FIREFISH was a multicentre, open-label, two-part, phase 2 trial that assessed the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of risdiplam in children with type 1 spinal muscular atrophy and two SMN2 copies, aged 1-7 months at enrolment. Part 1 assessed safety and determined the dose for part 2. In part 2, treatment with risdiplam for 24 months resulted in continual improvements in motor function and achievement of developmental motor milestones. The aim of the 3-year open-label extension reported here was to assess long-term safety and efficacy of risdiplam. The 3-year extension of the FIREFISH trial was a multicentre, open-label study in 17 centres across 12 countries (in Asia, Europe, Brazil, and the USA). Children with a confirmed genetic diagnosis of spinal muscular atrophy and two copies of the SMN2 gene who had completed 2 years of risdiplam treatment were eligible to continue in the open-label extension study, during which risdiplam was administered once daily by oral syringe or feeding tube at the pivotal dose of 0·20 mg/kg for infants younger than 2 years, at 0·25 mg/kg for children aged 2 years and older with bodyweight below 20 kg, and at 5 mg for children aged 2 years and older with bodyweight of 20 kg or more. Adverse events were assessed as part of physical assessments every 13 weeks. More comprehensive assessments every 26 weeks included level of respiratory support and efficacy assessments of motor function in addition to safety and physical assessments. Outcomes assessed during the open-label extension study included adverse events, survival and event-free survival (defined as being alive with no permanent ventilation), growth measures, swallowing, feeding, and motor function. Motor function was assessed with the Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND), the gross motor subscale of the Bayley Scales of Infant and Toddler Development, third edition (BSID-III), and the Hammersmith Infant Neurological Examination, Module 2 (HINE-2). For all children, safety data including adverse events, serious and non-serious adverse events of special interest; ophthalmology assessments; laboratory assessments; vital signs; ECGs; and other protocol-specified tests deemed critical to the safety evaluation of the study, were collected up to 30 days after the last dose. Survival and event-free survival are reported at year 5 for all enrolled children. Adverse events are reported at year 5 for all children who received at least one dose of risdiplam (safety population). All other efficacy endpoints are reported at year 5 for the population who received the pivotal dose. FIREFISH is registered with ClinicalTrials.gov, NCT02913482. Between Dec 23, 2016, and Nov 19, 2018, 62 children aged 1-7 months were enrolled in FIREFISH parts 1 and 2. After 2 years in the study (parts 1 and 2), 55 children continued risdiplam treatment into the open-label extension for a further 3 years. The study was completed on Dec 22, 2023, at which time 52 children (84%) had completed 5 years of risdiplam treatment. After 5 years of risdiplam treatment, 56 children (90%) were alive, and 50 (80%) were alive without the need for permanent ventilation. In the pivotal-dose population (n=58), 17 children (29%) did not require invasive or non-invasive respiratory support at year 5 and 13 (22%) did not require hospitalisations during the study. Upward trajectories for motor function responses were observed between years 1 and 5. The number of children who could sit without support for at least 5 s and 30 s was maintained during the 3-year extension, with 36 children (62%) and 34 children (59%), respectively, doing so by year 5. Similarly, for HINE-2, eight children (14%) reached the stable sit milestone and 26 (45%) met pivots at year 5. By year 5, four children (7%) could stand without support as assessed by both the BSID-III and HINE-2. No children could walk independently, but six (10%) met the HINE-2 cruising milestone. The mean HINE-2 total score increased from baseline (0·93 [90% CI 0·72-1·14]) to year 4 (14·22 [12·66-15·79]), remaining stable thereafter. Mean CHOP-INTEND scores increased from 22·47 at baseline (90% CI 20·97-23·96) and stabilised at around 50 at year 5, with scores of ≥40 for 38 children (66%), ≥50 for 29 children (50%), and ≥60 for 11 children (19%) at year 5. Most children maintained oral feeding (n=42; 72%) and swallowing (n=46; 79%) abilities, and growth was consistent with typical patterns seen in type 1 spinal muscular atrophy. Safety findings over 5 years were consistent with the results from the primary analysis. Most adverse events were mild or moderate. The most frequently reported adverse events were pyrexia (65% [n=40]), upper respiratory tract infection (63% [n=39]), and pneumonia (50% [n=31]). Pneumonia was the most frequently reported serious adverse event in 28 children (45%). No new safety findings were reported and no treatment-related events led to withdrawal from study treatment. Unlike the natural history of children with type 1 spinal muscular atrophy, after 5 years of risdiplam treatment, most children in FIREFISH were alive without needing permanent ventilation, were able to swallow and feed orally, and had reached motor milestones not typically observed in untreated patients. These results show long-term continuous efficacy and safety of risdiplam in children with type 1 spinal muscular atrophy. F Hoffmann-La Roche.
Malawi's paediatric and child health workforce faces a critical shortage. In response, the Kamuzu University of Health Sciences has been training clinical officers (29 to date) in a Bachelor of Science in Paediatric and Child Health (BSc PCH) program since 2012 to serve as paediatric and child health district leaders in mostly rural areas. However, little is known about their workplace reality, and how it affects their fulfilment professionally, and such information may have policy implications. Therefore, we aimed to investigate Malawian BSc PCH clinical officers' workplace satisfaction, retention and influencing factors. In a mixed-methods, sequential, explanatory design study, we used a quantitative workplace satisfaction survey (five-point Likert scale) covering 11 dimensions, and qualitative in-depth interviews. Analysis included paired t-tests applied to Herzberg's two-factor theory of individual needs dimensions contributing to workplace satisfaction. During data collection in 2022, a total of 27 (93%) clinical officers participated in the survey, and 15 clinical officers and 14 key informants participated in in-depth interviews. BSc PCH clinical officers worked at public district hospitals (n=13), public central hospitals (n=8), and at non-governmental organisations (NGOs) (n=6). Moral satisfaction and workplace/team harmony dimensions scored highest with means of 3.94±0.50 and 3.85±0.69, respectively. In contrast, career advancement and salary and benefits scored lowest with 2.01±0.99 and 2.46±0.80, respectively. Differences existed between clinical officers at public district hospitals compared to public central hospitals for the dimensions of tasks (eg variety, job description clarity) (p=0.034), work environment (p=0.006), and salary and benefits (p=0.032). Similarly, clinical officers at public district hospitals vs NGOs differed for the work environment (p=0.016), management style (p=0.003), and salary and benefits (p=0.002). Seventeen clinical officers considered leaving their current position, with salary being a significant reason (p=0.048). Moreover, delayed recognition and promotion, non-specific job descriptions, limited professional development opportunities, a lack of supervision, and career advancement barriers obstructed workplace satisfaction, according to the in-depth interviews. BSc PCH clinical officers program graduates' workplace satisfaction differs according to the workplace. Graduates working in public hospitals at district level, located mostly in rural areas, appear to be most critically affected and may leave government services. Changes in their specific work environment, career and professional development and remuneration may offer stakeholders opportunities to improve BSc PCH clinical officers' workplace satisfaction and retain them where they are needed most.
The WHO's Integrated Management of Childhood Illness (IMCI) strategy aims to reduce deaths among children under the age of five. Pulse oximetry, a simple non-invasive method for measuring peripheral oxygen saturation - an indicator of severe illness, can enhance IMCI implementation. In Nigeria, despite efforts to incorporate pulse oximetry into IMCI, its adoption is sub-optimal. We assessed healthcare workers' (HCWs) knowledge of pulse oximetry and IMCI in primary and secondary facilities to identify opportunities for improvement. We conducted a cross-sectional study in 54 primary health facilities and 8 secondary hospitals purposively selected across five different states in Nigeria: Oyo, Lagos, Rivers, Kano and Jigawa. The data was collected between 6 March 2025 and 30 June 2025 through an interviewer-assisted questionnaire, which was developed from the national oxygen policy and WHO guidelines. We used descriptive statistics to summarise our findings. Pulse oximetry knowledge scores were dichotomised, and we used bivariate logistic regression to assess associations between HCWs' cadre and pulse oximetry knowledge. For IMCI knowledge, we reported mean scores and used one-way analysis of variance to assess associations between mean scores and the HCWs' cadre. Of the 463 eligible HCWs recruited, 75.4% were female, 52.3% were community health workers (CHWs), and 73.8 and 26.2% were employed by the government and facility, respectively. Overall, 49.7% knew that pulse oximetry should be performed for all presenting patients, with substantial variation across cadres (39.4% among CHWs, 74.4% among doctors, 52.9% among nurses). Doctors had 3.1 times higher odds of having good pulse oximetry knowledge compared to other HCWs (95% CI: 1.17-8.04). Knowledge of IMCI general danger signs was mixed with convulsion (93.5%), lethargy (80.9%) and loss of consciousness (84.6%) being mostly reported as general danger signs compared to vomiting everything and being unable to eat/drink. Less than 10% of the HCWs knew the four main symptoms for assessment in children aged 2-59 months. IMCI Knowledge was generally poor across all HCW cadres, but pulse oximetry knowledge varied substantially. Strengthening pre-service and in-service IMCI and pulse oximetry training, particularly for CHWs and nurses is essential to improve childhood illness management and oxygen therapy practices in Nigeria.
To gain a preliminary understanding of the experiences of children with chronic urinary tract infection (UTI), and their parents, to inform advocacy for greater awareness of paediatric chronic UTI and improvements in its diagnosis and treatment. We conducted semi-structured interviews with the parents of four children (all girls under the age of ten) with chronic UTI symptoms and two adult sufferers whose symptoms began in childhood. Content analysis of the interview transcripts was used to identify key themes. Four key themes were identified: (1) 'diagnostic dismissal and misattribution' (a failure of medical practitioners to recognise and diagnose chronic UTI and their tendency to attribute symptoms to extraneous factors); (2) 'protracted and chaotic care pathways' (difficulty in finding a medical practitioner to appropriately diagnose and treat chronic UTI, and a lack of clear referral pathways); (3) 'impacts on children and families' (negative consequences of chronic UTI for children's health, development and emotional wellbeing, and distress caused to parents due to the child's suffering); and (4) 'the burden of parental advocacy' (parental distress, guilt and exhaustion caused by the struggle to get help for their children). Clinical guidelines, education and care pathways should be developed to help medical professionals recognise and better meet the needs of children with chronic UTI. As well as better UTI diagnostic and treatment approaches generally, child-specific research is required to develop safe and effective treatment protocols for paediatric patients.
This study aims to assess the content validity, face validity and comprehensiveness of the: (a) EQ-5D-5L, EQ-HWB, and ASCOT SCT4, for adults with rare genetic conditions; (b) the EQ-5D-5L, EQ-HWB, and ASCOT-carer for carers of adults or children with rare genetic conditions; and (c) the EQ-5D-Y-5L carer proxy-complete for children with rare genetic conditions. In total, 60 qualitative think-aloud interviews were conducted in Australia and England to understand individuals' thought process during the completion of the QoL measures. Participants were subsequently led through a semi-structured discussion. Transcripts were analysed for whether participants demonstrated understanding of the measures and thematic analysis was conducted on responses to the semi-structured discussion. The majority of participants showed good understanding and supported the validity of the measures for people experiencing rare conditions. For carers, however, a broader evaluative space than health-related QoL was preferred. Several non-health domains were identified as important to both patients and carers, including treatment availability, impact on employment and finance, information and uncertainty, medication and carer burden, impact of passing on a condition, relationships and social connection, and experience with the healthcare system. This study provides some support for the face validity and comprehensiveness of the measures for people experiencing rare conditions. However, several participants felt that the narrow health domains were inadequate to capture the breadth of their lived experience. Future research should explore the extent to which the measures capture differences and changes in the QoL domains identified as important to patients and carers.
Fear of progression (FoP) is a significant psychosocial burden for children with cancer and their parents, influencing their quality of life (HRQoL) and emotional adjustment. In this pilot randomised controlled trial (RCT) we present results on feasibility and preliminary efficacy of the family-based therapeutic intervention "Kinder-Progredienzangst" (KIPA), targeting FoP in pediatric oncology. N = 29 families with a child undergoing acute cancer treatment or follow-up care completed the program, with participation open to parents alone or to parents and children; ultimately, n = 6 children participated. Eligibility criteria included moderate or high FoP in at least one family member. Families were randomised to either an intervention group or a waitlist-control group receiving treatment as usual. KIPA consists of psychoeducation, anxiety confrontation, and resource activation. Participants completed questionnaires for FoP, anxiety, depression, HRQoL, and posttraumatic stress symptoms (PTSS) at different time points. Participation and retention rates were calculated, and we used Mann-Whitney U tests for between-group comparisons, Friedman and Wilcoxon tests for within-group comparisons and Hedges g for effect sizes. Feasibility results showed a participation rate of 23%, with higher participation in acute treatment (51%) compared to follow-up care (15%). Retention rates were 71% overall, with significant variability between settings. Efficacy analyses revealed significant differences in parental FoP between study conditions with high effect size (W = 65.5, p=.023, g=-0.855). Improvements were also noted in PTSS (W = 33, p<.001, g=-1.365), anxiety (W = 59, p=.017, g=-0.906) and mental (W = 217, p<.001, g = 1.619) and physical (W = 180, p=.042, g = 0.834) HRQoL. The pre-post analysis showed a significant reduction in parental FoP in both settings, and the follow-up data indicates sustainability. Effects on children's FoP were not significant. The study showed mixed feasibility-particularly regarding child participation, follow-up enrolment and drop-out during acute treatment-highlighting the need for optimisation before larger trials. Nevertheless, the data provide initial indications that KIPA may benefit parents of children with cancer across both acute and follow-up care settings, a group highly affected by FoP. Given the methodological limitations, the efficacy results should be viewed as preliminary. To demonstrate KIPA's effectiveness, especially in children, large multicentre trials using reliable RCT designs suitable for acute care settings are needed. The trial was retrospectively registered at the German Trial Registry (TRN: DRKS00024106, 04.05.2022).
This study aimed to examine sensory processing patterns, the frequency and impact of parent-reported pain in children who toe walk across a range of diagnoses using validated caregiver-report tools. An online cross-sectional survey was distributed internationally between July 2024 and March 2025. Parents of children who toe walk aged 3-14 years, regardless of diagnosis, completed the Short Sensory Profile (SSP) and the Child Activity Limitations Interview-9 (CALI-9). The survey collected responses from 176 parents of 187 children with toe walking gait. Sensory processing challenges were frequently reported, particularly in children with autism spectrum disorder (98%). Nearly half (48% of 187) of children were reported to experience pain, most frequently in the feet/ankles, lower legs, and hips/thighs. Most parents (94%) and their child's medical professional (81%) attributed their child's pain to toe walking. Parents of children experiencing pain described significantly greater limitations in activities such as sports (OR 3.10, p < 0.001), running (OR 3.46, p < 0.001), and social participation (OR 2.43, p < 0.001). Sensory processing differences and pain were frequently reported among children who toe walk in this sample. Routine assessment of sensory processing and pain in clinical practice may guide targeted interventions to improve participation, function, and quality of life. Emerging evidence suggests that toe walking gait in children may be related to disrupted sensory processing abilities. Parents in this study frequently reported sensory processing challenges among children who toe walked, regardless of the reason for toe walking. Limited research has examined the prevalence or impact of pain in children who toe walk. In this study, almost half of the children were reported to experience pain, which was associated with limitations in activities such as sports, running, and social participation. Routine assessment of sensory processing and pain in clinical practice may guide targeted interventions aiming to improve participation, function, and quality of life.
Child maltreatment is a major global public health concern with well-documented psychological consequences, but its associations with long-term physical health conditions remain less well understood. To examine the associations between childhood maltreatment and adult multimorbidity using electronic health records in Hong Kong. This population-based cohort study used electronic health record data from Hong Kong, comprising 7473 individuals with a first recorded episode of maltreatment between ages 0 and 19 years from 2001 to 2010 and 26 834 matched individuals without documented maltreatment. Individuals who reached adulthood (ie, after age 19 years) by December 31, 2024, were included. History of childhood maltreatment vs none. The primary outcome was the occurrence of multimorbidity during adulthood as documented in participants' diagnostic records through December 31, 2024. Cox proportional hazards models were used to estimate the risks of 16 disease categories and multimorbidity patterns diagnosed after age 19 years, both overall and stratified by preexisting physical, mental, or neurodevelopmental conditions in childhood. After exclusions, the final cohort comprised 32 674 individuals (16 986 female [52%]), including 7137 who were exposed to childhood maltreatment and 25 537 unexposed individuals. Their median (IQR) age was 9.0 (6.0-13.0) years at inclusion and 28.1 (24.2-31.6) years at the end of follow-up. The median (IQR) follow-up duration was 19.0 (16.6-21.3) years. Childhood maltreatment was associated with increased risks across 13 adult diagnostic categories. The highest risks were observed with mental and behavioral disorders (hazard ratio [HR], 2.78; 95% CI, 2.49-3.09), ear diseases (HR, 1.88; 95% CI, 1.27-2.78), injuries (HR, 1.81; 95% CI, 1.65-1.98), and blood diseases (HR, 1.67; 95% CI, 1.41-1.97). Maltreatment was also associated with a higher risk and earlier onset of complex multimorbidity (HR, 1.96; 95% CI, 1.68-2.27). The associations between childhood maltreatment and adult diagnoses were attenuated among individuals with mental health or neurodevelopmental conditions diagnosed in childhood. In this 19-year cohort study of 32 674 individuals, childhood maltreatment was associated with elevated risks for multiple health conditions in adulthood. These findings underscore the need for early prevention and sustained, trauma-informed health care support to reduce the risk of lifelong multimorbidity among children who have experienced maltreatment.
To describe the epidemiological characteristics of children and young people presenting to the emergency department (ED) with acute severe behavioural disturbance (ASBD) who were deemed to require oral sedative medication. Secondary analysis of a randomised controlled open-label multi-centre trial of oral olanzapine versus oral diazepam for the management of ASBD in children and young people aged nine to 17 years for whom epidemiological data were recorded. There were 348 participants enrolled in the randomised controlled trial (RCT). The majority were female (215/348, 62%) with a mean age of 14.6 years (standard deviation 2.2). The most common pre-existing medical or mental health condition was anxiety (122/299, 35%) followed by attention deficit hyperactivity disorder and autism spectrum disorder (33% and 32%, respectively). Two-thirds of the study population (216/348, 62%) had previously attended the ED for ASBD management and 61% (212/348) reported previous intentional self-harm. Nearly three-quarters (247/348, 71%) were accessing psychiatric care in the community prior to their ED presentation. Half of the study population (178/348, 52%) presented to the ED with emergency services (e.g., ambulance, police). The median length of stay in the ED was 5.7 h (interquartile range 3.9-10.2 h) and 28% (98/348) of study participants required admission to hospital. For children and young people presenting to the ED with ASBD who were deemed to require oral sedative medication to assist with behavioural containment, pre-existing mental health disorders were common. There is a need for focussed management procedures for more targeted, trauma-focussed care for children and young people presenting to the ED with ASBD and for support and education in the pre-hospital setting. The primary study (PEAChY-O) was registered with the Australian and New Zealand Clinical Trials Registry (ANZCTR) (ACTRN12621001236886) prior to commencement.
Severe anaemia is a major cause of hospital admission and mortality in children living in sub-Saharan Africa, but data on its association with invasive bacterial infection are sparse. We aimed to characterise severe anaemia and its association with invasive bacterial infection in Kenyan children. In this retrospective observational study, we systematically collected admission data for children aged 1 month to 14 years admitted to Kilifi County Hospital, Kenya, between Aug 1, 1998, and July 31, 2024. We excluded children with missing haemoglobin data. We examined prevalence, trends, and factors associated with severe anaemia and very severe anaemia, and the association between severe or moderate anaemia and odds of bacteraemia. Anaemia severity was defined using WHO age-specific thresholds. Of the 84 348 admissions, representing 64 499 unique children (28 232 [43·8%] females and 36 265 [56·2%] males), included in our analysis, 16 715 (19·8%) admissions had severe anaemia. Severe anaemia was associated with multiple, co-occurring risk factors, including sickle cell disease, malaria, HIV, and malnutrition. As malaria transmission declined (2004-09), the age distribution of severe anaemia shifted towards older children; cases in children aged 5-14 years increased from 16·4% during the high-transmission era (1998-2003) to 44·7% during the low-transmission era (2010-24). Compared with mild or no anaemia, severe anaemia was associated with higher odds of in-hospital mortality (adjusted odds ratio 2·1 [95% CI 1·9-2·3]) and bacteraemia (2·7 [2·5-3·0]), particularly with non-typhoidal Salmonella (6·1 [4·6-8·1]), Escherichia coli (5·7 [4·2-7·7]), Haemophilus influenzae (4·3 [3·0-6·4]), Streptococcus pneumoniae (3·5 [2·9-4·2]), and Klebsiella pneumoniae (2·4 [1·4-4·0]). Moderate anaemia was also associated with increased odds of bacteraemia and mortality. Severe anaemia in hospitalised Kenyan children is multifactorial, increasingly affects older children, and is strongly associated with pathogen-specific bacteraemia and increased mortality. These findings support prompt evaluation for bacteraemia and closer clinical management in severely anaemic children to improve survival. Wellcome and the Science for Africa Foundation to the DELTAS Africa programme.
Separation anxiety disorder (SAD) is a common childhood condition characterised by excessive distress related to separation from attachment figures, often leading to social, emotional, and academic difficulties. If untreated, SAD can increase the risk of future psychopathology, highlighting the importance of effective interventions. This scoping review systematically maps and analyzes the current evidence on intervention strategies to reduce separation anxiety and promote emotional and social well-being in children. Following Arksey and O'Malley's framework and PRISMA-ScR guidelines, a comprehensive search was conducted across multiple databases (PubMed, PsycINFO, Scopus, etc.) for studies published from 2005 to 2025. Inclusion criteria focused on original peer-reviewed research evaluating therapeutic, parental, or educational interventions targeting separation anxiety in children. Eight studies from diverse settings demonstrated that interventions such as child-centred group play therapy, attachment-based play, art therapy, storytelling, laughter yoga, and schedule-based paradoxical therapy effectively reduced separation anxiety symptoms. These approaches also improved social-emotional skills, resilience, and parent-child relationships. Combining therapies and involving parents enhanced outcomes, while the importance of school transition strategies was highlighted. Findings suggest that multimodal, relationship-focused interventions are most effective in alleviating separation anxiety and fostering broader emotional development. Incorporating expressive activities and strengthening attachment bonds are crucial, and tailored, culturally sensitive approaches are recommended. A comprehensive, developmentally sensitive framework integrating therapeutic, familial, and educational strategies holds promise for effective management of childhood separation anxiety. Further research with larger, diverse samples is needed to confirm long-term benefits.
This research aimed to gain an understanding of rheumatic fever related echocardiography experiences from Indigenous Maaori and Pacific children, adolescents and their whaanau (family members) in the Counties Manukau region of Aotearoa New Zealand to improve these services. The study used qualitative Indigenous Kaupapa Maaori consistent and Kakala methodologies. The research was based in the Counties Manukau region of Auckland, New Zealand. Data was collected through interviews with Maaori and Pacific children and adolescents with rheumatic fever or rheumatic heart disease who had recently had echocardiograms and their whaanau. Data was analysed using a general inductive thematic analysis. Seventeen Participants Were Included in the Study. Four Themes Were Identified in the General Inductive Analysis: (1) Communication; (2) Engagement; (3) Access; (4) Kaiaawhina. By identifying barriers and facilitators of echocardiography services for rheumatic fever, this research provided knowledge and insights that can assist in developing child, adolescent and whaanau-focused models of care that have the potential to improve patient experience and echocardiography services in Aotearoa New Zealand.
Dravet syndrome (DS) is a rare, severe developmental epileptic encephalopathy associated with lifelong clinical complexity and substantial caregiving demands. This study describes the quality of life and caregiver burden among family caregivers of individuals with DS across Europe. This cross-sectional, multinational study was conducted as part of the QoL4Dravet - Measuring Quality of Life in Dravet Syndrome project coordinated by the Dravet Syndrome European Federation. 379 family caregivers (322 females and 57 males) completed an anonymous, self-administered online questionnaire from 14 European countries. Caregiving for individuals with DS was associated with a high and sustained burden extending beyond the diagnostic period. Although diagnosis typically occurred early in life, most individuals with DS in this cohort were already adolescents or adults, underscoring the long-term and persistent nature of the disease. This prolonged trajectory was characterised not only by ongoing seizure activity, complex treatment regimens, and frequent need for rescue medication, but also by the continuous management and rehabilitation of associated neurodevelopmental comorbidities, including behavioural and motor difficulties, cognitive and communication impairments. Caregivers reported pronounced impairments in emotional well-being, sleep, fatigue, and stress, while physical functioning was relatively preserved. Only 26.4% of caregivers were employed full-time, whereas 25.6% identified as full-time caregivers, with marked cross-country variation. Women reported poorer emotional well-being, lower autonomy, and poorer social well-being than men, while no sex differences were observed in physical well-being. Significant cross-country differences were found in emotional well-being and autonomy, with poorer outcomes reported in Poland and more favourable profiles in the Netherlands and Germany. Caregiving burden in DS is long-term, multidimensional, and shaped by both gendered caregiving roles and national contexts. These findings underscore the necessity for integrated, gender-sensitive, and policy-informed support strategies that cater to caregivers' psychological, social, and occupational needs throughout their life course.
The morbidity and possible consequent mortality resulting from malaria in children are either due to delayed treatment-seeking or preceded by an inappropriate treatment-seeking process. This study aimed to assess the relationship between the pattern of family dynamics and treatment-seeking behaviour of caregivers of under-five children with uncomplicated malaria. This was a hospital-based cross-sectional study that recruited 350 child-caregiver pairs. An interviewer-administered questionnaire was used for data collection, and it was analysed using the Statistical Package for the Social Sciences (SPSS) software version 25. A p-value of < 0.05 was taken as statistically significant. The family dynamics of the caregivers revealed that 82.3% of them had strong family support, four-fifths (80.3%) had functional families, and a little over half of them (58.3%) had monthly family incomes that fell below the defined household poverty line. Only 41.7% of the caregivers had appropriate treatment-seeking behaviour. Caregivers from lower-income families had 2.5 times higher odds of exhibiting inappropriate treatment-seeking behaviour compared to those from higher-income families (OR, 2.494; 95% CI, 1.486-4.184; p = 0.001). Caregivers with dysfunctional families were about four times more likely to have inappropriate treatment-seeking behaviour compared to their counterparts with functional families (OR, 3.766; 95% CI, 1.445-9.813, p = 0.007). Significant inappropriate treatment-seeking behaviour existed among caregivers of under-five children with uncomplicated malaria. Family factors related to inappropriate treatment-seeking behaviour were family income and family functioning. Strengthening the family and counselling on appropriate treatment-seeking is crucial to reducing malaria morbidity and mortality among under-five children.
Sudden Unexplained Death in Youth (SUDY) requires thorough investigation to identify underlying causes and guide prevention strategies. In the Netherlands, cases are investigated using the standardized Postmortem Evaluation of Sudden Unexplained Death in Infants and Children (PESUDIC), in which autopsy is offered as a standard component. However, its invasive nature and associated time burden may limit parental acceptance. This study evaluated to what extent the cause of death can be established using a limited set of diagnostic tests compared to the standard procedure including autopsy. In this observational study, children > 2 years of age who died suddenly and unexpectedly and underwent PESUDIC, including imaging and autopsy, were included. Two expert panels, consisting of a forensic and a pediatric specialist, independently assessed early diagnostic tests available before autopsy. Cases were classified by level of diagnostic certainty and need for autopsy. Panel conclusions were compared with the reference standard: a multidisciplinary audit incorporating all available information, including autopsy findings. Sixty-six cases were included (median age 12 years (IQR 4-14.7), 63% male). Panels identified indicative information for a cause of death in 60 patients (91%). Nevertheless, autopsy was required in most cases (n = 59) to confirm the diagnosis. In 7 cases (10.6%), panels were sufficiently confident to establish the cause of death without autopsy with complete agreement with the reference standard. Causes included obstructive gastrointestinal pathology (n = 5) and diabetic ketoacidosis with dehydration (n = 2).  Only a small proportion of children > 2 years of age with sudden unexpected death have a cause of death that can be established with sufficient certainty at an early stage. In the vast majority of cases, the cause of death remains uncertain, supporting the recommendation to perform an autopsy. • Autopsy in sudden unexplained death in youth is worldwide recognized as the gold standard for postmortem examination. However, its invasive nature and associated time burden may limit parental acceptance. • In 7 of 60 sudden and unexplained deceased minors (10%), sufficient certainty regarding the cause of death can be obtained by other minimal invasive diagnostic test, so without the need of an autopsy. Causes without the need of an autopsy included obstructive gastrointestinal pathology and diabetic ketoacidosis with dehydration.
Functional assessment of children and adolescents is essential for monitoring motor development and for the prevention of deficits that may impact health and quality of life. In primary care contexts, the implementation of simple and cost-effective tests has been a pragmatic strategy, particularly in low- and middle-income countries. The sitting-rising test is noteworthy for its clinical and community relevance, although normative values for paediatric populations remain unavailable. To establish reference values for the Sitting-rising Test in children and adolescents aged 5-14 years, to investigate the influence of body mass index on functional performance, and to compare results between sexes. A cross-sectional study was conducted with 596 schoolchildren (aged 5-14 years) assessed using the Sitting-rising Test and anthropometric measures. Reference values were defined by age-specific percentiles. The association between body mass index and performance was analysed using linear regression, and sex differences were tested with the chi-square test. Sitting-rising Test performance was classified into three categories: below expected (≤ P10), adequate (> P10 and ≤ P90) and above expected (> P90). Body mass index accounted for 17.4% of the performance variation, indicating that greater body composition was associated with poorer test results. No significant sex differences were observed. This study provides, for the first time, normative Sitting-rising Test values for children and adolescents aged 5-14 years. The findings highlight the SRT as a simple, low-cost and effective tool for early detection of functional deficits, especially in resource-limited settings.
Infants exposed to alcohol and/or drugs (AoD) in utero are at considerable risk for developmental complications which are amenable to early intervention. There are no published guidelines in Australia for the allied health care of infants and toddlers (0-2 years) who have been exposed to AoD in utero. This study aimed to explore the current service delivery and follow-up health services available across Australia for this high-risk population group. A custom-designed survey was distributed via email to the leading contact of hospitals based in Australia that offered a neonatal-based follow-up service (n = 43). Reminders to prompt completion were emailed out at regular intervals over a two-month period. A total of 15 of 43 invited services (35%) provided responses with only 3 of 15 (20%) offering targeted care for infants exposed to AoD and 5 (33%) using any formal developmental or screening tools with this population. Responses indicated inconsistencies with allied health involvement including professions involved and time points for follow-up care. Funding was highlighted as a key barrier to conducting follow-up assessments, with 12 of 15 (80%) health services reporting they received no dedicated funding for this population. Within the responding services, our data reflects the lack of consensus on any standardised process for assessment or follow-up service delivery for this high-risk patient population. Although the response rate was low, it is consistent with previous health professional survey research. There remains a need for further research and the development of clinical care guidelines on best standard protocols for assessing and supporting the development of infants exposed to AoD in utero.
Intermittent skin-to-skin care (SSC) during phototherapy for neonatal jaundice improves breast milk consumption and reduces enterohepatic circulation. The primary objective of this study was to evaluate the effect of SSC on the rate of fall in bilirubin in neonatal jaundice. The secondary objectives were maximum TSB, duration of phototherapy, duration of hospital stay, weight gain, feeding and stooling pattern. In this randomised controlled trial, otherwise healthy late preterm and term neonates of birth weight ≥ 2 kg, who required phototherapy (AAP 2004 guidelines) and if the mother was available for giving SSC, were included. The intervention group received 2 h of SSC every 8 h while on phototherapy. The control group received only phototherapy. At the time of discharge, a feedback questionnaire was used to objectively assess the mother's experience. Of the 88 neonates enrolled (44 in SSC group; 44 in control group), the rate of fall of bilirubin was similar between the groups (0.18 mg/dL/h in SSC vs. 0.19 mg/dL/h in control; p = 0.49). The duration of phototherapy in hours (34.76 vs. 38.54; p = 0.19) and duration of hospitalisation in hours (38.8 vs. 42.0; p = 0.28) were also similar. The median total feedback questionnaire score was 37 for a maximum score of 40, indicating high maternal satisfaction and bonding. Intermittent skin-to-skin care during phototherapy did not affect the rate of fall of bilirubin. SSC is safe during phototherapy and is associated with high maternal satisfaction and bonding during the hospital stay. Clinical Trial Registry-India, www.ctri.nic.in: CTRI/2023/04/051798.
Stimulant medications dexamphetamine and methylphenidate are well-established for the treatment of Attention Deficit Hyperactivity Disorder (ADHD), but few studies directly compare their efficacy. We aimed to compare the therapeutic and adverse effects of immediate-release formulations of dexamphetamine and methylphenidate during the initial treatment of children with ADHD. This open-label study was conducted in public and private specialist paediatric clinics in New South Wales, Australia, between 2016 and 2020. One hundred stimulant-naïve children and adolescents with DSM-5-confirmed ADHD were randomised 1:1 to dexamphetamine or methylphenidate. ADHD diagnosis was based on detailed clinical assessment supported by parent and teacher DSM-5-based ratings. Dose titration followed a standardised 4-week weight-based protocol monitored with weekly school-based IOWA Conners ratings, after which dose adjustment or switching was made as clinically indicated. Participants were followed for 12 months. Study participants (mean age 9.08 [SD 2.87] years; range 4.5-16.4 years; 73% male) showed significant ADHD symptom improvement on either medication at all time periods (p = 0.001), with no significant between-group difference on repeated-measures ANOVA. At 12 months, 62 participants remained on their allocated medication, with no apparent preference between methylphenidate and dexamphetamine (34/50 [68%] vs. 28/50 [56%], p = 0.15). Both medications were associated with weight loss which was greater on dexamphetamine at 3 months (-1.44 [SD 2.42] kg vs. -0.31 [SD 0.97] kg; p = 0.005). Stimulant medications dexamphetamine and methylphenidate appear comparable in short-term symptom response and 12-month persistence after initial randomised allocation, but dexamphetamine may be associated with greater weight loss. Australian New Zealand Clinical Trials Registry number: ACTRN12616000569404.
The aim of this project was to develop and implement a working definition and ethical framework for use of innovative medicine in contemporary Australian paediatric practice. A mixed methods research methodology was undertaken using a Delphi process to establish a definition of innovative medicines in paediatric patients. This definition was used to quantitate innovative medicines use in a retrospective quantitative review of non-formulary medications in a tertiary paediatric hospital. An existing ethical framework from the United Kingdom was adapted to supplement standard clinical governance processes in contemporary Australian paediatric practice. A definition of innovative medicines and an ethical framework for their use in paediatric patients was developed after four rounds of feedback from 18 Australian paediatric experts. A total of 2906 individual patient medication approval applications were reviewed, 1447 of these were included for analysis, 120 of which were for medications not on the Australian Register of Therapeutic Goods and six applications were considered innovative using the newly developed definition. Evaluation of the use of innovative medicines in paediatric patients is essential to ensure patient safety and critical clinical governance of medication use. An adapted ethical framework for Australian practice is a valuable tool to assist clinical governance processes.