Safety of sibling cord blood cell infusion for children with cerebral palsy.

Cerebral palsy (CP) is a nonprogressive neurological disorder and the most common physical disability of childhood. There is no cure for CP, but stem cells have the potential to improve brain injury and hence function. This phase 1 clinical trial investigated the safety of the intravenous infusion of full-matched sibling cord blood cells for children with CP aged 1 to 16 years. Preliminary efficacy outcomes were also investigated. Twelve participants received 12/12 HLA-matched sibling cord blood cell infusions. One treatable serious adverse reaction to cryoprotectant was observed, and no adverse reactions occurred beyond 24 h after infusion. Gross motor function measure (GMFM-66) scores did not improve compared with baseline beyond what could be expected from developmental levels, and participants had varied changes in the Quality of Upper Extremity Skills Test (QUEST) and Vineland Adaptive Behavior Scales (VABS-II) scores. In conclusion, matched sibling cord blood cell infusion for children with CP is relatively safe when conducted in an appropriate facility. Australian and New Zealand Clinical Trials Registry (ACTRN12616000403437) and Clinicaltrials.gov (NCT03087110).

Physiotherapy departments in Australian tertiary hospitals regularly participate in and disseminate research results despite a lack of allocated staff: a prospective cross-sectional survey.

OBJECTIVES: To establish the level of research activity in physiotherapy departments of Australian tertiary hospitals. DESIGN: Prospective cross-sectional survey. PARTICIPANTS: Physiotherapy managers from 37 principal referral hospitals and specialist women's and children's hospitals as identified from the Australian Institute of Health and Welfare (2011). MAIN OUTCOME MEASURES: A purpose-designed predominantly open-response questionnaire investigating site demographics, research activity and research support was developed, piloted and administered. RESULTS: Thirty-seven surveys were completed (54% response rate). Median [IQR] respondent equivalent full-time staffing was 23.8 (19-39). Respondents represented a median [IQR] 6.5 (3-20) publication output in the past 2 years. Twelve respondents (32%) reported that staff had completed a doctorate in the past 5 years and 49% of respondents reported no staff had completed higher degrees. A total of 71 grants had been received and 73% of respondents indicated they had no allocated staffing for research activity. The most common indicators of research culture were organization-led research dissemination events and research training (i.e. manager attending research events and celebrating research achievements). CONCLUSIONS: This is the first study to report on research activity in hospital-based Australian physiotherapy departments. Few sites allocate staff to conduct or support research. Despite this, physiotherapy departments regularly publish and present research results. Future studies could investigate how hospital-based physiotherapy departments can optimize research culture and output.

Is physiotherapy effective in the management of child and adolescent conversion disorder? A systematic review.

Child and adolescent conversion disorder has the potential to impart significant burden on health-care services and affect quality of life. Clinically, physiotherapists are involved in conversion disorder management; however, no systematic reviews have examined physiotherapy effectiveness in its management. The aim of this review is to identify the efficacy of physiotherapy management of child and adolescent conversion disorder. A search of multiple databases (Medline, CINAHL, Embase, PsychINFO, PEDro and the Cochrane Library) was completed along with manual searching of relevant reference lists to identify articles including children 0-18 years with a diagnosis of conversion disorder who received physical management. Two independent reviewers screened titles and abstracts using criteria. Data were extracted regarding study characteristics, functional outcome measures, length of stay, physiotherapy service duration and resolution of conversion symptoms. Methodological quality was assessed using a tool designed for observational studies. Twelve observational studies were included. No functional outcome measures were used to assess the effectiveness of the treatment protocols in the case studies. Resolution of symptoms occurred in all but two cases, with conversion symptoms still present at 11 months and at 2 years. Length of stay varied from 3 days to 16 weeks, with similar variation evident in length of physiotherapy service provision (2.5 weeks to 16 weeks). There was limited and poor quality evidence to establish the efficacy of physiotherapy management of child and adolescent conversion disorders. More rigorous study designs with consistent use of reliable, valid and sensitive functional outcome measures are needed in this area.

Systematic review of interventions for low bone mineral density in children with cerebral palsy.

AIM: To systematically review the efficacy of interventions to improve low bone mineral density (LBMD) in children and adolescents with cerebral palsy (CP). METHODS: We performed a systematic search for published randomized, controlled trials (RCTs) and controlled clinical trials (CCTs) of children with CP (aged 0-20 years, all Gross Motor Function Classification System [GMFCS] levels) who received various medical and physical interventions for LBMD compared with no intervention or standard care. Study validity was evaluated by using the Physiotherapy Evidence Database (PEDro) scale. Standardized mean differences (SMDs) were calculated when data were sufficient. RESULTS: Eight of 2034 articles met the inclusion criteria (6 RCTs, 2 CCTs). There were 3 trials of bisphosphonates, one of which (Henderson RC, Lark RK, Kecskemethy HH, Miller F, Harcke HT, Bachrach SJ. J Pediatr. 2002;141[5]:644-651) revealed a large and significant effect on BMD in 1 of 3 sites in the distal femur (SMD: 1.88 [95% confidence interval (CI): 0.52-3.24]). There were 3 trials of weight-bearing through varying approaches, one of which (Caulton JM, Ward KA, Alsop CW, Dunn G, Adams JE, Mughal MZ. Arch Dis Child. 2004;89[2]:131-135) showed a large and significant effect on the lumbar spine when increasing static standing time (SMD: 1.03 [95% CI: 0.21-1.85]). The administration of vitamin D and calcium produced a large, nonsignificant effect on BMD in the lumbar spine (Jekovec-Vrhovsek M, Kocijancic A, Prezelj J. Dev Med Child Neurol. 2000;42[6]:403-405) (SMD: 0.88 [95% CI: -0.07 to 1.84). Growth hormone combined with vitamin D and/or calcium resulted in effects comparable with vitamin D and/or calcium on BMD in the lumbar spine (Ali O, Shim M, Fowler E, et al. J Clin Endocrinol Metab. 2007;92[3]:932-937) (SMD 0 [95% CI: -1.24 to 1.24]). CONCLUSIONS: Important effects on LBMD have been observed in small and diverse cohorts of children with CP. It is unclear whether small sample sizes or variable treatment responses account for nonsignificant findings. Additional large RCTs are needed of both physical and medical approaches.

Does parent report measure performance? A study of the construct validity of the Functional Mobility Scale.

AIM: Parental report is often relied on to measure performance of activities in children with cerebral palsy (CP). This study examined whether the Functional Mobility Scale (FMS) accurately reflects performance of mobility in children with CP. METHOD: Eighteen children with spastic CP (11 males, seven females; mean age 12 y 8 mo, SD 2 y 8 mo, range 8-17 y) were recruited from a special development school. Children were in Gross Motor Function Classification System (GMFCS) levels II (n=5), III (n=4), or IV (n=9), and had quadriplegia (n=9), diplegia (n=7), or hemiplegia (n=2). The children's mobility was observed directly around and outside the home and at school and their mobility methods were recorded. The parent's FMS rating was obtained on the telephone by a physiotherapist. Agreement between direct observation and the FMS rating was examined using quadratic weighted kappa (kappa) statistics. RESULTS: Agreement between direct observation and the FMS was as follows: FMS 5m kappa=0.71, 45%; FMS 50m kappa=0.76, 94%; FMS 500m kappa=0.74, 95%. Differences in the range and number of mobility methods were observed by GMFCS level across environmental settings. INTERPRETATION: Substantial agreement was found between FMS ratings and direct observation, particularly over longer distances, providing evidence of the validity of the FMS as a measure of performance in children with CP.