van Gelder EM, Temple SEL, Jefferson N
… +5 more, Brown D, Stratton-Gadke K, Rich M, Rose S, Blake K
J Paediatr Child Health
· 2026 May · PMID 42219920
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BACKGROUND: CHARGE syndrome (CS) is a rare multisystemic genetic condition caused by a pathogenic variant in the DNA-binding protein-7 CHD7 gene. The condition affects the development of neural crest cells, which give ri...BACKGROUND: CHARGE syndrome (CS) is a rare multisystemic genetic condition caused by a pathogenic variant in the DNA-binding protein-7 CHD7 gene. The condition affects the development of neural crest cells, which give rise to craniofacial structures, cranial nerves, ears, eyes and the heart, resulting in diverse and complex clinical features. CS is the most common genetic cause of congenital deafblindness. PURPOSE: This narrative review provides an updated and systematic approach to diagnosis, assessment and management of individuals with CS. METHODS: A broad literature search was performed using PubMed/MEDLINE, Embase and CENTRAL with search terms 'CHARGE syndrome' and 'CHD7'. No time restriction was placed on the literature search; however, we particularly focused on new/emerging evidence from within the last 10 years. Published evidence was supplemented by expert opinion from authors with extensive experience caring for individuals with this rare condition. RESULTS: The important role of cranial nerve dysfunction and its relevance to clinical features is explored. The review highlights several important aspects of CS which have significant impacts on quality of life and long-term health outcomes but may be missed, including vestibular function and balance, gastrointestinal dysmotility, bone health, pain and postural orthostatic tachycardia. Aspects of neurodevelopment and mental health are reviewed, with a focus on cognitive assessment and anxiety disorders and a multimodal approach to communication. Issues relating to adolescence and the transitional period are discussed, highlighting the importance of support groups and resources.
Mahar PD, Lee T, Orchard D
… +30 more, Gray A, Scardamaglia L, Robertson S, Bertinetti M, Teague W, Nizzero D, Grills C, Toholka R, Bao D, Kelly J, Grindlay J, Rozen T, Oliver M, Alexander N, Fernando A, Matthew A, Chan K, Grover SR, McDonald E, Wong LC, Rademaker M, Purvis D, Dear K, Weston S, Morgan V, Halbert A, Wargon O, Fischer G, Mewton E, King E
J Paediatr Child Health
· 2026 May · PMID 42219898
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BACKGROUND: Paediatric Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN) is a rare, severe mucocutaneous reaction requiring coordinated multidisciplinary care. Existing guidelines provide evidence-based recom...BACKGROUND: Paediatric Stevens-Johnson syndrome/toxic epidermal necrolysis (SJS/TEN) is a rare, severe mucocutaneous reaction requiring coordinated multidisciplinary care. Existing guidelines provide evidence-based recommendations, but implementation across tertiary paediatric hospitals requires practical consensus on local resources. AIM: To develop and evaluate multidisciplinary consensus-informed practical clinical guidance for assessment and management of paediatric SJS/TEN among clinicians from Australasian tertiary referral hospitals. METHODS: A draft guidance document was developed after review of SJS/TEN guidelines, systematic reviews and other relevant literature. Clinicians from one tertiary paediatric centre (Group A) and other Australian and New Zealand tertiary centres (Group B) completed a 130-item questionnaire. Continuous items were scored on a 1-9 Likert scale. Categorical items addressed referral urgency and whether investigations should be routine, reasonable but not routine, or not ordered. Quantitative responses and free-text comments informed revision. RESULTS: Twenty-two multidisciplinary clinicians from the internal centre and nine clinicians from eight external centres participated. Agreement was high for history, examination, supportive care, multidisciplinary involvement, discharge planning and follow-up. Uncertainty remained regarding fluid requirements compared with burns patients, the scope of initial investigations and systemic immunomodulatory therapy. The guidance was revised to emphasise individualised fluid management, directed differential diagnosis and testing, and case-by-case systemic therapy. CONCLUSIONS: This practical multidisciplinary consensus-informed guidance supports paediatric SJS/TEN care in Australasian tertiary referral hospitals. Its contribution is the quantified consultative process and identification of consistent, variable and uncertain practice. It complements, rather than replaces, existing guidelines.
Sarkies M, Srinivasan S, Horton A
… +7 more, Garton-Smith J, Watts GF, Tawtel C, Della-Vedova J, Nowak KJ, Pang J, Martin A
J Paediatr Child Health
· 2026 May · PMID 42219895
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OBJECTIVES: Familial hypercholesterolaemia (FH) affects 1 in 311 people. Left untreated, it elevates LDL-cholesterol from early life and drives premature atherosclerotic cardiovascular disease. Despite paediatric guideli...OBJECTIVES: Familial hypercholesterolaemia (FH) affects 1 in 311 people. Left untreated, it elevates LDL-cholesterol from early life and drives premature atherosclerotic cardiovascular disease. Despite paediatric guidelines for early lipid-lowering, detection and management in Australia remain suboptimal. Shared-care models across general practice, paediatrics, and FH-specialist services are promising but under-implemented. We co-developed implementation strategies to support a paediatric FH risk-reduction pathway. METHODS: Two qualitative focus-group workshops with clinical, policy and advocacy stakeholders were conducted. Transcripts underwent inductive and deductive thematic analysis. Barriers and facilitators were mapped to the Consolidated Framework for Implementation Research and strategies to the Expert Recommendations for Implementing Change, with structured specification of actor, action, target, temporality and dose. RESULTS: Barriers included unclear responsibility across settings, limited clinician confidence, constrained access to specialist dietary advice and treatment non-adherence. Facilitators included patient/family empowerment, existing financial reimbursement and engaged clinical champions. Nine strategies emerged: (1) treatment-guidance letters; (2) coordination and phone support; (3) individualised patient passports; (4) financial reimbursement; (5) diet and lifestyle resources; (6) patient and family engagement; (7) decision support systems; (8) audit and feedback; and (9) development of clinical champions. CONCLUSIONS: A co-designed suite of nine strategies could enable shared-care for paediatric FH in Australia. This structured, replicable package is adaptable across jurisdictions and can support earlier detection and improved management. Evaluation of effectiveness and scalability in routine care is warranted, with potential relevance to other paediatric lipid disorders.
Williamson A, Sudbury E, Khanal R
… +3 more, Woon J, Schaefer J, Paxton G
J Paediatr Child Health
· 2026 May · PMID 42216805
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AIM: To examine refugee health screening and services for Afghan children in the unique context of emergency expedited humanitarian resettlement in Melbourne, Australia. METHODS: Retrospective audit of Afghan children wh...AIM: To examine refugee health screening and services for Afghan children in the unique context of emergency expedited humanitarian resettlement in Melbourne, Australia. METHODS: Retrospective audit of Afghan children who attended a specialist child refugee health service between August 2021-April 2024. RESULTS: Participants included 218 children aged 2 months-18 years, 57.8% male. Most (82.6%) were part of an intact family unit, 11.5% were unaccompanied minors. At first clinic visit, more than half held a temporary humanitarian visa with variable medicare access, and 51.4% were in hotel quarantine or short-term accommodation due to the Covid-19 pandemic. Only 17.4% children reported offshore healthcare related to travel. While 65.1% children were linked with a general practitioner, 90.8% still required refugee health screening and 92.7% required catch-up vaccinations. The most common health issues were dental caries (28.4%), sleep concerns (17.0%), infections (14.2%) and developmental concerns (15.1%). Twenty children (9.2%) required hospital admission in the early post-arrival period, 10.1% had intellectual disability and 8.7% had complex physical disability. Refugee health screening identified high prevalence of vitamin D deficiency (64.7%), B12 deficiency (44.7%), iron deficiency (29.5%) and anaemia (25.5%), and a range of rare diagnoses and communicable diseases. CONCLUSIONS: Refugee health screening remains essential in post-arrival healthcare, allowing early identification and treatment of infections and micronutrient deficiencies and support for complex cases. Fragmented systems for refugee health screening in Melbourne are not meeting this need, and there is a strong case for specialist paediatric screening given the range of medical, developmental, disability, educational and social needs identified.
Kapoor V, Skellern C, McGrath P
… +3 more, Brosnan S, Gofer Y, Thorburn L
J Paediatr Child Health
· 2026 May · PMID 42200293
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AIM: To evaluate the impact of an artificial intelligence medical scribe (AIMS) on clinical documentation efficiency, document quality, clinician-patient interaction, clinician well-being, consumer perspectives, overtime...AIM: To evaluate the impact of an artificial intelligence medical scribe (AIMS) on clinical documentation efficiency, document quality, clinician-patient interaction, clinician well-being, consumer perspectives, overtime, and transcription costs. METHODS: Retrospective-prospective study of AIMS project cohort including medical, allied health and nursing practitioners at a quaternary children's hospital and health service (HHS). Outcome measures included time-to-finalise outpatient correspondence, clinicians' Reflections survey (1 month), document quality survey using modified Physician Documentation Quality Instrument-9 tool's domains (3 months), System Usability Scale (SUS) (3 months). Patient/consumer experience surveys, overtime and cost analyses. RESULTS: Total 131 participants including 89 medical, 36 allied health and 6 nurse practitioners used AIMS showing significant reduction in median time-to-finalise outpatient letters from 7.9 days (Interquartile range [IQR] 19 days) in pre-AIMS era to 14 min (IQR 6 days) (p < 0.001). In the Reflections survey participants reported improved: patient engagement (62/71, 87%), well-being (80%, 57/71) and work-life balance (66%, 47/71). The document quality survey showed acceptable document quality with median score 4, in 9 of the 10 survey domains (scale 1-to-5, n = 77). Mean SUS score was 74 (n = 77) suggesting above average usability. Study showed transcription cost savings of 16 903 AUD, but no definitive impact on overtime claims. Patient/consumer feedback (n = 333) was highly positive with over 90% reporting improved clinician-patient interaction and welcoming future use of AIMS. CONCLUSIONS: This study showed significantly improved documentation efficiency, acceptable document quality, and reduced transcription costs with significant benefits to clinician well-being and clinician-patient interaction with the use of an ambient AI medical scribe. TRIAL REGISTRATION: Australian New Zealand Clinical Trials Registry: ACTRN12625000432415.
Colligan T, Oei JL, Bajuk B
… +10 more, Burns L, Lawler K, Uebel H, Eastwood J, Page A, Lee E, Dicair L, Green C, Dickson M, Dronavalli M
J Paediatr Child Health
· 2026 May · PMID 42186151
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OBJECTIVE: Remote residents have worse health outcomes than metropolitan residents, but whether geography impacts the outcomes of children with prenatal drug exposure (PDE) is uncertain. DESIGN AND MAIN OUTCOME MEASURES:...OBJECTIVE: Remote residents have worse health outcomes than metropolitan residents, but whether geography impacts the outcomes of children with prenatal drug exposure (PDE) is uncertain. DESIGN AND MAIN OUTCOME MEASURES: Linked population data was used to compare rates of death, hospitalisation, emergency department (ED) encounters and placement in Out-of-Home Care (OOHC) for children with (n = 208 492) and without (n = 1 607 662) smoking, alcohol or substance exposure in NSW, Australia, born between 2001 and 2020. The relationship between remoteness of residence, PDE, service utilisation and death was determined with interaction terms after adjusting for confounders (First Nations status, maternal age, maternal mental illness, social disadvantage). Outcomes are reported as adjusted incidence rate ratios with 95% confidence intervals. RESULTS: Compared to other NSW children, children with PDE were more likely to die (2.90; 2.33-3.61), be placed in OOHC (52.43; 50.14-54.84), be hospitalised (1.22; 1.10-1.36) and attend ED (1.06; 1.03-1.09). Compared to metropolitan children with PDE, regional children were more likely to die (1.12; 0.78-1.61) but less likely to be hospitalised (0.82; 0.72-0.94), placed in OOHC (0.41; 0.37-0.44) and attend ED (0.93; 0.89-0.98). ED encounters increased up to 2.5-fold for regional children with PDE from 2001 to 2020. CONCLUSIONS: Children with PDE in regional areas are likely to use fewer hospital resources than their metropolitan counterparts, but are more likely to die, mainly from preventable causes. Equitable access to preventative health and psychosocial support must be prioritised to improve outcomes of these children.
Irie MN, de Menezes Jarry V, de Lima Ruffini M
… +5 more, de Souza AM, Dalaqua M, Duarte JÁ, Fraga A, Reis F
J Paediatr Child Health
· 2026 May · PMID 42186144
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BACKGROUND: Abusive head trauma (AHT) remains the leading cause of fatal traumatic brain injury in infants and young children. Diagnosis is frequently delayed or missed due to nonspecific clinical presentation and unreli...BACKGROUND: Abusive head trauma (AHT) remains the leading cause of fatal traumatic brain injury in infants and young children. Diagnosis is frequently delayed or missed due to nonspecific clinical presentation and unreliable histories, making neuroimaging a critical component of the diagnostic process. OBJECTIVE: This review synthesises computed tomography (CT) and magnetic resonance imaging findings (MRI) associated with abusive mechanisms and highlights patterns that may support clinical suspicion, multidisciplinary decision making and child protection interventions. METHODS: A narrative review of characteristic imaging features in AHT was conducted, focusing on the radiologic appearance of retinal haemorrhages, subdural hematomas, subarachnoid and epidural haemorrhages, bridging vein thrombosis, spinal subdural haemorrhages, hypoxic-ischemic damage, parenchymal lesions and skull injuries. RESULTS: Subdural hematoma was the most frequently reported imaging finding, often bilateral or asymmetric, typically involving the high convexities, the interhemispheric fissure and the posterior fossa. Additional findings commonly associated with AHT included retinal haemorrhages, bridging vein thrombosis, spinal subdural haemorrhage, HII injury, hemispheric hypodensity, traumatic axonal injury and complex skull fractures. MRI demonstrated superior sensitivity compared with CT for detecting small haemorrhages, early parenchymal injury and axonal damage. CONCLUSION: Recognition of characteristic neuroimaging patterns plays a central role in the evaluation of suspected AHT. Early identification by radiologists and paediatric clinicians is critical not only for guiding acute management but also for activating multidisciplinary child protection responses and preventing recurrent injury, long-term neurological impairment and fatal outcomes.
J Paediatr Child Health
· 2026 May · PMID 42186127
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AIM: To evaluate adherence to the Sydney Children's Hospital Network (SCHN) Asthma-Acute Management Practice Guideline in the management of paediatric patients presenting to a metropolitan emergency department (ED). METH...AIM: To evaluate adherence to the Sydney Children's Hospital Network (SCHN) Asthma-Acute Management Practice Guideline in the management of paediatric patients presenting to a metropolitan emergency department (ED). METHODS: A cross-sectional retrospective audit of 153 paediatric ED presentations with acute asthma between March 2023 and February 2024 was conducted. Data extracted from electronic medical records included demographics, documented asthma severity, investigations, treatment, length of stay and discharge planning. Asthma severity was inferred retrospectively from documentation, which was often incomplete, limiting interpretation of patient management. Outcomes were compared with SCHN guideline recommendations. Data were analysed descriptively using means and proportions, with subgroup comparisons by admission status and severity classification. RESULTS: Of 153 presentations, 56 (36.6%) resulted in admission. Documentation of key severity parameters was frequently incomplete, including speech (78%) and cyanosis (69%). Chest X-rays were performed in 34.6% of cases. Mean bronchodilator burst duration exceeded guideline recommendations (salbutamol 46.7 min; ipratropium 47.7 min). Systemic corticosteroids were administered in 92% of patients, with appropriate dosing in 63%. Among 27 severe/life-threatening cases, 8 (29.6%) received intravenous magnesium sulphate. Mean ED length of stay was longer in admitted patients than in discharged patients (6.5 vs. 4.3 h). CONCLUSIONS: Variation between local practice and SCHN guideline recommendations was identified in severity assessment, pharmacological management, imaging utilisation and discharge planning. Targeted quality improvement strategies, including implementation of ED short-stay models, structured electronic severity assessment tools and staff education initiatives, may support improved adherence to guideline-based care.
J Paediatr Child Health
· 2026 May · PMID 42178670
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AIM: Preterm birth is a major risk factor for attention-deficit/hyperactivity disorder (ADHD). Early recognition is crucial for academic and social development. Preschool identification remains challenging as it relies h...AIM: Preterm birth is a major risk factor for attention-deficit/hyperactivity disorder (ADHD). Early recognition is crucial for academic and social development. Preschool identification remains challenging as it relies heavily on parental observation. This study aimed to evaluate parental knowledge of ADHD and recognition of preschool ADHD symptoms in a high-risk preterm population. METHODS: Parents of children born preterm attending a tertiary outpatient clinic (January 2022-March 2023) completed a questionnaire in this cross-sectional study. The tool, validated by child psychiatry experts, assessed general ADHD knowledge and recognition of preschool manifestations. Responses were scored (correct = 1; incorrect/unsure = 0) and converted to a 0-100 scale. RESULTS: A total of 138 parents participated; 14% reported a family history of ADHD. Most participants had a university-level education (61%) and were employed (66%). The median total symptom recognition score was 38 (P25-P75: 19-52). Recognition was highest for hyperactivity [50 (0-50)] and lowest for impulsivity [25 (12-50)]. The median general ADHD knowledge score was 14 (14-28). Parents with a family history of ADHD showed significantly higher inattention recognition scores (p = 0.035), though total symptom recognition did not differ by family history (p = 0.24). General ADHD knowledge varied by occupation (p = 0.01), with teacher-parents scoring highest (p = 0.004). The overall model predicting general ADHD knowledge was significant (p = 0.020) in multivariable linear regression, but no single independent predictor remained significant. CONCLUSION: Recognition of preschool ADHD symptoms, particularly impulsivity, is low among parents of children born preterm. Targeted educational interventions are required to enhance early identification and improve developmental outcomes.
J Paediatr Child Health
· 2026 May · PMID 42169436
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AIM: To quantify the long-term associations between adolescent fertility (AFR), maternal mortality (MMR), immunization coverage, and under-five mortality (U5MR) across countries and regions. METHODS: A strongly balanced...AIM: To quantify the long-term associations between adolescent fertility (AFR), maternal mortality (MMR), immunization coverage, and under-five mortality (U5MR) across countries and regions. METHODS: A strongly balanced panel of 187 countries (1992-2023) was constructed using World Bank and WHO data. The primary outcome was U5MR (deaths before age five per 1000 live births). Exposures included AFR (births per 1000 women aged 15-19), MMR (per 100 000 live births), full immunization (%), and measles (MCV1) coverage (%). Two-way panel regression models with country and year effects were estimated under fixed and random effects, guided by the Hausman test. Inference used Driscoll-Kraay or cluster-robust standard errors. Robustness checks incorporated lagged covariates, correlated random effects, common-correlated-effects models, and a vaccination composite. RESULTS: Globally, higher AFR and MMR were consistently linked to increased U5MR, while full immunization was protective. Each 10-per-1000 increase in AFR corresponded to about 4.6 additional under-five deaths per 1000, each 10-percentage-point increase in full immunization to 4.9 fewer, and each 100-per-100 000 increase in MMR to 8.8 more. Measles coverage showed additional protective effects in Asia and the Americas. AFR was the strongest predictor in Africa, while MMR was a near-universal predictor across regions. CONCLUSION: U5MR remains strongly tied to adolescent fertility, maternal survival, and immunization. Region-specific, equity-focused interventions-expanding adolescent reproductive health services, high-quality maternal care, and comprehensive immunization (including at least 95% two-dose measles coverage)-are essential to accelerate progress towards Sustainable Development Goal 3.2.
Hamed O, Shrestha S, Bhurawala H
… +1 more, Poulton A
J Paediatr Child Health
· 2026 May · PMID 42157431
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AIM: To examine the predictive value of specific concerns documented in paediatric referral letters for subsequent diagnoses of autism spectrum disorder (ASD) and global developmental delay (GDD) within a tertiary child...AIM: To examine the predictive value of specific concerns documented in paediatric referral letters for subsequent diagnoses of autism spectrum disorder (ASD) and global developmental delay (GDD) within a tertiary child development clinic. METHODS: A retrospective audit was conducted of consecutive children assessed at the Nepean Hospital Child Development Clinic between 2015 and 2019. Referral letters, supporting documents, and medical records were reviewed to identify referral concerns and diagnostic outcomes. Chi-square analyses were used to explore associations between referral concerns and final diagnoses. RESULTS: Among 161 children assessed, 68% were under 5 years of age, and 78% were male. Speech delay (80%) and social interaction concerns (78%) were the most frequently documented referral issues. Following multidisciplinary assessment, 51% of children referred for ASD were diagnosed with ASD, and 57% referred for developmental concerns were diagnosed with GDD. Speech delay was the only referral concern significantly associated with an ASD diagnosis (p = 0.002). Behavioural concerns predicted attention deficit hyperactivity disorder (ADHD) and GDD (p < 0.001), while motor delay predicted GDD (p < 0.05). ADHD, although not a primary focus of the clinic during the study period, emerged as a clinically relevant co-occurring outcome. Ten percent of children received no neurodevelopmental diagnosis. CONCLUSIONS: Referral concerns varied widely in their diagnostic utility. Speech delay demonstrated the strongest predictive value for ASD, while motor delay and behavioural problems were associated with GDD. Other commonly cited concerns lacked diagnostic specificity. These findings highlight the clinical value of detailed symptom-based referral information and support the role of early speech pathology assessment in improving triage and prioritisation for ASD within tertiary developmental services.
J Paediatr Child Health
· 2026 May · PMID 42153406
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BACKGROUND: As parents increasingly seek information about child health on digital platforms, the risk of misinformation has also risen. AIMS: This study aimed to evaluate the performance of large language models (LLMs)...BACKGROUND: As parents increasingly seek information about child health on digital platforms, the risk of misinformation has also risen. AIMS: This study aimed to evaluate the performance of large language models (LLMs) in identifying, correcting and generating guideline-adherent, evidence-based responses to paediatric misinformation. METHOD: Twenty common paediatric misinformation statements across nine thematic areas were identified based on literature review and expert opinion. These statements were presented to four different LLMs (ChatGPT, Gemini Advanced, Claude, Microsoft Copilot) with three repetitions each. The generated responses were evaluated by one physician and two nurses in terms of accuracy, guideline adherence, explanation quality and risk. RESULTS: ChatGPT and Gemini demonstrated the highest and most consistent performance in accuracy and guideline adherence. Claude showed some deficiencies in certain explanations, while Copilot exhibited lower performance in guideline adherence and explanation depth compared to the other models. Risk scores were low across all models, and no hazardous content was observed. Furthermore, the models' abilities to correct misinformation varied in terms of guideline-compliant explanations and risk communication. CONCLUSION: Within the scope of the evaluated paediatric misinformation statements and current model versions, LLMs may provide conditionally safe and guideline-aligned information for parents. However, professional oversight and adherence to evidence-based paediatric guidelines remain essential. This study systematically highlighted the potential applications and limitations of LLMs in digital health safety and paediatric nursing practice.
Ludlow AK, Anderson S, Robinson S
… +2 more, Owen T, Hedderly T
J Paediatr Child Health
· 2026 May · PMID 42152473
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AIM: This study aimed to analyse the pathways to obtaining a diagnosis of functional tic-like behaviour (FTLBs) in the United Kingdom. METHODS: The primary caregivers of children with FTLB (n = 55) were recruited through...AIM: This study aimed to analyse the pathways to obtaining a diagnosis of functional tic-like behaviour (FTLBs) in the United Kingdom. METHODS: The primary caregivers of children with FTLB (n = 55) were recruited through charities and completed an anonymised survey. The authors established the pathway to any diagnosis, gaining information about their child's tics, their impact and the sequence of health-care professionals (HCPs) they had contacted that had either led or not led to a diagnosis and any support on offer. Mixed-method analyses were conducted on the surveys. RESULTS: Many of the characteristics of the children met the criteria for FTLBs, with over half of the children (n = 30; 55%) also showing self-harming and suicidal ideation behaviour. Mothers reported often finding themselves seeing more than three different services and being given differing labels for their child's symptoms (e.g., transient tics and PANDAS). There was little clear guidance on how to manage symptoms, with those who were able to access treatment offered CBT, counselling and/or medication. There were several families still waiting to be seen (n = 13; 24%); however, of those who had received a diagnosis, almost one-third had accessed support from private healthcare providers (n = 14; 25%). CONCLUSIONS: Pathways to diagnosis for young people showing FTLBs are complex, time-consuming and can involve multiple health-care contacts, with patients alternating between primary and specialised care. Our findings reflect failures in the diagnostic protocols and referral systems of health care in the United Kingdom and a general lack of understanding, specialist skill or knowledge relating to FTLBs.
J Paediatr Child Health
· 2026 May · PMID 42144874
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AIM: The role of systemic therapy in paediatric Stevens-Johnson Syndrome (SJS) and Toxic Epidermal Necrolysis (TEN) remains undefined. Management is primarily guided by observational data and variable practice patterns....AIM: The role of systemic therapy in paediatric Stevens-Johnson Syndrome (SJS) and Toxic Epidermal Necrolysis (TEN) remains undefined. Management is primarily guided by observational data and variable practice patterns. This systematic review aims to integrate recent evidence and evaluate clinical outcomes associated with systemic therapies in paediatric SJS/TEN. METHODS: A systematic search of PubMed, Embase and Scopus was conducted from January 1990 to February 2025. Studies were eligible if they included patients (< 18 years) with SJS, SJS-TEN overlap or TEN managed with supportive care alone or systemic therapy (corticosteroids, intravenous immunoglobulin (IVIg), cyclosporin, TNF-α inhibitors or plasmapheresis). Studies without defined diagnoses or outcomes were excluded. Primary outcomes were mortality and length of hospital stay. Risk of bias was assessed using CARE guidelines for case reports and the Joanna-Briggs checklist for observational studies. Studies providing individual-level data were synthesised quantitatively, while aggregated data were described narratively. RESULTS: A total of 260 studies comprising 1608 paediatric patients met inclusion criteria. Most patients received systemic therapy, most commonly corticosteroids or IVIg. Systemic therapy was not associated with a shorter length of stay or reduced mortality rate compared to supportive care alone. Treatment with cyclosporin and TNF-α inhibitors trended towards lower mortality rates, and for cyclosporin, shorter length of stay. However, these findings did not reach statistical significance. CONCLUSIONS: Evidence for systemic therapy remains limited by study heterogeneity and small sample sizes. Although emerging targeted therapies show early promise, firm conclusions regarding efficacy cannot be drawn. This highlights the need for further collaborative research.