BRAF is a component of the MAPK and PI3K/AKT/mTOR pathways that play a crucial role in cellular proliferation, differentiation, migration, and angiogenesis. Pediatric central nervous system tumors very often show mutations of the MAPK pathway, as demonstrated by next-generation sequencing (NGS), which now has an increasing role in cancer diagnostics. The MAPK mutated pathway in pediatric CNS tumors is the target of numerous drugs, approved or under investigation in ongoing clinical trials. In this review, we describe the main aspects of MAPK and PI3K/AKT/mTOR signaling pathways, with a focus on the alterations commonly involved in tumorigenesis. Furthermore, we reported the main available data about current BRAF and MEK targeted therapies used in pediatric low-grade gliomas (pLLGs), pediatric high-grade gliomas (pHGGs), and other CNS tumors that often present BRAF or MEK mutations. Further molecular stratification and clinical trial design are required for the treatment of pediatric CNS tumors with BRAF and MEK inhibitors.
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.
1 April 2021 in European Journal of Medical Genetics
Medulloblastoma represents a rare yet complex embryonal tumor of the posterior cranial fossa that, while predominantly affecting pediatric populations, occurs with increasing recognition among adolescents and young adults (AYAs, 15–39 years). The scarcity of medulloblastoma within this demographic creates substantial obstacles in diagnosis, treatment selection, and psychosocial management that differ markedly from established pediatric approaches. Emerging data reveal that AYA patients exhibit distinctive tumor biology, including altered molecular subgroup patterns, variable therapeutic responses, and unique survival trajectories when compared to younger patients. Current investigations examining autologous stem cell transplantation following intensive chemotherapy protocols in metastatic cases demonstrate encouraging preliminary results. Evidence increasingly supports adapting pediatric treatment paradigms for adult application, potentially improving therapeutic outcomes while reducing treatment burden. These cross-disciplinary approaches between pediatric and adult oncology demonstrate considerable promise for enhancing clinical results and minimizing therapy-associated morbidity, emphasizing the critical need for collaborative care models in managing this challenging malignancy across diverse age groups.
Healthcare professionals in pediatric oncology are at a high risk of burnout. Art therapy is being increasingly recognized as a potential tool for reducing stress and improving emotional well-being. The Art-Out pilot project aimed at nursing staff was initiated in a pediatric oncology unit. The staff members participating in the project were guided in a team-building course integrated with art and clay therapy, aiming to reduce burnout levels, improve emotional climate, and strengthen resilience. Methods: Burnout levels were assessed through the Maslach Burnout Inventory (MBI), alexithymia was measured with the Toronto Alexithymia Scale (TAS-20), and emotional regulation difficulties were evaluated through the Difficulties in Emotion Regulation Scale (DERS); these tests were assessed before (T0) and after (T1) the team-building course (Art-Out project). Results: Data analysis showed a significant reduction in burnout, alexithymia, and emotional dysregulation, highlighting the positive impact of this approach in improving team dynamics and emotional management. Conclusions: Our study confirms the high risk of burnout, alexithymia, and emotional dysregulation among pediatric oncology healthcare workers, underscoring the need for targeted interventions to prevent and mitigate these risks.
1 January 2025 in Case Reports in Oncological Medicine
Solid pseudopapillary neoplasms (SPNs) represent a tumor of the exocrine pancreas, belonging to the group of pancreatic cystic neoplasms. It is exceedingly uncommon for SPNs to manifest in extrapancreatic locations, such as the mesocolon, greater omentum, jejunum, and ovary. SPNs are considered very rare, constituting about 0.17%–2.5% of all pancreatic neoplasms and 6% of cystic pancreatic neoplasms. We present two pediatric cases of SPNs: one involving a 16‐year‐old female patient and the other a 14‐year‐old girl, both diagnosed and treated in our center with surgical resection. The experience of our center confirms that wide margin surgery, with associated metastasectomy if necessary, represents the therapy of choice for SPNs, ensuring effective control of the disease.