Large datasets in paediatric oncology are inherently rare. Therefore, it is paramount to fully exploit all available data, which are distributed over several resources, including biomaterials, images, clinical trials, and registries. With privacy-preserving record linkage (PPRL), personalised or pseudonymised datasets can be merged, without disclosing the patients\' identities. Although PPRL is implemented in various settings, use case descriptions are currently fragmented and incomplete. The present paper provides a comprehensive overview of current and future use cases for PPRL in paediatric oncology. We analysed the literature, projects, and trial protocols, identified use cases along a hypothetical patient journey, and discussed use cases with paediatric oncology experts. To structure PPRL use cases, we defined six key dimensions: distributed personalised records, pseudonymisation, distributed pseudonymised records, record linkage, linked data, and data analysis. Selected use cases were described (a) per dimension and (b) on a multi-dimensional level. While focusing on paediatric oncology, most aspects are also applicable to other (particularly rare) diseases. We conclude that PPRL is a key concept in paediatric oncology. Therefore, PPRL strategies should already be considered when starting research projects, to avoid distributed data silos, to maximise the knowledge derived from collected data, and, ultimately, to improve outcomes for children with cancer.

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OBJECTIVE: The terminal phase of childhood cancer poses profound physical and mental challenges for children, simultaneously influencing parents and rendering them particularly susceptible to psychosocial issues.
METHODS: This review included studies exploring the experiences of either: (1) paediatric terminal oncology patients aged under 18 years, (2) parents with a child facing terminal cancer undergoing palliative care, or (3) parents with a child who had undergone palliative care and died. English language, qualitative journal studies or grey literature of any care settings, geographical locations and publication years were included. Studies exploring the experiences of (1) paediatric terminal oncology not receiving palliative care from qualified healthcare professionals, and (3) non-biological parents or non-parental family members, were excluded.
METHODS: A total of 22 studies were included, published between January 2000 and December 2023. Seventy-two children (aged between 5 and 18 years old) and 236 parents (aged between 24 and 57 years old) participated across all studies. Palliative care settings mostly comprised oncology centres, hospitals and homes.
RESULTS: Two themes were identified from the 22 included studies: (1) Navigating rough waters and enduring hardships, and (2) Preparing for end-of-life amidst the looming threat of death.
CONCLUSIONS: This review underscored the importance of integrating palliative childhood cancer care in a holistic, age-specific, family-centred, person-centred and timely manner.
CONCLUSIONS: Paediatric oncology nurses should attend to physical and psychosocial needs of children and parents, fostering familial and social ties while recognising cultural and spiritual needs. Future research could recruit participants of varying ages, genders, and cultures.

BACKGROUND: Taurolidine-citrate(-heparin) lock solutions (TCHL) are suggested as a promising and safe method for the prevention of central line-associated bloodstream infections (CLABSI).
OBJECTIVE: To investigate the efficacy TCHL for the prevention of CrLABSI in paediatric oncology patients.
METHODS: An assessor blinded randomized controlled trial at the Princess Máxima Centre for paediatric oncology, the Netherlands, was performed from 2020-2023. Paediatric oncology patients receiving a tunnelled central venous access device (CVAD) were eligible. A total of 462 patients was required to compare the TCHL to the heparin-only lock (HL). Patients were followed-up for the first 90 days after CVAD insertion. The primary outcome was the incidence of the first CLABSI from CVAD insertion until the end of follow-up. Intention-to-treat and per-protocol analyses were performed.
RESULTS: In total, 232 were randomized in the HL and 231 in the TCHL-group. A total of 47 CLABSIs were observed. The intention-to-treat analysis showed that a CLABSI was observed in 26 (11.2%) of the HL-group patients versus 21 (9.1%) of the TCHL-group patients; incidence rate ratio (IRR) of 0.81 (CI95%0.46-1.45), in favour of the TCHL-group. The per-protocol analysis showed that a CLABSI was observed in 10 (7.9%) of the HL-group patients versus 6 (4.8%) of the TCHL-group patients; IRR of 0.59 (CI95%0.21-1.62) in favour of the TCHL-group. Adverse events were more common in the TCHL-group but rarely reported.
CONCLUSIONS: No difference was detected between the TCHL and HL in the incidence of CLABSI in paediatric oncology patients.
BACKGROUND: ClinicalTrials.gov NCT05740150.

OBJECTIVE: In our study, we aimed to characterise adult childhood cancer survivors (ACCS), assess their health issues, gauge health-related quality of life (HRQOL) and evaluate visit satisfaction.
METHODS: Prospective cohort study using data from clinical visits and questionnaires.
METHODS: Interdisciplinary follow-up programme for ACCS based on the long-term follow-up (LTFU) guidelines of the Children\'s Oncology Group and overseen by internists in two Swiss hospitals.
METHODS: ACCS attending our LTFU clinics between April 2017 and January 2022 were eligible.
METHODS: We documented medical history, current health status and assessed HRQOL using Short Form-36 V.2, comparing it with Swiss general population (SGP) norms (T mean=50, SD=10; age stratified). 3 months post visit, a feedback questionnaire was distributed.
RESULTS: Among 102 ACCS (mean age: 32 years (range: 18-62 years), 68% women), 43 had no prior follow-up (36 ACCS>28 years, 7 ACCS≤28 years). A notable 94% had health issues, affecting an average of 6.1 (SD=3.3) organ systems. HRQOL was lower in ACCS>28 years than the SGP>28 years (physical: 44.8 (SD=11.65) vs 49.3 (SD=10.29), p=0.016; mental: 44.4 (SD=13.78) vs 50.53 (SD=9.92), p=0.004). Older ACCS (>28 years) reported inferior physical (44.8 vs 50.1 (SD=9.30), p=0.017) and mental HRQOL (44.4 vs 50.3 (SD=7.20), p=0.009) than younger ACCS. The majority of respondents reported high levels of satisfaction with the consultation, exceeding 90%.
CONCLUSIONS: ACCS attending LTFU clinics face diverse health issues impacting multiple organ systems and exhibit lower HRQOL compared with the SGP. Thus, internist-led LTFU clinics are crucial for optimising follow-up care.

Phosphatidylinositol 3-kinase (PI3-K) signalling pathway is a crucial path in cancer for cell survival and thus represents an intriguing target for new paediatric anti-cancer drugs. However, the unique clinical toxicities of targeting this pathway (resulting in hyperglycaemia) difficulties combining with chemotherapy, rarity of mutations in childhood tumours and concomitant mutations have resulted in major barriers to clinical translation of these inhibitors in treating both adults and children. Mutations in PIK3CA predict response to PI3-K inhibitors in adult cancers. The same mutations occur in children as in adults, but they are significantly less frequent in paediatrics. In children, high-grade gliomas, especially diffuse midline gliomas (DMG), have the highest incidence of PIK3CA mutations. New mutation-specific PI3-K inhibitors reduce toxicity from on-target PI3-Kα wild-type activity. The mTOR inhibitor everolimus is approved for subependymal giant cell astrocytomas. In paediatric cancers, mTOR inhibitors have been predominantly evaluated by academia, without an overall strategy, in empiric, mutation-agnostic clinical trials with very low response rates to monotherapy. Therefore, future trials of single agent or combination strategies of mTOR inhibitors in childhood cancer should be supported by very strong biological rationale and preclinical data. Further preclinical evaluation of glycogen synthase kinase-3 beta inhibitors is required. Similarly, even where there is an AKT mutation (∼0.1 %), the role of AKT inhibitors in paediatric cancers remains unclear. Patient advocates strongly urged analysing and conserving data from every child participating in a clinical trial. A priority is to evaluate mutation-specific, central nervous system-penetrant PI3-K inhibitors in children with DMG in a rational biological combination. The choice of combination, should be based on the genomic landscape e.g. PTEN loss and resistance mechanisms supported by preclinical data. However, in view of the very rare populations involved, innovative regulatory approaches are needed to generate data for an indication.

UNASSIGNED: The burden of advanced and metastatic cancer is high among children in developing countries, and palliative care (PC) services for children are sparsely available and poorly accessed. To estimate the burden of PC requirements in children with metastatic neuroblastoma (NB), and to evaluate the PC services offered.
UNASSIGNED: Retrospective analysis of case records of children 1-14 years diagnosed with metastatic NB from 1 January 2008 to 31 December 2017.
UNASSIGNED: One hundred and nineteen patients with metastatic NB were included, of which 87 patients received PC consultation. Early PC referral occurred only in 13 patients (14.9%), and pain was the most prominent symptom. Shifting of care from oncology to PC occurred at disease relapse in 58 patients (66.6%) and at end-of-life in 16 patients (18.3%). Nausea/vomiting, constipation and abdominal distension were the most common symptoms during end-of-life. Seventy-one patients (85%) died of disease, median time to death being 9 months from diagnosis and 4 months from relapse. The mean time from initiation of PC to death was 4.2 months.
UNASSIGNED: Timely integration of PC and shared care incorporating the oncology team, PC team and local paediatricians can ease out transition in care, ensure a continuum of care and improve the quality of treatment delivered to children with metastatic cancer.

Rhabdomyosarcomas are the most common soft-tissue sarcomas, found usually in the younger age group. Histologically, they are subdivided into embryonal, alveolar, pleomorphic and not otherwise specified. They have a heterogenous appearance on imaging with few additional characteristic features based on the subtype. Botryoid variant of embryonal rhabdomyosarcoma commonly involves the genitourinary and the biliary system. They can be multifocal. Most of these lesions have a heterogenous appearance on imaging with areas of necrosis and haemorrhage. On ultrasound, they are polypoidal with cystic areas and are vascular. The lesions are hyperintense on T2 sequences, isointense to the skeletal muscle on T1 sequences and show heterogenous enhancement. Surgery is the mainstay of treatment along with radiotherapy or chemotherapy depending on the site and the stage of the tumour. We report a case of botryoid variant of rhabdomyosarcoma involving the vagina and the urinary bladder.

BACKGROUND: Paediatric oncology/haematology patients and their families are confronted with a life-threatening situation for which music therapy can be a cross-linguistic field of action. The creative act of making music together offers the possibility to strengthen competences and make conflicts tangible. Besides its complementing of evidence-based biomedical care, there is little research on the feasibility and efficacy of interactive music therapy including the diagnosed child and their significant others.
METHODS: We conducted an assessor blind, prospective, multicentric feasibility randomized controlled trial (RCT) with subsequent intervention. Including overall 52 child-significant other dyads, INMUT investigates interaction-focused music therapy with cancer-affected children and their significant others (INMUT-KB; n = 21) compared to music therapy only with the child (MUT-K; n = 21) and a wait-list group (WLG; n = 10). The measurement points include the screening for a cancer diagnosis, psychometric baseline (pre-T1), initial assessment (T1/T2), music therapy sessions (T3-T9), final assessment (T10), final psychometric evaluation (post-T10), and 3-month follow-up (cat-T11). Feasibility and acceptability of the (1) research methodology, (2) intervention and (3) estimation of effect sizes will be assessed using qualitative and quantitative data. The proposed primary outcome includes the parent-child interaction (APCI), and the proposed secondary outcomes refer to subjective goal achievement (GAS), quality of life (KINDL), system-related functional level (EXIS), psychosocial stress (BAS), psychosomatic complaints (SCL-9k), and resources (WIRF). We plan to investigate the efficacy of INMUT-KB and MUT-K post-intervention (post-T10) within the RCT design and at 3-month follow-up (cat-T11).
CONCLUSIONS: This study will provide insights into the feasibility of INMUT and the final sample needed for a confirmatory RCT. We will reflect on successfully implemented study procedures and, if necessary, provide recommendations for changes considering the design, procedures, measures, and statistical analyses. The discussion will conclude with an evaluation whether a confirmatory RCT is worth the investment of future resources, including the calculated number of child-significant other dyads needed based on the efficacy trends derived from this feasibility study.
BACKGROUND: ClinicalTrials.gov: NCT05534282; date of registration: June 23, 2022.