PURPOSE:This study aimed to evaluate the cardiovascular medical management of young adults who had viewed the massive open online course (MOOC) "Childhood Cancer, Living Well, After" four years earlier during the feasibility study (START-MOOC1), compared with those who had not. METHODS:A retrospective, descriptive, French multicenter study was conducted involving 322 childhood cancer survivors (CCS) (range, 20-36 years) eligible to view the cardiovascular health module of the MOOC. Participants were divided into two groups those who had viewed the MOOC (MOOC+) four years earlier and those who had not (MOOC-). We collected data on medical follow-up, cardiovascular care, and lifestyle habits using a short questionnaire. We analyzed it using descriptive statistics and comparative tests. RESULTS:Of the 322 eligible CCS, 95 completed the questionnaire (34 MOOC+ and 61 MOOC-). The participation rate was significantly higher in the MOOC+ group (55.7% vs. 23.4%, p < 0.0001). We found no significant difference between the groups regarding general medical care and cardiologist follow-up. However, 20% of MOOC+ participants began cardiological follow-up after the MOOC, 15% increased their physical activity, and 6% improved their diet. In addition, 25% of MOOC+ participants began complementary therapies. CONCLUSION:The MOOC "Childhood Cancer, Living Well, After" did not result in any significant difference in cardiovascular follow-up behaviors compared with patients who did not view the MOOC. However, the MOOC+ group promoted positive lifestyle changes. Further research with a larger cohort is necessary to assess the full impact of the MOOC on the long-term medical follow-up.

Background Medulloblastoma (MB) is one of the most prevalent embryonal malignant brain tumors. Current classification organizes these tumors into 4 molecular subgroups (WNT, SHH, Group 3, and Group 4 MB). Recently, a comprehensive classification has been established, identifying numerous subtypes, some of which exhibit a poor prognosis. It is critical to establish effective subtyping methods for accurate diagnosis and patient's management that strikes a delicate balance between improving outcomes and minimizing the risk of comorbidities.Methods We evaluated the ability of Nanopore sequencing to provide clinically relevant methylation and copy number profiles of MB. Nanopore sequencing was applied to an EPIC cohort of 44 frozen MB, benchmarked against the gold standard EPIC array, and further evaluated on an integrated diagnosis cohort of 116 MB.Results Most MB of both cohorts (42/44; 95.5% and 106/116; 91.4%, respectively) were accurately subgrouped by Nanopore sequencing. Employing Flongle flow cells for 18 MB allowed a more rapid and cost-effective analysis, with 94.4% (17/18) being correctly classified. Nanopore sequencing enabled us to accurately subtype 28/30 (93.3%) MB.Conclusion This study, conducted on the largest cohort of MB analyzed with Nanopore sequencing to date, establishes the proof of concept that this modern and innovative technology is well-suited for MB classification. Nanopore sequencing demonstrates a robust capacity for precise subtyping of MB, a critical advancement that holds significant potential for enhancing patient stratification in future clinical trials. Its ability to deliver quick and cost-effective results firmly establishes it as a game-changer in the field of MB classification.

Childhood and adolescent cancer survivors (age at diagnosis: 0-21 years) face increased risk for long-term health complications and less favourable outcomes in terms of functioning and social participation. Moreover, they often experience inadequate health-care transitions to appropriate services after leaving paediatric or adolescent services, while the prevalence and severity of late effects increase as they become adults. To address transition challenges, we developed evidence-based recommendations as part of the European Network of Youth Cancer Survivors project with the goal to improve health-care transitions to both long-term survivorship and adult care, ensuring continuity and addressing survivors' unique needs. Using evidence-based methods, an international and multidisciplinary guideline panel developed a clinical practice guideline for health-care transitions. Patient representatives were included at all steps of the development process. The guideline panel systematically reviewed data from PubMed from Jan 1, 1990, to April 8, 2025, and graded the evidence with the GRADE methodology. In addition, existing guidelines and perspectives from patients, parents, and health-care providers were considered when formulating recommendations. Of 2538 citations identified, 86 articles met the inclusion criteria, focusing on health-care transitions for cancer survivors up to age 21 years at diagnosis or for patients with chronic conditions (eg, diabetes and asthma) to extrapolate insights from these populations. The quality of evidence varied from very low to moderate. Unique needs and preferences were captured, ensuring a comprehensive and patient-centred approach to the recommendations. In total, 44 strong recommendations were formulated for health-care transitions of childhood and adolescent cancer survivors. We integrated existing evidence and multistakeholder expertise and developed actionable recommendations that support smooth transitions for childhood and adolescent cancer survivors and improve their lives as adults. Implementing this guideline will enhance the quality of care and improve quality of life by addressing the specific health-care transition needs of this vulnerable population.

Bloom syndrome (BS) is a rare genetic disorder associated with an elevated risk of cancer. In a national multicentre study, nine paediatric patients with BS and cancer were analysed. Median age at cancer diagnosis was 12 years. Four of the nine patients were diagnosed with BS prior to cancer detection. Six presented with solid tumours, whilst three had haematological malignancies. Six received polychemotherapy, often with dose reductions. Complications included prolonged aplasia, sepsis and early treatment discontinuation. Two patients received radiotherapy. Four relapsed, and four died, including one toxic death. However, five achieved remission, highlighting the possibility of curative treatment despite significant toxicities.

Patients diagnosed with osteosarcoma undergo intensive multimodality treatment that can lead to long-term adverse effects, significantly impacting various aspects of daily living. To objectively assess the Health-Related Quality of Life (HR-QoL) in pediatric and adult populations, several Patient-Reported Outcome Measurements (PROMs) are available. However, these questionnaires often exhibit substantial variability in the domains and items they encompass, frequently failing to address aspects that are particularly important after osteosarcoma treatment. A systematic review was conducted to identify the most frequently used questionnaires concerning QoL in pediatric and adult patients with osteosarcoma and to examine the diverse domains and subdomains of QoL assessed by these questionnaires to identify gaps in their coverage, to recommend suitable instruments for an upcoming European trial within the Fighting Osteosarcoma Through European Research (FOSTER) Consortium. English-language literature published since 1980 in PubMed was reviewed. One hundred twenty-eight articles were initially screened for eligibility. Sixty-three original articles were included in the qualitative synthesis. An overview from review articles was given. Selected studies displayed substantial heterogeneity in terms of their objectives, target populations, age ranges, follow-up time, and number of patients included. None of the questionnaires covered all age groups and addressed all important aspects following osteosarcoma treatment. To comprehensively address as many relevant aspects as possible, a combination of questionnaires is suggested. For the adult population, it is recommended to use the EORTC-QLQ-C30 questionnaire together with the Body Image Scale (BIS), while for pediatric patients, the PedsQl-generic and PedsQl-cancer-specific questionnaires and BIS (> 16 years) are suggested. The use of Patient-Reported Outcome Measurement Information Systems (PROMIS) can provide a comprehensive assessment of symptoms such as anxiety, pain, and fatigue. The development of new bone sarcoma-specific, pediatric and adult self-reported questionnaires, or the validation and translation of existing bone sarcoma-specific questionnaires, along with the utilization of new digital possibilities, holds great value for upcoming trials.

Background Hemorrhagic episodes may have physical or psychological effects on children with von Willebrand disease (VWD) and on their families. These effects can be measured by health-related quality of life (HRQoL). Objectives The Willebrand Study Health-related Quality of Life study aimed at filing this knowledge gap, using generic or disease-specific patient-reported outcome (PRO) questionnaires in France. Methods This prospective observational study included 117 children (<18 years) with all VWD types (type 1 with basal von Willebrand factor [VWF]:Ag < 30%; HRQoL [DISABKIDS, VWD-QoL]; and treatment satisfaction, and family burden) were assessed at baseline and 24 months via child and/or parent questionnaires. Results The DISABKIDS questionnaire revealed age-specific patterns: preschoolers (4-7 years) showed predominant physical limitations, while older children (8-17 years) faced independence challenges. The VWD-QoL questionnaire identified early and persistent impacts on peer/family relationships. Parent-child concordance was observed in total DISABKIDS/VWD-QoL scores, although adolescents were more optimistic than parents in some domains, while children aged 4-7 years reported slightly poorer physical abilities. Gender differences emerged, with boys (8-17 years) reporting more school difficulties and younger girls (4-7 years) perceiving greater social/sports restrictions. Disease severity (VWF:Ristocetin co-factor <15 IU/dL in types 1/2) significantly worsened overall HRQoL. Parents reported some dissatisfaction regarding treatment ease and burden. Parents also reported significant disease-related impacts on both family dynamics and personal well-being, particularly in type 3 VWD families. Conclusion HRQoL was reduced in French VWD children and their families secondary to physical, mental, and social health impacts, especially (but not only) in adolescents and in children with severe VWD types.

Introduction > Advances in knowledge about late effects of childhood cancer treatments have led to implement long-term follow-up care. This raises the question of the ethical issues involved in providing information to survivors, and in proposals for long-term follow-up consultations. Method > We conducted a two-part qualitative study: (1) A semi-directive interview survey to explore survivors' experiences of medical proposals for follow-up consultations; (2) The creation of a multidisciplinary ethical reflection group aimed at identifying the ethical issues associated with the systematization of follow-up care. Results > The study identified five key issues related to medical requests and the implementation of follow-up care: (1) The needs and expectations of former patients regarding information; (2) The temporality of after-cancer; (3) The ambivalence of categorizations in light of the diversity of post-cancer experiences; (4) The role of various professionals and the responsibility for follow-up; (5) The plurality of needs and proposed approaches. Discussion > In light of these issues, five areas of concern emerge: (1) Provide information about a risk in life after the disease? (2) When should follow-up be proposed? (3) The category of "former patients": relevant or problematic? (4) Who can or should assume the role and responsibility for follow-up? (5) The diversity of follow-up approaches: balancing standardization with reliance on informal networks.

Introduction L’avancée des connaissances sur les risques de complications et séquelles appelle la mise en place d’un suivi à long terme après la fin des traitements de cancers pédiatriques. Se pose alors la question des enjeux éthiques liés à l’information des anciens patients et aux sollicitations médicales visant à leur proposer une consultation de suivi à long terme. Méthode Nous avons mené une étude qualitative en deux volets : (1) Une enquête par entretiens semi-directifs visant à explorer le vécu des anciens patients à l’égard des sollicitations médicales de suivi ; (2) La constitution d’un groupe de réflexion éthique pluridisciplinaire visant à faire émerger les enjeux éthiques liés à la systématisation du suivi. Résultats L’étude a fait émerger cinq enjeux liés aux sollicitations médicales et à la mise en place du suivi : (1) Les besoins et les attentes des anciens patients en termes d’information ; (2) La temporalité de l’après-cancer ; (3) L’ambivalence des catégorisations au regard de la pluralité du vécu de l’après-cancer ; (4) Le rôle des différents professionnels et la responsabilité du suivi ; (5) La pluralité des besoins et propositions. Discussion Au regard de ces enjeux, cinq points de vigilance émergent : (1) Informer sur un risque dans une vie après la maladie ; (2) Quand proposer un suivi après cancer ? (3) Les « anciens patients », une catégorie en question ? (4) Qui peut/doit endosser le rôle et la responsabilité du suivi ? (5) La pluralité du suivi : entre homogénéisation et appui sur réseaux informels.

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.

OBJECTIVES:To assess the real-world efficacy and safety of recombinant factor IX albumin fusion protein (rIX-FP) in patients with hemophilia B (HB) in France. METHODS:Data on dosing frequency, weekly consumption, and bleeds before-and-after switching to rIX-FP, were collected from December 2021 to February 2024. Annualized (spontaneous) bleeding rates [A(s)BRs] were calculated only in patients on prophylaxis with a follow-up ≥ 6 months. RESULTS:This interim analysis focused on 77 patients ≥ 12 years; 62 (81%) had severe HB. After switching to rIX-FP, the infusion interval was 14 (7-14) days. Weekly consumption was 43 (35.5-53) IU/kg. ABRs and AsBRs were 0.5 (0-1.9) and 0 (0-0.7) (n = 63) at 18.2 (12.3-21.9) months of follow-up. Prophylactic efficacy of rIX-FP was considered 'Excellent'/'Good' in 65/68 (95%) patients. Among the 43 patients previously treated with rFIXFc, 21 increased the infusion interval from 7 (7-11) days with rFIXFc to 14 (7-14) days with rIX-FP; 33/43 (77%) reduced weekly factor IX (FIX) consumption from 59.95 (46.35-77.93) to 42.5 (35.88-50.25) IU/kg. Patients maintained good protection against bleeds. CONCLUSION:This analysis confirmed that switching to rIX-FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.

Background:Childhood cancer survivors are at increased risk of developing cardiovascular diseases, presenting as the main causes of morbidity and mortality within this group. Besides the usual primary and secondary prevention in combination with screening during follow-up, the modifiable lifestyle factors of physical activity, nutrition, and body weight have not yet gained enough attention regarding potential cardiovascular risk reduction.Objective:These practical recommendations aim to provide summarised information and practical implications to paediatricians and health professionals treating childhood cancer survivors to reduce the risk of cardiovascular late effects.Methods:The content derives from either published guidelines or expert opinions from Association of European Paediatric and Congenital Cardiology working groups and is in accordance with current state-of-the-art.Results:All usual methods of prevention and screening regarding the risk, monitoring, and treatment of occurring cardiovascular diseases are summarised. Additionally, modifiable lifestyle factors are explained, and clear practical implications are named.Conclusion:Modifiable lifestyle factors should definitely be considered as a cost-effective and complementary approach to already implemented follow-up care programs in cardio-oncology, which can be actively addressed by the survivors themselves. However, treating physicians are strongly encouraged to support survivors to develop and maintain a healthy lifestyle, including physical activity as one of the major influencing factors. This article summarises relevant background information and provides specific practical recommendations on how to advise survivors to increase their level of physical activity.

Objective To discover new variants associated with low ovarian reserve after gonadotoxic treatment among adult female childhood cancer survivors using a genome-wide association study approach. Design Genome-wide association study. Setting Not applicable. Patients A discovery cohort of adult female childhood cancer survivors from the pan-European PanCareLIFE cohort (n = 743; median age: 25.8 years), excluding those who received bilateral ovarian irradiation, bilateral oophorectomy, central nervous system or total body irradiation, or stem cell transplantation. Replication was attempted in the US-based St. Jude Lifetime Cohort (n = 391; median age: 31.3 years). Exposure Female childhood cancer survivors are at risk of therapy-related gonadal impairment. Alkylating agents are well-established risk factors, and the interindividual variability in gonadotoxicity may be explained by genetic polymorphisms. Data were collected in real-life conditions, and cyclophosphamide equivalent doses were used to quantify alkylation agent exposure. Main Outcome Measure Anti-M & uuml;llerian hormone (AMH) levels served as a proxy for ovarian function, and the findings were combined in a meta-analysis. Results Three genome-wide significant (<5.0 x 10(-8)) and 16 genome-wide suggestive (<5.0 x 10(-6)) loci were associated with log-transformed AMH levels, adjusted for cyclophosphamide equivalent dose of alkylating agents, age at diagnosis, and age at study in the PanCareLIFE cohort. On the basis of the effect allele frequency (EAF) (>0.01 if not genome-wide significant), and biologic relevance, 15 single nucleotide polymorphisms were selected for replication. None of the single nucleotide polymorphisms were statistically significantly associated with AMH levels. A meta-analysis indicated that rs78861946 was associated with borderline genome-wide statistical significance (reference/effect allele: C/T; effect allele frequency: 0.04, beta (SE): -0.484 (0.091). Conclusion This study found no genetic variants associated with a lower ovarian reserve after gonadotoxic treatment because the findings of this genome-wide association study were not statistically significant replicated in the replication cohort. Suggestive evidence for the potential importance of 1 variant is briefly discussed, but the lack of statistical significance calls for larger cohort sizes. Because the population of childhood cancer survivors is increasing, large-scale and systematic research is needed to identify genetic variants that could aid predictive risk models of gonadotoxicity as well as fertility preservation options for childhood cancer survivors.