Clinical features, therapeutic approaches and prognosis of this rare tumour are discussed with regard to the known 30 cases in the literature.

The survival rates of patients who complied with all the inclusion criteria for INES trials are higher compared to those that included also not registered patients. Five-year EFS and OS for INES 99.2 were 87.8 and 95.7 %, while our stage 4s population obtained 78 and 87 %. Concerning 99.3, 5-year EFS and OS were 86.7 and 95.6 %, while for stage 4 we registered 61 and 68 %. MYCN amplification had a strong impact on prognosis and therefore we consider it unacceptable that many patients were not studied for MYCN and probably inadequately treated. Ten-year survival rates were shown to decrease: EFS from 73 to 70 % and OS from 81 to 74 %, indicating a risk of late events, particularly in stage 4s. Population-based registries like European ENCCA WP 11-task 11 will possibly clarify these data.

Childhood intrinsic brain-stem gliomas have a dismal prognosis. Different treatment strategies have been adopted over the years without changing the final outcome of this ominous disease. Due to this grim prognosis, experimental therapeutic designs are worthwhile. Vinorelbine is a semi-synthetic vinca alkaloid that has demonstrated a broad spectrum of activity both in in vitro and in vivo experimental systems. By adopting vinorelbine during and after focal radiotherapy in the last two years, we have tried to evocate its known synergistic effect in brain-stem tumour control. Vinorelbine was administered intravenously before, during and after radiotherapy on tumour bed for a total duration of 10 months. All the consecutive patients whose clinical and radiological features corresponded to the diagnosis of an intrinsic brain-stem tumour, i.e., diffuse pontine glioma, have been accrued to this treatment protocol since July 2002. A histological assessment was not required. All patients were treated during hospital stay or in the outpatient clinic at the Istituto Nazionale Tumori of Milan (n=12) and at the Pediatric Clinic of Policlinico in Catania (n=1). Two of the thirteen patients so far treated have developed multiple subsequent, and transitory, episodes of monolateral peripheral facial nerve palsy during vinorelbine administration. The palsy always completely and spontaneously resolved at a short interval-around 30 min-after the end of the drug infusion. Obvious tumour progression was excluded by means of MRI; therefore the drug was administered as scheduled until the end of the treatment. We describe possible neurological and oncological implications of this unusual side effect, until now not reported in any other series dealing with vinorelbine as adjuvant treatment.

10% of all pediatric brain tumors arise in the brainstem. Amongst these gliomas are the most common while other entities are rare and infrequently described in the literature. In this study we investigated the prevalence of non-gliomatous tumors in the brainstem. Amongst the 1323 embryonal tumours received at the RBTC, we identified 17 cases of ETMRs (17/165) that presented as brainstem primaries. Previously grouped within CNS-PNETs, ETMR, is a new WHO diagnostic entity, characterized by C19MC alterations. ETMR is a disease of infancy, the clinical spectrum of which is poorly understood. ETMRs arise at multiple CNS locations including cerebrum being most common (60%,) followed by cerebellum (18%) and midline structures (6%); notably 10% were brainstem primaries, mimicking DIPG radiologically. All patients presented with a short history of progressive neurological symptoms, with most common signs and symptoms of cranial neuropathies, long tract signs and gait disturbance. Median age at diagnosis was 27 months (range 16-75months) with a male to female ratio of 0.9:1. Predominantly localized (M0-94%, M2-3 -6%) majority of patients underwent upfront biopsy or partial resection (15/17:88%), while complete tumor resection was achieved in 2 cases. All patients received heterogenous combination of chemotherapy with and without radiotherapy. Majority of patients progressed rapidly with median time to progression of 4 months and overall survival of < 13 months. The only long-term surviving patient had complete resection dose intensified chemotherapy and radiation (OS 202months). Primary ETMRs in the brainstem are under recognised entities and carry a dismal prognosis. Although rapidly progressive, prompt recognition, maximal resection and management with multimodal adjuvant therapy should be considered in cases with brainstem disease.