Trends in the off-label use of ß-blockers in pediatric patients.

The use of US Food and Drug Administration (FDA)-approved drugs for the treatment of an unapproved indication or in an unapproved age group, or at doses or route of administration not indicated on the label is known as off-label use. Off-label use may be beneficial in circumstances when the standard-of-care treatment has failed, and/or no other FDA-approved medications are available for a particular condition. In pediatric patients, off-label use may increase the risk of adverse events as pharmacokinetic and pharmacodynamic data are limited in children. Approximately 73% of off-label drugs currently prescribed for various conditions do not have sufficient scientific evidence for safety and efficacy. For example, ß-blockers are a class of drugs with FDA-approval for very few indications in pediatrics but are commonly used for various off-label indications. Interestingly, the proportion of off-label use of ß-blockers in adults is at about 52% (66.2 million) of the total number of ß-blockers prescribed. The frequency of off-label use of ß-blockers in children is also high with limited data on the indications as well as safety and efficacy. We present trends in off-label use of ß-blockers in children to discuss drug safety and efficacy and include recommendations for pediatric providers.

Pulmonary arteriovenous malformations leading to hypoxemia in child with primary ciliary dyskinesia.

Primary ciliary dyskinesia (PCD) is a rare genetic condition characterized by respiratory tract infections, situs inversus or heterotaxy, and male infertility. Chronic respiratory infections begin in childhood and result in complications such as bronchiectasis. As hypoxemia is often attributed to bronchiectasis, other etiologies for desaturation in this setting are not routinely evaluated. The development of pulmonary arteriovenous malformations (PAVMs) in PCD is not an established association. PAVMs as the etiology for hypoxemia may have been overlooked due to the lack of awareness of this rare association. We present a child with diagnosis of PCD with significant hypoxemia in the absence of bronchiectasis, found to have diffuse bilateral PAVMs and discuss possible physiopathologic mechanisms.

Determination of the Frequency of Right and Left Internal Mammary Artery Embolization in Single Ventricle Patients: A Two-Center Study.

Embolization of systemic to pulmonary artery collaterals to regulate pulmonary arterial flow or pressure of the cavopulmonary circulation in patients with single ventricle is a common practice. The relative incidence and impact of this practice on future interventions like coronary artery bypass grafting is poorly understood. This study aims to evaluate the frequency and implications of internal mammary artery (IMA) embolization in the single ventricle (SV) population. A retrospective chart review was performed of SV patients who underwent cardiac catheterization before and after Fontan procedure between February 2007 and 2017. Data were collected from two tertiary care centers in the Midwest. Of the 304 SV patients, 62 (20.4%) underwent embolization of one or more IMAs, whereas 242 (79.6%) did not. The rate of embolization of IMA was 40.5% in one center and 14.5% in the second center. Among patients who received IMA embolization, left internal mammary artery (LIMA) embolization was seen in 6 (9.7%) patients. Majority of patients underwent either right internal mammary artery (RIMA) embolization (n = 25; 40.3%) or RIMA and LIMA embolization (n = 27; 43.5%). IMA embolization in SV patients is common. Embolizing IMAs early in life will likely eliminate a valuable graft option for coronary artery bypass grafting should it be required in the future care of these patients. Multi-center, prospective, nation-wide studies are warranted to examine coronary artery disease in the SV population and true frequency of IMA embolization. Delineation of which IMAs were embolized is a necessary in surgical and cardiac intervention national data, such as Society of Thoracic Surgeons (STS) database. All measures should be taken to preserve IMAs patency, if deemed feasible and safe.