Archive

Infantile-Onset Pompe Disease: Current Therapeutic Landscape, Clinical Challenges, and Future Horizons

No trial data reported. Conventional enzyme replacement therapy improves cardiac function, motor development and survival, but is limited by lifelong intravenous infusions, immune responses and poor blood-brain barrier passage.

Paediatrics · Cureus · 22 Sept

Histopathology Versus Imaging: A Comparative Study of Liver Biopsy, Ultrasonography, and Triphasic Computed Tomography in Paediatric Patients With Chronic Liver Disease

In children with chronic liver disease, ultrasound and triphasic CT correlate imperfectly with biopsy, so imaging alone may understage fibrosis and delay referral.

Paediatrics · Cureus · 22 Sept

Severe Pulmonary Hypertension in Critically Ill Children: A 10-Year Single-Center Case Series and Practical Management Considerations

All four presented with respiratory distress, hypoxaemia and cardiovascular compromise after recent respiratory infection. Median PICU stay 15 days (range 10-24). Two survived to discharge; two died of refractory pulmonary hypertensive crises.

Paediatrics · Cureus · 22 Sept

Clinical Profile and Outcomes of Children With Congenital Heart Disease Undergoing Surgery at a Tertiary Care Cardiac Center in Central Gujarat and Their Anthropometric Profiles During Follow-Up: A Prospective Longitudinal Observational Study

Baseline growth and surgical outcome data from non-metropolitan India help you counsel families on expected catch-up growth after CHD repair.

Paediatrics · Cureus · 21 Sept

Correlation Between Serum Ferritin and Electrocardiographic and Echocardiographic Markers of Cardiac Dysfunction in Children With Transfusion-Dependent β-Thalassemia Major

Ferritin negatively correlated with LVEF (r = -0.684), E/A ratio (r = -0.582), lateral e' (r = -0.658), TAPSE (r = -0.512); positively with E/Ea (r = 0.726), QTc (r = 0.648), MPI (r = 0.672), all p < 0.001.

Paediatrics · Cureus · 20 Sept

Growth Profile and Endocrine Dysfunction in Children Aged 2-10 Years With Transfusion-Dependent β-Thalassemia Major

78.7% had at least one endocrine abnormality; thyroid dysfunction 63.3%, vitamin D deficiency 62.0%, gonadal dysfunction 46.7%, cortisol abnormalities 41.3%, low growth hormone 38.7%.

Paediatrics · Cureus · 20 Sept

Phototherapy-Associated Changes in Ionized Calcium Among Neonates Born at ≥35 Weeks of Gestation

Neonates with iCa ≥1.40 mmol/L fell from 109/145 (75.2%) to 30/145 (20.7%) after 24 hours; among those without low baseline iCa, 33.1% developed <1.20 mmol/L.

Paediatrics · Cureus · 20 Sept

FSSAI takes legal action against Nestle India over sub-standard infant formula

Two Nestle infant formulas face regulatory action over HMO and whey protein claims, so verify what you tell parents about these products' composition.

Paediatrics · ETHealthworld · 18 Sept

Empirical Antibiotic Therapy for the Management of Empyema Thoracis in Children: A Systematic Review.

Cloxacillin was empirical therapy in 10/17 studies (638 children), appropriate in six (419 children) as Staphylococcus aureus dominated. Third-generation cephalosporins used in 9/17 studies (444 children), appropriate in four (283 children).

Paediatrics · Indian pediatrics · 18 Sept

Time to diagnosis of acute kidney injury and its predictors among outborn neonates in India: a competing-risk analysis of tele-ICU network data (2022-2024).

AKI occurred in 170 (29.8%); median time to diagnosis 2 days (IQR 1-5); five-day cumulative incidence 0.23 (95% CI 0.20-0.27). Dehydration, ventilation and vasopressors predicted early AKI.

Paediatrics · Lancet regional health. Southeast Asia · 18 Sept

FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A

First approved therapy for MPS IIIA, a fatal neurodegenerative lysosomal disorder; early diagnosis now becomes actionable rather than purely supportive.

Paediatrics · FDA — Press Announcements · 17 Sept

ARPA-H launches SPECTRA program, new autism research initiative

A US-funded push to standardise early autism risk detection and referral could shape the screening pathways and trial options you discuss with families.

Paediatrics · Fierce Biotech · 17 Sept

Marked Increase in Influenza-Associated Acute Myositis Among Children During the 2024–2025 Season: A Two-Season Retrospective Cohort Study From Riyadh, Saudi Arabia

Influenza in children can cause acute myositis; a marked seasonal rise means consider it in any child with sudden calf pain and refusal to walk during flu season.

Paediatrics · Cureus · 17 Sept

Switch to injectable cabotegravir-rilpivirine given every 8 weeks in adolescents living with HIV with virological suppression in sub-Saharan Africa (LATA): a randomised, open-label, multicentre, 96-week non-inferiority trial.

Every-8-week injectable ART kept adolescents virologically suppressed as well as daily pills, offering an adherence-sparing option for a group with poor outcomes.

Paediatrics · Lancet · 17 Sept

Trial of a Maternal Diet Rich in Eggs and Peanuts to Reduce Infant Allergy.

If maternal egg and peanut consumption prevents infant sensitisation, antenatal dietary advice shifts from avoidance to deliberate early allergen exposure.

Paediatrics · NEJM · 17 Sept

Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial.

If adolescent hemophilia B patients sustain factor IX levels after one infusion, they may stop prophylactic injections through the highest-risk bleeding years.

Paediatrics · Nature Medicine · 16 Sept

Sickle Cell Disease in Latin America and the Caribbean: Where the Care Cascade Breaks Down

The care cascade breaks at every step: screening panels often omit the disease, transcranial Doppler implementation is uneven, and hydroxyurea use proportions are not comparable because denominators differ.

Paediatrics · Cureus · 16 Sept

Van Wyk-Grumbach Syndrome: A Rare Triad of Precocious Puberty, Pituitary Pseudomacroadenoma, and Multicystic Ovaries in an Eight-Year-Old Girl

Bone age was delayed to 3.5-4 years and thyroid profile confirmed autoimmune primary hypothyroidism; the patient was managed purely medically with cautious levothyroxine, avoiding surgery

Paediatrics · Cureus · 15 Sept

Prevention and Management of Pediatric Sensitive Skin Conditions in India: A Modified Delphi Consensus

Experts unanimously agreed diaper dermatitis, xerosis and atopic dermatitis are the most prevalent paediatric sensitive skin conditions; emollients were recommended as the cornerstone of management, with colloidal oatmeal emollients favoured in xerosis and atopic dermatitis.

Paediatrics · Cureus · 15 Sept

Pediatric Rosai-Dorfman-Destombes Disease: From Isolated Cervical Lymphadenopathy to Multisystem Disease

Consider RDD in a child with persistent cervical lymphadenopathy, since the same disease can progress from a single node to multisystem involvement.

Paediatrics · Cureus · 15 Sept

Congenital Bile Acid Synthesis Defect Type 2 Mimicking Biliary Atresia: A Case Report of Delta-4-3-Oxosteroid 5-Beta-Reductase Deficiency in a Two-Month-Old Infant

Urine LC-MS/MS showed trace primary bile acids with Δ4-3-oxo intermediates; NGS confirmed CBASD type 2. Liver biopsy showed giant cell hepatitis without ductal proliferation, excluding BA.

Paediatrics · Cureus · 15 Sept

Molecular Etiology and Mortality Risk Stratification of Non-SARS-CoV-2 Severe Acute Respiratory Infection in a Predominantly Pediatric Cohort: A Retrospective Study From a Tertiary Care Hospital in North India

In North Indian children with non-COVID SARI, the ViVI severity score may help you flag those at higher mortality risk early and target escalation.

Paediatrics · Cureus · 14 Sept

Late-Onset Neonatal Cyanosis Secondary to Methemoglobinemia in a Preterm, Small-for-Gestational-Age Infant: A Case Report

In a preterm neonate with unexplained desaturations unresponsive to oxygen, check methemoglobin — methylene blue and vitamin C correct it, and pulse oximetry alone will mislead you.

Paediatrics · Cureus · 14 Sept

Transient Post-infectious Multisystem Inflammatory Overlap Presentation in a Pediatric Patient With Neurological, Hepatic, Gastrointestinal, and Autoimmune Manifestations: A Rare Case From Azerbaijan

Transient autoantibody positivity and incidental neuroinflammatory imaging changes, hepatic steatosis and gastrointestinal inflammation appeared disproportionate to clinical status and resolved without immunosuppression or invasive intervention

Paediatrics · Cureus · 13 Sept

When Fever Hides Fire: A Systematic Review of Clinical and Digital Approaches to Early Recognition of Pediatric Sepsis

Approaches performed well ruling out sepsis in low-risk children, but case detection and alert burden varied by thresholds, settings, and sepsis definitions; no approach was consistently superior

Paediatrics · Cureus · 13 Sept

Racial and Ethnic Disparities in Receipt of Behavioral Treatment Among Children With Autism Spectrum Disorder in the United States

564 of 790 (71.4%) received behavioural treatment. Non-Hispanic Black children were less likely than White children to receive it (adjusted OR 0.47, 95% CI 0.28-0.79). Asian, Hispanic and multiracial differences were not significant.

Paediatrics · Cureus · 12 Sept

Hyperglycemic Hyperosmolar State As the Initial Presentation of Type 1 Diabetes Mellitus in an Adolescent: A Case Report

Glucose exceeded 1,000 mg/dL with hyperosmolality, dehydration and only mild acidosis; treatment caused marked glucose swings needing repeated insulin and dextrose titration.

Paediatrics · Cureus · 11 Sept

Pediatric Headache: A Practical Approach to Clinical Diagnosis and Management

No numerical results reported. Diagnosis rests on structured history, disability assessment and examination; red flags prompt risk stratification rather than reflexive neuroimaging

Paediatrics · Cureus · 11 Sept

Immunogenicity of fractional one-fifth and one-half doses of 17DD yellow fever vaccine compared with full dose in children aged 9-23 months in Uganda: a phase 4, single-blind, randomised clinical trial.

Both fractional doses met non-inferiority (5 percentage-point margin) for seroconversion at 4 weeks; one-half versus full dose difference was -0.17 (95% CI -0.52 to 0.17), with no difference between one-fifth and full doses.

Paediatrics · Lancet · 11 Sept

Profile of Invasive Fungal Infections in a Paediatric Haemato-Oncology Unit: A Five-Year Retrospective Cohort Study From a Tertiary Care Centre in South India

33 IFI episodes in 23 patients; Candida and Aspergillus each 43.5%. All 20 Candida episodes were fungemia, 60.0% Candida tropicalis, 100% fluconazole-susceptible. Mortality 26.1% overall: 0 Candida, 30% aspergillosis, 100% mucormycosis.

Paediatrics · Cureus · 10 Sept

Oxygen vs Air at Birth for Moderate- to Late-Preterm Infants: The AIROPLANE Cluster Randomized Crossover Trial.

Ongoing respiratory support when leaving the delivery room: 700 of 964 (72.6%) with Fio2 0.30 vs 626 of 854 (73.3%) with Fio2 0.21; risk difference -0.83 (95% CI, -4.33 to 2.67).

Paediatrics · JAMA · 9 Sept

PIL challenges Centre’s HPV vaccination drive, seeks Supreme Court intervention over consent, safety and accountability

School-based HPV vaccination may pause; verify consent protocols and legal status before counselling parents or administering doses.

Paediatrics · ETHealthworld · 7 Sept

FDA Takes Steps to Maintain Newborn Access to Life-Saving Starter Nutrition Products

Ensures neonatal intensive care units can keep stocking starter PN for premature infants during potential shortages, preventing treatment delays.

Paediatrics · FDA — Press Announcements · 5 Sept

FSSAI plans curbs on high-fat food sales within 50 metres of schools, CEO Rajit Punhani says

This may reduce childhood obesity and metabolic risk; paediatricians should counsel families on healthier snack choices near schools.

Paediatrics · ETHealthworld · 4 Sept

Safe Sleep Video Intervention via Text Messaging to Low-Income Families

Text-delivered safe sleep videos may reduce unsafe infant sleep practices in low-income families—consider integrating into prenatal and postnatal outreach.

Paediatrics · JAMA · 1 Sept

Trouble Lies Ahead for Global Immunization Coverage

Expect more vaccine-preventable disease outbreaks in your practice; check and catch up children behind on routine immunizations.

Paediatrics · JAMA · 1 Sept

FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

Now offers a one-time cure option for children as young as 2 with recurrent crises, potentially ending years of transfusions and pain.

Paediatrics · FDA — Press Announcements · 2 Jul

UnitedHealthcare eliminates prior authorization requirements on 2K pediatric services

If you bill UnitedHealthcare, 2,000+ paediatric codes no longer need prior auth from Dec 1 — fewer approval calls before scans, procedures and referrals.

Paediatrics · Fierce Healthcare · 29 May