Selective dorsal rhizotomy in non-ambulant children with cerebral palsy: a multi-center prospective study.

PURPOSE: Assess the effects of selective dorsal rhizotomy (SDR) on motor function and quality of life in children with a Gross Motor Function Classification System (GMFCS) level of IV or V (non-ambulatory). METHODS: This is a prospective, observational study in three tertiary neurosurgery units in England, UK, performing SDR on children aged 3-18 with spastic diplegic cerebral palsy, and a GMFCS level of IV or V, between 2012 and 2019. The primary outcome measure was the change in the 66-item Gross Motor Function Measure (GMFM-66) from baseline to 24 months after SDR, using a linear mixed effects model. Secondary outcomes included spasticity, bladder function, quality of life, and pain scores. RESULTS: Between 2012 and 2019, 144 children who satisfied these inclusion criteria underwent SDR. The mean age was 8.2 years. Fifty-two percent were female. Mean GMFM-66 score was available in 77 patients (53.5%) and in 39 patients (27.1%) at 24 months after SDR. The mean increase between baseline and 24 months post-SDR was 2.4 units (95% CI 1.7-3.1, p < 0.001, annual change 1.2 units). Of the 67 patients with a GMFM-66 measurement available, a documented increase in gross motor function was seen in 77.6% (n = 52). Of 101 patients with spasticity data available, mean Ashworth scale decreased after surgery (2.74 to 0.30). Of patients' pain scores, 60.7% (n = 34) improved, and 96.4% (n = 56) of patients' pain scores remained the same or improved. Bladder function improved in 30.9% of patients. CONCLUSIONS: SDR improved gross motor function and reduced pain in most patients at 24 months after surgery, although the improvement is less pronounced than in children with GMFCS levels II and III. SDR should be considered in non-ambulant patients.

Intrathecal baclofen pumps in the management of hypertonia in childhood: a UK and Ireland wide survey.

BACKGROUND: Intrathecal baclofen (ITB) is a useful treatment for hypertonia where non-invasive treatments have been ineffective or poorly tolerated. There is an absence of national guidance on selection criteria and a lack of literature regarding patient characteristics and treatment details for children and young people (CYP) receiving ITB therapy in the UK and Ireland. We aimed to gather patient and treatment characteristics for CYP receiving ITB in the UK and Ireland. METHODS: An electronic survey was sent to all paediatric ITB centres in the UK and Ireland. Anonymised data were returned between December 2019 and April 2020. CYP >16 years and those awaiting ITB pump removal were excluded from the dataset. RESULTS: 176 CYP were identified as receiving ITB therapy across the UK and Ireland. The majority of CYP with ITB pumps were non-ambulant (93%) with a diagnosis of cerebral palsy (79%). Median age of ITB insertion was 9 years; median current age was 14 years. 79% of CYP had significant spasticity, 55% had significant dystonia. The most commonly used ITB dosing modes were continuous (73%) and flexible (23%). CONCLUSIONS: ITB pumps were most frequently used for non-ambulant CYP with cerebral palsy and existence of spasticity and/or dystonia in the UK and Ireland. Most CYP were receiving a continuous dose of ITB. There is significant variation in the number of paediatric ITB pumps across UK and Ireland. There is a need for development of nationally accepted paediatric referral criteria and clinical standards for ITB use.

Incidence and prognostic factors of ataxia in children with posterior fossa tumors.

BACKGROUND: There is minimal literature specific to motor outcomes in children with posterior fossa tumors (PFTs) despite ataxia being a significant problem in this group. This study aims to report children's physical outcomes following management of PFT and determine which factors affect severity of ataxia and functional limitations. METHODS: Forty-two children aged between 5 and 17 and between 1 and 4 years following surgery for PFT were assessed using the Scale for the Assessment and Rating of Ataxia (SARA), the Brief Ataxia Rating Scale (BARS), and the mobility Pediatric Evaluation of Disability Inventory (PEDI) subscale to determine prevalence and severity of ataxia and a measure of physical function. Analysis was undertaken comparing impact of tumor location, tumor histology, adjuvant treatment, age at diagnosis, presence of preoperative ataxia, and presence of cerebellar mutism syndrome (CMS) on ataxia and physical function scores. RESULTS: Seventy-one percent of children demonstrated a SARA and BARS score greater than 2. A total of 48% of children had a PEDI-m score greater than 90. There was no correlation between age at diagnosis or preoperative ataxia and assessment scores. There was a significant difference in SARA/BARS and PEDI-mobility scores depending on tumor histology, tumor location, and presence of CMS. CONCLUSIONS: A high proportion of children (>1 year) following surgery for PFT continue to present with ataxia. Higher ataxia and lower physical function scores were demonstrated in children with medulloblastoma and midline tumors and those diagnosed with CMS. The high prevalence of ataxia demonstrates the need for further research regarding rehabilitation management in this population.

Selective dorsal rhizotomy in ambulant children with cerebral palsy: an observational cohort study.

BACKGROUND: Selective dorsal rhizotomy (SDR) is an irreversible surgical procedure involving the division of selected sensory nerve roots, followed by intensive physiotherapy. The aim is to improve function and quality of life in children with cerebral palsy and a Gross Motor Function Classification System (GMFCS) level of II or III (walks with or without assistive devices, respectively). We assessed gross motor function before and after SDR and postoperative quality of life in a study commissioned by NHS England. METHODS: We did a prospective observational study in five hospitals in England who were commissioned to perform SDR on children aged 3-9 years with spastic diplegic cerebral palsy. The primary outcome was score changes in the 66-item Gross Motor Function Measure (GMFM-66) and seven domains of the Cerebral Palsy Quality of Life Questionnaire ([CP-QoL] social wellbeing and acceptance, feelings about functioning, participation and physical health, emotional wellbeing and self-esteem, access to services, family health, and pain and impact of disability) from before to 24 months after SDR. FINDINGS: From Sept 4, 2014, to March 21, 2016, 137 children underwent SDR. The mean age was 6·0 years (SD 1·8). The mean GMFM-66 score increased after SDR with an annual change of 3·2 units (95% CI 2·9 to 3·5, n=137). Of the seven CP-QoL domains, five showed significant improvements over time: feelings about functioning mean annual change 3·0 units (95% CI 2·0 to 4·0, n=133), participation and physical health 3·9 units (2·5 to 5·3, n=133), emotional wellbeing and self-esteem 1·3 units (0·2 to 2·3, n=133), family health 2·0 units (0·7 to 3·3, n=132), and pain and impact of disability -2·5 units (-3·9 to -1·2, n=133). 17 adverse events were reported in 15 children, of which none were severe and 15 (88%) resolved. INTERPRETATION: SDR improved function and quality of life in the 24 months after surgery in children with cerebral palsy classified as GMFCS levels II and III. On the basis of these findings, an interim national policy decision was made that SDR would be funded for eligible children in England from 2018. FUNDING: National Institute for Health and Care Excellence, National Institute for Health Research Biomedical Research Centre, NHS England.

Indwelling intrathecal catheter with subcutaneous abdominal reservoir: a viable baclofen delivery system in severely cachectic patients.

Intrathecal baclofen (ITB) is a reversible treatment that reduces muscle tone to ameliorate spasticity and dystonia in patients with cerebral palsy (CP). The resulting decrease in energy expenditure allows patients to gain much-needed weight, albeit temporarily. Modern techniques require sufficient abdominal musculature and subcutaneous fat to permit the implantation of an indwelling pump. In patients with extremely low muscle bulk, visceral pumps may be impractical or impossible, with increased risks of dehiscence and infection. The authors describe a variation of the classical procedure in a young patient with severe cachexia. A 10-year-old boy with spastic-dystonic quadriplegic CP was admitted to the neuromedical unit. Numerous drug trials had failed, and surgical intervention was deemed necessary but was complicated by his cachectic body habitus. The authors inserted a lumbar intrathecal catheter and subcutaneously tunneled it to the anterolateral abdomen, where it was connected to a subcutaneous injection port. Baclofen was continuously infused into the subcutaneous port using a noncoring needle connected to an external pump. The needle and line were changed every 5 days to minimize the risk of sepsis. Although other techniques, such as intraventricular baclofen delivery, have been described, these are largely dependent upon sufficient musculature to support a visceral pump. A subcutaneous injection port system represents an alternative approach that reduces the risk of sepsis and may be better tolerated in cachectic patients.