Is the search for cerebral palsy 'cures' a reasonable and appropriate goal in the 2020s?

In the field of disability research and advocacy, the notion of 'cures' is contentious. Cerebral palsy (CP) is no exception. In this narrative review, we combine perspectives gained during community consultation undertaken for the Australian and New Zealand Cerebral Palsy Strategy, 2020 with those published in the scientific and grey literature to understand whether 'cures for CP' is a reasonable and appropriate goal. We frame these perspectives through the lens of several ethical principles central to the discussion. These include maintaining hope while also being realistic, sensitivity to sharply different viewpoints amongst people with disability and their families, and responding to community priorities, societal attitudes, and identity. Through this exploration of the literature and perspectives, we arrived at a definition of 'cures for CP' that is pluralized and focuses on functional improvement and/or symptom reduction whilst still acknowledging the potential for neural repair/regeneration strategies.

An Ideal Multidisciplinary Cleft Lip and Cleft Palate Care Team.

We emphasize the benefits of a multidisciplinary team approach to the systemic management of patients with cleft lip and cleft palate (CL/CP) and their families. An ideal team offers support to families at each stage of their child's development and adapts to address new issues that are encountered throughout growth. We suggest the addition of a registered dietitian to the initial phase of the care team to ensure the child consumes adequate macronutrient intake, and to maximize growth and development. An ideal cleft palate care team focuses on a comprehensive, collaborative, and family-centered approach to the complex medical and surgical needs of infants with CL/CP as they grow to young adulthood. Although care teams may differ slightly depending on context and location, there are central tenets that make an ideal team. After reviewing CL/CP care team guidelines and recommendations from a variety of different countries, this article reflects what an ideal organization structure and care team composition could be comprised of.

Mapping the interaction between eukaryotic initiation factor 4A (eIF4A) and the inhibitor hippuristanol using carbene footprinting and mass spectrometry.

Protein-ligand interactions are central to protein activity and cell functionality. Improved knowledge of these relationships greatly benefits our understanding of key biological processes and aids in rational drug design towards the treatment of clinically relevant diseases. Carbene footprinting is a recently developed mass spectrometry-based chemical labelling technique that provides valuable information relating to protein-ligand interactions, such as the mapping of binding sites and associated conformational change. Here, we show the application of carbene footprinting to the interaction between eIF4A helicase and a natural product inhibitor, hippuristanol, found in the coral Isis hippuris. Upon addition of hippuristanol we identified reduced carbene labelling (masking) in regions of eIF4A previously implicated in ligand binding. Additionally, we detected hippuristanol-associated increased carbene labelling (unmasking) around the flexible hinge region of eIF4A, indicating ligand-induced conformational change. This work represents further development of the carbene footprinting technique and demonstrates its potential in characterising medicinally relevant protein-ligand interactions.

Consumer involvement in research - parent perceptions of partnership in cerebral palsy research: a qualitative study.

PURPOSE: Identify perceptions of parents and caregivers of children with cerebral palsy about being consumer research partners and identify strategies to inform involvement of parents in cerebral palsy research. MATERIALS AND METHODS: Twenty-two parents in New South Wales and Victoria (Australia) participated in this qualitative study. Seven interviews and three focus groups were completed. Interpretive description guided data analysis. Methodological rigor was enhanced through involving two consumer investigators in the research team, member checking, and multiple researchers completing data analysis and theme generation. RESULTS: Participants identified a range of factors that may influence their involvement in research partner roles. Main topics emerging from the data included "Research Is Better with Parents" and "Parents Benefit from Being Research Partners." A third, "Parents as Research Partners," contained the themes "Flexible Involvement," "Starting Partnerships," and "Building and Sustaining Partnerships." CONCLUSION: This study has provided a rich insight into how parents perceive and describe engaging as research partners. Parent-identified guidance will inform future partnerships aiming to enhance the quality of cerebral palsy research and outcomes for people with cerebral palsy and their families. The involvement of consumer investigators in this study was considered valuable for enhancing the quality and applicability of the research.IMPLICATIONS FOR REHABILITATIONParents believed that parent partnership in research has benefits for the research and for the consumers involved.Parents provided guidance about the importance of starting, building and sustaining relationships in involving parents as research partners.Understanding the parent context, investing in relationships and acknowledgement of, and recognition for, contributions were considered important for building and sustaining effective partnerships.Flexible approaches to supporting parents as research partners was considered necessary for effective partnership.

More than just having fun! Understanding the experience of involvement in physical activity of adolescents living with cerebral palsy.

Purpose: To explore the experiences of involvement of adolescents living with cerebral palsy, and their parents, while participating in physical activity. Understanding involvement in physical activity may be used to guide future participation.Methods: Eight adolescents (mean age 13 years 11 months, SD 1 year 6 months) with cerebral palsy participated in a New Zealand-based high-level mobility programme (HLMP) focused on running skills, twice per week for 12 weeks. The adolescents and 12 parents were interviewed before, after the 12 weeks and 9-months following the HLMP. Guided by interpretative description, 38 interviews were coded, analysed, and interpreted.Results: Four themes were: "Turning up is not enough" ("There's no point being there if you're not involved"); "In it all the way", "Changes on a dime", and "What works for me." Perceptions of involvement varied between adolescents and parents. Being "very involved" related to high levels of focus, concentration, effort; but not always enjoyment.Conclusions: Focusing on enjoyment as the key experience of involvement understates the complexity and dynamic nature of involvement. "Being involved" is not always easy and may not mean the absence of discomfort or effort. Optimising the individuals' involvement continuum during physical activity may be essential to promote lifelong participation.IMPLICATIONS FOR REHABILITATIONAdolescents living with cerebral palsy and their parents have differing perspective of involvement and utilise different strategies to encourage being and staying active.Teaching adolescents living with cerebral palsy about their involvement continuum and optimal level of involvement for each activity, context and environment could promote sustained participation.To ensure adolescents are "being involved" in physical activity, opportunities for engagement, motivation and persistence are important; enjoyment is a possible, but not essential attribute of involvement.Encouraging involvement in physical activity can be a source of family conflict from a young age therefore clinicians have a role as an essential supporter, motivator and educator.

Effects of a 12 week community-based high-level mobility programme on sustained participation in physical activity by adolescents with cerebral palsy: a single subject research design study.

PURPOSE: To assess if a high-level mobility programme (HLMP) can promote sustained participation in physical activity by adolescents with cerebral palsy. METHODS: Eight adolescents with cerebral palsy, Gross Motor Function Classification System levels I-II, 11-16 years, participated in 24 community-based group HLMP sessions across 12 weeks. Participants set attendance, involvement, and physical performance goals, completed activity diaries over 58 weeks and undertook physical capacity tests. Measures of activity frequency and diversity (attendance) and involvement level were collected weekly across baseline (4-6 weeks), intervention (12 weeks), and nine months follow-up (including Covid lockdown). RESULTS: Median attendance was 23 of 24 HLMP sessions. Attendance goal/s attainment was highest during COVID lockdown. Involvement goals were consistently attained throughout all phases. Physical performance goal/s attainment was highest during intervention phase but reduced during nine months follow-up. Frequency of participation in physical activities varied greatly across study phases (range 0-33 episodes/week) with stable variety of activities and generally high 'involvement.' During the intervention, seven participants improved physical capacity and six maintained, or increased, the gains six months later. CONCLUSION: Most participants improved physical capacity post-intervention but only some had sustained attendance and involvement in physical activity, highlighting the complexity of physical activity participation.

Health professionals' promotion of sustained participation in physical activity needs to consider individual preferences for frequency, diversity and duration.Supporting and measuring involvement in physical activity should be prioritised as a key outcome of an intervention.Physical activity interventions should be followed up for longer than six months to determine sustained changes in participation outcomesMeasuring physical capacity and performance gains alone is insufficient to determine sustained, meaningful participation.

The Journey to Sustainable Participation in Physical Activity for Adolescents Living with Cerebral Palsy.

PURPOSE: To understand adolescents' and their parents' perspectives on 'being active', this study explored the experience of participation in physical activity (PA), the role of long-term participation in PA, and the importance of remaining active for life. METHODS: Eight ambulant adolescents with CP (aged 11-16 years, seven male) participated in a high-level mobility programme twice per week for 12 weeks. Guided using interpretive description, adolescents and 12 of their parents were interviewed before, after and nine months following the programme. Thirty-eight interviews were coded, analysed, and interpreted, informed by audit information, reflective journaling, and team discussions. RESULTS: Adolescents and their parents highly value being active now and into adulthood. Sustainable participation in PA requires adolescents and families to navigate complex environments (interpersonal, organisational, community, and policy). Core themes were: 'Just Doing it', 'Getting the Mix Right' (right people, right place, right time), 'Balancing the Continua' and 'Navigating the Systems'. The continua involved balancing intra-personal attributes: 'I will try anything' through to 'I will do it if I want to' and 'It's OK to be different' through to 'It sucks being disabled'. CONCLUSIONS: The journey to sustainable participation was complex and dynamic. Experiences of successful journeys are needed to help adolescents with CP "stay on track" to sustainable participation.

Do physical activity interventions influence subsequent attendance and involvement in physical activities for children with cerebral palsy: a systematic review.

PURPOSE: To investigate if children with cerebral palsy have sustained attendance and involvement in physical activities after completing physical activity interventions. METHODS: The Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines were followed. Seven databases were searched for the period 2001-2020 with hand-searching of pertinent reference lists. Criteria for study inclusion were participants aged 0-18 years and ≥50% with cerebral palsy; follow-up ≥1 month beyond completion of the physical activity intervention; and measurement of attendance and/or involvement in any physical activity post-intervention. Study selection, data extraction, and risk of bias assessments (Physiotherapy Evidence Database (PEDro) or tool for non-randomised studies) were completed independently by paired reviewers. Results were compiled by narrative synthesis. RESULTS: Thirteen studies were included (11 randomised controlled trials (RCTs), two non-randomised case series; intervention sample sizes: 6-34). All study participants had cerebral palsy and were aged 4-16.7 years. PEDro scores for the RCTs ranged from 5 to 10; 10 did not blind one or more therapist, participant, or assessor. Two case series showed high risk of bias. Twelve studies reported on attendance, with positive changes in three studies. At 4-14 weeks post-intervention, two studies demonstrated positive changes were maintained. Four studies included involvement outcomes; one reporting positive changes in physical activity involvement four weeks after intervention completion. CONCLUSIONS: Physical activity attendance may be influenced by physical activity interventions in the short term, but more robust research designs are required to investigate whether gains can be sustained. Activity involvement, which may influence ongoing participation, is under-researched.Implications for RehabilitationPositive changes in attendance and involvement following physical activity interventions appear short term at best.Physical activity interventions should have longer follow-up periods to determine the effect on sustained physical activity participation.Careful selection and reporting of attendance and involvement outcome measures is required.The optimal physical activity intervention to increase attendance or involvement in physical activities remains uncertain.

Age of Autism Spectrum Disorder Diagnosis and Comorbidity in Children and Adolescents with Autism Spectrum Disorder.

AIM: Research is required to study the relationship between age of autism spectrum disorder (ASD) diagnosis and the presence of comorbidities. METHOD: The Gastrointestinal Symptom Inventory, Autism Spectrum Disorder-Comorbid for Children, Behavior Problem Inventory-Short Form and Social Communication Questionnaire were completed by parents of 129 children and adolescents with a diagnosis of ASD. RESULTS: Results revealed significant relationships between the age of ASD diagnosis, the presence of comorbidities and intellectual disability. Significant correlations were found between the age of ASD diagnosis and self-injurious and stereotyped behavior. Comorbid psychopathology significantly predicted the presence of GI symptoms. In addition, the relationship between comorbid psychopathology and challenging behavior in this study was reported as bi-directional as both comorbidities predicted one another in the sample. CONCLUSION: Future research needs to consider the role of comorbidities in relation to ASD diagnosis.

Educating health science students about disability: Teachers' perspectives on curricular gaps.

BACKGROUND: People living with disabilities are significantly more likely than their peers to find health professionals' skills and facilities inadequate. The 66th World Health Assembly called for better health care for people with disabilities including more inclusive health services and a stronger focus on professional training. OBJECTIVE: To explore how teachers at a New Zealand university perceived the need, approaches, and systemic challenges to enhance disability education for health professionals in training. METHODS: Qualitative analysis of interviews with 11 key informants teaching in population health, medicine, nursing, pharmacy, and optometry training programmes. Transcribed interview recordings were analysed using a general inductive approach. RESULTS: The participants described a range of teaching approaches that they used to increase disability awareness among their students. However, these were largely ad hoc individually driven initiatives reflecting personal interests. Participants identified a critical need to develop and implement a systematic, integrated approach to enhance disability education particularly from a social justice perspective among students in health disciplines. Engaging people with lived experience of disability in teaching and course design, and senior administrative commitment were identified as necessary to address current gaps in education. CONCLUSIONS: In order to develop a health professional workforce competent to respond to the needs of people with disabilities, greater attention is required at a strategic level to enhance the profile of disability education in health curricula. Meaningful engagement of people with disability and senior leadership commitment are critical components that can enable effective progression of this agenda.

"It Should Have Been Given Sooner, and We Should Not Have to Fight for It": A Mixed-Methods Study of the Experience of Diagnosis and Early Management of Cerebral Palsy.

Listening to the family experience is integral to identifying areas of strength and for improvement in health service delivery around diagnosis and early management of cerebral palsy (CP). Families of children with a diagnosis of CP were invited to complete a purpose-developed electronic survey that included items around the timing of diagnosis, their experiences and satisfaction. It also allowed families to expand on their experiences through free text. Of the 57 families responding, 49% of children functioned at Gross Motor Function Classification System (GMFCS) levels I or II, 8% at GMFCS level III and 23% at GMFCS levels IV or V. 51% of participants were satisfied or very satisfied with the diagnosis experience, 18% were neutral about the experience and 31% were dissatisfied or very dissatisfied. Though the findings of this study may be subject to selection bias, perceived delays in the receipt of diagnosis of CP appeared common with 60% of participants indicating concerns about their child by <6 months of age but only 21% provided with a diagnosis of CP <6 months of age. Approximately 18% of families experienced a delay of more than 12 months. Thirty-four (61%) participants noted a delay between referrals to a service and receipt of service management/therapy. Common themes impacting on families' experience in the diagnosis and health service delivery journey related to provision of information, and the style of communication, with both direct and ongoing communication styles common for greater family satisfaction. Overall, families desired the diagnosis experience to be informative and timely, with early follow up support and assistance with health sector navigation.

Disabled people say 'Nothing about us without us'.

BACKGROUND: This study explored disability community representatives' perspectives on why and how health professional education could be strengthened to address the prevalent challenges in health care experienced by disabled people. METHODS: A total of 14 people from the disability community (disabled people, caregivers and disability advocates) in Auckland, New Zealand, participated in three focus groups moderated by a disabled community researcher. Audiorecordings were transcribed and analysed thematically to characterise broad themes. FINDINGS: Participants described many barriers to health care experienced by disabled people, with service providers' inadequate knowledge of disability issues being a key contributor. Participants viewed educational approaches incorporating disabled peoples' diverse lived realities as critical to improving health system responses to these inequities. They recommended broadening concepts and teaching methods to shift common deficit framing of disability, engaging disabled people to develop and deliver curricula, improving communication, and promoting empathic provider-patient partnerships in care. Study participants strongly advocated inclusive participatory approaches across training pathways using assistive and multimedia technologies that optimise the engagement of disabled people and reduce respondent burden. DISCUSSION: Disability community participants urged strengthening health professional training to address the prevalent inadequacies of health systems in responding to disabled people's needs. They viewed a greater awareness of the lived realities of disabled people and critical consciousness to overcome barriers to care as essential attributes of a competent workforce. These findings indicate the need to engage, empower and work in partnership with disabled people to develop reflexive health professional curricula. Core competencies should be considered mandatory given the increasing prevalence of disability globally. This study explored disability community representatives' perspectives on why and how health professional education could … address … challenges in health care experienced by disabled people.

High-density electromyographic data during isometric contractions of the ankle joint in children with cerebral palsy pre and post BoNT-A treatment.

Understanding the underlying mechanisms leading to progressive muscle pathologies in spastic Cerebral Palsy remains a challenging field of research. Furthermore, Botulinum Neurotoxin-A (BoNT-A) is a frequent intervention to treat spasticity in CP but its effects on neuromuscular properties are not yet fully explored. High-density Electromyographic (HD-EMG) data have been collected before and after BoNT-A injections from children aged 5-15 years during isometric contractions of the ankle joint together with torque output, clinical assessments and demographic details. Data collected from a total of 13 children with and 29 children without spastic CP allow for between-group comparisons and are made available using Mendeley Data (https://doi.org/10.17632/3sbptrk54c.2 and https://doi.org/10.17632/3b98g5fyff.1).