The latest medical research on Neuromuscular Medicine

The research magnet gathers the latest research from around the web, based on your specialty area. Below you will find a sample of some of the most recent articles from reputable medical journals about neuromuscular medicine gathered by our medical AI research bot.

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Speech and communication classification of children with cerebral palsy: Novice rater agreement and clinical utility.

Cerebral Palsy

To examine novice inter-rater agreement and clinical utility perspectives for speech and communication classification of children with cerebral palsy (CP).

Twenty-one clinicians (speech-language pathologists [SLPs] n = 11; physiotherapists [PTs] n = 5; occupational therapists [OTs] n = 5) novice to the Viking Speech Scale (VSS), Functional Communication Classification System (FCCS), and Communication Function Classification System (CFCS) rated eight unfamiliar children with CP (8-16 years) following classification orientation. Inter-rater agreement was examined between (a) novices, (b) novice SLPs vs. PTs and OTs, and (c) novice vs. expert (kappa statistics). Utility perceptions were scored regarding classification terminology, ease of use, assistive decision-making resources, and construct validity and were analysed using Kruskal-Wallis H-tests.

Rating agreement between novices was substantial (VSS, k = 0.72, 95% CI [0.53-0.92]) to moderate (FCCS, k = 0.44, 95% CI [0.23-0.65]; CFCS, k = 0.45, 95% CI [0.18-0.71]), and almost perfect between novice and expert ratings (VSS, kw = 0.89, 95% CI [0.86-0.92]; FCCS, kw = 0.89, 95% CI [0.86-0.92]; CFCS, kw = 0.86, 95% CI [0.82-0.91]). Statistically significant differences, presented highest to lowest, were found for clinical utility: terminology (VSS, FCCS, CFCS; p = 0.02), assistive decision-making resources (FCCS, VSS, CFCS; p = 0.009), and construct validity (FCCS, CFCS, VSS; p < 0.001).

Novice raters achieved substantial agreement for speech classification, supporting utilisation in clinical, research, and CP register activities. Orientation to communication classification constructs, content, and instructions is recommended for novice raters.

A pilot study proposing an algorithm for pubertal induction in cerebral palsy.

Cerebral Palsy

To explore delayed puberty in cerebral palsy (CP) and to test the acceptability of an interventional puberty induction algorithm.

A two phase cohort study in children and adolescents diagnosed with CP who have delayed puberty. Phase 1: Retrospective review of clinical records and interviews with patients who have been treated with sex-steroids and Phase 2: Prospective interventional trial of pubertal induction with a proposed algorithm of transdermal testosterone (males) or oestrogen (females). Phase 1 examined experiences with sex-steroid treatment. Phase 2 collected data on height adjusted bone mineral density (BMAD), fractures, adverse effects, mobility and quality of life over two years during the induction.

Phase 1, treatment was well tolerated in 11/20 treated with sex-steroids; phase 2, using the proposed induction algorithm, 7/10 treated reached Tanner stage 3 by nine months. One participant reached Tanner stage 5 in 24 months. Mean change in BMAD Z-scores was +0.27 % (SD 0.002) in those who could be scanned by dual-energy X-ray absorptiometry (DXA).

Delayed puberty may be diagnosed late. Treatment was beneficial and well tolerated, suggesting all patients with severe pubertal delay or arrest should be considered for sex hormone supplementation.

Can RESPiratory hospital Admissions in children with cerebral palsy be reduced? A feasibility randomised Controlled Trial pilot study protocol (RESP-ACT).

Cerebral Palsy

Australian New Zealand Clinical Trials Registry (ACTRN12620000114943).

Mixed-method feasibility pilot randomised controlled trial with economic analysis. Twenty children with CP aged 0-12 years who are at risk of respiratory disease will be followed up for 1 year. All children will receive baseline assessments for comparison. The control group will receive usual care from their treating teams. The intervention group will receive comprehensive assessments from physiotherapy, speech pathology and respiratory medicine. An individualised investigation and treatment plan will then be made. Participants in both groups will complete fortnightly patient-reported outcome surveys to assess symptoms and health service use. Analysis will include assessments of acceptability through qualitative interviews, implementation by ability to recruit, randomise and retain, practicality including costs of intervention and hospitalisation, and explore efficacy through quality-of-life surveys and decreased health service use for respiratory-related symptoms.

Saudi Cerebral Palsy Register (SCPR): Protocol on the Methods and Technical Details.

Cerebral Palsy

This protocol presents a comprehensive proposal for the establishment of the Saudi Cerebral Palsy Register (SCPR), a crucial project for investigat...

Excellent response to anti-CD38 therapy with daratumumab in a patient with severe refractory CANOMAD.

Neurology, Neurosurgery and Psychiatry

Intravenous immunoglobulin (IVIG) and rituximab are considered the first-line and second-line treatments for Chronic Ataxic Neuropathy and Ophthalmoplegia with IgM-paraprotein, cold Agglutinins, and anti-Disialosyl antibodies (CANOMAD), with an overall clinical response around 50%. New anti-CD38 daratumumab, targeting long-lived plasma cells, has been reported as a promising therapy for treatment-refractory antibody-mediated disorders. We report the first case of a severe refractory CANOMAD, successfully treated with daratumumab.

A patient in their 70s with severe relapsing CANOMAD, refractory to IVIG, steroids, rituximab and ibrutinib developed severe tetraparesis and respiratory failure. Plasma exchange (PE) improved motor and ventilatory function; however, after 6 weeks, patient remained PE dependent. Intravenous daratumumab was initiated at 16 mg/kg weekly for 3 weeks, every 2 weeks for the second and third month, and monthly afterwards.

After 3 weeks of starting daratumumab, PE was discontinued and, since then, the patient evolved to complete recovery. Antidisialosyl antibody titres decreased after PE and remained stable during daratumumab. Serum neurofilament light-chain levels were elevated in the exacerbation phase and normalised after daratumumab. The patient remains in clinical remission under monthly daratumumab, 12 months after initiation.

The first patient with aggressive treatment-refractory CANOMAD treated with daratumumab provides proof-of-principle evidence that daratumumab may be an effective treatment in IgM-related neuropathies.

Assessment of fetal growth trajectory identifies infants at high risk of perinatal mortality.

Cerebral Palsy

To analyze perinatal risks associated with three distinct scenarios of fetal growth trajectory in the latter half of pregnancy compared with a referent group.

This cohort study included women with singleton pregnancies that birthed between 32+0 and 41+6 gestational weeks and had two or more ultrasound scans at least four weeks apart from 18+0 weeks. We evaluated three different scenarios of fetal growth against a referent, defined as appropriate for gestational age-sized fetuses with appropriate forward growth trajectories. The comparator growth trajectories were categorized as Group 1: Small for gestational age (SGA) fetuses (EFW or AC <10th centile) with appropriate forward growth; Group 2: Decreased growth trajectory fetuses (decrease of ≥50 centiles) with EFW or AC ≥10th centile (i.e., non-SGA) at their final scan; and Group 3: Decreased growth trajectory fetuses with EFW or AC <10th centile (i.e., SGA) at their final scan. The primary outcomes were perinatal mortality (stillbirth or neonatal death). Secondary outcomes included stillbirth, birth of an SGA infant, preterm birth, emergency cesarean section (CS) for non-reassuring fetal status (NRFS), and composite severe neonatal morbidity. Associations were analyzed using logistic regression.

The final study cohort comprised 5319 pregnancies. Compared to the referent group, the adjusted odds of perinatal mortality were significantly increased in Group 2 (odds ratio [OR] 4.00, 95%CI 1.36-11.22) and Group 3 (OR 7.71, 95%CI 2.39-24.91). Only Group 3 had increased odds of stillbirth (OR 5.69, 95%CI 1.55-20.93). In contrast, infants in Group 1 did not have significantly increased odds of demise. The odds of an SGA infant at birth increased in all three groups but were highest in Group 1 (OR 111.86, 95%CI 62.58-199.95) and Group 3 (OR 40.63, 95%CI 29.01-56.92). In both groups, more than 80% of infants were born SGA, and nearly half had a birth weight <3rd centile. Likewise, the odds of preterm birth were increased in all three groups, being the highest in Group 3 with an OR of 4.27 (3.23-5.64). Lastly, the odds of severe neonatal morbidity were increased in Groups 1 and 3, whereas the odds of emergency CS for NRFS were only increased in Group 3.

Assessing the fetal growth trajectory in the latter half of pregnancy can help identify infants at increased risk of perinatal mortality and birth weight <3rd centile for gestation. This article is protected by copyright. All rights reserved.

Health literacy in adolescents and young adults with cerebral palsy: a mixed methods systematic review.

Cerebral Palsy

To identify evidence of health literacy in young people with cerebral palsy (13-38 years), describe current strategies they use to access and build their health knowledge, and explore associations between health literacy and quality of life (QoL).

Four electronic databases were systematically searched (2001 to June 2023) to identify studies describing components of health literacy in this population. Two reviewers screened for eligibility, then extracted data and assessed methodological quality of included studies. Data were synthesised using a convergent integrated analysis framework and summarised with a narrative synthesis.

Eleven studies were included (N = 363). Evidence of health literacy was demonstrated through a range of strategies young people employed to identify their specific information needs, develop health literacy skills, and learn experientially. The preferred method for building health knowledge was obtaining information from trusted sources. Information gaps were identified in topics such as ageing with cerebral palsy, sexuality and navigating intimate relationships. There were minimal data on health literacy and QoL.

Young people with cerebral palsy want tailored and credible health information to increase participation in making informed health-related decisions. Building capacity and development of self-efficacy may assist with the identification of emerging health literacy needs.

Development and content validation of the Upper Limb-Motor Learning Strategy Tool for cerebral palsy.

Cerebral Palsy

To describe the development and content validation of the Upper Limb-Motor Learning Strategy Tool (UL-MLST) that aims to guide clinicians on how to implement and document the motor learning strategies used in the upper limb therapy approaches for children with cerebral palsy.

The study consists of two main stages (1) item generation and development and (2) content validation and refinement. The UL-MLST Online Training Program, Manual and Checklist were developed by the authorship group in stage one. In stage 2, two experts evaluated the UL-MLST regarding the Relevance, Coherence, and Significance of the individual strategies and whether the tool is Relevant, Comprehensive, and Clinically useful.

Of sixty-two strategies included in the UL-MLST, 52 strategies were rated as being either "Moderately" or "Highly" Relevant, Coherent, and Significant. Ten strategies did not achieve mutual agreement; however, they did not meet the criteria for deletion and were revised according to expert feedback. Overall, the UL-MST was judged to be Relevant, Comprehensive, and Clinically useful.

The UL-MLST provides a valid tool to support clinicians in the implementation of the motor learning strategies for children with cerebral palsy.IMPLICATIONS FOR REHABILITATIONThe Upper Limb- Motor Learning Strategy Tool (UL-MLST) Online Training Program, Manual, and Checklist provide a comprehensive package of resources to support the application of motor learning strategies in upper limb therapy for children with cerebral palsy.The UL-MLST provides clinicians with a valid tool for self-appraising the implementation of motor learning-based therapies.The tool has the potential to improve fidelity, enhance the quality, and ensure consistency of evidence-based, task-focused approaches of therapy.

ON/OFF non-motor evaluation: a new way to evaluate non-motor fluctuations in Parkinson's disease.

Neurology, Neurosurgery and Psychiatry

NMF are currently poorly evaluated in therapeutic decisions. A quantification of their severity would facilitate their integration. The objective of this study was to validate an autoquestionnaire evaluating the severity of non-motor fluctuations (NMF) in Parkinson's disease (PD).

Patients with PD were included in presurgical situation for deep brain stimulation of subthalamic nuclei. They participated in the PREDISTIM cohort (a study evaluating the predictive factors for therapeutic response of subthalamic stimulation in PD) in 17 centres in France. Our questionnaire, resulting from previous phases of development, included 11 non-motor symptoms (NMS). Their severity ranged from 0 to 10 and was assessed in OFF and then ON-Dopa to study their fluctuations.

310 patients were included, of whom 98.8% had NMS and 98.0% had NMF. Each NMS was significantly improved by L-Dopa (decrease in severity score ranging from 43.1% to 69.9%). Fatigue was the most frequent and most severe NMS. NMS were considered more bothersome than motor symptoms by 37.5% of patients in OFF-Dopa and 34.9% in ON-Dopa.

This is the first questionnaire allowing a real-time quantification of the severity of NMS and their fluctuation with levodopa. It was able to confirm and measure the effect of L-dopa and show differences according to the patients and the NMS. It differs from other questionnaires by its measurement at a precise moment of the severity of the NMS, allowing its use during pretherapeutic assessments.Our questionnaire has been validated to measure the severity of NMF. It will be able to quantify the non-motor effect of anti-parkinsonian treatments and could facilitate the integration of NMF in therapeutic decisions.

Novel Modular Walking Orthosis (MOWA) for Powerful Correction of Gait Deviations in Subjects with a Neurological Disease.

Cerebral Palsy

This article introduces a novel concept where advanced technologies have been leveraged to produce a modular walking orthosis (MOWA) within a compl...

Prioritized strategies to improve diagnosis and early management of cerebral palsy for both Māori and non-Māori families.

Cerebral Palsy

To identify prioritized strategies to support improvements in early health service delivery around the diagnosis and management of cerebral palsy (CP) for both Māori and non-Māori individuals.

Using a participatory approach, health care professionals and the parents of children with CP attended co-design workshops on the topic of early diagnosis and management of CP. Health design researchers facilitated two 'discovery' (sharing experiences and ideas) and two 'prototyping' (solution-focused) workshops in Aotearoa, New Zealand. A Māori health service worker co-facilitated workshops for Māori families.

Between 7 and 13 participants (14 health care professionals, 12 parents of children with CP across all functional levels) attended each workshop. The discovery workshops revealed powerful stories about early experiences and needs within clinician-family communication and service provision. The prototyping workshops revealed priorities around communication, and when, what, and how information is provided to families; recommendations were co-created around what should be prioritized within a resource to aid health care navigation.

There is a critical need for improved communication, support, and guidance, as well as education, for families navigating their child with CP through the health care system. Further input from families and health care professionals partnering together will continue to guide strategies to improve health care service delivery using experiences as a mechanism for change.

Adolescent health care and perceptions in a provincial hospital in Papua New Guinea.

Cerebral Palsy

Adolescents make up an estimated 22 % of the 11.8 million population of Papua New Guinea (PNG), yet, as in many low and middle income countries, there are limited specific services for adolescents, who in traditional health service design fall between paediatric and adult patients. With more survivors of chronic illnesses, including tuberculosis and HIV, congenital and acquired heart disease, epilepsy and cerebral palsy, diabetes, cancer and other chronic conditions, there are gaps in the care of such young people, and in their eventual transition to adult services. Moreover, traditional health service design rarely addresses many adolescent health concerns - mental health, self-esteem, substance use, and adolescent-friendly sexual and reproductive health. This study, amongst a cohort cared for in a provincial hospital in Milne Bay Province, Papua New Guinea, aimed to explore the perceptions of adolescents on the health care they receive, and their views on their health priorities.

A qualitative study was carried out from April to August 2022, amongst adolescents (persons aged 10-19 years) attending or admitted to Alotau Provincial Hospital. Data were collected via semi-structured wide-ranging interviews, and contextual data obtained from their medical charts. Thematic data analysis was done.

Fifty-four adolescents were interviewed. Adolescents were generally content with the care they received, although many raised concerns regarding feeling out of place in a ward with older adults or infants, and staff attitudes towards them including not directly communicating. Forty four adolescents preferred an adolescent-friendly setting, as opposed to the adult or children's wards, stating that it would allow for positive peer-interaction, improve their experience in the hospital, and improve the quality of health care received. Many adolescents with chronic illnesses had concerns regarding loneliness and isolation, and uncertainty of their future. Pregnant adolescents highlighted the lack of education on sexual and reproductive health, and experiences of gender-based violence.

This study shows that adolescents can perceive the need for adolescent-friendly health services and advocate for improvement in the quality of health care they receive, and the breadth of adolescent health care concerns.