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Platinum-induced ototoxicity constitutes a significant adverse effect in pediatric oncology, frequently resulting in permanent hearing impairment with profound implications for quality of life, language acquisition, and scholastic performance. This comprehensive review critically evaluates contemporary ototoxicity monitoring practices across various pediatric oncology settings, analyzes current guideline recommendations, and formulates strategies for implementing standardized surveillance protocols. Through examination of recent literature—encompassing retrospective cohort investigations, international consensus recommendations, and functional outcome assessments—we present an integrated analysis of challenges and opportunities in managing chemotherapy-associated hearing loss among childhood cancer survivors. Our findings demonstrate marked heterogeneity in monitoring methodologies, substantial implementation obstacles, and considerable impact on survivors’ functional status across multiple domains. Particularly concerning is the persistent absence of an evidence-based consensus regarding the appropriate duration of audiological surveillance for this vulnerable population. We propose a structured framework for comprehensive ototoxicity management emphasizing prompt detection, standardized assessment techniques, and integrated long-term follow-up care to minimize the developmental consequences of platinum-induced hearing impairment. This approach addresses critical gaps in current practice while acknowledging resource limitations across diverse healthcare environments.

Background/Objectives: Diencephalic syndrome (DS) is an uncommon pediatric disorder presenting with severe failure to thrive despite adequate caloric intake and preserved linear growth. First characterized by Russell, this condition predominantly affects infants under 12 months and remains diagnostically challenging. Methods: We performed a comprehensive literature review examining clinical presentation, underlying pathophysiology, associated pathology, diagnostic approaches, and long-term outcomes of DS. Results: DS typically manifests in the first year of life with profound cachexia, normal or increased appetite, preserved height velocity, and characteristic features including hyperactivity, euphorism, and visual pathway involvement. Low-grade gliomas of the hypothalamic–chiasmatic region, particularly pilocytic astrocytomas, represent the predominant underlying pathology. The pathophysiological mechanisms remain incompletely understood but likely involve complex dysregulation of hypothalamic energy homeostasis. While overall survival exceeds 90% at five years, most patients experience significant long-term morbidity including visual impairment, multiple endocrine deficiencies, and hypothalamic obesity. Diagnostic delays averaging 11 months contribute to irreversible complications. Conclusions: Early recognition of DS is essential to prevent permanent visual loss and optimize outcomes. Multidisciplinary management incorporating chemotherapy as first-line treatment for underlying gliomas has improved survival while reducing radiation-associated toxicities. However, survivors face substantial lifelong sequelae requiring comprehensive monitoring and intervention. Future research should focus on elucidating precise pathophysiological mechanisms, developing targeted molecular therapies, and improving management of hypothalamic obesity and other late effects.

Vincristine-induced peripheral neurotoxicity (VIPN) is a very common side effect of vincristine chemotherapy among pediatric patients with cancer. Neuropathy may be sensory, motor and/or autonomic, with consequent reduction, delay or discontinuation of vincristine-chemotherapy, but also pain, disability, reduced quality of life of patients and an increase in medical costs. Vincristine acts out its antineoplastic function by altering the normal assembly and disassembly of microtubules, with their consequent mitosis block and death. Vincristine leads to VIPN through a complex mechanism of damage, which occurs not only on the microtubules, but also on the endothelium and the mitochondria of nerve cells. Furthermore, both patient-related risk factors (age, race, ethnicity and genetic polymorphisms) and treatment-related risk factors (dose, time of infusion and drug–drug interactions) are involved in the pathogenesis of VIPN. There is a lack of consensus about the prophylaxis and treatment of VIPN among pediatric oncologic patients, despite several molecules (such as gabapentin, pyridoxine and pyridostigmine, glutamic acid and glutamine) having been already investigated in clinical trials. This review describes the molecular mechanisms of VIPN and analyzes the risk factors and the principal drugs adopted for the prophylaxis and treatment of VIPN in pediatric patients with cancer.

Aminoglycosides are broad-spectrum antibiotics largely used in children, but they have potential toxic side effects, including ototoxicity. Ototoxicity from aminoglycosides is permanent and is a consequence of its action on the inner ear cells via multiple mechanisms. Both uncontrollable risk factors and controllable risk factors are involved in the pathogenesis of aminoglycoside-related ototoxicity and, because of the irreversibility of ototoxicity, an important undertaking for preventing ototoxicity includes antibiotic stewardship to limit the use of aminoglycosides. Aminoglycosides are fundamental in the treatment of numerous infectious conditions at neonatal and pediatric age. In childhood, normal auditory function ensures adequate neurocognitive and social development. Hearing damage from aminoglycosides can therefore strongly affect the normal growth of the child. This review describes the molecular mechanisms of aminoglycoside-related ototoxicity and analyzes the risk factors and the potential otoprotective strategies in pediatric patients.

The central nervous system (CNS) may be considered as a sanctuary site, protected from systemic chemotherapy by the meninges, the cerebrospinal fluid (CSF) and the blood-brain barrier (BBB). Consequently, parenchymal and CSF exposure of most antineoplastic agents following intravenous (IV) administration is lower than systemic exposure. In this review, we describe the different strategies developed to improve delivery of antineoplastic agents into the brain in primary and metastatic CNS tumors. We observed that several methods, such as BBB disruption (BBBD), intra-arterial (IA) and intracavitary chemotherapy, are not routinely used because of their invasiveness and potentially serious adverse effects. Conversely, intrathecal (IT) chemotherapy has been safely and widely practiced in the treatment of pediatric primary and metastatic tumors, replacing the neurotoxic cranial irradiation for the treatment of childhood lymphoma and acute lymphoblastic leukemia (ALL). IT chemotherapy may be achieved through lumbar puncture (LP) or across the Ommaya intraventricular reservoir, which are both described in this review. Additionally, we overviewed pharmacokinetics and toxic aspects of the main IT antineoplastic drugs employed for primary or metastatic childhood CNS tumors (such as methotrexate, cytosine arabinoside, hydrocortisone), with a concise focus on new and less used IT antineoplastic agents.

Background During the last decade, there has been a growing number of cases of children born from pregnancy-associated cancer (PAC), however there are currently insufficient data on the follow up to be observed in this category of newborns. Objective of the study was to evaluate the neonatal outcomes of infants born to mother with PAC, the potential adverse effect of chemotherapy during pregnancy and the risk of metastasis to the fetus. Methods Maternal clinical data and neonatal outcomes of child born to mothers diagnosed with PAC were collected; infants were divided into those were and were not exposed to chemotherapy during fetal life and their outcomes were compered. Results A total of 37 newborn infants from 36 women with PAC were analyzed. Preterm delivery occurred in 83.8% of the cases. No significant differences in neonatal outcomes were found between infants who were and were not exposed to chemotherapy during pregnancy. The median follow-up period was 12 months. Conclusions PAC treatment during the second or third trimester does not seem to be dangerous for the fetus, however infants born from PAC must be carefully evaluated for to rule out the consequences of chemotherapy and exclude the presence of metastasis. Long-term follow-up, especially in children exposed to chemotherapy, should be encouraged to obtain relevant data on long-term toxicity.

The human oral cavity harbors the second most abundant microbiota after the gastrointestinal tract, with over 700 species currently identified in the oral microflora. The oral microbiota develops from intrauterine life and after birth is continuously shaped by several influencing factors. The perturbation of the diversity and proportions of species within the oral microbiota leads to dysbiosis and associated increased risk of local and systemic diseases. In children who receive chemotherapy for cancer, oral mucositis is a common and painful side effect that decreases quality of life (QoL) and treatment adherence. The oral microbiota undergoes a substantial dysbiosis as an effect of cancer and its treatment, characterized by lower richness and less diversity. Furthermore, this dysbiosis seems to promote pro-inflammatory cytokine release and pro-apoptotic mediators, enhancing the oral tissue damage. Further studies on the role of the oral microbiota in the pathogenesis of oral mucositis should be performed among children with cancer who receive chemotherapy, to find preventive and protective factors against the pathogenesis of oral mucositis.

Brain cancer is the second most common childhood malignancy and is the leading cause of death among all pediatric cancers [...].Brain cancer is the second most common childhood malignancy and is the leading cause of death among all pediatric cancers [...].

Purpose Prenatal ultrasound screening has increased detection of adrenal masses during the fetal and neonatal periods, creating management dilemmas between immediate surgical intervention and observation. This study evaluates the outcomes of selective conservative management in perinatal adrenal masses. Methods We conducted a single-institution retrospective case series of nine patients with perinatal adrenal masses diagnosed at our institution between 2015 and 2025. Clinical presentation, imaging characteristics, biochemical markers, treatment decisions, and outcomes were analyzed. The cohort included five males and four females, with masses detected prenatally in five cases. All patients were initially managed conservatively with serial imaging surveillance to assess lesion volume over time. Results Neuroblastoma was confirmed in four patients; other diagnoses included four benign lesions and one adrenocortical carcinoma. Given the small sample size of this case series, the following proportions should be interpreted with caution and are not intended to reflect population-level outcomes. Spontaneous regression occurred in six cases, two remained stable, and one increased in size. Four patients underwent surgical intervention based on growth patterns or radiological suspicion of malignancy. Median follow-up was 42 months (range: 6 months to 10 years), with no mortality recorded. Conclusion Among the nine case, we observed spontaneous regression in six of nine patients. Temporal evolution of lesion volume proved more informative for clinical decision-making than initial imaging characteristics. The adrenocortical carcinoma case illustrates that radiological trend alone does not always determine management, and that clinical context—including genetic predisposition—must guide decisions. Although our small cohort precludes generalizable conclusions, this institutional experience contributes to the growing evidence base supporting observational approaches in appropriately selected cases. Larger prospective studies incorporating molecular and genetic characterization remain essential to identify reliable predictors of lesion behavior and to optimize patient selection for conservative versus surgical management.

Background Falls are a common adverse event in both elderly inpatients and patients admitted to rehabilitation units. The Hendrich Fall Risk Model II (HIIFRM) has been already tested in all hospital wards with high fall rates, with the exception of the rehabilitation setting. This study’s aim is to address the feasibility and predictive performances of HIIFRM in a hospital rehabilitation department. Methods A 6 months prospective study in a Italian rehabilitation department with patients from orthopaedic, pulmonary, and neurological rehabilitation wards. All admitted patients were enrolled and assessed within 24 h of admission by means of the HIIFRM. The occurrence of falls was checked and recorded daily. HIIFRM feasibility was assessed as the percentage of successful administrations at admission. HIIFRM predictive performance was determined in terms of area under the Receiver Operating Characteristic (ROC) curve (AUC), best cutoff, sensitivity, specificity, positive and negative predictive values, along with their asymptotic 95% confidence intervals (95% CI). Results One hundred ninety-one patents were admitted. HIIFRM was feasible in 147 cases (77%), 11 of which suffered a fall (7.5%). Failures in administration were mainly due to bedridden patients (e.g. minimally conscious state, vegetative state). AUC was 0.779(0.685–0.873). The original HIIFRM cutoff of 5 led to a sensitivity of 100% with a mere specificity of 49%(40–57%), thus suggesting using higher cutoffs. Moreover, the median score for non-fallers at rehabilitation units was higher than that reported in literature for geriatric non fallers. The best trade-off between sensitivity and specificity was obtained by using a cutoff of 8. This lead to sensitivity = 73%(46-99%), specificity = 72%(65-80%), positive predictive value = 17% and negative predictive value = 97%. These results support the use of the HIIFRM as a predictive tool. Conclusions The HIIFRM showed satisfactory feasibility and predictive performances in rehabilitation wards. Based on both available literature and these results, the prediction of falls among all hospital wards, with high risk of falling, could be achieved by means of a unique tool and two different cutoffs: a standard cutoff of 5 in geriatric wards and an adjusted higher cutoff in rehabilitation units, with predictive performances similar to those of the best-preforming pathology specific tools for fall-risk assessment.