High-risk neuroblastoma is challenging to treat, requiring multimodal therapy including differentiation treatment with isotretinoin. Pseudotumour cerebri (PC) has been underreported as a complication of isotretinoin during neuroblastoma maintenance therapy. We identified 22 patients receiving isotretinoin for neuroblastoma at a single tertiary paediatric centre. Overall, 91% of patients developed isotretinoin-related toxicity, with 27% experiencing Grade 3 or higher toxicity. Two patients treated with the liquid formulation were diagnosed with PC, of which one was asymptomatic. The asymptomatic case highlights possible under-recognition of this condition and prospective studies should evaluate the value of early ophthalmic assessment during isotretinoin treatment.
In the EU, paediatric development of anticancer medicines is regulated by the requirement for an agreed paediatric investigation plan (PIP) or waiver for each new medicinal product. This indication-based framework may overlook products whose mechanism of action (MoA) is relevant to paediatric cancers beyond the adult indication. Evolving EU legislation and regulatory practice increasingly support mechanism-driven paediatric decisions. Using HER2-targeting antibody–drug conjugates as examples, we show how integrated, case-by-case evaluation of mechanism, target relevance, and proof-of-concept data can guide scientifically robust paediatric oncology development and regulatory decision-making.
Background Advances in cancer treatment have improved survival among children, adolescents, and young adults (CAYAs). However, CAYA cancer survivors are at increased risk of long-term physical, cognitive, psychological, and social consequences of cancer and its treatment, including challenges with educational attainment and employment. Education and career support programs have the potential to improve social participation among this population. Aim The study aim was to identify gaps and needs in education and career support programs for CAYA cancer survivors. The findings are intended to inform the development and refinement of interventions that enhance social participation and quality of life. Methods This study was conducted as part of the European Network of Youth Cancer Survivors (EU-CAYAS-NET) project. A qualitative design was used, involving two focus groups with CAYA cancer survivors in Austria and the Netherlands. Participants were recruited through clinics, patient organizations, and social media. Discussions were audio-recorded, transcribed, and analyzed using thematic content analysis. A brief questionnaire captured demographic and clinical characteristics. Results Eleven survivors participated (Austria: 7; Netherlands: 4), all with experience in education and career support programs. Analysis of 557 codes revealed three main categories: individual behaviors (18%), sociodemographic and health factors (8%), and environmental conditions (74%). Environmental conditions dominated and included the domains infrastructure, support providers, employers, healthcare professionals, and interpersonal relationships. Participants emphasized the need for accessible systems, tailored support, proactive providers, employer engagement, and better integration between stakeholders such as healthcare professionals and vocational services. Conclusion CAYA cancer survivors experience gaps in education and career support. Beyond program availability, clear structures, effective referral pathways, and specialized, coordinated support from multiple stakeholders are needed. Our findings provide implications to guide interventions that improve social participation and quality of life.
Introduction Advances in childhood cancer treatment have improved survival rates, shifting attention towards treatment-related physical limitations. However, evidence on gait alterations and motor correlates in pediatric oncology remains scarce. This cross-sectional correlation study explores relationships between physical performance and gait in childhood cancer patients. Materials and Methods Sixty-four childhood cancer patients (mean age 9.75 ± 4.01 years, range 4–20) underwent gait analysis and physical performance assessments during and post-treatment. Wilcoxon signed-rank tests compared participants with age- and sex-matched normative values. Spearman’s rank correlations assessed associations between physical performance and gait, adjusted for age, height, and weight. Results Participants’ gait differed significantly from norms, with reduced step length (−11.33%) and walking velocity (−10.59%) and increased base of support (+19.68%; all p < .001). Lower limb strength (−41.60% to −56.65%, p < .001), joint range of motion (−2.56% to −47.03%, p < .05), and BBS and FGA scores were reduced (−1.79 and −2.67 points, p < .001). Several associations between physical performance and gait remained significant after adjustment for age, height, and weight, with the strongest observed for knee flexor strength and step length (ρ = .439), BBS and base of support (ρ = −.448), as well as BBS and FGA with step duration variability (ρ = −.535 and ρ = −.507, all p < .01). Conclusions These findings emphasize the relationship between physical performance and gait in childhood cancer patients. Clinically feasible assessments may help identify motor performance limitations associated with altered gait and guide rehabilitation strategies in pediatric oncology. Trial registration NCT05867186 (registered on: 06.04.2023)
Aim Intravascular extension of renal tumours to the inferior vena cava (IVC)/right atrium (RA) represent a major surgical challenge. We report outcomes of IVC patch reconstruction (IVC-PR) following tumour-thrombus resection (TTR) in children. Methods Retrospective review of all patients after TTR IVC-PR (2007–2023): doppler ultrasound assessed IVC patency. Secondary outcomes: 30-day surgical mortality/morbidity, relapse or death. Results Wilms tumour 19/21(90%) and renal Ewings sarcoma 2/21(10%) prompted TTR IVC-PR in 21 patients, aged 2–16years (8/21(38%) metastatic, 1/21(5%) bilateral). Tumour Thrombus (TT) extended to RA 8/21(38%), hepatic veins 9/21(43%) and infra-hepatic IVC 4/21(19%). After neo-adjuvant chemotherapy, TT regressed in 8/21(38%). Noteworthy venous collaterals were present in 18/21(86%). Pre-operatively, 5/21(24%) received anticoagulation, 4/5(80%) with bleeding complications. TTR IVC-PR - on cardio-pulmonary bypass in 17/21(81%) - occurred alongside tumour nephrectomy in 19/21(90%) and as a secondary procedure after disease progression/relapse in 2/21(10%). A macroscopically complete TTR was achieved for all, en-bloc in 17/21(81%). Stage III was assigned to 19/21(90%), with viable TT in 16/21(76%). There was no 30-day mortality. Complications occurred in 10/21(48%), necessitating return to theatre for 4/21. Post-operative anticoagulation for a median 5(2−14)months ensued without bleeding complications. At median 4.5(1−15)years follow-up, IVC-PR was patent in 15/21(71%): the luminal calibre normal in 11/15(73%) and reduced in 4/11(27%) patients. Although asymptomatic, 6/21(29%) had a non-patent IVC-PR. Relapse occurred in 2/21(10%) patients (1 died), both after secondary TTR. Conclusions TTR at radical nephrectomy appears important for survival. IVC-PR was patent in the majority, without clinically significant adverse effects during childhood for those without IVC-PR patency.
