Search Results (216)

Background/Objectives: Functional abilities are fundamental for social participation. However, functional abilities assessment tools validated for children/and adolescents undergoing cancer treatment are lacking. A preliminary validation of the Gross Motor Function Measure-88 (GMFM-88) scale for children with cancer found that some items are unable to discriminate or distinguish between different gross motor function levels. This study aims to develop and validate the Functional Abilities Assessment in Paediatric Oncology (FAAP-O), starting with GMFM-88. Methods: This multicentre study (ClinicalTrials.gov Identifier: NCT04862130) involved children and adolescents (6 months–18 years old) diagnosed with cancer, in any phase of treatment or less than 1 year of therapy. A multiphase mixed-methods design was employed. The content validity of each item of GMFM-88 for the paediatric oncology population was calculated with the Content Validity Ratio (CVR). Items with a CVR score > 0.70 were included in the FAAP-O scale. Other items with a score between 0 and 0.70 were selected for their relevance by consensus with five focus groups and a plenary meeting. The FAAP-O items set was organised in five dimensions by exploratory factor analyses. The calculation of FAAP-O internal consistency was made using Cronbach’s α while and inter/intra-rater reliability was assessed by intraclass correlation coefficients (ICCs). Results: The study involved 217 subjects. The FAAP-O included 36 items; its internal consistency was good in each dimension (0.90 ≤ α ≤ 0.96) and its inter/intra-rater reliability was excellent (ICC > 0.90). Conclusions: A new specifically validated scale to assess functional abilities in children and adolescents with cancer is provided. Validated tools are necessary for specific, objective rehabilitation assessments, which are fundamental in clinical practice and research. Full article

Didactic digital tools can commence, enhance, and strengthen reading fluency in children undergoing long-term hospitalization due to oncology conditions. However, resources specifically designed to support rapid naming and decoding in Spanish remain scarce. This study presents the design, development, and evaluation of a game prototype aimed at addressing this gap among Spanish-speaking preschoolers in hospital settings. Developed using Unity through a design-based research methodology, the game comprises three narratively linked levels targeting rapid naming, decoding, and fluency. A sequential exploratory mixed-methods design (QUAL-quan) guided the evaluation. Qualitative data were obtained from a focus group of hospital teachers (N = 6) and interviews with experts (N = 30) in relevant fields. Quantitative validation involved 274 experts assessing the game’s contextual, pedagogical, and technical quality. The prototype was also piloted with four end-users using standardised tests for rapid naming, decoding, and fluency in Spanish. Results indicated strong expert consensus regarding the game’s educational value, contextual fit, and usability. Preliminary findings suggest potential for fostering and supplementing early literacy skills in hospitalised children. Further research with larger clinical samples is recommended to validate these outcomes. Full article

Objective: Dinutuximab beta (DB) and naxitamab (NAXI) with GM-CSF are used for maintenance treatment of relapsed/refractory neuroblastoma. The objective of this study was to systematically assess comparative efficacy of the two therapies within their designated indications in accordance with established clinical guidelines. Methods: Relevant evidence was identified in systematic literature review. Individual patient data (IPD) from prospective clinical trials of DB were assessed and data on patients with disease in bone or bone marrow, as assessed in MRI, CT, mIBG or biopsy, with incomplete response to previous therapy were included. Patients with complete response, progressive disease and/or soft tissue disease were excluded. DB population was adjusted for sex, MYCN amplification, disease type (relapsed, refractory), and disease site (bone marrow and/or bone) to balance aggregated characteristics of NAXI population. More characteristics were included in sensitivity analyses, including DB treatment without interleukin-2, as currently recommended. Overall response rate (ORR) was assessed as best response. Results: Aggregated data for NAXI from Study 201 (n = 52) and Study 230 (n = 38) and IPD from DB studies (APN311-202, APN311-304, c = 77) met the inclusion criteria. Compared to NAXI, DB significantly extended progression-free survival (PFS): hazard ratio, DB vs. NAXI of 0.47 (95% CI: 0.26 to 0.87, p = 0.015). ORR was 60.1% (95% CI: 48.5% to 71.6%) for DB vs. 43.3% (33.1% to 53.6%) for NAXI (ORR odds ratio, DB vs. NAXI was 1.97, 95% CI: 1.02 to 3.80, p = 0.044). Sensitivity analyses and unadjusted comparisons supported the results. Conclusion: In the indirect comparison, dinutuximab beta significantly extended PFS and increased ORR compared to naxitamab. Full article

Conventional melanoma is exceedingly rare in the pediatric population, particularly among prepubescent children, and its diagnosis and management necessitate a multidisciplinary approach. The objective of this present report is to delineate the diagnostic pathway and therapeutic management of a 4-year-old girl with conventional melanoma, with particular focus on the molecular context. A pigmented lesion located on the auricle was surgically excised, and subsequent histopathological and immunohistochemical analyses confirmed the diagnosis of malignant melanoma (pT3b). Radiologic investigations revealed no evidence of metastatic disease, and comprehensive genetic testing utilizing next-generation sequencing (NGS) identified no pathogenic variants in the germline genes examined, nor in the BRAF, NRAS, KRAS, and TP53 genes within the excised lesion. The patient remains in good general health. This case report adds to the limited body of literature on melanoma in pediatric patients and underscores the importance of thorough diagnostic evaluation in this age group. Full article

Background: Staphylococcus spp. represent the most prevalent Gram-positive organisms in children with malignancies or undergoing haematopoietic cell transplantation (HCT), contributing to significant morbidity and mortality. This study aimed to assess the epidemiology, risk factors, treatment strategies, and outcomes of staphylococcal infections (SIs) in paediatric haemato-oncology (PHO) and HCT patients in Poland over a 12-year period. Methods: A retrospective, multicentre study was conducted across 17 paediatric oncology centres in Poland. The clinical and microbiological data of patients under the age of 18, diagnosed with malignancies or post-HCT, were analysed for confirmed SI between 2012 and 2023. The variables assessed included demographics, underlying conditions, infection type and source, antimicrobial susceptibility, treatment, and 30-day infection-free survival. Results: Among 1725 patients with SI, 1433 were PHO and 292 were HCT patients. The cumulative incidence of SI was 12.7% in PHO and 14.3% in HCT patients (p = 0.008). The 30-day survival rate was significantly higher in PHO compared to HCT patients (98.4% vs. 93.2%, p < 0.001). Most deaths were caused by S. epidermidis, S. haemolyticus, and S. hominis, predominantly involving methicillin-resistant coagulase-negative Staphylococci (MRCNS). Multivariate Cox regression identified undergoing HCT (HR = 3.0, 95% CI: 1.6–5.6, p < 0.001) and treatment of infection > 10 days (HR = 2.0, 95% CI: 1.1–3.6, p = 0.019) as independent risk factors for mortality. Conclusions: Staphylococcal infections pose a significant challenge in paediatric oncology and transplant populations. Optimising prevention, diagnostics, and antimicrobial therapy is crucial for improving outcomes in these high-risk groups. Full article

Background/Objectives: Childhood cancer, although relatively rare, has a profound impact on the quality of life of affected children and their families. Technological advances have facilitated the development of mobile applications (apps) aimed at enhancing symptom monitoring and improving communication with healthcare teams. This systematic review aimed to analyse the effect of mobile applications on the health of children with cancer, with a specific focus on health-related quality of life (HRQoL). Methods: A systematic review was conducted in accordance with PRISMA 2020 guidelines. Searches were performed in PubMed (Medline), CINAHL, Cochrane and Scopus databases using MeSH terms such as Smartphone, Mobile Applications, Child Health, Neoplasms, and Digital Health, with no date restrictions, and including studies published in English, Spanish or Portuguese. We included original research studies that examined the use of mobile apps in paediatric oncology patients. The search was completed in January 2025. Results: Of the 324 records initially identified, 14 studies (mainly pilot studies, early-phase clinical trials, and observational designs) met the inclusion criteria. Interventions commonly focused on symptom tracking (pain, nausea, fatigue), promoting treatment adherence, and delivering educational content. Several studies reported high user acceptance and a potential positive impact on HRQoL, particularly when gamification strategies were incorporated to sustain children’s engagement. Conclusions: Despite the preliminary nature and small sample sizes of most studies, mobile applications appear to be effective in supporting symptom management, communication, and health education in paediatric oncology. Their use may contribute to improvements in HRQoL. Further high-quality research involving younger children and diverse socio-cultural contexts is required to confirm their effectiveness. Full article

Objectives: A simple method for quantifying fluconazole in small blood volumes has been developed using volumetric absorptive microsampling (VAMS^®) technology and isocratic high-performance liquid chromatography (HPLC) with ultraviolet (UV) detection. Methods: For sample collection, Mitra^® devices are used to keep the sample volume at 10 µL. For the quantitative determination of fluconazole, the Mitra^® samples are extracted using acetonitrile as the extraction agent, containing 2-(4-chlorophenyl)-1,3-bis(1,2,4-triazol-1-yl)propan-2-ol as the internal standard. A Synergi 4 μm Polar-RP 80 Å (150 × 2 mm) column forms the stationary phase, and a mixture of acetonitrile and phosphate buffer is the mobile phase. The UV detection is set at a wavelength of 210 nm. The therapeutic concentration range of 5 to 160 mg/L is covered, and the linear equation with 1/x² weighting is used to determine unknown samples. This method has been validated according to the current EMA and FDA guidelines for bioanalytical methods. Results: The validation data obtained after analysing whole blood samples (EDTA) showed within- and between-run accuracy between 94.4% and 115% and precision between 0.4% and 9.4%, respectively. A lower limit of quantification (LLOQ) of 5 mg/L was sufficient for therapeutic drug monitoring in paediatric patients receiving fluconazole as antifungal prophylaxis after haematopoietic cell transplantation. Conclusions: So far, 211 samples from 49 patients were successfully analysed, and concentrations between 5.84 mg/L and 107 mg/L were determined for whole blood Mitra^® samples. To our knowledge, this is the first application of VAMS^® technology using simple and cheap HPLC-UV quantification. Full article

Approximately 5% to 8% of patients with Wilms tumour have bilateral disease. The prevalence of bilateral Wilms tumour (BWT) is higher in individuals with genetic predisposition syndromes than in those without. The goal of therapy is to preserve as much renal tissue as possible without compromising the overall oncological outcomes, utilising neoadjuvant chemotherapy followed by nephron sparing surgery (NSS) if possible. The Children’s Oncology Group (COG) in North America and the International Society of Paediatric Oncology (SIOP) in Europe have developed the main protocols for the treatment of BWT. Both protocols are similar: initial biopsies are not indicated, and they both recommend surgical resection at week 6 or no later than week 12. Chemotherapy includes the use of vincristine and actinomycin-D in both protocols, but the COG approach also includes the use of doxorubicin, which is a cardiotoxic drug with important long-term effects on the cardiac function of childhood cancer survivors. What doxorubicin adds to patients with BWT in terms of radiological tumour response, resectability, long-term renal function and overall survival, is still not very well described and it may be variable depending on the tumour biology. This article describes the current approach for BWT in North America and Europe, focusing on the potential effect that doxorubicin may have on patient outcomes. Full article

Background/Objectives: Targeted and non-targeted fluorescent molecular probes (FMPs) can be used intra-operatively to visualise tumour tissue. Multiple probes have been clinically approved for fluorescence-guided surgery (FGS) in adult oncology, and the translation of these technologies to paediatric neuroblastoma may provide novel strategies for optimising tumour resection whilst minimising morbidity. We aimed to identify clinically approved FMPs with potential utility for FGS in neuroblastoma. Methods: A systematic review of the literature was performed in accordance with the PRISMA guidelines (PROSPERO CRD42024541623). PubMed and Web of Science databases were searched to identify studies investigating clinically approved FGS probes and/or their targets in the context of neuroblastoma. Pre-clinical and clinical studies looking at human neuroblastoma were included. The primary outcomes were that the FGS probe was tested in patients with neuroblastoma, the probe selectively accumulated in neuroblastoma tissue, or that the target of the probe was selectively over-expressed in neuroblastoma tissue. Results: Forty-two studies were included. Four were clinical studies, and the remainder were pre-clinical studies using human neuroblastoma cell lines, human tumour tissue, or xenograft models using human neuroblastoma cells. The only FMP clinically evaluated in neuroblastoma is indocyanine green (ICG). FMP targets that have been investigated in neuroblastoma include poly-ADP ribose polymerase (PARP) (targeted by PARPiFL), endothelial growth factor receptor (EGFR) (targeted by Panitumumab-IRDye800CW, Cetuximab-IRDye800CW, Nimotuzumab-IRDye800CW and QRHKPRE-Cy5), vascular endothelial growth factor receptor (VEGFR) (targeted by Bevacizumab IRDye800CW), and proteases such as cathepsins and matrix metalloproteinases that activate the fluorescent signal of FMPs, such as LUM015 and AVB-620. Of the clinical studies included, all were found to have a high risk of bias. Conclusions: ICG is the only clinically approved fluorescent dye currently used for FGS in neuroblastoma; however, studies suggest that its ability to recognise neuroblastoma tissue is inconsistent. There are several clinically approved FMPs, or FMPs in clinical trials, that are used in adult oncology surgery that have targets expressed in neuroblastoma. Further research should validate these probes in neuroblastoma to enable their rapid translation into clinical practice. Full article

Background: Dried ivy leaf extract EA 575^® (Prospan^®) is commonly used to treat coughs and may help reduce inappropriate antibiotic use for the common cold. This retrospective study investigated whether prescribing EA 575 is associated with reduced subsequent antibiotic use in children and adolescents with the common cold. Repeated EA 575 prescriptions were also analyzed to estimate treatment satisfaction. Methods: Data were sourced from the IQVIA Disease Analyzer database, including patients under 18 diagnosed with a common cold and prescribed either EA 575 or antibiotics between 2017 and 2020 (index date). Propensity score matching controlled for confounding factors. Antibiotic prescriptions were assessed 4–30 and 31–365 days after the index date, along with bacterial infections 4–40 days post-index. Repeated EA 575 prescriptions 2–5 years post-index were analyzed as a proxy for treatment satisfaction. Results: Overall, 10,390 children and adolescents were included in each matched cohort. Compared to antibiotics, EA 575 prescriptions were associated with significantly lower odds of antibiotic use 4–30 days (OR: 0.56; 95% CI: 0.49–0.64; p < 0.001) and 31–365 days (OR: 0.58; 95% CI: 0.54–0.62; p < 0.001) after the index date. The odds of bacterial infection 4–30 days after EA 575 prescription were also lower (OR: 0.67; 95% CI: 0.45–0.99; p = 0.047). Of the 42,677 patients in the EA 575 analysis, 50.5% had at least one repeated prescription, with the highest rates among children aged 0–2 years (54.7%) and the lowest in those aged 13–17 years (19.9%). Conclusions: EA 575 prescription was associated with reduced subsequent antibiotic use in children and adolescents with common colds. Frequent repeated prescription rates emphasize the therapeutic value of EA 575 as a treatment option for cold symptoms, especially in younger children. Full article

Background: Childhood, adolescent, and young adult cancer survivors (CAYACS) face significant long-term health risks, yet adherence to long-term follow-up (LTFU) care remains inconsistent. This study explores the concept of akrasia (i.e., acting against one’s better judgment by engaging in behaviors known to be harmful or counterproductive) to understand the psychological, cognitive, and systemic barriers influencing survivor engagement in LTFU. Method: Using an ethical reflection approach based on a literature review, we discussed survivor experiences, behavioral science insights, and ethical principles to identify solutions that balance patient autonomy with supportive interventions. A narrative approach was used to summarize the key points discussed during the ethics reflection group meetings. Results: Our findings highlight key barriers such as trauma, avoidance behaviors, and cognitive constraints that contribute to non-adherence. Strategies such as shared decision-making, digital health tools, and nudge-based interventions are proposed to enhance survivor engagement. Ethical considerations emphasize the need for personalized and flexible care approaches that respect survivor agency while mitigating obstacles to adherence. Conclusions: Addressing akrasia through ethical and behavioral frameworks could improve LTFU adherence, ultimately enhancing survivorship care and long-term health outcomes. Full article

Background/Objectives: Chronic non-bacterial osteomyelitis (CNO) is a rare autoinflammatory disease characterized by chronic sterile uni- or multifocal osteomyelitis. The treatment of CNO is mostly empirical and the outcome of the disease has not yet been standardized. The aims of this study were to correlate clinically active lesions with radiological signs of inflammation and to evaluate the outcomes in terms of symptoms and radiological signs with Whole Body Magnetic Resonance Imaging (WB-MRI) based on the treatment line used. Methods: A retrospective, observational cohort study of 20 CNO patients, recruited from a single tertiary center in southern Italy, was conducted. Patients included in the study were treated based on the “step-up” approach and were guided by the “treat-to-target” strategy as well as by the response to therapy. The outcome measure was stratified into four different groups, defined by a “Delphy consensus”, depending on the symptoms and the presence of bone lesions in WB-MRI, compared with the therapy carried out. Results: Pain was the most common presenting symptom of the disease. Only 15% of our patients reported long-term complications. WB-MRI was performed for each patient both at diagnosis and during follow-up. At onset, the site most affected by the disease was the tibia. All patients who reached a 5-year follow-up (30%, n = 6) achieved a complete disease remission. Conclusions: The standardized “step-up” treatment approach in our cohort proved effective in disease management with disease control or remission in nearly 90% of patients at one year from diagnosis. Full article

Background/Objectives: In bilateral nephroblastoma and high-risk neuroblastoma in children with extensive tumor involvement of the renal vessels or pedicle, complete tumor resection with preservation of healthy renal tissue is not feasible with in situ nephron-sparing surgery or vascular replacement. The aim of this study was to present our experience with ante situ tumor resection and renal autotransplantation (RATX) in these children. Methods: A retrospective study of children with bilateral nephroblastoma and high-risk neuroblastoma who underwent tumor resection and RATX at an international referral center for pediatric surgical oncology between 2006 and 2024 was performed. RATX was performed by transection of renal vessels, ante situ mobilization, and perfusion of the kidney with Bretschneider’s solution. Tumor resection was performed on a bloodless kidney under hypothermia. Results: Ante situ tumor resection and RATX were performed at a median age of 36 months (range 13–62) in 4 children with bilateral nephroblastoma and 4 children with high-risk neuroblastoma. Complete tumor resection was achieved in all patients. One patient with neuroblastoma died of sepsis after 14 days. The 7 surviving patients showed no evidence of disease and normal to slightly decreased glomerulofiltration rates at a median follow-up of 20 months (range 3–155). Limitations include the retrospective design, small sample size, and heterogeneity of the study population due to very rare indication. Conclusions: Ante situ tumor resection and RATX is a feasible surgical approach for children with multifocal bilateral nephroblastoma or high-risk neuroblastoma who are ineligible for in situ nephron-sparing surgery or vascular reconstruction. Full article

Background: Advances in diagnosis and treatment have significantly increased survival rates for childhood cancer, leading to a growing population of long-term survivors. However, these survivors face substantial physical and psychological sequelae that affect both the child and their family. We developed the RECOVER model of care to support childhood cancer survivors as they transition from the end of their planned treatment to survivorship, addressing the broader health and wellness needs beyond medical surveillance. The primary objectives are to assess the feasibility and acceptability of the RECOVER model of care in routine paediatric oncology practice. Secondary objectives include evaluating preliminary efficacy outcomes and identifying factors that influence the successful adoption and integration of the model. Methods: The study comprises a Type 2 Hybrid Implementation/Effectiveness non-randomised controlled trial to compare historical and prospective data. Quantitative data will assess feasibility, reach, effectiveness, adoption, maintenance, and implementation. The qualitative component will assess end-user acceptability and appropriateness through focus groups, surveys, and interviews. Quantitative and qualitative results will be integrated during the interpretation phase to provide complementary insights into the interconnected contextual factors that facilitate the model uptake. Discussion: The RECOVER model of care aims to offer a robust approach to survivorship care, facilitating the continuous monitoring and management of long-term and late effects in childhood cancer survivors. This model has the potential to significantly improve the quality of life and health outcomes for this vulnerable population by addressing their comprehensive needs in a timely and systematic manner. Full article