- Childhood Cancer Survivors' Quality of Life
- Ocular Oncology and Treatments
- Respiratory viral infections research
- COVID-19 and healthcare impacts
- Respiratory Support and Mechanisms
- Cancer Genomics and Diagnostics
- Cancer-related Molecular Pathways
- Histiocytic Disorders and Treatments
- Neonatal Respiratory Health Research
- Statistical Methods in Clinical Trials
- Pharmaceutical studies and practices
- Teratomas and Epidermoid Cysts
- Metastasis and carcinoma case studies
- Vascular Malformations and Hemangiomas
- Healthcare cost, quality, practices
- Ocular Infections and Treatments
- Renal and related cancers
- Viral-associated cancers and disorders
- Gastrointestinal Tumor Research and Treatment
- Immunotherapy and Immune Responses
- Optimal Experimental Design Methods
- Meta-analysis and systematic reviews
- Effects of Radiation Exposure
- Nutrition, Genetics, and Disease
- Cutaneous lymphoproliferative disorders research
Birmingham Children's Hospital
2017-2025
Birmingham Women’s and Children’s NHS Foundation Trust
2019-2024
Cancer Research UK Clinical Trials Unit
2020-2021
University of Birmingham
2020-2021
NIHR Birmingham Biomedical Research Centre
2021
NIHR Clinical Research Network
2017
Heartlands Hospital
2014
Heart of England NHS Foundation Trust
2012
Good Hope Hospital
2011
Abstract Background Children with cancer are frequently immunocompromised. While children generally thought to be at less risk of severe SARS-CoV-2 infection than adults, comprehensive population-based evidence for the in is unavailable. We aimed produce incidence and outcomes from attending all hospitals treating this population across UK. Methods Retrospective prospective observational study UK under 16 diagnosed through data collection providing care population. Eligible patients tested...
The identification of somatic RB1 variation is crucial to confirm the heritability retinoblastoma. We and others have previously shown that, when tumour DNA unavailable, cell-free (cfDNA) derived from aqueous humour (AH) can be used identify pathogenic variation. Here we report variant detection, as well cfDNA concentration in an extended cohort 75 AH samples 68 patients. show highly variable significantly correlated with collection point AH. Cell-free concentrations above 5 pg/µL enabled...
Clinical trials can be separated into phase I (dose finding and safety), II (activity or early efficacy), III (efficacy compared with current standard of care) occasionally IV (postmarketing studies). A new compound would usually have to go through I–III studies sequentially all the financial regulatory hurdles this poses. recent study has estimated that only 13.8% compounds tested will successful in achieving a marketing license.1 Adaptive designs are an extensive class flexible tools...
Children with cancer are not at increased risk of severe SARS-CoV-2 infection; however, adults haematological malignancies have infections compared non-haematological malignancies.
e22016 Background: Novel anticancer therapies are regularly prescribed outside their marketing authorization or through compassionate use programs to children, adolescents and young adults. Until recently, safety efficacy data on those prescriptions were neither prospectively nor systematically collected pharmacovigilance declarations largely underreported. Methods: SACHA-INTERNATIONAL (NCT04477681) is a prospective international non-interventional study developed within the Innovative...
Abstract Most children with high-risk Langerhans cell histiocytosis (LCH) have BRAFV600E mutation. alleles are detectable in myeloid mononuclear cells at diagnosis but it is not known if the cellular distribution of mutation evolves over time. Here, profiles 16 patients disease were analyzed. Two received conventional salvage chemotherapy, 4 on inhibitors tracked intervals 3 to 6 years, and 10 patients, also given inhibitors, analyzed more than 2 years after diagnosis. In contrast responding...
Abstract Background Retinoblastoma is the most common intra-ocular malignancy in children and frequently presents very young patients who commonly require intravenous carboplatin. Delivering this challenging due to a lack of uniform dosing recommendations, rapid changes physiological function risk side-effects. Methods We conducted retrospective review neonates infants UK with retinoblastoma, have undergone carboplatin therapeutic drug monitoring (TDM). report on pharmacokinetic, treatment...
Early phase trials provide crucial information about new medicines that allow them to be taken forward into larger confirmatory studies. Paediatric early studies are becoming more common, particularly in the era of precision therapy. There almost 600 active paediatric I/II listed on clinicaltrials.gov. Conventionally, dose-escalation use rule-based designs such as 3+3 to guide dose decisions. A trial is considered have a design if predefined rules used decisions escalate, continue or...
Abstract Juvenile Xanthogranuloma (JXG), the commonest type of non-Langerhans cell histiocytosis, is regarded as a benign self-limiting condition infancy, characterised by asymptomatic yellow brown papulonodular lesions, usually confined to skin but occasionally affecting eyes or internal organs. Skin lesions characteristically regress spontaneously within 3-6 years. We describe an unusually aggressive cutaneous presentation requiring input from paediatric oncology. A 6 year old boy...
Intracranial germ cell tumors are a heterogeneous group of tumors, broadly classified into germinomatous, nongerminomatous, or teratoma subtypes. Treatment has evolved over recent decades to include multimodal therapy combining surgery, radiotherapy, and chemotherapy. Although the majority intracranial germs treated successfully, management can be fraught with complexities present significant clinical challenges. Bifocal disease is well described, but rare, therefore its behavior not...
e21509 Background: Participation of children with advanced solid cancers in phase I trials raises ethical and logistic dilemmas. Life-expectancy beyond 8-12 weeks is a common inclusion criterion, but it can be difficult to gauge. This multicentric European study assessed the mortality survival rates pediatric trials. Methods: Retrospective patients aged < 18 years tumors enrolled ITCC centres between 2015-2017. Outcome variables were described prognostic factors analysed. Results: 256...
10020 Background: Phase I/II trials play a key role for children with relapsed/refractory tumors. Life expectancy beyond 8-12 weeks is common eligibility criterion. However, there are no objective means to determine life trial candidates. A better understanding of the risk factors associated early mortality on could maximize efficiency such trials, whilst ensuring ethical aspects recruitment. Methods: Patients solid tumors or lymphomas aged < 18 years and participating in interventional...
<h3>Aim</h3> The 2012 RCPCH position statement described vitamin D deficiency as a significant health risk<sup>1</sup>. 2016 Scientific Advisory Committee on Nutrition (SACN) report now recommends starting supplements at birth until 5 years of age in all children living the UK. A citywide intervention 2013 reduced incidence symptomatic deficiency<sup>2</sup>. regional survey aimed to explore parental awareness Vitamin supplementation and their mothers, compare large urban area with other...
A 6 month old girl was referred to the paediatric assessment unit with a haemoglobin of 12 g/L. She had no history weight loss, fever or night sweats, but three week black stools. pale, 1 × cm blue lesion on her upper gum and apparent hepatomegaly 4–5cm. petechiae, lymphadenopathy splenomegaly. The FBC showed microcytic anaemia very low ferritin marked thrombocytopenia platelet count 7. blood transfusion arranged. Her total differential white cell counts were normal. renal liver function...