Central nervous system tumours in neonates: what should the neonatologist know?

Central nervous system (CNS) tumours in neonates are relatively rare and present differently when compared with those occurring later in childhood in terms of aetiology, clinical features, location, histology and prognosis. The clinical presentation is extremely variable. Even if the most frequent clinical sign is a macrocephaly, there are many other non-specific symptoms associated. The prognosis is usually poor with overall survival of less than 30%. Surgery continues to be the primary treatment for neonatal CNS tumours, aiming for a gross total resection, directly correlated with prognosis and the overall outcome. The chemotherapy is the only adjuvant therapy whereas the radiotherapy is avoided under three years of age because of the severe sequelae. Hence the importance of molecular characterization of these neoplasms in order to improve the accuracy of the diagnosis and identify new therapeutic targets. The aim of this review is to describe the main characteristics of these tumours and the recent advances in their treatment in order to recognize these pathologies in the prenatal period and create a multidisciplinary team providing the best possible treatment while minimising the risk of long-term complications. Neonatologists play a key role in the early detection, diagnostic evaluation, management and supportive care of these neonates.  Conclusion: The aim of this review is to describe the main characteristics of these tumours and the recent advances in their treatment in order to ensure the essential knowledge that will help the neonatologist identify them and create a multidisciplinary team providing the best possible treatment while minimising the risk of long-term complications. What is Known: • Neonatal CNS tumours are relatively rare and their early identification is important to identify the best diagnostic-therapeutic management. • Surgery is the main treatment of neonatal CNS tumours. The extent of surgical resection directly correlates with prognosis and outcome. What is New: • Predisposing conditions such as Cancer Predisposition Syndromes must be considered. • Targeted drugs and other therapeutic strategies can be identified through molecular characterization.

Unraveling the impact of upfront chemotherapy and proton beam therapy on treatment outcome and follow-up in central nervous system germ cell tumors: a single center experience.

Background: Germ cell tumors (GCT) account for a minority of central nervous system (CNS) malignancies, highly prevalent in adolescents and young adults. Despite their aggressive biological behavior, prognosis is excellent in most cases with risk stratified treatment, consisting in a combination of chemotherapy and radiotherapy. Whole ventricular irradiation (WVI) and craniospinal irradiation, the treatment of choice for localized and metastatic disease, pose significant risk of collateral effects, therefore proton beam radiation (PBT) has been recently proposed for its steep dose fallout. Materials and methods: We report our experience in a consecutive series of 17 patients treated for CNS GCT at our Institution from 2015 to 2021. Results: Most frequent lesion location were sellar/suprasellar (35%) and bifocal germinoma (35%), followed by pineal (18%) and thalamic (12%). Two patients (12%), had evidence of disseminated disease at the time of diagnosis. At the latest follow-up all but one patient showed complete response to treatment. The only relapse was successfully rescued by additional chemotherapy and PBT. PBT was well tolerated in all cases. No visual, neurological or endocrinological worsening was documented during and after treatment. Neuropsychological evaluation demonstrated preservation of cognitive performance after PBT treatment. Conclusions: Our data, albeit preliminary, strongly support the favourable therapeutic profile of PBT for the treatment of CNS germ cell tumors.

Impact of compounded drugs on the caregivers' burden of home therapy management in pediatric palliative care: A descriptive study.

BACKGROUND: Children with medical complexity need complex assistance, that considerably affects caregivers' quality of life. They often need multiple medications, with a consequent relevant risk of errors or poor compliance. Galenic (or compounded) drugs are blended in the pharmacy's laboratory worldwide according to different rules and tailoring the patient's needs. While their use may sometimes simplify these therapies, little is known about parents' attitude about this issue. AIM: This study aimed at investigating the complexity of the daily therapy management and exploring the parents' opinions about galenic compounds. DESIGN: Parents were interviewed by using a structured questionnaire. SETTING: Children followed by the Pediatric Palliative Care Network in Friuli Venezia Giulia, Italy, were included from November 2021 to April 2022. Those diagnosed with malignancies were excluded, since therapies are mainly administered through a central venous catheter. RESULTS: Thirty-four parents were interviewed. Fourteen patients took drugs orally, one via nasogastric tube (NGT), 18 via gastrostomy, and one orally + NGT. The mean number of drugs taken every day was six (2-14), in mean 10 (3-18) administrations, that overall required a mean of 44 (8-180) minutes to be delivered. Twenty-eight parents used galenic compounds, and 24 reported relevant advantages, because of a ready-to-use and safe formulation. CONCLUSIONS: The therapy management of children with medical complexity relies on parents. Galenic compounds may improve both patients' and caregivers' quality of life, either in terms of shorter time of administration or smaller risk of errors. Therefore, their use should be encouraged worldwide, according to the different reference rules.