Author: Kayla Korzekwinski
PTN Post: Issue 16 (March 2016)
Check out the Spring 2016 issue of the PTN Post, the newsletter for the Pediatrics Trials Network. This issue highlights the continuation of the Sildenafil Cohort 2 program, the new role of Child and Family Advocate, and PTN’s presentations at the upcoming Pediatric Academic Society meeting in Baltimore.
We hope you enjoy this issue, and as always, we welcome your comments and feedback.
Optimizing operational efficiencies in early phase trials: The Pediatric Trials Network experience
Contemporary Clinical Trials • March 2016
England A, Wade K, Smith PB, Berezny K, Laughon M; Best Pharmaceuticals for Children Act — Pediatric Trials Network Administrative Core Committee.
Performing drug trials in pediatrics is challenging. In support of the Best Pharmaceuticals for Children Act, the Eunice Kennedy Shriver National Institute of Child Health and Human Development funded the formation of the Pediatric Trials Network (PTN) in 2010. Since its inception, the PTN has developed strategies to increase both efficiency and safety of pediatric drug trials. Through use of innovative techniques such as sparse and scavenged blood sampling as well as opportunistic study design, participation in trials has grown. The PTN has also strived to improve consistency of adverse event reporting in neonatal drug trials through the development of a standardized adverse event table. We review how the PTN is optimizing operational efficiencies in pediatric drug trials to increase the safety of drugs in children.
Insulin, Hyperglycemia, and Severe Retinopathy of Prematurity in Extremely Low-Birth-Weight Infants
American Journal of Perinatology • March 2016
Lee JH, Hornik CP, Testoni D, Laughon MM, Cotton CM, Maldonado RS, Belcastro MR, Clark RH, Smith PB
This study aimed to determine the association between hyperglycemia, insulin therapy, and severe retinopathy of prematurity (ROP) in extremely low-birth-weight (ELBW) infants. Hyperglycemia alone was not associated with severe ROP in ELBW infants. However, researchers did observe a possible trend between the use of insulin and severe ROP.
Pharmacokinetics and bioequivalence of a liquid formulation of hydroxyurea in children with sickle cell anemia
The Journal of Clinical Pharmacology • March 2016
Estepp JH, Melloni C, Thornburg CD, Wiczling P, Rogers Z, Rothman JA, Green NS, Liem R, Brandow AM, Crary SE, Howard TH, Morris MH, Lewandowski A, Garg U, Jusko WJ, Neville KA; Best Pharmaceuticals for Children Act-Pediatric Trials Network Administrative Core Committee.
Hydroxyurea (HU) is a crucial therapy for children with sickle cell anemia, but its off-label use is a barrier to widespread acceptance. We found HU exposure is not significantly altered by liquid vs capsule formulation, and weight-based dosing schemes provide consistent exposure. HU is recommended for all children starting as young as 9 months of age with sickle cell anemia; however; a paucity of pediatric data exists regarding the pharmacokinetics (PK) or the exposure-response relationship of HU.
Sildenafil and retinopathy of prematurity risk in very low birth weight infants
American Journal of Perinatology • February 2016
Samiee-Zafarghandy S, van den Anker JN, Laughon MM, Clark RH, Smith PB, Hornik CP; Pharmaceuticals for Children Act – Pediatric Trials Network Administrative Core Committee.
We identified premature infants who were discharged from Pediatrix Medical Group neonatal intensive care units from 2003–2012 and who received an ophthalmologic exam. We matched each infant exposed to sildenafil prior to first eye exam to three non-exposed infants using propensity scoring to control for differences in baseline infant characteristics. We evaluated the association between sildenafil exposure and development of severe ROP using conditional logistic regression.
Dosing in neonates: Special considerations in physiology and trial design
Pediatric Research • January 2016
Ku LC, Smith PB
Determining the right dose for drugs used to treat neonates is critically important. Neonates have significant differences in physiology affecting drug absorption, distribution, metabolism, and elimination that makes extrapolating dosages from adults and older children inappropriate. Fortunately, specialized analytical techniques, such as the use of dried blood spots, scavenged sampling, population pharmacokinetics analyses, and sparse sampling, have helped investigators better define doses that maximize efficacy and safety. Through the use of these methods, successful clinical trials have resulted in recent changes to drug dosing in this population.
PTN Post: Issue 15 (December 2015)
Check out the Fall-Winter 2015 issue of the PTN Post, the newsletter for the Pediatrics Trials Network. This issue highlights four exciting PTN projects: timolol in infants with hemangiomas; anti-epileptics and pantoprazole for obese children; and furosemide for BPD. We hope you enjoy this issue and as always, we welcome your comments and feedback.
Effect of Catheter Dwell Time on Risk of Central Line–Associated Bloodstream Infection in Infants
Pediatrics • December 2015
Greenberg RG, Cochran KM, Smith PB, Edson BS, Schulman J, Lee HC, Govindaswami B, Pantoja A, Hardy D, Curran J, Lin D, Kuo S, Noguchi A, Itmann P, Duncan S, Gupta M, Piccarillo A, Karna P, Cohen M, Giuliano M, Carroll S, Page B, Guzman-Cottrill J, Walker
Central venous catheters in the NICU are associated with significant morbidity and mortality because of the risk of central line–associated bloodstream infections (CLABSIs). The purpose of this study was to determine the effect of catheter dwell time on risk of CLABSI. Increased dwell time was not associated with increased risk of CLABSI for PICCs. For tunneled catheters, infection incidence was significantly higher in weeks 7 and 9 compared with week 1.
Enteral Feeding With Human Milk Decreases Time to Discharge in Infants Following Gastroschisis Repair
The Journal of Pediatrics • December 2015
Gulack BC, Laughon MM, Clark RH, Burgess T, Robinson S, Muhammad A, Zhang A, Davis A, Morton R, Chu VH, Arnold CW, Hornik CP, Smith PB
This study reviewed a multi-institutional database to assess the effect of enteral feeding with human milk on duration from initiation of feeds to discharge after gastroschisis repair. The use of human milk for enteral feeding of infants following repair of gastroschisis significantly reduces the time to discharge from initiation of feeds.