCNU, was given. Event free survival (EFS) in two groups and in high-risk patients of both groups were compared. RESULTS: In the earlier treated group 4 yrs EFS was 50% versus 78% in recently treated patients. In high-risk patients 4 yrs EFS was 40% (with no plateau on the curve) in the historical group and 76% in currently treated. CONCLUSIONS: Introduction of our protocol resulted in marked improvement of treatment results, especially since most of our patients were in the high-risk group.