At-a-Glance
Clopidogrel dosing in infants with congenital heart disease: Therapeutic doses followed an age-related gradient rather than a fixed 0.2 mg/kg/day, with a proposed escalation to 0.9-1 mg/kg/day by 24 months.¹
Neonatal needle-based analgesia: For IM injections and IV placement, combine oral sucrose with topical EMLA cream for improved pre-emptive analgesia.
Gene editing for Duchenne muscular dystrophy: FDA clearance of an in vivo gene-editing trial marks a shift from gene addition to mutation excision strategies.²
New & Notable
In 44 pediatric cardiac patients 0-24 months of age (median 2.1 months), the median clopidogrel dose associated with therapeutic P2Y12 reaction unit (PRU) values was 0.22 mg/kg/day (IQR 0.19-0.34). This conflicts with the fixed 0.2 mg/kg/day recommended by the PICOLO trial.
Modeling of doses that achieved therapeutic PRUs demonstrated an age-related gradient, with exponential models fitting better than linear models, suggesting dose requirements increase with age over the first two years of life.
A proposed age-based dosing scheme recommends starting at 0.2-0.3 mg/kg/day in infants ≤ 3 months and increasing every three months to 0.9-1 mg/kg/day by 21-24 months; concordance with observed therapeutic dosing was approximately 80% in this cohort.
Hemorrhage occurred in 13.6% of patients and thrombosis in 6.8%. Events were not clearly attributable to specific dosing thresholds, and CYP2C19 pharmacogenomic status was not assessed.
Interpretation is limited by the retrospective, single-center design, small sample size, and minimal representation of patients aged 12-24 months (n=2), resulting in weaker model fit in older infants. The proposed dosing scheme should be viewed as a reference framework with continued reliance on PRU monitoring and clinical context.¹
Clinical Pearl
For neonatal needle-based procedures such as intramuscular injections, venipuncture, and peripheral IV insertion, sucrose alone is often insufficient for pain prevention.
Topical EMLA cream (2.5% lidocaine/2.5% prilocaine) should be applied about 45 minutes before the procedure and used with oral sucrose. It is not effective for heel sticks. Doses up to 1 gram, about one-fifth of a tube, have not been shown to significantly increase methemoglobin levels in term infants.
Pre-emptive analgesia for mildly painful procedures is reviewed fully in How to Improve Neonatal Pain Management for Mildly Painful Procedures.
Pediatric Pulse
Precision BioSciences Receives U.S. FDA Clearance for Investigational Gene Editing Therapy PBGENE-DMD
On February 11, 2026, the FDA granted “Study May Proceed” clearance for an Investigational New Drug application for PBGENE-DMD, an in vivo gene-editing therapy for Duchenne muscular dystrophy.² Unlike gene addition therapies that deliver truncated micro-dystrophin, this strategy uses gene excision to restore production of a near full-length dystrophin protein.
For pediatric clinicians, this represents a shift in therapeutic approach. The program is designed to address mutations affecting up to 60% of boys with DMD. As an early-phase investigational therapy, long-term durability, off-target effects, and cardiac outcomes remain key considerations.
References
Anton-Martin P, Coalter C, DeAvilla K, et al. Clopidogrel dosing scheme in pediatric cardiac patients 0–24 months old using P2Y12 reaction unit monitoring. J Pediatr Pharmacol Ther. 2026;31(1):30-36. doi:10.5683/JPPT-25-00024.
Precision BioSciences. Precision BioSciences receives U.S. FDA clearance for investigational new drug application for PBGENE-DMD. Business Wire. 2026. https://investor.precisionbiosciences.com/news-releases/news-release-details/precision-biosciences-receives-us-fda-clearance-investigational
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— Dr. Su