Leeds Teaching Hospitals NHS Trust
The Children’s Neuromuscular service in Leeds was established in 1992 and provides support to the 1 million or so children in what was the old ‘Yorkshire Health Authority’. Over the years, the service has grown and developed considerably. We offer full diagnostic facilities including multidisciplinary clinical assessment, muscle biopsy and histopathology, neurophysiology (electromyography and nerve conduction), muscle and cardiac MRI (magnetic resonance imaging), genetic evaluation and testing.
The NM service includes 4 consultant paediatric neurologists, 2 specialist care advisors, 3 specialist neuromuscular physiotherapist, a specialist NM occupational therapist and 2 specialist NM nurses. In-patient care is supported by senior therapists from the Paediatric Neurosciences and Respiratory service. There is a neonatal and paediatric ICU on site, in addition to paediatric cardiologists, hepatologists, orthopaedics including spinal surgeons and the long term ventilation service. The team have access to support from SALT, dieticians, psychologists based at the Leeds Children’s Hospital. As the Children’s Hospital is co-located with adult services, the full range of adult services is also readily accessible.
Paediatric Neurology
The service carries a caseload of 400+ children with neuromuscular disorders and undertake 3-4 NM clinics/week. Our caseload includes a high percentage of families from a South Asian background, with a high prevalence of NM disorders, particularly those inherited in an autosomal recessive manner as a result of consanguineous marriage. These conditions are often more challenging to diagnose and manage and require greater resources from the clinical team. We work closely with the regional neuroscience network and Yorkshire and Humber NM forum to streamline referral pathways and ensure consistent management across our network.
The Leeds NM team has always been actively involved in the UK Neuromuscular Consortium, contributing to national audits: NorthStar and SMA REACH (iSMAC) for which Dr Childs is a steering group member, developing guidelines and protocols, working to support service developments, and access to novel interventions/treatments in Yorkshire and across the UK.
The NM team in Leeds have been recognised by MDUK as a “Centre of Excellence” in the management of Paediatric NM disorders in every audit cycle, and in 2024 was recognised as a centre of excellence for clinical care and research.
Since becoming a Hub site in 2020, we have continued to expand our research portfolio, using commercial income to appoint and secure dedicated NM research staff to support a wide range of clinical trials in DMD.
Experience of running trials
Since participating in the FOR DMD study, we have been a site for 15 trials in DMD and 4 in Spinal SMA and are in set up for a further 4 commercial studies.
We have benefited from charitable funding and have used this to ‘pump -prime’ developing research portfolio we have secured additional funding for 2 specialist NM nurses, 0.8 WTE specialist NM physiotherapist, 3 clinical research fellow, a research nurse, a NM well-being practitioner, 2 NM coordinators and most recently a band 6 NM trial coordinator.
All these posts, with the exception of the well-being post, which is being negotiated at present, have been adopted as substantive posts by Leeds TH in recognition of the additional benefit to the patient community and LTHT. This has enabled us to expand the range of clinical trials we can deliver, ensured effective retention and recruitment and enhanced the range of trials and number of patients we can consider.
We have excellent support from our Children’s research faculty and trust R+D department. Trials are conducted in the dediciated clinical trials facility, co located in the with all the children’s hospital facilities . Children requiring 24 hr observation are admitted to the neurosciences ward enabling us to deliver more intense phase 1 studies.
We have access to muscle and cardiac MR for clinical trials. We are ‘a surgical site’ for studies and can undertake muscle biopsy and IV port insertion. Indeed, we have supported other centres who cannot perform muscle biopsies at their site.
Leeds TH has expertise in delivering ATIMP and gene therapy trials in a number of conditions. Effective SOPs are in place within the trust and research pharmacy and there is an active programme for training staff engaged in these studies. All relevant paediatric specialities to support a child/young person following gene therapy are on site at the Leeds General Infirmary.
We are also conducting non commercial studies, leading a study with the Northstar network to look at the impact of deprivation and ethnicity on outcomes in DMD across the UK and a regional study looking at the application of the DMD QoL in clinical practice.
Other relevant projects
We also work closely with MDUK who continue to support the Yorkshire and Humber Muscle forum following the ‘Bridging the Gap’ project and the Awaaz forum supporting South Asian families
We have collaborated on a number of publications with our colleagues in the Clinical genetics department, defining the clinical and genetic basis of novel neuromuscular disorders, i.e. MICU1 and EMARD, and with colleagues in other centres to expand the clinical phenotyping of rare NM conditions.
We have always worked closely with our colleagues in Palliative Care, particularly the Research team at Martin House Children’s Hospice, with whom we have undertaken a number of projects looking at the role of palliative care in supporting families living with NM disorders.
We are actively involved in the DMD Hub and worked with the team in Newcastle to develop the pilot for the central recruitment database (CRD). This has been a really positive development for families and sites and we look forward to continuing to be engaged in its development as the project moves into a definitive stage.
Through the CRD we have been able to recruit and retain patients from other UK sites expanding access to trials for families in a transparent and equitable manner.
Dr Martos is a member of the national MDT that assesses and advises on gene therapy in SMA.
Contact Information
F Floor
Martin Wing
Leeds General Infirmary
Leeds
LS1 3EX
Fiona Halstead, fiona.halstead@nihr.ac.uk
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