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"Aquilina, Kristian"
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Mixed-methods study exploring medium to longer-term outcomes following selective dorsal rhizotomy in ambulatory children with cerebral palsy at a tertiary hospital in the UK: MOSAiC study protocol
by
Gimeno, Hortensia
,
Main, Eleanor
,
Aquilina, Kristian
in
Botulinum toxin
,
Cerebral palsy
,
Child
2025
IntroductionSelective dorsal rhizotomy (SDR) is one of the treatment options available for spasticity management in ambulatory children and young people with cerebral palsy (CYPwCP). Although improvements in gross motor function one to two years after surgery have been established, evidence of longer-term benefit requires further investigation. Given the irreversible nature of SDR and the increased rehabilitation commitments required from families and clinicians, providing evidence of longer-term benefits is essential to support their decision-making. This study aims to investigate medium (3–5 years) and long-term (6–10 years) SDR outcomes in ambulatory children with CP and how SDR affects families’ lives over time.Methods and analysisThis is a convergent parallel mixed-methods study using the International Classification of Functioning, Disability and Health as a theoretical framework. The study aims to recruit 90 CYPwCP participants, who had SDR at a tertiary hospital in the UK when aged between 3 and 14 years. Participants (parents and CYPwCP) will be invited to complete an online survey and attend the hospital for one follow-up visit 3 or more years after SDR. Comparisons will be made with existing data on objective measures and parent-reported outcomes collected in clinical practice at baseline, 6, 12 and 24 months to understand the trajectory of changes. Semistructured interviews will be conducted with 18–20 parents/carers and 25–30 CYPwCP to understand their perspectives on the outcomes of SDR compared with their prior expectations. The Framework Method will be used to analyse qualitative data both inductively and deductively. Qualitative and quantitative study data will be integrated using joint displays.Ethics and disseminationEthical approval has been obtained through the Coventry and Warwick Research and Ethics Committee (24/WM/0078). Findings will be shared through international conferences, peer-reviewed journals, social media and dissemination events for families and CYP.Trial registration numberNCT06518889.
Journal Article
An international study presenting a federated learning AI platform for pediatric brain tumors
by
Grant, Gerald A.
,
Kuwabara, Michael
,
Szymanski, Kathryn A.
in
59/57
,
631/114/1305
,
631/67/1922
2024
While multiple factors impact disease, artificial intelligence (AI) studies in medicine often use small, non-diverse patient cohorts due to data sharing and privacy issues. Federated learning (FL) has emerged as a solution, enabling training across hospitals without direct data sharing. Here, we present FL-PedBrain, an FL platform for pediatric posterior fossa brain tumors, and evaluate its performance on a diverse, realistic, multi-center cohort. Pediatric brain tumors were targeted due to the scarcity of such datasets, even in tertiary care hospitals. Our platform orchestrates federated training for joint tumor classification and segmentation across 19 international sites. FL-PedBrain exhibits less than a 1.5% decrease in classification and a 3% reduction in segmentation performance compared to centralized data training. FL boosts segmentation performance by 20 to 30% on three external, out-of-network sites. Finally, we explore the sources of data heterogeneity and examine FL robustness in real-world scenarios with data imbalances.
Federated learning (FL) has emerged as a potential solution to train machine learning models in multiple clinical datasets while preserving patient privacy. Here, the authors develop an MRI-based FL platform for pediatric posterior fossa brain tumors—FL-PedBrain—and evaluate it on a diverse multi-center cohort.
Journal Article
Surgical approaches in pediatric neuro-oncology
2019
Tumors of the central nervous system comprise nearly a quarter of all childhood cancers and are the most frequent solid tumor in the pediatric population. The most common location is in the posterior fossa, but tumors can occur anywhere intracranially. The spectrum of lesions encountered varies, from being completely benign and requiring surveillance alone to being highly malignant and requiring aggressive treatment in the form of surgery and adjuvant therapy. The extent of resection plays a crucial role in the oncological outcome of many of these tumors. A variety of surgical approaches are available for the spectrum of lesions encountered. This review focuses on summarizing the location, types, and neurosurgical management strategies for pediatric brain intracranial brain tumors. Here, we discuss neurosurgical approaches for a variety of brain tumors and regions, including the management of tumors of the posterior fossa, brainstem, pineal region, intraventricular region, sellar and suprasellar regions, optic pathway and hypothalamus, and supratentorial hemispheres.
Journal Article
Correction: Surgical Site Infection after Craniotomy in Neuro-Oncology (SINO): A protocol for an international prospective multicentre service evaluation across the United Kingdom and Ireland
2025
[This corrects the article DOI: 10.1371/journal.pone.0316237.].
Journal Article
Surgical Site Infection after Craniotomy in Neuro-Oncology (SINO): A protocol for an international prospective multicentre service evaluation across the United Kingdom and Ireland
2025
Given its proximity to the central nervous system, surgical site infections (SSIs) after craniotomy (SSI-CRAN) represent a serious adverse event. SSI-CRAN are associated with substantial patient morbidity and mortality. Despite the recognition of SSI in other surgical fields, there is a paucity of evidence in the neurosurgical literature devoted to skin closure, specifically in patients with brain tumors. The primary objective of this service evaluation is to ascertain the incidence and the risk factors associated with SSI-CRAN. The secondary objectives would be a) to ascertain the incidence of SSI-CRAN in sutured versus stapled wounds, after accounting for patient, surgical and hospital confounders of SSI-CRAN and b) to determine the percentage of patients with gliomas that begin adjuvant oncological treatment in patients with infection versus those without infection.
Surgical Site Infection after Craniotomy in Neuro-Oncology (SINO) is a international prospective multicentre service evaluation that will include patients with an intracranial neoplasm, both primary and secondary neoplasms, treated with cranial surgery (including biopsy). Consecutive paediatric (<18 years) and adult (≥18 years) patients diagnosed with a brain tumour, undergoing cranial surgery between 1st October 2024 and 1st December 2024 will be included. Prospective data will be collected with a follow-up of 90 days.
Journal Article
Endoscopic lavage after intraventricular haemorrhage in neonates in the UK (ENLIVEN-UK): study protocol for a national randomised-controlled trial
2025
Background
Intraventricular haemorrhage (IVH) is a common and severe complication of preterm birth, affecting nearly 500 neonates annually in the UK. Over 50% of infants with IVH develop post-haemorrhagic ventricular dilatation (PHVD), which is associated with significant long-term neurodevelopmental impairment. Current treatment strategies involve the use of temporary CSF diversion, through options such as ventricular access devices (VADs) or ventricular subgaleal shunts (VSGS).
Neuroendoscopic lavage (NEL) is an emerging technique that aims to directly reduce the load of intraventricular blood and its breakdown products, potentially reducing the risk of secondary brain injury. The ENLIVEN-UK trial aims to assess whether the addition of NEL to standard temporising device placement improves neurodevelopmental outcomes at 2 years of corrected age compared to temporising device placement alone.
Methods
ENLIVEN-UK is a national, multicentre, parallel-group, assessor-blinded, superiority randomised controlled trial (RCT) that aims to enrol 100 preterm infants with severe IVH and PHVD across UK paediatric neurosurgical centres. Infants will be randomised in a 1:1 ratio to receive either standard temporising device placement or NEL in addition to temporising device placement. Randomisation will be performed using a secure online system (Sealed Envelope), with outcome assessors and statisticians blinded to treatment allocation.
The primary outcome measure will be cognitive quotient (CQ) at 2 years of corrected age, assessed using the Bayley Scales of Infant and Toddler Development (4th edition, Bayley-IV). Secondary outcomes will include motor and language development, the requirement for permanent CSF diversion with a ventriculoperitoneal (VP) shunt, surgical complications, health-related quality of life (EQ-5D-5L, TAPQOL), and healthcare costs.
Discussion
This study aims to provide level 1 evidence regarding the efficacy and safety of NEL in preterm infants with IVH and PHVD. If successful, this trial has the potential to change the standard of care and improve long-term neurodevelopmental outcomes in this cohort of patients.
Trial registration
ISRCTN Trial Registration: ISRCTN14018410.
Journal Article
The COMBAT Project: study protocol for the development of a core outcome set for morbidity following surgery in paediatric brain tumour patients
by
Trichinopoly Krishna, Sandhya
,
Jenkinson, Michael D.
,
Hartley, Helen
in
Biomedicine
,
Biopsy
,
Brain cancer
2025
Background
Central nervous system tumours affecting the brain and spine are the most common solid tumour site in the paediatric population and the most common causes of cancer death in children and young people. They are associated with high morbidity both from the tumour and the interventions used to treat them. Postoperative morbidity reporting following surgery for paediatric brain tumours is poor. This is due to variability of outcomes measured and reported and the lack of a common language when reporting adverse outcomes. One solution is to develop a core outcome set which will stipulate the minimum postoperative outcomes that should be reported. The COMBAT (Core Postoperative Morbidity Set for Paediatric Brain Tumours) Project will develop a core set of adverse outcomes that can be applied to paediatric brain tumour patients undergoing surgery.
Methods and analysis
This protocol has been developed using the COS-STAD (Core Outcome Set-Standards for Development) recommendations and the COS-STAP (Core Outcome Set-STAndardised Protocol Items) statement. A systematic review will identify adverse outcomes reported in the literature and how they are measured. Outcomes of importance to patients and their carers will be identified from semi-structured qualitative interviews with patients and their carers from Alder Hey Children’s Hospital, Liverpool, UK. Consensus on the most important harms will be sought using a two-round eDelphi survey completed by national and international participants including health professionals, researchers, patients and their carers. Results of the eDelphi survey will be assessed against a pre-defined definition of consensus and discussed at an international consensus meeting attended by participants of the eDelphi survey.
Discussion
There is a clear need for a common language to harmonise measurement and reporting of morbidity following surgery for paediatric brain tumour patients. This project will define postoperative adverse outcomes that are of critical importance to key stakeholders. It will standardise surgical morbidity outcome measurement and reporting in both research studies and routine clinical practice, enabling comparison across different trials, studies and clinical services. It will lay the groundwork for future research in paediatric brain tumour surgical morbidity.
Study registration
This study is registered with the COMET database as Study 1968 (
https://www.comet-initiative.org/Studies/Details/1968
), registration date: 26/10/2021.
Journal Article
Spectrum of neuroimaging findings post-proton beam therapy in a large pediatric cohort
2021
Purpose
Proton beam therapy (PBT) is now well established for the treatment of certain pediatric brain tumors. The intrinsic properties of PBT are known to reduce long-term negative effects of photon radiotherapy (PRT). To better understand the intracranial effects of PBT, we analyzed the longitudinal imaging changes in a cohort of children with brain tumors treated by PBT with clinical and radiotherapy dose correlations.
Materials and methods
Retrospective imaging review of 46 patients from our hospital with brain tumors treated by PBT. The imaging findings were correlated with clinical and dose parameters.
Results
Imaging changes were assessed by reviewing serial magnetic resonance imaging (MRI) scans following PBT over a follow-up period ranging from 1 month to 7 years. Imaging changes were observed in 23 patients undergoing PBT and categorized as pseudoprogression (10 patients, 43%), white matter changes (6 patients, 23%), parenchymal atrophy (6 patients, 23%), and cerebral large vessel arteriopathy (5 patients, 25%). Three patients had more than one type of imaging change. Clinical symptoms attributable to PBT were observed in 13 (28%) patients.
Conclusion
In accordance with published literature, we found evidence of varied intracranial imaging changes in pediatric brain tumor patients treated with PBT. There was a higher incidence (10%) of large vessel cerebral arteriopathy in our cohort than previously described in the literature. Twenty-eight percent of patients had clinical sequelae as a result of these changes, particularly in the large vessel arteriopathy subgroup, arguing the need for angiographic and perfusion surveillance to pre-empt any morbidities and offer potential neuro-protection.
Journal Article
MRI-Guided Focused Ultrasound for the Treatment of Dystonia: A Narrative Review
by
Natalwala, Ammar
,
Momin, Sheikh Muktadir Bin
,
Kalia, Suneil
in
Ablation
,
Cerebral palsy
,
Dysarthria
2024
Contemporary surgical management of dystonia includes neuromodulation via deep brain stimulation (DBS) or ablative techniques such as radiofrequency (RF) ablation. MRI-guided focused ultrasound (MRgFUS) is an emerging modality that uses high-intensity ultrasound to precisely ablate targets in the brain; this is incisionless, potentially avoiding the surgical risks of a burr hole and transcortical tract to reach the anatomical target. There is some evidence of efficacy in essential tremor and Parkinson's disease (PD), but, to date, there is no study aggregating the evidence of MRgFUS in dystonia. In this narrative review, we searched Medline, Embase, CINAHL, EBSCO, and ClinicalTrials.gov for primary studies and clinical trials on MRgFUS in the treatment of dystonia. Data were analyzed concerning dystonia phenotype, reported outcomes, and complications. PD-related dystonia was also included within the scope of the review. Using our search criteria, six articles on the use of MRgFUS in adult dystonia and three articles on the use of FUS in dystonia in PD were included. Four trials on the use of FUS in dystonia were also found on ClinicalTrials.gov, one of which was completed in December 2013. All included studies showed evidence of symptomatic improvement, mostly in focal hand dystonia; improvements were also found in dystonia-associated tremor, cervicobrachial dystonia, and dystonia-associated chronic neuropathic pain as well as PD-related dystonia. Reported complications included transient neurological deficits and persistent arm pain in one study. However, the evidence is limited to level-4 case series at present. MRgFUS is an emerging modality that appears to be safe and effective, particularly in focal hand dystonia, without major adverse effects. However, the quality of evidence is low at present, and long-term outcomes are unknown. High-quality prospective studies comparing MRgFUS to other surgical techniques will be useful in determining its role in the management of dystonia.
Journal Article
Intraventricular haemorrhage and posthaemorrhagic ventricular dilatation: moving beyond CSF diversion
by
Mallucci, Conor
,
Chari, Aswin
,
Aquilina, Kristian
in
Annual Issue Paper
,
Blood clots
,
Blood pressure
2021
Advances in medical care have led to more premature babies surviving the neonatal period. In these babies, germinal matrix haemorrhage (GMH), intraventricular haemorrhage (IVH) and posthaemorrhagic ventricular dilatation (PHVD) are the most important determinants of long-term cognitive and developmental outcomes. In this review, we discuss current neurosurgical management of IVH and PHVD, including the importance of early diagnosis of PHVD, thresholds for intervention, options for early management through the use of temporising measures and subsequent definitive CSF diversion. We also discuss treatment options for the evolving paradigm to manage intraventricular blood and its breakdown products. We review the evidence for techniques such as drainage, irrigation, fibrinolytic therapy (DRIFT) and neuroendoscopic lavage in the context of optimising cognitive, neurodevelopmental and quality of life outcomes in these premature infants.
Journal Article