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Renal function-based carboplatin dosing is used routinely in paediatric oncology clinical practice. It is important that accurate assessments of renal function are carried out consistently across clinical centres, a view supported by recently published British Nuclear Medicine Society (BNMS) guidelines for measuring glomerular filtration rate (GFR). These guidelines recommend the use of a radioisotope method for GFR determination, with between two and five blood samples taken starting 2 h after radioisotope injection and application of the Brochner-Mortensen (BM) correction factor. To study the likely impact of these guidelines, we have investigated current practices of measuring GFR in all 21 Children's Cancer and Leukaemia Group (CCLG) paediatric oncology centres in the United Kingdom. This information was used to evaluate the potential impact on renal function-based carboplatin dosing using raw 51Cr-EDTA clearance data from 337 GFR tests carried out in children with cancer. A questionnaire survey revealed that between two and four samples were taken after isotope administration, with BM and Chantler corrections used in 38% (8/21) and 28% (6/21) of centres, respectively. A change from Chantler to BM correction, based on the BNMS guidelines, would result in a >10% decrease in carboplatin dose in at least 15% of patients and a >25% decrease in 2% of patients. A greater proportion of patients would have an alteration in carboplatin dose when centres not using any correction factor implement the BM correction. The increase in estimated 51Cr-EDTA half-life observed by omitting the 1 h sample decreases carboplatin dose by >10% in 23–52% of patients and by >25% in 3% of patients. This study highlights current variations in renal function measurement between clinical centres and the potential impact on carboplatin dosing. A standard methodology for estimating GFR should be followed to achieve uniform dosing in children with cancer.British Journal of Cancer (2008) 99, 894–899. doi:10.1038/sj.bjc.6604612 www.bjcancer.com Published online 19 August 2008 [ABSTRACT FROM AUTHOR]

The aim was to produce evidence-based guidelines on mouth care for children, teenagers and young adults receiving chemotherapy and/or radiotherapy. Systematic reviews were undertaken and research was graded according to the methods of the Scottish Intercollegiate Guidelines Network. Where no relevant research was identified, an opinion-gathering process was undertaken. 'Best practice' recommendations were developed with regard to appropriate dental care and basic oral hygiene. An evaluation of oral assessment tools identified seven which had been assessed for reliability and/or validity. Only Eilers' Oral Assessment Guide was felt to be relevant for daily clinical practice. A variety of interventions have been used for the management of oral mucositis, candidiasis, xerostomia and herpes simplex virus; few are supported by research evidence. Careful oral management of children treated for cancer can improve the quality of life during treatment. The guidelines have the potential to improve patient care by promoting interventions of proven benefit and discouraging use of ineffective or potentially harmful practices which may result in adverse patient outcomes. [ABSTRACT FROM AUTHOR]

Survival from Wilms tumour is excellent. Hence, better markers are required to restrict treatments causing late sequelae to those at highest risk of relapse. We investigated the prognostic significance of loss of heterozygosity (LOH) on 1p and 16q in 426 favourable histology Wilms tumours treated with either immediate nephrectomy (63%) or preoperative chemotherapy (37%). Four years RFS and OS were 84.6% and 92.0%, respectively. 10.3% tumours had LOH 1p, 14.6% LOH 16q, with 2.6% at both loci. In multivariate analysis, LOH 16q was associated with an increased risk of relapse (hazard ratio (HR) 2.69, 95%CI: 1.47-4.92) and death (HR 2.67, 95%CI: 1.17-6.06). LOH 1p showed no significant associations. These results were not influenced by treatment approach. LOH 16q is an adverse risk factor in favourable histology Wilms tumour, regardless of initial approach to therapy. Its relationship with histological risk groups defined after neo-adjuvant chemotherapy requires analysis in a larger series, and is the subject of the current SIOP WT 2001 trial. [ABSTRACT FROM AUTHOR]

Background: In the United Kingdom, healthcare data is collected on all patients receiving National Health Service (NHS) care, including children and young people (CYP) with cancer. This data is used to inform service delivery, and with special permissions used for research. The use of routinely collected health data in research is an advancing field with huge potential benefit, particularly in CYP with cancer where case numbers are small and the impact across the life course can be significant. Patient and public involvement (PPI) exercise aims: Identify current barriers to trust relating to the use of healthcare data for research. Determine ways to increase public and patient confidence in the use of healthcare data in research. Define areas of research importance to CYP and their carers using healthcare data., Methods: Young people currently aged between 16 and 25 years who had a cancer diagnosis before the age of 20 years and carers of a young person with cancer were invited to take part via social media and existing networks of service users. Data was collected during two interactive online workshops totalling 5 h and comprising of presentations from health data experts, case-studies and group discussions. With participant consent the workshops were recorded, transcribed verbatim and analysed using thematic analysis., Results: Ten young people and six carers attended workshop one. Four young people and four carers returned for workshop two. Lack of awareness of how data is used, and negative media reporting were seen as the main causes of mistrust. Better communication and education on how data is used were felt to be important to improving public confidence. Participants want the ability to have control over their own data use. Late effects, social and education outcomes and research on rare tumours were described as key research priorities for data use., Conclusions: In order to improve public and patient trust in our use of data for research, we need to improve communication about how data is used and the benefits that arise., (© 2023. BioMed Central Ltd., part of Springer Nature.)

Background: Children with cancer are not at increased risk of severe SARS-CoV-2 infection; however, adults with haematological malignancies have increased risk of severe infections compared with non-haematological malignancies., Methods: We compared patients with haematological and non-haematological malignancies enrolled in the UK Paediatric Coronavirus Cancer Monitoring Project between 12 March 2020 and 16 February 2021. Children who received stem cell transplantation were excluded., Results: Only 2/62 patients with haematological malignancy had severe/critical infections, with an OR of 0.5 for patients with haematological compared with non-haematological malignancies., Interpretation: Children with haematological malignancies are at no greater risk of severe SARS-CoV-2 infection than those with non-haematological malignancies., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2022. Re-use permitted under CC BY. Published by BMJ.)

Purpose: Service development to improve patient safety and experience, and improve staff safety and confidence when managing telephone calls from parents or carers of children with cancer in the UK., Method: A multi-layered mixed methods approach broadly based on sequential PDSA (plan, do, study, act) cycles, to a series of quality initiative projects spanning 14 years. Various project styles and methods are described., Results: A Telephone Triage Toolkit for children's cancer services was piloted, reviewed and rolled out across the UK. Similarities were identified between adult and paediatric cancer services when identifying the case for need, enabling partnership working. A scheduled review completed in 2020 included new developments in cancer treatment, building on user experience, local audits and national feedback, leading to a 2nd edition being implemented., Conclusions: Ground up, quality initiatives and collaborative working across organisations can be complex but draws on a wider pool of expertise and can lead to improved parent and staff experience of services. This initiative has improved practice and has application outside the UK., (Copyright © 2021 The Authors. Published by Elsevier Ltd.. All rights reserved.)

Background: Children with cancer are frequently immunocompromised. While children are generally thought to be at less risk of severe SARS-CoV-2 infection than adults, comprehensive population-based evidence for the risk in children with cancer is unavailable. We aimed to produce evidence of the incidence and outcomes from SARS-CoV-2 in children with cancer attending all hospitals treating this population across the UK., Methods: Retrospective and prospective observational study of all children in the UK under 16 diagnosed with cancer through data collection from all hospitals providing cancer care to this population. Eligible patients tested positive for SARS-CoV-2 on reverse transcription polymerase chain reaction (RT-PCR). The primary end-point was death, discharge or end of active care for COVID-19 for those remaining in hospital., Results: Between 12 March 2020 and 31 July 2020, 54 cases were identified: 15 (28%) were asymptomatic, 34 (63%) had mild infections and 5 (10%) moderate, severe or critical infections. No patients died and only three patients required intensive care support due to COVID-19. Estimated incidence of hospital identified SARS-CoV-2 infection in children with cancer under 16 was 3%., Conclusions: Children with cancer with SARS-CoV-2 infection do not appear at increased risk of severe infection compared to the general paediatric population. This is reassuring and supports the continued delivery of standard treatment.

The prognostic value of minimal residual disease (MRD) in Philadelphia-chromosome-positive (Ph+) childhood acute lymphoblastic leukemia (ALL) treated with tyrosine kinase inhibitors is not fully established. We detected MRD by real-time quantitative polymerase chain reaction (RQ-PCR) of rearranged immunoglobulin/T-cell receptor genes (IG/TR) and/or BCR/ABL1 fusion transcript to investigate its predictive value in patients receiving Berlin-Frankfurt-Münster (BFM) high-risk (HR) therapy and post-induction intermittent imatinib (the European intergroup study of post-induction treatment of Philadelphia-chromosome-positive acute lymphoblastic leukemia (EsPhALL) study). MRD was monitored after induction (time point (TP)1), consolidation Phase IB (TP2), HR Blocks, reinductions, and at the end of therapy. MRD negativity progressively increased over time, both by IG/TR and BCR/ABL1. Of 90 patients with IG/TR MRD at TP1, nine were negative and none relapsed, while 11 with MRD<5×10 -4 and 70 with MRD≥5×10 -4 had a comparable 5-year cumulative incidence of relapse of 36.4 (15.4) and 35.2 (5.9), respectively. Patients who achieved MRD negativity at TP2 had a low relapse risk (5-yr cumulative incidence of relapse (CIR)=14.3[9.8]), whereas those who attained MRD negativity at a later date showed higher CIR, comparable to patients with positive MRD at any level. BCR/ABL1 MRD negative patients at TP1 had a relapse risk similar to those who were IG/TR MRD negative (1/8 relapses). The overall concordance between the two methods is 69%, with significantly higher positivity by BCR/ABL1. In conclusion, MRD monitoring by both methods may be functional not only for measuring response but also for guiding biological studies aimed at investigating causes for discrepancies, although from our data IG/TR MRD monitoring appears to be more reliable. Early MRD negativity is highly predictive of favorable outcome. The earlier MRD negativity is achieved, the better the prognosis., (Copyright© 2018 Ferrata Storti Foundation.)

The prognostic significance of early response to treatment has not been reported in relapsed pediatric acute myeloid leukemia. In order to identify an early and easily applicable prognostic factor allowing subsequent treatment modifications, we assessed leukemic blast counts in the bone marrow by morphology on days 15 and 28 after first reinduction in 338 patients of the international Relapsed-AML2001/01 trial. Both day 15 and day 28 status was classified as good (≤20% leukemic blasts) in 77% of patients. The correlation between day 15 and 28 blast percentages was significant, but not strong (Spearman correlation coefficient = 0.49, P<0.001). Survival probability decreased in a stepwise fashion along with rising blast counts at day 28. Patients with bone marrow blast counts at this time-point of ≤5%, 6-10%, 11-20% and >20% had 4-year probabilities of survival of 52%±3% versus 36%±10% versus 21%±9% versus 14%±4%, respectively, P<0.0001; this trend was not seen for day 15 results. Multivariate analysis showed that early treatment response at day 28 had the strongest prognostic significance, superseding even time to relapse (< or ≥12 months). In conclusion, an early response to treatment, measured on day 28, is a strong and independent prognostic factor potentially useful for treatment stratification in pediatric relapsed acute myeloid leukemia. This study was registered with ISRCTN code: 94206677., (Copyright© Ferrata Storti Foundation.)

Background: The highest reported metastatic response rate to induction chemotherapy in patients with neuroblastoma has been achieved by Kushner et al. [Kushner et al.: J Clin Oncol 12:2607-2613,1994; Cheung et al.: Med Pediatr Oncol 36: 227-230, 2001; Kushner et al.: J Clin Oncol 22:4888-4892, 2004] using their N6 and subsequently N7 protocols. This N7 induction was adopted by UKCCSG for new patients in 1999., Methods: Forty-seven children with metastatic neuroblastoma were recruited between 1999 and 2002. They received the N7 intensive chemotherapy protocol, after full staging including evaluation by I123mIBG imaging where possible., Results: Thirty patients with positive mIBG scans were evaluable for response, and complete resolution of metastatic disease was obtained in 16 (53.3%). Fourteen patients without positive mIBG scans were evaluated for response according to bone marrow and bone scan data and 11 (78.6%) cleared metastatic disease. The toxicity of this induction therapy was similar to that seen in previous UK protocols, although 14 patients had Brock grade 3 or 4 ototoxicity. Thirty-three patients proceeded to high-dose therapy with no unanticipated toxicities. For the whole group of 44 evaluable patients, the 3-year event-free survival (EFS) and overall survival (OS) were 38.3 and 46.7%, respectively., Conclusions: Although feasible in terms of delivery, when used in the UKCCSG centers, this protocol achieved a much lower response rate than in the previously published series in the US [Kushner et al.: J Clin Oncol 12:2607-2613,1994; Cheung et al.: Med Pediatr Oncol 36: 227-230, 2001; Kushner et al.: J Clin Oncol 22:4888-4892, 2004]., (Copyright (c) 2007 Wiley-Liss, Inc.)