Chapter 2: Pediatrics Pharmacotherapy

Content Update

May 06, 2024

New Precision Therapy for Patients with Cystic Fibrosis: Recent U.S. FDA approvals of cystic fibrosis transmembrane conductance regulator (CFTR) potentiator agents in younger children and infants are reviewed. In May 2023, ivacaftor was approved for patients 1 month or older. Another updated approval in 2023 (April) was for elexacaftor/tezacaftor/ivacaftor for patients ages 2 through 5 years. In September 2022, lumacaftor/ivacaftor approval was updated to include children aged 12 through less than 24 months. Current standard of care recommends CFTR modulator therapy for all patients qualifying for therapy based on genotype and age-based approvals. These recent approvals expand the number of patients who can now qualify to receive these therapies.

Content Update

December 21, 2023

Reacting to the Rise of Respiratory Syncytial Virus: An Update on Prevention: In 2023, the U.S. Food and Drug Administration (FDA) approved several prophylactic measures against the respiratory syncytial virus (RSV): 1) Nirsevimab (Beyfortus®) for the pediatric population; 2) a recombinant, adjuvanted RSVPreF3 vaccine (Arexvy®) for the geriatric population; and 3) a bivalent RSVpreF vaccine (Abrysvo®) for the pregnant and geriatric populations. Compared to palivizumab, the previous standard of care, nirsevimab has an extended half-life and convenient single-dosing schedule. Abrysvo® and Arexvy® are recommended by the Centers for Disease Control and Prevention (CDC) as single dose RSV vaccine options for adults 60 and older with shared decision making. Additionally, Abrysvo® is recommended in pregnant women at 32-36 weeks gestational age. Adverse effects (AEs) were mostly minor, although neurological AEs (e.g. Guillain-Barre syndrome) were noted in a small number of patients.

Content Update

August 16, 2022

Albuterol–Budesonide Combination for Asthma Exacerbation: Asthma exacerbations are increasing in prevalence and result in serious complications such as hospitalizations and death. For acute treatment of flare-ups, the benefit of short-acting β₂-agonists may be limited because they do not address the underlying airway inflammation compared to inhaled corticosteroids. A Phase 3, multinational, randomized, double-blinded trial demonstrated an albuterol-budesonide combination at higher and lower doses was effective compared to albuterol monotherapy for patients aged 4 years and older with uncontrolled moderate to severe asthma. The frequency of adverse events was similar among the three treatment groups. With these positive results, the first-in-class combination was recently submitted to the U.S. Food and Drug Administration for approval and may provide better access and outcomes for patients.

LEARNING OBJECTIVES

Upon completion of the chapter, the reader will be able to:

1. Define different age groups within the pediatric population.

2. Identify factors that affect selection of safe and effective drug therapy in pediatric patients.

3. Explain general pharmacokinetic and pharmacodynamic differences in pediatric versus adult patients.

4. Develop strategies for appropriate and effective medication administration to infants and young children.

5. Determine approaches to effectively communicate with patients and caregivers about appropriate medication use including ...