共找到 20 条结果
In paediatric oncology, imaging biomarkers play an increasing role in diagnostic imaging and research. They can be used for prediction, detection, staging, and grading of diseases, as well as for assessment of response to treatment. Imaging biomarkers are complementary to tissue-based biomarkers, enabling a more personalised approach in oncology care. In this white paper by the European Society of Paediatric Radiology (ESPR) Oncology Taskforce and European Association of Nuclear Medicine (EANM) Paediatrics Committee, an overview is given of the current knowledge on the use of imaging biomarkers in general and per tumour group.
BACKGROUND: To compare paediatric oncologic vascular access ports located on the anterior thoracic wall to ports on the lower lateral thoracic wall, in terms of perceived port-related hindrance and scar-quality. METHODS: A cross-sectional survey study including paediatric oncology patients (≥8-<19 yrs), caregivers (in patients <8 yrs), survivors (>22 yrs with only anterior ports) and nurses of the Princess Máxima Center, the Netherlands, was performed. The survey consisted of questions regarding satisfaction, hindrance during daily life, and port position preference. For survivors, scar-quality was assessed using the validated Patient and Observer Scar Assessment Scale (POSAS 2.0); a high score (i.e., a displeasing scar) was defined as a score higher than the third quartile of the median for that question. RESULTS: In total, 147 participants were included; 83 patients/caregivers, 31 survivors, and 33 nurses. Overall, 81 % was satisfied with the position of their port. Satisfaction, hindrance and complications did not differ between anterior and lower lateral ports. For the anterior position, minimal pressure on the port during daily life was a mentioned reason to prefer this position. For the lower lateral position, less visibility of the scar and easiest access were mentioned. Of all survivors with an anterior port scar, one in five had a displeasing scar and all scars observed were widened. Female patients preferred a lower lateral port, and scar-quality was better for left-sided port scars. CONCLUSION: The port position should be chosen together with patients/caregivers based on the (dis-)advantages of each position, as identified by this study. LEVEL OF EVIDENCE: II.
Parents of children with cancer often face complex decisions about clinical trial participation and treatment options. This cross-sectional survey of 100 parents of pediatric oncology patients explored their perspectives on accessing clinical trials, focusing on influencing factors and perceived barriers. While most parents expressed interest in clinical trial participation, common obstacles included inadequate information, geographic constraints and financial challenges. Many parents emphasized the need for improved communication with healthcare providers to better understand trial risks and benefits. These findings suggest that addressing informational and logistical barriers can enhance access to clinical trials and support informed decision-making among parents of pediatric oncology patients.
Molecular imaging with positron emission tomography (PET) offers significant potential for improving diagnostic accuracy, staging and treatment monitoring in paediatric solid tumours, by using radiopharmaceuticals more specific than [ 18 F]fluorodeoxyglucose ([ 18 F]FDG). While non-[ 18 F]FDG tracers have already improved diagnostic abilities in adult solid cancers such as prostate and neuroendocrine tumours, their use in the paediatric population has been underexplored. This narrative review summarises clinical evidence regarding the use, advantages, and limitations of these more specific PET tracers in paediatric patients. In neuroblastoma, [ 18 F]mFBG, [ 18 F]F-DOPA, and SSTR-targeting peptides stand out as the most evolved and promising tracers for the clinical setting, with encouraging results regarding feasibility, safety and detection rates. SSTR-targeting peptides have also consistently outperformed other imaging methods (both conventional and functional) and carry the benefits of theragnostic applications. For brain tumours, amino acid-based tracers in general stand out due to their ability to surpass the blood-brain barrier/blood-tumour barrier (BBB/BTB) and their specific accumulation in malignant tissue. Other paediatric solid tumours, such as sarcoma and bone tumours, suffer from a clear lack of clinical evidence that should be addressed in the near future. The studies performed to date show high accuracy, evident prognostic value, and significant clinical impact of non-[ 18 F]FDG tracers in paediatric patients with solid tumours. However, prospective studies with longer follow-up times are warranted to provide high-level evidence regarding the impact of these tracers on patient management and prognosis, to consolidate the encouraging results obtained so far. Further research and international collaboration will be essential to overcome current challenges related to low incidence of paediatric solid tumours, logistical barriers and concerns about radiation exposure.
Artificial intelligence (AI) is rapidly transforming pediatric oncology by creating new means to improve the accuracy and efficacy of cancer diagnosis and treatment in children. This review critically examines current applications of AI technologies like machine learning (ML) and deep learning (DL) to the main types of pediatric cancers. However, the application of AI to pediatric oncology is prone to certain challenges, including the heterogeneity and rarity of pediatric cancer data, rapid technological development in imaging, and ethical concerns pertaining to data privacy and algorithmic transparency. Collaborative efforts and data-sharing schemes are important to surpass these challenges and facilitate effective training of AI models. This review also points to emerging trends, including AI-based radiomics and proteomics applications, and provides future directions to realize the full potential of AI in pediatric oncology. Finally, AI is a promising paradigm shift toward precision medicine in childhood cancer treatment, which can enhance the survival rates and quality of life for pediatric patients.
Wearable devices (WDs) are capable of collecting large volumes of objective and clinically relevant patient data that is not yet routinely captured.This ability to collect continuous, real-time data offers a unique opportunity to gather health information in new and insightful ways.In paediatric oncology, advancement in treatment have led to significant improvements in survival rates.However, aggressive therapies often result in a range of distressing side effects, which can severely impact quality of life, and even can become life-threatening themselves.Supportive care plays a crucial role in mitigating these symptoms, aiming to prevent and manage side effects.Patient-reported outcomes should be used to guide initiation and choice of supportive care treatment whenever possible.In this context, continuous monitoring of vital signs, physical activity and other health parameters using WDs could add individual, patient specific information regarding a patient's current condition.In this article we discuss the requirements of non-invasive WDs for their use in paediatric oncology, give an overview on possible areas of application in children with cancer and discusses challenges that must be addressed.Also we identify key research gaps and speculate on future perspectives.
Artificial intelligence (AI) has recently garnered significant public attention. Among the various fields where AI can be applied, medicine stands out as one with immense potential. In particular, AI is transforming precision oncology by providing innovative approaches to customize cancer treatments for individual patients. This article examines the latest developments in AI-powered tools designed to improve cancer diagnosis accuracy and predict treatment outcomes. The integration of AI into precision oncology is transforming cancer care by enabling more personalized and effective treatments, minimizing treatment-related toxicities, and enhancing patient survival rates. As AI advances, it will be pivotal in developing more targeted and successful cancer therapies. The field is still in its early stages, and future progress will benefit from establishing standards and guidelines to promote rigorous methodological design and uphold ethical principles. This research highlights the transformative potential of AI in addressing the challenges posed by cancer heterogeneity.
Purpose This article provides an overview of the scientific content on tirbanibulin 1 % ointment, summarizing key Phase IV results and real-life experiences with tirbanibulin shared by dermatologists across Europe. Materials and Methods Summary of posters, oral presentation and symposium discussions related to tirbanibulin presented at the 11th World Congress of Melanoma in conjunction with 21st European Association of Dermato Oncology (EADO) Congress held between 3rd and 5th of April 2025 in Athens, Greece. Results We report presented data on the efficacy/effectiveness, safety and tolerability of tirbanibulin 1 % ointment for the treatment of actinic keratosis (AK) on the face, scalp and décolleté. We also collected evidence supporting its use in AK in organ transplant recipients (OTR), solar damaged skin, pigmented AK (PAK), proliferative AK, hypertrophic AK, actinic cheilitis (AC), keratinocyte carcinoma (KC) and lentigo maligna (LM). Conclusions Overall, data from a Phase IV trial and real-world clinical practice suggest that tirbanibulin is an effective, safe and well-tolerated treatment for AK. Tirbanibulin may also be an option for OTR, patients with PAK, proliferative or hypertrophic AK, and even for conditions such as AC or KC, although the sample size is small in some of these groups and further evidence is needed.
Antimicrobial and diagnostic stewardship initiatives have become increasingly important in paediatric settings. The value of qualitative approaches to conduct stewardship work in paediatric patients is being increasingly recognized. This article seeks to provide an introduction to basic elements of qualitative study designs and provide an overview of how these methods have successfully been applied to both antimicrobial and diagnostic stewardship work in paediatric patients. A multidisciplinary team of experts in paediatric infectious diseases, paediatric critical care and qualitative methods has written a perspective piece introducing readers to qualitative stewardship work in children, intended as an overview to highlight the importance of such methods and as a starting point for further work. We describe key differences between qualitative and quantitative methods, and the potential benefits of qualitative approaches. We present examples of qualitative research in five discrete topic areas of high relevance for paediatric stewardship work: provider attitudes; provider prescribing behaviours; stewardship in low-resource settings; parents' perspectives on stewardship; and stewardship work focusing on select high-risk patients. Finally, we explore the opportunities for multidisciplinary academic collaboration, incorporation of innovative scientific disciplines and young investigator growth through the use of qualitative research in paediatric stewardship. Qualitative approaches can bring rich insights and critically needed new information to antimicrobial and diagnostic stewardship efforts in children. Such methods are an important tool in the armamentarium against worsening antimicrobial resistance, and a major opportunity for investigators interested in moving the needle forward for stewardship in paediatric patients.
Oncology drug discovery and development has always been an area facing many challenges. Phase 1 oncology studies are typically small, open-label, sequential studies enrolling a small sample of adult patients (i.e., 3-6 patients/cohort) in dose escalation. Pediatric evaluations typically lag behind the adult development program. The pediatric starting dose is traditionally referenced on the recommended phase 2 dose in adults with the incorporation of body size scaling. The size of the study is also small and dependent upon the prevalence of the disease in the pediatric population. Similar to adult development, the dose is escalated or de-escalated until reaching the maximum tolerated dose (MTD) that also provides desired biological activities or efficacy. The escalation steps and identification of MTD are often rule-based and do not incorporate all the available information, such as pharmacokinetic (PK), pharmacodynamic (PD), tolerability and efficacy data. Therefore, it is doubtful if the MTD approach is optimal to determine the dosage. Hence, it is important to evaluate whether there is an optimal dosage below the MTD, especially considering the emerging complexity of combination therapies and the long-term tolerability and safety of the treatments. Identification of an optimal dosage is also vital not only for adult patients but for pediatric populations as well. Dosage-finding is much more challenging for pediatric populations due to the limited patient population and differences among the pediatric age range in terms of maturation and ontogeny that could impact PK. Many sponsors defer the pediatric strategy as they are often perplexed by the challenges presented by pediatric oncology drug development (model of action relevancy to pediatric population, budget, timeline and regulatory requirements). This leads to a limited number of approved drugs for pediatric oncology patients. This review article provides the current regulatory landscape, incentives and how they impact pediatric drug discovery and development. We also consider different pediatric cancers and potential clinical trial challenges/opportunities when designing pediatric clinical trials. An outline of how quantitative methods such as pharmacometrics/modelling & simulation can support the dosage-finding and justification is also included. Finally, we provide some reflections that we consider helpful to accelerate pediatric drug discovery and development.
Even though children's malignant bone tumours are rare, it is crucial to understand how to identify and stage them accurately to develop an appropriate treatment plan. Ewing's sarcoma and osteosarcoma are the two main paediatric bone malignancies and require multidisciplinary treatment involving radiologists, orthopaedists, oncologists, pathologists, and paediatricians. These neoplasms may be associated with genetic syndromes but typically occur in patients with no known germline abnormalities. With a frequency of 4.4 per million, osteosarcoma is the most common malignant bone tumour in children. Ewing's sarcoma has an incidence of 2.5 to 3 per million, making it the second most prevalent. Clinically, these neoplasms present with pain and inflammation in the bones and joints, nocturnal pain unresponsive to drug therapy, systemic symptoms such as fever or weight loss, and persistent symptoms-all of which should prompt clinicians to initiate further diagnostic investigations. The gold standard for diagnosis includes X-ray examination and MRI, which provide an accurate assessment of tumour extension into the medullary canal and surrounding soft tissues. Fluorine-18-labelled FDG-PET scans or fluoro-deoxyglucose positron emission tomography are valuable for evaluating tumour aggressiveness and excluding metastases. A biopsy is mandatory once all other diagnostic tests have been completed. Accurate diagnosis and timely referral to an experienced clinic are essential for ensuring prompt access to treatment and improving patient outcomes.
Chemotherapy-induced toxicities remain challenging in pediatric oncology, affecting patient outcomes, hospital stays, and quality of life. Genetic variation can partly explain these toxicities, and pharmacogenomics could potentially optimize treatment. This review provides an overview of pharmacogenomic studies in relation to chemotherapy-induced toxicity in children with solid tumors. A systematic literature search was performed in PubMed, Embase, and Web of Science following PRISMA guidelines. Two independent reviewers assessed eligibility, risk of bias using ROBINS-I, and extracted data. Out of 9000 articles screened, 279 were deemed relevant, and 59 met the inclusion criteria by focusing on children with solid tumors and pharmacogenomics in relation to chemotherapy-induced toxicity. Following risk of bias assessment, 24 articles with low to moderate risk of bias were summarized. Identifying specific SNPs associated with toxicities proved challenging due to variability across studies. For methotrexate, the genes ABCC2, MTHFR, and SXR were associated with myelosuppression and hepatotoxicity. The genes ABCC3, COMT, ERCC2, GSTP1, GSTT1, LRP2, SLC22A2, and TPMT showed associations with ototoxicity due to platinum-based drugs. Anthracycline-induced cardiotoxicity was associated with CBR2, CELF4, GSTM1, HAS3, RARG, and SLC28A3, and further with HNMT and SLC22A2 in younger children, with ABCB4 in females, and with SULT2B1 in males. A dose-dependent effect of CELF4 on cardiotoxicity was noted with anthracycline doses over 300 mg/m². This review highlights the complexity and variability of pharmacogenomic associations with chemotherapy-induced toxicities in pediatric oncology. While certain genetic variants show associations with specific toxicities, larger multinational/center studies are needed to strengthen the associations and improve clinical guidelines.
Pediatric oncology and hematopoietic stem cell transplant (HSCT) patients may fail to mount an appropriate immune response to COVID-19 vaccinations. The immunologic response to initial SARS-CoV-2 vaccination was studied in 93 pediatric patients with hematologic malignancy, solid tumors, or post-HSCT. The majority of patients who were not receiving chemotherapy showed a positive humoral and cell-mediated response to COVID-19 vaccination. In contrast, variable responses to vaccination were seen in patients receiving chemotherapy. Patients with a normal lymphocyte count showed higher rates of seroconversion than their lymphocytopenic counterparts. This report details pediatric oncology and HSCT patients' immunologic responses to COVID-19 vaccination during all phases of treatment.
The National Cancer Institute designated models of comprehensive cancer care centres endeavour to enable the delivery of high-quality, holistic cancer care, informed by research evidence across the cancer care trajectory. These comprehensive cancer centers have typically been adult-oncology-focused, leaving an important gap and opportunity to consider what a model of comprehensive cancer care might look like for children and young people. With the advent of the opening of the first comprehensive children's cancer center in Australia and the southern hemisphere, this commentary considers the important role that psychosocial oncology needs to play in driving high-quality, person-centered comprehensive cancer care for all.
Bevacizumab is often used off-label in pediatric neuro-oncology, and evidence for indications of bevacizumab use in pediatric neuro-oncology is often fragmented. Therefore, this review aims to provide an organized summary of efficacy across different types of tumors, highlight outcomes, and link findings to the underlying biology. Gaps in the literature were also identified to guide future research. We narratively synthesized various pediatric studies, and the following tumor categories were identified for discussion: low-grade glioma, high-grade glioma, diffuse intrinsic pontine glioma, schwannoma, medulloblastoma, radiation necrosis, and cerebral edema. Key outcomes considered included overall survival, event-free survival, progression-free survival, vision and/or hearing improvements, steroid use, quality of life, and toxicity. The greatest benefits were observed in cases such as recurrent medulloblastoma in combination with temozolomide and irinotecan, optic pathway glioma visual function, and diminished steroid use in radiation necrosis. Results were poorer in cases of newly diagnosed high-grade gliomas and diffuse intrinsic pontine gliomas. The medication was overall well tolerated, with adverse events like hypertension, proteinuria, and epistaxis often being manageable with surveillance. In consideration of the results, bevacizumab should be considered based on the tumor profile, and its outcome measured along functional endpoints, besides radiological evolution. Continued investigations into outcome measures, as well as combination with targeted treatments and optimizing therapy, will contribute to improving outcomes in this vulnerable population.
Radiotherapy is an indispensable treatment modality in the management of rhabdomyosarcoma. Numerous efforts have been made to improve outcomes. The current thinking and future developments in the radiation oncology field about how to raise cure rates, especially in the highest-risk patients, are presented.
PURPOSE: Within the Paediatric Rare Tumours Network-European Registry (PARTNER) project, we aimed to evaluate the situation on the registration and management of paediatric patients affected by very rare tumours (VRT) in the European low health expenditure average rates (LHEAR) countries. METHODS: A survey regarding infrastructure, organisation, and clinical decision-making information on VRT was designed. This survey was distributed to the representatives of LHEAR countries involved in the activities of the PARTNER Work Package 7. RESULTS: Eighteen answers from 17 countries were collected regarding the national organisation, methods of registration of VRT cases, the availability of medical experts in VRT, the access to updated diagnostic and therapeutic procedures (such as proton therapy, immunotherapy and, targeted therapies), and research on paediatric VRT. A high variability in the registration and management of patients with VRT has been observed with additional wide inequalities in pathology review, uniformity of clinical decisions, availability of selected procedures, and diagnostic and research tools. CONCLUSION: In the majority of LHEAR countries, no clinical or research structures have been implemented for children and adolescents with VRT. Therefore, VRT still have an orphan status in these countries. These significant differences on the technology access and use between European regions need to be addressed.
With the continuous development of genetics in healthcare, there has been a significant contribution to the development of precision medicine, which is ultimately aimed at improving the care of patients. Generally, drug treatments used in Oncology are characterized by a narrow therapeutic range and by their potential toxicity. Knowledge of pharmacogenomics and pharmacogenetics can be very useful in the area of Oncology, as they constitute additional tools that can help to individualize patients' treatment. This work includes a description of some genes that have been revealed to be useful in the field of Oncology, as they play a role in drug prescription and in the prediction of treatment response.
BACKGROUND: Consensus statements and clinical practice guidelines are widely available for enhancing the care of cancer patients. Despite subtle differences in their definition and purpose, these terms are often used interchangeably. We systematically assessed the methodological quality of consensus statements and clinical practice guidelines published in three commonly read, geographically diverse, cancer-specific journals. Methods Consensus statements and clinical practice guidelines published between January 2005 and September 2013 in Current Oncology, European Journal of Cancer and Journal of Clinical Oncology were evaluated. Each publication was assessed using the Appraisal of Guidelines for Research and Evaluation II (AGREE II) rigour of development and editorial independence domains. For assessment of transparency of document development, 7 additional items were taken from the Institute of Medicine's standards for practice guidelines and the Journal of Clinical Oncology guidelines for authors of guidance documents. METHODS: Consensus statements and clinical practice guidelines published between January 2005 and September 2013 in Current Oncology, European Journal of Cancer and Journal of Clinical Oncology were evaluated. Each publication was assessed using the Appraisal of Guidelines for Research and Evaluation II (AGREE II) rigour of development and editorial independence domains. For assessment of transparency of document development, 7 additional items were taken from the Institute of Medicine's standards for practice guidelines and the Journal of Clinical Oncology guidelines for authors of guidance documents. FINDINGS: Thirty-four consensus statements and 67 clinical practice guidelines were evaluated. The rigour of development score for consensus statements over the three journals was 32% lower than that of clinical practice guidelines. The editorial independence score was 15% lower for consensus statements than clinical practice guidelines. One journal scored consistently lower than the others over both domains. No journals adhered to all the items related to the transparency of document development. One journal's consensus statements endorsed a product made by the sponsoring pharmaceutical company in 64% of cases. CONCLUSION: Guidance documents are an essential part of oncology care and should be subjected to a rigorous and validated development process. Consensus statements had lower methodological quality than clinical practice guidelines using AGREE II. At a minimum, journals should ensure that that all consensus statements and clinical practice guidelines adhere to AGREE II criteria. Journals should consider explicitly requiring guidelines to declare pharmaceutical company sponsorship and to identify the sponsor's product to enhance transparency.
Background: During the EpSSG RMS2005 trial, organ-sparing surgery (OSS) with brachytherapy (BT) became the local therapy (LT) of choice for selected patients with bladder-prostate rhabdomyosarcoma (BP-RMS). We compare this LT technique with surgical resection and/or external-beam radiotherapy. Methods: Patients with BP-RMS without nodal or metastatic spread enrolled in RMS2005 were categorized by their LT, differentiating OSS from organ-depleting surgery (ODS) and BT from external-beam radiotherapy (EBRT). Progressive disease, relapse or death were considered events for progression-free survival (PFS) and all deaths for overall survival (OS). Results: The cohort comprised 176 patients, aged 10days-21.8years (median 2.5years). Median follow-up was 6.5years (22months-12.5years): 5year-PFS was 80.3% (95%CI:73.6-85.5%); 5year-OS was 90.7% (95%CI:85.3-94.2%). Patients selected for surgery alone or BT with/without OSS (BT+/-OSS) differed significantly in age, tumour size and location from those offered EBRT alone or any other surgery and radiotherapy. Nevertheless, 5year-PFS was similar for the LT groups. However, 5year-OS differed significantly, being highest in patients suitable for surgery alone (100%; by ODS in 55%) or BT+/-OSS (98.1%; 95%CI:87.4-99.7%). Patients with local tumour progression/relapse after EBRT failed salvage: 5year-OS was 81.8% (95%CI:58.5-92.8%) for EBRT alone and 85.3% (95%CI:71.6-92.7%) for surgery and radiotherapy. Postponing LT until after chemotherapy cycle 7 did not significantly impact 5year-PFS or OS. Conclusions: The risk of events was similar for different LT modalities; poor salvage after EBRT significantly reduced 5year-OS. Although not feasible for all, BT+/-OSS offers an excellent prospect of cure, the best chance of organ retention while avoiding EBRT, and may be delayed for chemotherapy responsive tumours.