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    The ResearchWorks Podcast

    The Research Works podcast is designed for health professionals in the area of child health, where we discuss emerging, modern, evidence based research - the behind the scenes stories, interviews with world renowned authors and researchers, material that never made the papers and a breakdown on how you can implement this into your clinical practice.

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    Episodes (127)

    Episode 129 (Dr Jacqui Barfoot)

    Episode 129 (Dr Jacqui Barfoot)

    Including a relationship-focus in paediatric occupational therapy interventions: Introducing the PAIR Model


    The importance of parent-child relationships for child developmental outcomes suggests a need to incorporate a relationship focus into early intervention programs for children with developmental delays. 

    Nevertheless, confusion exists about the definition and application of relationship-focussed interventions, and occupational therapists remain more developmentally- and child-focussed. 

    There is a need to operationalize relationship-focussed interventions to make these approaches clinically accessible. This report defines, and provides a rationale for, including parent-child relationship-focussed interventions in early childhood occupational therapy interventions. 

    A new conceptual model, the “Phased Approach to Incorporating a Relationship-focus” (PAIR), is detailed for consideration in pediatric therapy settings. The PAIR model can support professional education, inform practice, and guide future research regarding relationship-focussed interventions. 

    Research is needed to test the usefulness of this model in occupational therapy practice.

    Episode 128 (Professor Iona Novak)

    Episode 128 (Professor Iona Novak)

    The potential of cell therapies for cerebral palsy: where are we today?

    Iona Novak, Madison Cb Paton, Alexandra R Griffin, Michelle Jackman, Remy K Blatch-Williams, Megan Finch-Edmondson

    No abstract available

    Keywords: Cerebral palsy; efficacy; inflammation; mesenchymal stem cells; safety; stem cells; umbilical cord blood.


    Professor Novak is the Cerebral Palsy Alliance Chair of Allied Health, and co-founder of the Cerebral Palsy Alliance Research Institute, affiliated with the Brain and Mind Centre and Faculty of Medicine and Health at The University of Sydney.

    Episode 125 (A thematic analysis)

    Episode 125 (A thematic analysis)

    Season 3 Wrap Up

    Final episode of the season?!?

    As Ash and Dayna reflect on this incredible year, we pull together some key themes of the episodes and the ever so wonderful and brilliant guests we have had on the show. We are so grateful for all of the researchers who so generously gave their time to help spread the word on the current state of science in the field. We have learnt so much and we hope you have had some great takeaways too!

    In true Ash style, the qualitative researcher in her couldn’t help but to carry out a thematic analysis of the episodes in 2023. We love that she did that though - you can see the transdiagnostic approach really set in because of the common themes that run through so many different areas. We truly believe that if you can grasp all of the themes of the episodes this year, you can so easily translate this knowledge into your practice - staying up to date with the latest and waiting ever so hopefully for more to come. 

    In this episode, we talk about the powerhouses of the industry who joined us on the show. They are thought provokers for sure - titans of the industry with years of experience, extensive global collaborations and wisdom to impart to clinicians and researchers alike. These researchers include Professor Peter Rosenbaum, Professor Diane Damiano, Professor Andrew Whitehouse, Professor Mark Belgrove and Professor Laurent Servais and of course some of our returning guests which include Professor Ben Jackson, Dr Bhooma Aravamuthan, Dr Ros Ward and Ginny Paleg just to name a few. All of their messaging was around the importance of the ‘F-words’!  

    We as health professionals need to “be humble” as Bhooma so eloquently described and the real importance of being person centred was a common theme. Starting the season with Emily Prior and later Connor Johnstone truly reinforced this - both incredible young people with lived experience. It always comes down to the consumers and what a compelling reason to bring forth evidence based interventions and approaches. Their lives matter, their dreams matter - and again, the ‘F-words’ framework brings that home.

    We loved our conversations with other thought provokers such as Associate Professor Daniel Navon regarding the sociological take on genetic testing (who asked us more questions then we of him!) and most recently Dr Jessica Stokes-Parish on the world of misinformation, disinformation and the value of debunking! (remember the CRABs mnemonic!)

    Then there was the whole theme of brain activation and mobility - we loved talking about the functional MRI results with Dr Yannick Bleyenhueff and that self-initiation is vital! More than just the talk about dosage and the number of hours children engage in therapy - it is about ensuring that the right ingredients are in the intervention. This led nicely into a little series with Andrina Sabet, Dr Heather Feldner and Dr Sam Logan on mobility as a human right. For Dayna, as a clinician, this whole area was incredibly exciting. We now have these papers to reference and use as a resource when we have to advocate for change.

    Then we just talked about our highlights and what a highlight reel too! Going international and bringing the podcast to EACD in Slovenia was an amazing experience. We loved meeting so many people and the opportunity to help bring their work into the broader community. 

    Also, our very first Minister visit with the Honourable Bill Shorten was a highlight for sure. The clinic and our studio was a hive of activity and it was so encouraging to hear the Minister describe the value of evidence based practice and that it is be the way forward for a sustainable scheme.

    That was 2023! We are so excited for what 2024 holds! We look forward to meeting more of you at EACD in Bruge and AusACPDM in Cairns in 2024. Have a wonderful holiday season and we will talk to you again soon!

    Episode 124 (Dr Jessica Stokes-Parish)

    Episode 124 (Dr Jessica Stokes-Parish)

    Navigating the Credibility of Web-Based Information During the COVID-19 Pandemic: Using Mnemonics to Empower the Public to Spot Red Flags in Health Information on the Internet


    Jessica Stokes-Parish

    Free PMC article


    Abstract

    Misinformation creates challenges for the general public in differentiating truth from fiction in web-based content. During the COVID-19 pandemic, this issue has been amplified due to high volumes of news and changing information. Evidence on misinformation largely focuses on understanding the psychology of misinformation and debunking strategies but neglects to explore critical thinking education for the general public. This viewpoint outlines the science of misinformation and the current resources available to the public. 

    This paper describes the development and theoretical underpinnings of a mnemonic (Conflict of Interest, References, Author, Buzzwords, Scope of Practice [CRABS]) for identifying misinformation in web-based health content. Leveraging evidence-based educational strategies may be a promising approach for empowering the public with the confidence needed to differentiate truth from fiction in an infodemic.

    Keywords: COVID-19; critical appraisal; digital literacy; health literacy; infodemic; infodemiology; misinformation; online health; science communication; social media.



    Episode 123 (Associate Professor Asha Bowen)

    Episode 123 (Associate Professor Asha Bowen)

    Bivalent Prefusion F Vaccine in Pregnancy to Prevent RSV Illness in Infants


    List of authors.

    • Beate Kampmann, M.D., Ph.D., 
    • Shabir A. Madhi, M.B., B.Ch., Ph.D., 
    • Iona Munjal, M.D., 
    • Eric A.F. Simões, M.D., 
    • Barbara A. Pahud, M.D., M.P.H., 
    • Conrado Llapur, M.D., 
    • Jeffrey Baker, M.D., 
    • Gonzalo Pérez Marc, M.D., 
    • David Radley, M.S., 
    • Emma Shittu, Ph.D., 
    • Julia Glanternik, M.D., 
    • Hasra Snaggs, M.D., 
    • et al.,
    •  for the MATISSE Study Group*

    Abstract


    BACKGROUND

    Whether vaccination during pregnancy could reduce the burden of respiratory syncytial virus (RSV)–associated lower respiratory tract illness in newborns and infants is uncertain.


    METHODS


    In this phase 3, double-blind trial conducted in 18 countries, we randomly assigned, in a 1:1 ratio, pregnant women at 24 through 36 weeks’ gestation to receive a single intramuscular injection of 120 μg of a bivalent RSV prefusion F protein–based (RSVpreF) vaccine or placebo. The two primary efficacy end points were medically attended severe RSV-associated lower respiratory tract illness and medically attended RSV-associated lower respiratory tract illness in infants within 90, 120, 150, and 180 days after birth. A lower boundary of the confidence interval for vaccine efficacy (99.5% confidence interval [CI] at 90 days; 97.58% CI at later intervals) greater than 20% was considered to meet the success criterion for vaccine efficacy with respect to the primary end points.


    RESULTS

    At this prespecified interim analysis, the success criterion for vaccine efficacy was met with respect to one primary end point. Overall, 3682 maternal participants received vaccine and 3676 received placebo; 3570 and 3558 infants, respectively, were evaluated. Medically attended severe lower respiratory tract illness occurred within 90 days after birth in 6 infants of women in the vaccine group and 33 infants of women in the placebo group (vaccine efficacy, 81.8%; 99.5% CI, 40.6 to 96.3); 19 cases and 62 cases, respectively, occurred within 180 days after birth (vaccine efficacy, 69.4%; 97.58% CI, 44.3 to 84.1). 

    Medically attended RSV-associated lower respiratory tract illness occurred within 90 days after birth in 24 infants of women in the vaccine group and 56 infants of women in the placebo group (vaccine efficacy, 57.1%; 99.5% CI, 14.7 to 79.8); these results did not meet the statistical success criterion. No safety signals were detected in maternal participants or in infants and toddlers up to 24 months of age. The incidences of adverse events reported within 1 month after injection or within 1 month after birth were similar in the vaccine group (13.8% of women and 37.1% of infants) and the placebo group (13.1% and 34.5%, respectively).


    CONCLUSIONS

    RSVpreF vaccine administered during pregnancy was effective against medically attended severe RSV-associated lower respiratory tract illness in infants, and no safety concerns were identified. 


    Episode 122 (Professor Ana Carolina de Campos)

    Episode 122 (Professor Ana Carolina de Campos)

    F-words and early intervention ingredients for non-ambulant children with cerebral palsy: A scoping review

    Ana Carolina De Campos, Álvaro Hidalgo-Robles, Egmar Longo, Claire Shrader, Ginny Paleg


    Abstract

    Aim: To explore the ingredients of early interventions provided to young children with cerebral palsy (CP) who are classified in Gross Motor Function Classification System (GMFCS) levels IV and V, and to identify the 'F-words' addressed by the interventions.

    Method: Searches were completed in four electronic databases. Inclusion criteria were the original experimental studies that fitted the following PCC components: population, young children (aged 0-5 years, at least 30% of the sample) with CP and significant motor impairment (GMFCS levels IV or V, at least 30% of the sample); concept, non-surgical and non-pharmacological early intervention services measuring outcomes from any of the International Classification of Functioning, Disability and Health domains; and context, studies published from 2001 to 2021, from all settings and not limited to any specific geographical location.

    Results: Eighty-seven papers were included for review, with qualitative (n = 3), mixed-methods (n = 4), quantitative descriptive (n = 22), quantitative non-randomized (n = 39), and quantitative randomized (n = 19) designs. Fitness (n = 59), family (n = 46), and functioning (n = 33) ingredients were addressed by most experimental studies, whereas studies on fun (n = 6), friends (n = 5), and future (n = 14) were scarce. Several other factors (n = 55) related to the environment, for example, service provision, professional training, therapy dose, and environmental modifications, were also relevant.

    Interpretation: Many studies positively supported formal parent training and use of assistive technology to promote several F-words. A menu of intervention ingredients was provided, with suggestions for future research, to incorporate them into a real context within the family and clinical practice.


    Episode 121 (Loren West, Marissa Smith, Dr Dayna Pool, Dr Ashleigh Thornton)

    Episode 121 (Loren West, Marissa Smith, Dr Dayna Pool, Dr Ashleigh Thornton)

    A roundtable discussion about the clinical application of Mobility as a Human Right!

    Mobility is a fundamental human right and is supported by the United Nations and the ON Time Mobility framework.

    W Logan, Bethany M Sloane, Lisa K Kenyon, Heather A Feldner 

    PMID: 37232636 
    PMCID: PMC10215286 
    DOI: 10.3390/bs13050399 

    Free PMC article 

    Abstract 

    Mobility is a fundamental human right and is supported by the United Nations and the ON Time Mobility framework. The purpose of this study was to understand the effect of a powered mobility intervention on developmental changes of children with cerebral palsy (CP). 

    This study was a randomized, crossover clinical trial involving 24 children (12-36 months) diagnosed with CP or with high probability of future CP diagnosis based on birth history and current developmental status. Children received the Explorer Mini and a modified ride-on car in randomized order, each for 8 weeks. 

    The Bayley Scales of Infant and Toddler Development-4th Edition was administered at baseline, mid-study, and end-of-study. Raw change scores were used for analysis. 

    Total minutes of use per device was categorised as low or high use for analysis based on caregiver-reported driving diaries. 

    Explorer Mini: The high use group exhibited significantly greater positive change scores compared to the low use group on receptive communication, expressive communication, and gross motor sub scales (p less than 0.05). 

    Modified ride-on car: No significant differences between low and high use groups. Regardless of device, low use was associated with no significant developmental change and high use was associated with positive developmental changes. Mobility access is critical to maximize the development of children with CP and may be augmented by using powered mobility devices. 

    Results may have implications for the development of evidence-based guidelines on dosage for powered mobility use. 

    Keywords: cerebral palsy; disability; mobility; technology.

    Episode 120 (Associate Professor Samuel Logan)

    Episode 120 (Associate Professor Samuel Logan)

    Powered Mobility Device Use and Developmental Change of Young Children with Cerebral Palsy


    Mobility is a fundamental human right and is supported by the United Nations and the ON Time Mobility framework.

    The purpose of this study was to understand the effect of a powered mobility intervention on developmental changes of children with cerebral palsy (CP).

    This study was a randomized, crossover clinical trial involving 24 children (12-36 months) diagnosed with CP or with high probability of future CP diagnosis based on birth history and current developmental status. Children received the Explorer Mini and a modified ride-on car in randomized order, each for 8 weeks. The Bayley Scales of Infant and Toddler Development-4th Edition was administered at baseline, mid-study, and end-of-study. Raw change scores were used for analysis. Total minutes of use per device was categorized as low or high use for analysis based on caregiver-reported driving diaries.

    Explorer Mini: The high use group exhibited significantly greater positive change scores compared to the low use group on receptive communication, expressive communication, and gross motor subscales (p < 0.05). 

    Modified ride-on car: No significant differences between low and high use groups. Regardless of device, low use was associated with no significant developmental change and high use was associated with positive developmental changes. 

    Mobility access is critical to maximize the development of children with CP and may be augmented by using powered mobility devices. 

    Results may have implications for the development of evidence-based guidelines on dosage for powered mobility use.

    Episode 119 (Assistant Professor Heather Feldner and Andrina Sabet)

    Episode 119 (Assistant Professor Heather Feldner and Andrina Sabet)

    ON Time Mobility: Advocating for Mobility Equity

    Abstract


    Mobility is a human right. The traditional definition of mobility in physical therapy practice is centered on translocation and, while accurate, is not comprehensive. 

    In this article, we propose the ON Time Mobility framework: that all children have the right to be mobile throughout their development to explore, engage in relationships, and develop agency to cocreate their lives. 

    This perspective highlights interconnected principles of timing, urgency, multimodal, frequency, and sociability to begin discussions on supporting the right to hours of active mobility each day for all children. 

    We propose critical evaluation and discussion of these principles followed by a call to action to shift our conceptualization and enactment of mobility. 

    This mobility rights perspective challenges current medical systems, industry, and government to collaborate with children with disabilities, their families and communities to support mobility as a source of physical and social interactions that define and develop individuals 

    (see Supplemental Digital Content 1, the Video Abstract, available at: http://links.lww.com/PPT/A398 ).

    Episode 118 (Dr Yannick Bleyenheuft)

    Episode 118 (Dr Yannick Bleyenheuft)

    Brain activation changes following motor training in children with unilateral cerebral palsy: An fMRI study


    Rodrigo Araneda, Laurance Dricot, Daniela Ebner-Karestinos, Julie Paradis, Andrew M Gordon, Kathleen M Friel, Yannick Bleyenheuft

    Free article

    Abstract

    Background:
    Intensive motor-learning-based interventions have demonstrated efficacy for improving motor function in children with unilateral spastic cerebral palsy (USCP). Although this improvement has been associated mainly with neuroplastic changes in the primary sensori-motor cortices, this plasticity may also involve a wider fronto-parietal network for motor learning.

    Objective: To determine whether hand-arm bimanual intensive therapy including lower extremities (HABIT-ILE) induces brain activation changes in an extensive network for motor skill learning and whether these changes are related to functional changes observed after HABIT-ILE.

    Methods: In total, 25 children with USCP were behaviourally assessed in manual dexterity and everyday activities before and after HABIT-ILE. Functional imagery monitored brain activity while participants manipulated objects using their less-affected, more-affected or both hands. Two random-effects-group analyses performed at the whole-brain level assessed the brain activity network before and after therapy. Three other random-effects-group analyses assessed brain activity changes after therapy. Spearman's correlations were used to evaluate the correlation between behavioural and brain activity changes.

    Results: The same fronto-parietal network was identified before and after therapy. After the intervention, the more-affected hand manipulation elicited a decrease in activity on the motor cortex of the non-lesional hemisphere and an increase in activity on motor areas of the lesional hemisphere. The less-affected hand manipulation generated a decrease in activity of sensorimotor areas in the non-lesional hemisphere. Both-hands manipulation elicited an increase in activity of both hemispheres. Furthermore, we observed an association between brain activity changes and changes in everyday activity assessments.

    Conclusion: Brain activation changes were observed in a fronto-parietal network underlying motor skill learning with HABIT-ILE in children with USCP. Two different patterns were observed, probably related to different phases of motor skill learning, representing an increased practice-dependent brain recruitment or a brain activation refinement by more efficient means. CLINICALTRIALS.GOV: NCT01700777 &NCT02667613.

    Keywords: Cerebral palsy; FMRI; HABIT-ILE; Intensive therapy; Neuroplasticity.


    Episode 116 (Minister Bill Shorten)

    Episode 116 (Minister Bill Shorten)

    The NDIS (National Disability Insurance Scheme) and evidence based practice.

    A very special ResearchWorks episode as we speak with (Australian) Federal Minister Bill Shorten - the Minister for the National Disability Insurance Scheme (the NDIS). 

    The NDIS is a very unique Australian treasured, national health care service - there may be no other equivalent of it's kind in the world and though it has had it's share of critics, the NDIS is ever changing and adapting to the needs of Australian families - old and young alike.

    The discussion over the NDIS and evidence based practices is at a crossroads and under the stewardship of Minister Bill Shorten, there is a galvanised effort to bring evidence based practices and research to the fore.

    The Healthy Strides Foundation was proud to host the Minister as the Foundation is an  unequivocal supporter of merging evidence and research into the clinical environment.

    We hope you enjoy the mini-episode. It is exciting to note what the future holds for research and evidence fused clinical practices and how the NDIS fits into this model. A very nice blueprint that goes beyond the borders of Australia and is food for thought for every clinician and researcher throughout the developed world. 

    Episode 115 (Professor Ben Jackson)

    Episode 115 (Professor Ben Jackson)

    Persuasion and Communication in Sport, Exercise, and Physical Activity

    How can we use persuasion methods to make people more physically active and improve their sport and exercise experiences? 

    How can instructors, coaches, athletes, and practitioners most effectively communicate their messages to others? Persuasion and Communication in Sport, Exercise, and Physical Activity is the first book to consider the applications of persuasion frameworks within activity-related contexts, whilst also summarising the major developments relating to communication topics in these settings. 

    It provides a state of the art review of the key developments, challenges, and opportunities within the field. It brings together international experts from the fields of social, health, and sport and exercise psychology, to give theoretical overviews, insights into contemporary research themes and practical implications, as well as agendas for future research. 

    Covering topics such as changing attitudes towards exercise, social influence, persuasive leadership and communicating with people with physical disabilities, this book provides a contemporary approach to persuasion and communication in a sport, exercise and physical activity setting. 

    It is an important text for upper-level undergraduate and postgraduate students, as well as academics in the fields of Sport and Exercise Science, Kinesiology, Health and Physical Activity Promotion and related areas of Psychology.

    https://www.researchgate.net/publication/316698536_Persuasion_and_Communication_in_Sport_Exercise_and_Physical_Activity

    https://www.routledge.com/Persuasion-and-Communication-in-Sport-Exercise-and-Physical-Activity/Jackson-Dimmock-Compton/p/book/9780367407759

    Episode 114 (Dr Saranda Bekteshi)

    Episode 114 (Dr Saranda Bekteshi)

    Eye movements and stress during eye-tracking gaming performance in children with dyskinetic cerebral palsy


    Saranda Bekteshi, Petra Karlsson, Lieselot De Reyck, Karen Vermeerbergen, Marco Konings, Patrick Hellin, Jean-Marie Aerts, Hans Hallez, Bernard Dan, Elegast Monbaliu

    Affiliations expand

    Abstract

    Aim: This study aimed to explore eye movements and stress during eye-tracking gaming performance in children with dyskinetic cerebral palsy (CP) compared with typically developing children, and associations between eye-tracking performance, eye movements, stress, and participants' characteristics.

    Method: This cohort study included 12 children with dyskinetic CP aged 5 to 12 years (mean age 8 years 7 months, standard deviation [SD] 2 years 3 months) and 23 typically developing children aged 5 to 13 years (mean age 9 years 0 months, SD 2 years 7 months). Participants played 10 eye-tracking games. Tobii X3-120 and Tobii Pro Lab were used to record and analyse eye movements. Stress was assessed through heart rate variability (HRV), recorded during rest, and eye-tracking performance using the Bittium Faros360° ECG Holter device. Eye-tracking performance was measured using gaming completion time. Fixation and saccade variables were used to quantify eye movements, and time- and frequency-domain variables to quantify HRV. Non-parametric statistics were used.

    Results: Gaming completion time was significantly different (p < 0.001) between groups, and it was negatively correlated with experience (rs = -0.63, p = 0.029). No significant differences were found between groups in fixation and saccade variables. HRV significantly changed from rest to eye-tracking performance only in typically developing children and not in children with dyskinetic CP.

    Interpretation: Children with dyskinetic CP took longer to perform the 10 games, especially the inexperienced users, indicating the importance of the early provision of eye-tracking training opportunities. It seems that eye-tracking tasks are not a source of increased stress and effort in children with dyskinetic CP.

    What this paper adds: Participants with dyskinetic cerebral palsy (CP) took twice as long to perform 10 eye-tracking games than typically developing peers. Participants with dyskinetic CP with previous eye-tracking experience performed the games faster. Fixation and saccade variables were not significantly different between children with and without dyskinetic CP. Heart rate variability showed no differences between rest and performance in participants with dyskinetic CP. Gross Motor Function Classification System, Manual Ability Classification System, and Viking Speech Scale levels were not correlated to the eye movements or stress variables.


    Episode 113 (Associate Professor Dana Anaby)

    Episode 113 (Associate Professor Dana Anaby)

    Reliability and Validity of the Youth and Young-Adult Participation and Environment Measure (Y-PEM): An Initial Evaluation

    Saeideh Shahin, Sara Ahmed, Briano DiRezze, Dana Anaby

    Abstract

    Aim: To examine psychometric properties and aspects of utility of the Youth and young-adult Participation and Environment Measure (Y-PEM).


    Methods: Young people with and without physical disabilities (n = 113) aged 12 to 31 (x¯ = 23; SD = 4.3) completed an online survey containing the Y-PEM and QQ-10 questionnaire. To examine construct validity, differences in participation levels and environmental barriers/facilitators were examined between those with (n = 56) and without disabilities (n = 57) via t-test. Internal consistency was computed using Cronbach's alpha. To examine test-retest reliability, a sub-sample of 70 participants completed the Y-PEM a second time, 2-4 weeks apart. The Intraclass correlation coefficient (ICC) was calculated.


    Results: Descriptively, participants with disabilities had lower levels of frequency and involvement across all four settings: home, school/educational, community, workplace. Internal consistency were 0.71 and above (up to 0.82) across all scales with the exception of home (0.52) and workplace frequency (0.61). Test-retest reliability were 0.70 and above (up to 0.85) across all settings except for environmental supports at school (0.66) and workplace frequency (0.43). Y-PEM was perceived as a valuable tool with relatively low burden.


    Conclusions: Initial psychometric properties are promising. Findings support Y-PEM's use as a feasible self-reported questionnaire for individuals aged 12-30 years old.


    Keywords: Assessment; environment; participation measure; transition-aged; workplace participation.


    Episode 112 (Professor Laurent Servais)

    Episode 112 (Professor Laurent Servais)

    Financial cost and quality of life of patients with spinal muscular atrophy identified by symptoms or newborn screening


    Tamara Dangouloff, Mickael Hiligsmann, Nicolas Deconinck, Adèle D'Amico, Andreea M Seferian, François Boemer, Laurent Servais

    Free article


    Abstract

    in English and Spanish

    Aim: To compare the societal financial costs and quality of life (QoL) of untreated patients with spinal muscular atrophy (SMA) and treated patients identified because they presented symptoms or were identified by early testing (sibling or newborn screening).

    Method: Data from two different sources were used: data collected prospectively in untreated patients from 2016 to 2018 and data collected during a prospective follow-up study from 2018 to 2021. Patients or their caregiver completed a questionnaire that included questions on direct medical and non-medical costs, indirect non-medical costs, and health-related QoL.

    Results: Data (median; range) were available for 149 patients (93 untreated - 10 years; 2 years-59 years), 42 patients (6 years 3 months; 9 months-58 years) treated after presenting with symptoms, and 14 patients (1 year 7 months; 5 months-2 years) treated after early diagnosis. Total costs were lower in untreated patients due to the high cost of drugs used in treated patients. Costs were lower for treated patients who were identified by early testing than for treated patients identified because they presented with symptoms. In all groups, patients with two SMN2 copies had higher costs than those with more copies.

    Interpretation: Early patient identification and treatment offer the opportunity to reduce the total societal costs of SMA where treatments are available for presymptomatic and postsymptomatic patients.

    What this paper adds: Untreated patients with spinal muscular atrophy had lower total financial costs than treated patients. Total financial costs were lower for treated patients identified by early screening than for treated patients identified after symptom onset. Direct financial costs excluding treatment were much lower in treated patients identified by early screening. Hospitalization costs were much lower in patients identified by early screening.


    Episode 111 (Dr Katrina Kelso)

    Episode 111 (Dr Katrina Kelso)

    Poor comprehenders have difficulty with reading comprehension despite adequate word reading accuracy and fluency. 

    Weaknesses have been identified with lower-level vocabulary and grammar skills, and higher-level language skills such as inference making. It is important that speech-language pathologists (SLPs) tailor intervention to meet the specific needs of individuals; however, there is a lack of research on intervention for poor comprehenders, who comprise a heterogenous group. 

    This case study aimed to explore whether a pilot 8-week novel vocabulary intervention was 
    (a) effective in improving word knowledge, and 
    (b) if gains generalised to reading comprehension. 

    Following intervention, significant improvements were found on the semantic subtasks and in word knowledge for treated words on the Word Knowledge Profile measure; improvement was also seen for untreated words at six-month follow-up. 

    There were also gains on the standardised word and reading comprehension measures, providing promising preliminary evidence for the usefulness of the intervention.