Following the approval of elexacaftor/tezacaftor/ivacaftor (ETI), there have been post-marketing reports and published cases of papilledema and intracranial hypertension in children with cystic fibrosis (CF) taking ETI. In those reports, the patients often presented with marked symptoms and concomitant hypervitaminosis A. In this multicenter case series, we report eight cases of papilledema in children with CF taking ETI that were diagnosed via routine eye exam, the majority of whom presented with minimal to no symptoms and all had normal serum Vitamin A levels.

Use of proton-pump inhibitors (PPIs) is common among people with cystic fibrosis (pwCF) both for the management of suspected GERD, as well as pancreatic enzyme replacement therapy augmentation. Despite their use, limited data exist to demonstrate a clinically significant impact of PPIs on key endpoints in pwCF. Furthermore, the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy may modify the need for use. These notions, coupled with the potential for adverse outcomes associated with long-term PPI use in pwCF, should facilitate re-evaluation of long-term PPI use in pwCF and promote potential deprescribing. Despite limited data on PPI deprescribing in pwCF, it intuitively mirrors the existing guidance in adults in the general population, but with added consideration given to tapering strategy, and monitoring for CF-specific outcomes such as nutritional and respiratory status. The development of a monitoring and re-initiation plan is key to reducing deprescribing inertia. This review aims to summarize the evidence that details the concern for long-term use of PPIs and provide CF clinicians with rationale and guidance on how to approach deprescribing in their practice.

Purpose The purpose of this review is to summarize the management of asthma in children and to highlight different guideline-based approaches. This review also discusses literature regarding the use of corticosteroids, both inhaled and systemic, as well as biologic agents, in asthma management. Summary Asthma is a common chronic respiratory condition in the pediatric population and has evolved into a highly patient-specific disease. Of the 2 main asthma guidelines, one developed by the National Asthma Education and Prevention Program was recently published as a focused update in 2020. The other, from the Global Initiative for Asthma, focuses on a global strategy for management and prevention, with the most recent update in 2023. Both reports discuss diagnosis, assessment, and treatment of asthma in adults and children. Treatment is designed as a stepwise approach in both reports, although there are key differences. This article focuses on gaps in these guidelines, including the use of bronchodilators and inhaled corticosteroids with single maintenance and reliever therapy and long-acting muscarinic antagonists in children. It also reviews treatment in children under 5 years of age, although recommendations are limited due to a lack of evidence in this age group. Finally, this review discusses considerations for emerging treatments, including biologics, for patients who are difficult to treat. Conclusion New treatment strategies and agents have emerged in the treatment of pediatric asthma. Pharmacists play a key role in providing education about, dispensing, and recommending the newest evidence-based treatment options for children.

Over the last several decades, substantial treatment advances have improved the quality of life and median predicted survival in people with cystic fibrosis (PwCF). It is critical for CF clinicians to begin to discuss health considerations related to an aging and overall healthier CF population. Such considerations include family planning, reproductive health, and contraception. CF care teams are trusted sources of medical information and therefore often have initial discussions related to contraception for PwCF. The purpose of this article is to review the available pharmacologic contraceptive methods, with a specific focus on the benefits and risks that may be more relevant to PwCF.

•Caregivers are overall satisfied with an interdisciplinary clinic.•Interprofessional education is well-received by caregivers.•Rarely, a large interprofessional team may intimidate patients.

Purpose The aim of this article is to provide an overview of the current literature for direct-acting oral anticoagulant (DOAC) use in pediatric patients and summarize ongoing trials. In treatment of venous thromboembolism (VTE) in pediatric patients, evidence supports use of both dabigatran and rivaroxaban. Dabigatran has been shown to be noninferior to standard of care (SOC) in terms of efficacy, with similar bleeding rates. Similarly, treatment with rivaroxaban in children with acute VTE resulted in a low recurrence risk and reduced thrombotic burden, without increased risk of bleeding, compared to SOC. Treatment of pediatric cerebral venous thrombosis as well as central venous catheter-related VTE with rivaroxaban appeared to be both safe and efficacious and similar to that with SOC. Dabigatran also has a favorable safety profile for prevention of VTE, and rivaroxaban has a favorable safety profile for VTE prevention in children with congenital heart disease. Many studies with several different DOACs are ongoing to evaluate both safety and efficacy in unique patient populations, as well as VTE prevention. Conclusion The literature regarding pediatric VTE treatment and prophylaxis is growing, but the need for evidence-based pediatric guidelines remains. Additional long-term, postauthorization studies are warranted to further elucidate safety and efficacy in clinical scenarios excluded in clinical trials. Additional data on safety, efficacy, and dosing strategies for reversal agents are also necessary, especially as the use of DOACs becomes more common in the pediatric population.

Vaccines are a daycare and school requirement for children. The Public Readiness and Emergency Preparedness (PREP) Act 3rd amendment grants pharmacists and pharmacy interns the capability to vaccinate children at ages 3-18 years according to the Centers for Disease Control and Prevention Advisory Committee on Immunization Practices schedule of recommended vaccines. Before this, only some state boards of pharmacy allowed pharmacists to administer pediatric vaccines. Despite these opportunities, immunization training programs lack the comprehensive training of pediatric vaccines and administration for pharmacists and pharmacy interns without additional financial cost. All pharmacists should be adequately trained and pharmacies be reimbursed to provide vaccines to all ages of pediatric patients. Advocacy is necessary to ensure children have reliable, affordable, and convenient access to lifesaving vaccines today and after the PREP Act expires. (c) 2023 American Pharmacists Association((R)). Published by Elsevier Inc. All rights reserved.

Antibiotics are the most widely used treatment for infections yet there has been minimal antibiotic discovery in recent years despite rising drug resistance and treatment failures. A better understanding of antibiotic pharmacokinetics (PK) and pharmacodynamics (PD), along with the development of novel dosing strategies, such as extended (EI) and continuous infusions (CI), has helped to overcome these barriers. Studies have consistently demonstrated that EI/CI administration of beta-lactams and vancomycin in particular, improves PK-PD target attainment compared to intermittent infusions. However, the effects of EI/CI on clinical outcomes, including efficacy and safety, remain controversial. Emerging data focus on patient populations with altered PK that may benefit from EI/CI beta-lactams or vancomycin (e.g., critically ill, cystic fibrosis, or outpatient parenteral antibiotic therapy). Logistical barriers limit EI/CI use in practice, including intravenous access, drug stability and drug incompatibility. This review highlights beta-lactam and vancomycin PK-PD, EI/CI dosing strategies and relevant evidence for practice.

Clostridioides difficile infection guidelines were published in final format on April 1, 2018. Among 4962 and 3545 C. difficile infection cases in children the year before and after publication, oral metronidazole use decreased from 63.0% to 44.3% (P < 0.001) and oral vancomycin use increased from 27.3% to 47.7% (P < 0.001). Quarterly metronidazole utilization decreased postguidelines among 117 institutions, incidence rate ratios 0.86 (95% confidence intervals: 0.78-0.96).

The study investigated the safety and efficacy of two antithrombin III (ATIII) products in pediatric patients receiving extracorporeal membrane oxygenation (ECMO) by performing a retrospective analysis of patients who received either recombinant ATIII (rATIII) or human-derived ATIII (hATIII). Twenty-two patients were included in the study from January 2014 to September 2015 and all received unfractionated heparin (UFH) as anticoagulation during ECMO. In total, 86 doses of ATIII were included in the analysis in which 37 doses (43%) were rATIII and 49 doses (57%) were hATIII. Unfractionated heparin rates were also evaluated for all cases (n = 86) at 24 hours post-ATIII supplementation. The UFH rate decreased after the administration of both types of ATIII. However, neither the reduction in UFH rate between the two ATIII products ( p = 0.52) nor the UFH rates pre- and post-ATIII supplementation at 24 hours ( p = 0.08) reached statistical significance. There was a significant difference in cost favoring the rATIII product ( p < 0.0001). An ad-hoc estimation of waste associated with ATIII supplementation showed >$100,000 in financial loss of unused drug. Future studies are warranted to evaluate the efficacy of ATIII supplementation in pediatric ECMO.

Pharmacists working in outpatient settings are in a prime position to improve pediatric vaccination rates by recommending, administering, or educating families and patients about vaccines. Pediatric vaccination rates are less than optimal in the United States (U.S.), many falling short of the goals set forth by Healthy People 2020, which may be contributing to pediatric morbidity and mortality. For the 2018-2019 influenza season, less than half of children indicated for vaccination with the influenza vaccine were vaccinated by December and 101 influenza-associated pediatric deaths have occurred as of April 2019. For the adolescent population, fewer than 50% received the complete series of human papillomavirus (HPV) vaccine in 2017 and this same demographic had the lowest influenza vaccination rate of all pediatric age groups at 47.4% in the 2017-2018 influenza season.