Volume 29: Issue 5

Postural orthostatic tachycardia syndrome (POTS) is a chronic illness with unknown mortality and high morbidity, often diagnosed in the adolescent years. Published literature regarding POTS primarily focuses on the adult population, and guidance on treatment in pediatrics is sparse. The purpose of this clinical review is to evaluate the current literature on the management of POTS in pediatric patients. A search was conducted using the Cochrane database, Google Scholar, and PubMed. Studies were included if they evaluated the management of POTS, primarily in pediatric patients. Case reports and series were excluded. Eight published studies met the inclusion and exclusion criteria. To date, there are no US Food and Drug Administration-approved agents for the treatment of POTS. However, select pharmacological therapies have shown positive outcomes by addressing symptom origins, such as providing heart rate control, peripheral autonomic modulation, and targeting hypovolemia. Targeted pharmacological therapies studied in children and young adults include ivabradine, metoprolol, midodrine, pyridostigmine, intravenous crystalloid fluids, and fludrocortisone. Before adding pharmacotherapeutic interventions, non-pharmacologic interventions such as patient education, avoidance of symptom-triggering environments and medications, dietary fluid and sodium supplementation, exercise, and use of compression garments should be first attempted. Although the body of evidence for the management of POTS is expanding, additional research is needed to determine safe and efficacious dosing and establish clear guidelines for POTS in the pediatric population.

OBJECTIVE

Chylous effusion and chylous ascites are rare but serious conditions that affect both fetuses and neonates. Previous studies have documented chylous effusions or chylous ascites treatment with medications as an adjunct to respiratory support and dietary modifications, but no formal recommendations have been made. New literature suggests propranolol as an effective and safe treatment option, though no randomized clinical studies have been published to date. This review aims to assess the efficacy and safety of propranolol in the treatment of chylous effusion and chylous ascites in fetuses and newborns from case reports.

METHODS

A comprehensive search of 10 databases and grey literature was completed. The inclusion criteria for articles were age at diagnosis less than 40 days old and case report/series. Articles were excluded if they were animal studies or not published in English.

RESULTS

Following Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines, 4 articles were ultimately included in the study for a total of 10 reported cases. Propranolol administered to mother and neonates was effective in 100% of cases. The most common oral dose for mothers was 20 mg, 4 times daily, titrated to 40 mg, 4 times daily. The maximum dosage varied for administration orally to neonates, with the median being 3 mg/kg/day. Side effects, including bradycardia and transient hypoglycemia, were seen in 20% of the cases and resolved with dose adjustment.

CONCLUSION

Propranolol is a relatively effective and safe treatment option for chylous effusion and chylous ascites that can be administered prenatally or to neonates.

OBJECTIVE

Anthracycline chemotherapy agents have significant dose-dependent cardiotoxic effects. Carnitine, a non-essential amino acid, is involved in long chain fatty acid oxidation, and carnitine deficiency can result in cardiomyopathy and cardiac arrhythmias. If administered concurrently with chemotherapy, carnitine supplementation could be a potential strategy to prevent cardiotoxicity. However, the association between serum carnitine concentrations and anthracycline cardiotoxicity during cancer treatment in the childhood, adolescent, and young adult (CAYA) age range has not been established.

METHODS

This prospective pilot cohort study characterized changes in serum carnitine concentrations and cardiac function before, during, and approximately 1 year after large-dose anthracycline therapy in newly diagnosed CAYA cancer patients.

RESULTS

Among 21 patients with a mean cumulative anthracycline dose exposure of 409 mg/m² of doxorubicin equivalents, left ventricular ejection fraction and relative wall thickness decreased, indicating an overall decline in cardiac function. A reversible decrease in serum carnitine concentrations was also observed. A non-statistically significant positive correlation was observed; for every 1 mmol/L decrease in serum carnitine concentration, there was a 0.09% decrease in LVEF (p = 0.2).

CONCLUSIONS

These findings from this small pilot study suggest that there may be a relationship between serum carnitine concentrations and cardiac function after anthracycline therapy that should be evaluated in larger studies.

With the use of risk-adapted chemotherapy based treatments, outcomes for children and adolescents with hematological malignancies have significantly improved in industrialized countries. For example, in acute lymphoblastic leukemia (ALL), the most common pediatric cancer, with a peak incidence in preschool-children, long-term survival steadily improved in US Children’s Oncology Group (COG) clinical trials; 499 patients treated from 1970–1972 had a survival rate at 10 years of ∼20% versus >90% in 11,806 patients treated from 2010–2015.¹ Similar or better results have been achieved in major single-institution clinical trials including the Total Therapy protocols at St. Jude Children’s Research Hospital (SJCRH).

Myalgia and Rigidity as Adverse Effects of Trametinib Therapy

Charles J WeeksBS,

Victoria FramePharmD,

Rohan VuppalaBS,

David ThomasBS,

Abigayle B SimonBS,

Michael StokesMD, and

Colleen McDonoughMD